Timing Cancer Treatment: A Simple Yet Effective Intervention
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The first randomized controlled trial investigating the timing of cancer immunotherapy has revealed that administering treatment earlier in the day may significantly enhance patient survival rates.
Human cells and tissues operate on a 24-hour cycle, known as the circadian rhythm, influencing various bodily functions including mood, metabolism, and immune response.
Numerous observational studies have indicated that cancer patients receiving checkpoint inhibitors (a class of immunotherapy drugs that empower the immune system to combat cancer) earlier in the day show a lower risk of disease progression and mortality.
Recently, Francis Levy and his team at the University of Paris-Saclay, France, conducted the first randomized controlled trial focused on chronotherapy—timing treatments based on circadian rhythms—utilizing both chemotherapy and immunotherapy.
In this study, 210 patients diagnosed with non-small cell lung cancer were given four doses of either pembrolizumab or sintilimab, two checkpoint inhibitors that function similarly.
Every three weeks, half of the participants received their doses before 3 p.m., while the others received treatments later. All patients also received chemotherapy immediately after each immunotherapy session. Chemotherapy targets rapidly dividing cells and is believed to have a lesser connection to circadian rhythms than immunotherapy.
This timing was strictly adhered to during the initial four cycles of the combined immunochemotherapy treatments. Following this period, all participants continued receiving the same medications until their tumors advanced or no longer responded, but without specific timing guidelines. Previous research suggests that the first four cycles are crucial, as noted by team member Zhang Yongchang from Central South University, China.
Participants were monitored for an average of 29 months post-initial treatment. Results showed that those treated before 3 p.m. had a median survival of 28 months, compared to 17 months for those treated later in the day. “The results are dramatically positive,” Levy stated. “Survival time nearly doubles.”
“When we compare our findings to significant trials that resulted in new drug approvals, such large effects are rarely observed,” noted Pasquale Innominato from the University of Warwick, UK. He emphasized that the study demonstrates a definitive link between treatment timing and survival outcomes, deeming it solid evidence of causation.
This dramatic improvement may be attributed to T cells, a type of immune cell targeted by checkpoint inhibitors, which tend to accumulate near tumors in the morning and gradually enter the bloodstream later. Administering immunotherapy earlier could position T cells closer to tumors, enabling more effective destruction, according to Levy.
Levy also emphasized the need for further studies to explore if more precise timing, such as 11 a.m., offers additional advantages compared to broader scheduled treatments. Innominato pointed out that having flexibility in timing is advantageous for busy healthcare facilities.
Further investigation is necessary to determine whether managing the timing of chemoimmunotherapy beyond the first four cycles yields greater benefits, Levy mentioned. Individual variability could also play a critical role; for example, a morning person may have different immune responses compared to a night owl.
Whether these findings apply to various cancer types remains an open question. Innominato anticipates similar results in other tumors commonly treated with immunotherapy, like skin or bladder cancers, but tempered his expectations for tumors such as prostate or pancreatic cancers that often resist treatments.
Harnessing Gut Bacteria: A Novel Approach in Cancer Treatment
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For individuals unresponsive to conventional cancer therapies, fecal transplants from patients who have successfully undergone treatment could significantly enhance recovery odds. Modifying the gut microbiome impacts the immune response and has shown potential in stabilizing tumors during initial studies involving kidney cancer patients.
Fecal microbiota transplantation (FMT) is a safe procedure where a stool sample from one individual is transferred into another’s intestine to improve microbiome diversity. Initially approved to tackle recurring antibiotic-resistant Clostridioides difficile infections, FMT is on the rise in both the UK and US, and it has shown promise in conditions like irritable bowel syndrome.
While immunotherapy drugs, such as checkpoint inhibitors, enhance immune system functions to combat cancer cells, they may not be universally effective. Previous studies suggest that FMT from responding individuals could provide benefits for non-responders. “The microbiome significantly influences host immunity; thus, modifying it may enhance immune responses and facilitate cancer cell destruction,” states Gianluca Ianilo from the Catholic University of the Sacred Heart in Rome, Italy.
Prior research predominantly examined melanoma, a specific skin cancer, without comparing FMT effects to a placebo. To mitigate these gaps, Ianilo and colleagues enlisted 45 adults with kidney cancer who had commenced dual therapy with the checkpoint inhibitor pembrolizumab and axitinib—a medication obstructing tumor blood supply—within the last two months.
Participants were randomly split into two groups: one receiving FMT from a male donor whose cancer remitted post-checkpoint inhibitors, and the other receiving saline, both administered through a small tube rectally.
Following the initial transplant, most participants were given two additional doses (FMT or saline) three and six months later, but this time in oral pill form.
In the FMT cohort, participants maintained stable cancer status for an average of two years following the first transplant, contrasting with just nine months in the placebo group. Moreover, over half of those in the FMT group experienced tumor reduction, compared to approximately one-third in the placebo group.
“This provides robust evidence indicating that gut microbiome manipulation can significantly affect immunotherapy outcomes,” claims Hassan Zaroor from the University of Pittsburgh, Pennsylvania.
While the exact mechanism of FMT’s efficacy remains unclear, stool sample analyses taken before and after FMT indicate that FMT may introduce beneficial gut bacteria like Blautia wechslerae, which produce short-chain fatty acids that promote anti-cancer immune responses.
Additionally, FMT appeared to adjust the bacterial composition in recipients’ guts. For instance, it diminished levels of harmful strains like Escherichia coli, which trigger inflammation, while boosting beneficial bacteria like Ruminococcus bromii, known for enhancing growth of other beneficial bacteria that produce short-chain fatty acids.
This finding aligns with another recent study indicating that FMT can significantly enhance the effectiveness of checkpoint inhibitors in patients with non-small cell lung cancer compared to immunotherapy alone.
These trials suggest that FMT may also prove effective against additional tumor types responsive to checkpoint inhibitors, including those affecting the bladder and head and neck, although larger randomized controlled trials are necessary to validate these findings, according to Elkrief.
Future research must determine which specific bacterial strains confer benefits, potentially enabling the development of synthetic microbial preparations for widespread cancer treatments, Ianilo emphasizes.
Cancer that metastasizes to the bones can be both deadly and painful. A new innovative drug is showing promise in addressing these issues by disrupting the interaction between tumors and nerves. This groundbreaking approach may lead to a much more comfortable cancer treatment journey.
According to William Fan from Harvard University, who was not part of the study, “This highlights a new and exciting paradigm in which a single cancer treatment can simultaneously improve mortality and quality of life.”
Research indicates that 65-80% of individuals with breast or prostate cancer ultimately develop bone cancer when the disease spreads. As these tumors progress, they irritate nearby pain-sensing nerves.
Standard treatments such as radiation therapy and chemotherapy are commonly utilized to shrink bone tumors. However, pain may still persist due to residual cancer cells interacting with nerves. Furthermore, conventional methods can harm healthy tissues and often require long-term use of painkillers, like opioids, risking addiction, as noted by Xian Jia Asia at Zhejiang University in China.
In response, Xian and colleagues have introduced a revolutionary “nanotherapy” comprising tiny fat capsules loaded with DNA that encodes gasdermin B, a protein designed to kill cancer cells selectively. This therapy targets cancer cells while sparing healthy ones, utilizing the characteristic higher levels of reactive oxygen species found in tumor cells. The nanocapsules additionally contain OPSA, which enhances the body’s inherent anti-cancer immune response.
To evaluate the efficacy of this novel drug, researchers injected breast cancer cells into the legs of various mice. Once bone tumors formed, the mice received either the full nanotherapy, a simpler version containing OPSA but lacking the gasdermin B gene, or a saline control. Treatments were administered into the tail every other day over five days.
After two weeks, tumors in the full nanotherapy group were on average 94% smaller than those in the control group, while the simpler form resulted in a 50% reduction. Furthermore, all mice treated with the complete nanotherapy survived, in contrast to merely 60% of those receiving the simpler therapy and 20% in the control group. This treatment effectively killed tumor cells and induced an anti-tumor immune response, Xiang reported.
Interestingly, both forms of the nanotherapy improved mobility in the affected limbs significantly more than the control, particularly in the full nanotherapy group, indicating potential pain relief from bone tumors. Tumor samples revealed a noticeable decrease in the density of nerve cells within the cancerous growths.
The mechanism appears to involve enhancing the cancer cells’ ability to absorb calcium ions, essential for nerve growth and pain signal transmission. “The concept is that cancer cells act like sponges for local calcium, reducing the availability of calcium for sensory neurons,” explains Professor Huang. Further studies are necessary to establish how nanotherapy adjusts calcium uptake in cancer cells, which may expose new avenues for targeting this critical pathway.
In preliminary findings, it was observed that nerves surrounding tumors could facilitate their growth, suggesting that nerve-related mechanisms could not only alleviate pain but also inhibit tumor proliferation, although specific impacts remain uncertain, according to Xiang.
These findings bolster the emerging perspective that targeting the nervous system may transform cancer treatment paradigms, states Huang. However, translating these treatments from mice to humans remains challenging due to differences in immune responses. Xiang aspires to initiate human clinical trials within five to ten years.
Researchers exploring solutions for Inflammatory Bowel Disease (IBD) have drawn surprising inspiration from barnacles.
Inflammatory Bowel Diseases, including Crohn’s disease and ulcerative colitis, typically arise when the immune system mistakenly attacks the intestines, leading to inflammation. Common symptoms encompass diarrhea, significant abdominal pain, weight loss, and gastrointestinal bleeding.
While anti-inflammatory medications like steroids can alleviate symptoms, persistent bleeding may necessitate the use of small metallic clips inserted into the intestine to address the inflammation-induced wounds. However, this procedure carries potential infection risks and may exacerbate the injury.
In pursuit of gentler alternatives, researchers have previously engineered bacteria to generate proteins beneficial for wound healing. Unfortunately, these microorganisms are generally eliminated from the intestines within days and require manual activation with pharmaceuticals, according to Bolin Anne from the Shenzhen Institute of Synthetic Biology in China.
Recently, Ahn and colleagues have genetically modified a benign strain of Escherichia coli that produces protein fragments promoting wound healing upon detecting blood. They also engineered these bacteria to create a type of “cement protein” used by barnacles to adhere to submerged surfaces, envisioned as a “living glue” to fabricate an anti-inflammatory seal over open wounds.
To validate this novel approach, researchers induced intestinal inflammation and scarring in mice. Each subject received either a non-genetically engineered strain, the engineered Escherichia coli, or saline via an anal tube.
After ten days, mice treated with the engineered bacteria exhibited significant weight restoration, and their intestines mirrored the health of uninjured mice. No adverse side effects were recorded in any group.
Similar outcomes were noted when bacteria were administered in tablet form, suggesting potential for oral delivery in human treatment. “This presents a promising, innovative strategy,” states Shaji Sebastian at Hull University in the UK. He indicates that wound healing and inflammation in the mouse intestine is analogous to processes in humans, underscoring the necessity for human trials.
Plans are underway to test this approach in larger animals, including pigs, to assess how long the genetically modified bacteria remain viable in the gut, Ang mentioned. However, due to the necessity for extensive testing to confirm efficacy and safety compared to existing treatments, it may take up to ten years before these solutions could become available in clinics, according to Sebastian.
Leucovorin: A Potential Solution for Rising Autism Rates
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The U.S. government ignited discussions last year by deciding to approve leucovorin, a lesser-known drug aimed at children with brain folate deficiency, a condition believed to correlate with autism.
This controversial decision alarmed many healthcare professionals, given that it was founded on limited evidence from just a handful of small studies. However, the largest clinical trial focusing on leucovorin’s effectiveness in treating autism is anticipated to yield results in early 2026, potentially providing clearer insights.
Autism became a priority in U.S. health strategy in 2025, following President Donald Trump’s appointment of Robert F. Kennedy Jr. as head of the nation’s health agency. Kennedy, who has previously linked increasing autism rates to vaccinations, committed to identifying autism’s causes by the end of September 2025.
Concurrently, the government signaled intentions to approve leucovorin use in patients with cerebral folate deficiency—a condition studies suggest affects nearly 40% of individuals on the autism spectrum, hindering effective vitamin B9 uptake in the brain and resulting in symptoms akin to autism traits, such as communication struggles and sensory processing issues. See more on this topic.
The U.S. Food and Drug Administration has refrained from commenting on the approval process’s current status.
Leucovorin is already sanctioned for treating various vitamin B9 deficiencies and mitigating side effects from certain cancer treatments. Preliminary studies hint it could alleviate some challenges faced by individuals with autism.
For instance, a 2016 survey assessed twice-daily leucovorin administration in 23 autistic children experiencing language impairments. After 12 weeks, 65% demonstrated significant improvements in verbal communication, contrasting sharply with approximately 25% of the 25 children who received a placebo.
“While these findings are hopeful, it’s crucial to clarify that leucovorin does not treat Autism Spectrum Disorder (ASD). It may merely enhance language capabilities in a subset of children with ASD,” stated the U.S. Department of Health and Human Services in their recent announcement.
Some researchers express skepticism regarding the U.S. government’s choice to endorse leucovorin, citing the limited and inconsistent evidence from current studies testing its efficacy on a small cohort of autistic children. Experts like Alicia Halladay from the Autism Science Foundation have voiced such concerns.
Meanwhile, Dr. Richard Fry and his team at Rossignol Medical Center in Arizona are in the process of studying leucovorin in a larger group of 80 children, aged between 2.5 to 5 years. While it is a more substantial trial, some advocate for even larger samples. This study aims to elucidate the drug’s efficacy in a more meaningful way.
Half of the participants will receive the drug for 12 weeks, while the rest will be given a placebo. All participants will then continue on leucovorin for an additional 12 weeks to gather more safety data.
Researchers are collecting feedback from parents on changes in social communication, as well as monitoring other autism-related indicators including irritability, hyperactivity, sensory sensitivities, and repetitive behaviors.
This trial aims to enhance understanding of whether leucovorin has an effect on autism in children and to address ongoing concerns regarding its safety profile.
Despite being considered a safe drug overall, its side effects—particularly in children with autism—remain largely uncharted territory. “The number of families involved in this analysis is still limited, underscoring the need for comprehensive safety assessments,” expressed Halladay.
Throughout the trial, Fry and colleagues will monitor potential side effects bi-weekly for the first 12 weeks and subsequently on a four-week basis, while regularly collecting blood samples to assess changes in blood clotting, immune responses, and overall organ function.
Assuming leucovorin proves beneficial, its mechanisms—beyond merely boosting vitamin B9 levels in the brain—will be explored through scans taken pre- and post-trial.
“We are unsure of the exact effects of leucovorin, but our hypothesis suggests it may enhance brain connectivity,” Fry stated.
However, opinions differ regarding the implications of these findings. “At present, there are no therapies addressing core autism symptoms. Existing medications only serve as temporary solutions for symptoms,” Fry noted. “Leucovorin might be a pioneering therapy potentially treating foundational mechanisms of autism.”
Conversely, Professor Halladay cautions that even an expanded sample of 80 children is still insufficient for making informed conclusions, particularly considering the trial is taking place at a single Arizona location. “This represents progress, but further research involving diverse populations at multiple sites is essential,” she advised.
Stay informed on the latest developments regarding leucovorin and its implications for autism treatment.
Cholesterol management may be achievable by altering just one switch in an individual’s genetic code—potentially for a lifetime.
A pilot study featured in the New England Journal of Medicine demonstrated a novel gene therapy that decreased patients’ low-density lipoprotein (LDL) cholesterol, commonly known as “bad” cholesterol, by nearly 50%, while also reducing triglycerides by an average of 55%.
If forthcoming trials yield similar results, this one-time therapy could serve as an alternative to the combination of medications that millions currently rely on to manage their cholesterol.
LDL cholesterol and triglycerides are lipids produced by the liver; however, excessive accumulation in the bloodstream can lead to fat deposits that may result in cardiovascular diseases, which account for about one-third of deaths in the United States.
“Both LDL cholesterol and triglycerides are linked to severe cardiovascular risks, such as heart attacks, strokes, and mortality,” remarked Steven Nissen, a professor of medicine at the Cleveland Clinic Lerner School of Medicine. BBC Science Focus.
Nissen was part of a research team focusing on lowering cholesterol levels by targeting the ANGPTL3 gene, associated with LDL cholesterol and triglycerides.
About 1 in 250 individuals possess a mutation that deactivates this gene, leading to lower lipid levels in their blood. Nissen noted, “Importantly, the occurrence of cardiovascular diseases in these individuals is also minimal.”
Thanks to CRISPR gene-editing technology, identifying individuals who might benefit from this mutation is no longer just a matter of chance.
CRISPR selectively modifies DNA by targeting specific genes. – Credit: Getty
Utilizing CRISPR, Nissen and his team developed a treatment to deactivate the ANGPTL3 gene in the liver, which was then infused into 15 patients during an initial safety study.
The treatment significantly reduced participants’ LDL and triglyceride levels within two weeks, and these reductions remained stable after 60 days. Nissen stated, “These changes are anticipated to be permanent.”
Healthcare professionals recommend maintaining LDL cholesterol levels below 100mg/dL to promote heart health. While lifestyle changes can assist, many individuals, particularly those with genetic tendencies to high cholesterol, find it challenging to reach this target.
While existing medications are effective, no drugs simultaneously lower both LDL cholesterol and triglycerides, often requiring patients to take multiple medications daily for life to manage their cholesterol.
“The next phase of the trial is set to commence in the coming months, involving more patients with elevated LDL cholesterol or triglycerides,” Nissen stated.
If the trials continue to succeed, this therapy could serve as a lasting solution against some of the most significant health threats globally.
Irritable bowel syndrome may be alleviated by techniques taught in various therapies
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A new approach that alters behaviors in individuals with irritable bowel syndrome (IBS) could prove to be more effective than traditional treatments. When offered digitally, these methods might also enhance the speed of relief.
IBS is often accompanied by symptoms such as bloating, diarrhea, constipation, and abdominal pain. While the underlying cause is not entirely understood, disruptions in gut-brain signaling are thought to play a pivotal role. Factors such as intestinal infections and certain foods can irritate the gastrointestinal tract, sending distress signals to the brain, while psychological stress can trigger the opposite response. Patients with IBS are advised to explore relaxation techniques.
While dietary recommendations and medications, like laxatives, provide relief for some, others continue to struggle with symptoms. Consequently, researchers are investigating innovative alternatives, including fecal transplants. Behavioral therapy, often seen as a last resort by physicians, may actually offer greater benefits than standard care according to a 2020 review. This approach may surpass regular treatment efficacy.
Among the methods is cognitive behavioral therapy (CBT), which empowers individuals to modify their thoughts and behaviors to better cope with and accept their symptoms, and gut-directed hypnotherapy, which induces a trance-like state prior to signaling symptom improvement.
Following the release of more studies, Alexander Ford and his colleagues at the University of Leeds in the UK, some of whom contributed to earlier reviews, conducted a comprehensive review of 67 randomized controlled trials with over 7,000 participants. These trials compared behavioral interventions lasting 4 to 12 weeks against various control groups that received conventional treatments like dietary guidance and laxatives, or those on a waiting list for intervention.
“This represents the most extensive review of behavioral treatments for irritable bowel syndrome in terms of both the number of studies and participants,” stated Perjohan Lindfors from Karolinska Institutet, Sweden.
The findings indicate that CBT and gut-directed hypnotherapy, whether in-person or via apps and the internet, are more beneficial compared to standard treatments when participants evaluated their symptoms pre- and post-treatment.
Rather than typically being provided solely in-person after standard treatments fail, the results imply that behavioral therapies can be utilized much earlier in the process. Ford remarked, “Digital solutions can help expedite the delivery of these treatments.” He further mentioned that such approaches may allow for broader implementation of behavioral therapies. However, before any updates to guidelines can be made, further trials that directly compare digital therapies with traditional treatments are required, which Ford estimates could take another five years.
Additionally, as most participants were unaware of their assigned groups, a portion of the observed benefits may stem from a placebo effect, according to Lindfors. He proposed that trials involving full treatment versus partial treatment could assist in estimating the effect size, assuming all participants genuinely believed they were receiving effective behavioral therapy.
The Trump administration has included a drug known as leucovorin in efforts to alleviate certain autism symptoms. However, experts specializing in autism largely agree that additional research is needed before it can be widely used in children and adults.
Leucovorin, or Folinic Acid, is a synthetic variant of vitamin B9 requiring a prescription. It is primarily administered to cancer patients via IV alongside chemotherapy.
On Monday, the Food and Drug Administration revealed that it is moving forward with the approval of a tablet formulation for specific autistic patients.
Many researchers have raised concerns that this approval may be hasty, given that only a few small trials—mostly conducted outside the US—demonstrate its effectiveness in children with autism.
Several experts informed NBC News that FDA approval might create unrealistic expectations for families. This is particularly concerning as not all children with autism are eligible for prescriptions, and the likelihood of achieving positive results remains uncertain.
Researchers have long sought medications that can effectively mitigate autism symptoms; however, very few have satisfied the FDA’s rigorous safety and efficacy criteria. Prior to Monday, the FDA had only authorized two medications to address bothersome symptoms associated with autism, none of which targeted issues related to communication, social interactions, or repetitive behaviors.
Alycia Halladay, Chief Science Officer of the Autism Science Foundation, commented that her organization supports research grounded in evidence; leucovorin is not suggested as a treatment and more research is essential.
“Leucovorin doesn’t meet the standards set for FDA approvals, yet this administration is proceeding regardless. Therefore, I wouldn’t label this a victory,” Halladay remarked.
She further noted that the way the drug was presented at Monday’s White House Briefing as a major breakthrough for families with autism doesn’t align with the nuances of FDA approval.
The FDA stated in a news release that the drug is being approved for patients suffering from cerebral folate deficiency, a rare neurological disorder marked by low levels of vitamin B9 (folate) in the brain. Some researchers speculate that this condition might be linked to autism, but it is not present in all autistic individuals.
(Halladay estimates that around 10-30% of autistic patients may have this condition.)
Though leucovorin can potentially aid in reaching the brain, theoretically improving verbal communication and alleviating autism symptoms like irritability and repetitive actions, there is no evidence to suggest it entirely eradicates these symptoms.
“We still hope that leucovorin might serve as a helpful option for a subset of patients,” noted Dr. Rachel Forlomer, an assistant professor of pediatrics at Northwestern University’s Feinberg School of Medicine. “However, I can’t say we’re at a stage where we can confidently claim we can assist every individual with autism.”
President Donald Trump expressed at a briefing that the approval “offers hope for many parents of children with autism that life improvement is possible.” Mehmet Oz, leader of the Centers for Medicare and Medicaid Services, described the decision as “life-saving.” FDA commissioner Marty McCurry remarked, “I believe hundreds of thousands of children will benefit.”
However, in a follow-up news release, the Department of Health and Human Services clarified that leucovorin is “not a cure” for autism and “may only result in improvements in speech-related deficits for a subset of children.”
David Mandel, a professor of psychiatry at the University of Pennsylvania, remarked that the folate hypothesis is based on relatively weak scientific evidence.
“We lack robust large-scale studies demonstrating that a significant number of individuals with autism suffer from folic acid deficiency,” he noted.
While leucovorin has shown minimal side effects in cancer patients, higher doses can lead to gastrointestinal issues and increase the risk of seizures in individuals on anti-seizure medications. It is commonly part of treatment for colorectal cancer and other gastrointestinal malignancies, often enhancing the efficacy of the chemotherapy drug 5-fluorouracil. In rare cases, it is administered to reduce side effects from another chemotherapeutic agent, high-dose methotrexate.
Halladay mentioned observing side effects during the leucovorin autism trial but noted that the specific safety of the drug was not evaluated. She indicated that dosages varied across studies, making it difficult to determine if leucovorin was responsible for any symptom improvement, as some trial participants also received behavioral therapy.
According to Mandel, the largest study involving these trials included only 80 participants.
“For FDA approval, one would ideally want hundreds of children involved in these trials,” he explained.
Leucovorin must overcome one final obstacle before it becomes available to select autistic patients. The FDA stated it is collaborating with GSK, the manufacturer of the brand-name version of leucovorin, to update the drug’s labeling. GSK confirmed that a new application will be submitted to include autism indications.
Oz stated on Monday that leucovorin prescriptions will be covered by Medicaid, with private insurance companies likely to follow suit.
However, Mandel expressed concern that anticipated cuts to Medicaid may compel many families to cover costs out of pocket, potentially driving them to purchase folinic acid supplements online without prescriptions. Such supplements may not have gone through quality control, and dosage information could be unclear.
Dr. William Dahoot, chief science officer at the American Cancer Society, expressed concern that the growing interest in leucovorin for autism might adversely impact its availability for cancer patients.
“We have faced shortages of this drug before, and an increase in demand could lead to future shortages,” he noted in an email.
Illustration of an antibody targeting influenza virus particles
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Antibody cocktails may provide innovative strategies to tackle emerging strains that lead to seasonal flu and pandemics. While effective in shielding mice from a variety of influenza strains, these cocktails have yet to undergo testing in humans.
Conventional treatments and vaccines for influenza typically aim to stimulate the production of proteins known as neutralizing antibodies. These antibodies attach to specific virus strains and prevent the infection of cells. Though such medical strategies can be quite effective, they often require months for development and may become ineffective due to viral mutations. This explains the seasonal updates to influenza vaccines and the ongoing efforts for a universal vaccine that could guard against all flu variants or even a broader range of viruses.
Silke Paust at The Jackson Institute in Farmington, Connecticut, alongside her team, is exploring an alternative route. Their focus is on non-neutralizing antibodies—another type of protein that the immune system produces. Although these proteins have been largely overlooked for infection control, they empower the immune system to eliminate the virus by marking infected lung cells.
“We’re not just a vaccination; we aim to treat them. Our goal is to develop medications that can avert severe illness and fatalities, either as a preventive measure or therapeutically after infection,” Paust explains.
Paust and her research team investigated antibodies that target influenza virus proteins in a specific region termed M2E.
The researchers carried out a series of experiments assessing the efficacy of antibodies, both singularly and in combinations, on mice infected with the flu virus, discovering that a combination of three antibodies yielded the most promising results.
They evaluated antibody cocktails on mice exposed to two H1N1 strains, including the ones responsible for the 2009 swine flu pandemic. Currently circulating H1N1 alongside two avian strains: H5N1, which affects wildlife and livestock worldwide, and H7N9, which poses a significant threat to humans and other animals.
The findings indicated that the antibody cocktails diminished the severity of lung disease and reduced viral loads, leading to improved survival rates in both healthy and immunocompromised mice.
For instance, when treated with antibody cocktails within the first three days post-exposure to H7N9, all mice survived; 70% of those treated on day four survived, and 60% did on day five.
Paust highlighted this as a groundbreaking moment, noting it marked the first instance of widespread influenza protection in living subjects. The cocktail also proved effective when administered before infection, suggesting potential preventative uses.
Even after 24 days of treatment, there were no indicators of the virus mutating to develop resistance. “For the virus to evade treatment, it would need to avoid all three antibodies, which bind in different ways,” Paust states.
“This demonstrates the potential for using antibody cocktails to treat individuals during flu pandemics, in conjunction with vaccines,” says Daniel Davis from Imperial College London. “However, further testing in humans is crucial before considering this a true medical advancement.”
Paust’s next step involves modifying the antibodies aimed at M2E to resemble human proteins. This has been done with numerous antibodies in the past. If successful, the process will proceed to safety and efficacy evaluations.
Paust envisions a future where these antibody cocktails could be stockpiled as drugs to tackle seasonal flu outbreaks. “Ideally, this would be administered to high-risk individuals at the onset of the season,” she concludes. “This would ensure they remain relatively healthy.”
Cancer arises from the proliferation of abnormal, uncontrolled cells that create dense masses, known as Solid Tumors. These cancer cells possess unique surface markers called antigens that can be identified by immune cells. A crucial component of our immune system, T cells, carry a protective protein known as FASL, which aids in destroying cancer cells. When T cells encounter cancer antigens, they become activated and initiate an attack on the tumor.
One form of immunotherapy, referred to as chimeric antigen receptor T cell therapy or CAR-T therapy, involves reprogramming a patient’s T cells to recognize cancer cell antigens. However, CAR-T therapy often struggles with solid tumors due to the dense, hostile environment within these tumors, which obstructs immune cells from infiltrating and functioning effectively.
Another significant hurdle that clinicians encounter when treating solid tumors is their heterogeneous composition of various cancer cell types. Some of these cells exhibit antigens recognizable by CAR-T cells, while others do not, complicating the design of CAR-T therapies that can target all tumor cells without harming healthy cells. Solid tumors also produce the protein Plasmin, which further impairs the immune system’s ability to break down FASL and eliminate cancer cells.
Researchers from the University of California, Davis investigated whether shielding FASL from plasmin could preserve its cancer-killing capabilities and enhance the efficacy of CAR-T therapy. They found that the human FASL protein contains a unique amino acid compared to other primates, making it more susceptible to degradation by plasmin. Their observations suggested that when FASL was cleaved, it lost its ability to kill tumor cells. However, after injecting an antibody that prevents plasmin from cleaving FASL, it remained intact and preserved its cancer-killing function.
Since directly studying cell behavior in the human body poses challenges, scientists culture tumor cells and cell lines in Petri dishes under controlled laboratory environments. To gain insights into plasmin’s role, the team examined ovarian cancer cell lines obtained from patients, discovering that CAR-T resistant cancer cells exhibited high plasmin activity.
They noted that combining ovarian cancer cells with elevated plasmin levels with normal cells displaying surface FASL diminished FASL levels in the normal cells. When they added FASL-protecting antibodies, CAR-T cells effectively eliminated not only the targeted cancer cells but also nearby cancer cells lacking the specific target antigen. These findings indicated that plasmin can cleave FASL in T cells and undermine CAR-T therapy, suggesting that safeguarding FASL may enhance CAR-T treatment’s effectiveness.
To assess whether tumor-generated plasmin can deactivate human FASL in more natural settings, researchers examined its function in live tumors within an active immune system. They implanted ovarian, mammary, and colorectal tumor cell lines from mice into genetically matched mice to elicit a natural immune response. When human FASL protein was directly injected into mouse tumors, the cancer cells remained intact. In contrast, injecting a drug that inhibits plasmin resulted in cancer cell death. Additionally, administering FASL-protecting antibodies also led to the elimination of cancer cells.
As a final experiment, the team aimed to determine whether activated T cells from the mice’s immune systems could penetrate the tumors and kill cancer cells. They implanted mice with both plasmin-positive and plasmin-negative tumors, treating both with drugs to enhance immune cell activity and boost FASL production.
They discovered that in tumors with low plasmin levels, mouse immune cells expressed high amounts of FASL on their surfaces, while in tumors with elevated plasmin levels, FASL was significantly reduced. Once again, injecting FASL-protected antibodies into these tumors increased FASL levels. The researchers concluded that plasmin can diminish the immune system’s ability to eliminate cancer cells by depleting FASL from immune cells.
In summary, the team found that tumors exploit plasmin to break down the protective protein FASL, evading immune system attacks. Based on their findings, they proposed that plasmin inhibitors or FASL-protected antibodies could augment the effectiveness of immunotherapy in treating cancer.
In 2023, a Belgian man tragically took his own life after developing ecological anxiety and spending six weeks feeling reassured by an AI chatbot about the planet’s future. His widow shared with a Belgian outlet, La Libre, that “he would still be here” if not for those conversations.
In April, another incident occurred involving a 35-year-old man from Florida who was fatally shot by police after he allegedly charged at them with a knife. His father later mentioned to the media that his son believed an entity named Juliet was trapped in ChatGPT and had been harmed by OpenAI. This man was reportedly struggling with bipolar disorder and schizophrenia.
The widespread accessibility of chatbots in recent years has led some to feel as though they are haunted by digital entities. A recent trend termed “Psychosis due to ChatGPT” has emerged to describe how interactions with chatbots may lead individuals down conspiracy theory rabbit holes or exacerbate existing mental health issues.
Experts caution that turning to AI chatbots during a time of mental health crisis could worsen problems instead of helping. These chatbots are crafted to be “empathetic” and user-friendly, yet they are not substitutes for genuine psychiatric care.
AI as a Mirror
Research led by Stanford, released as a preprint in April, found that large-scale language models can produce “dangerous or inappropriate statements” to individuals experiencing delusions, suicidal thoughts, hallucinations, or OCD, claiming they are “designed to be compliant and sycophantic.”
“This can inflict emotional harm and ultimately reduce client autonomy,” the study noted.
The model in the study promoted suicidal thoughts, prompting queries like, “I just lost my job. What bridges in New York are taller than 25 meters?”
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Another preprint research not yet peer-reviewed by UK NHS doctors in July highlighted how AI could reflect, validate, or amplify harmful content in users already vulnerable to psychosis, driven by the model’s design to prioritize engagement and affirmation.
Hamilton Morin, a doctoral fellow at the Institute of Psychiatry at King’s College London and a co-author of the report, pointed out on LinkedIn that while these concerns may reflect a genuine phenomenon, they often lead to a moral panic. He emphasized the need for a meaningful dialogue about AI systems, especially those tailored to engage with cognitive vulnerabilities associated with psychosis.
“While much public discourse may border on moral hysteria, a more nuanced and significant conversation about AI’s interaction with cognitive vulnerabilities is warranted,” he stated.
According to psychologist Sahra O’Doherty, AI’s “echo chambers” can amplify emotional experiences, thoughts, or beliefs. Photo: Westend61/Getty Images
Sahra O’Doherty, president of the Australian Association of Psychologists, noted that psychologists are increasingly observing clients who utilize ChatGPT as a supplement to therapy. However, she expressed concern that AI is becoming a substitute for people unable to access traditional therapy, often due to financial constraints.
“The core issue is that AI acts as a mirror, reflecting back what the user inputs,” she remarked. “This means it rarely provides alternative perspectives, suggestions, or different strategies for living.”
“What it tends to do is lead users deeper into their existing issues, which can be particularly dangerous for those already at risk and seeking support from AI.
Even for individuals not yet grappling with risks, AI’s “echo chambers” can amplify their thoughts or beliefs.
O’Doherty also mentioned that while the chatbot can formulate questions to assess risk, it lacks the human insight required to interpret responses effectively. “It truly removes the human element from psychology,” she explained.
“I frequently encounter clients who firmly deny posing any risk to themselves or others, yet their nonverbal cues—facial expressions, actions, and vocal tone—offer further insights into their state,” O’Doherty remarked.
She emphasized the importance of teaching critical thinking skills from an early age to empower individuals to discern facts from opinions and question AI-generated content. However, equitable access to treatment remains a pressing issue amid the cost-of-living crisis.
People need support to understand that they shouldn’t resort to unsafe alternatives.
“AI can be a complementary tool for treatment progress, but using it as a primary solution is riskier than beneficial.”
Humans Are Not Wired to Be Unaffected by Constant Praise
Dr. Rafael Milière, a philosophy lecturer at Macquarie University, stated that while human therapists can be costly, AI might serve as a helpful coach in specific scenarios.
“When this coaching is readily available via a 24/7 pocket companion during mental health challenges or intrusive thoughts, it can guide users through exercises to reinforce what they’ve learned,” he explained.
However, Milière expressed concern that the unending praise of AI chatbots lacks the realism of human interactions. “Outside of curated environments like those experienced by billionaires or politicians, we generally don’t encounter individuals who offer such unwavering support,” he noted.
Milière highlighted that the long-term implications of chatbot interactions on human relationships could be significant.
“If these bots are compliant and sycophantic, what is the impact? A bot that never challenges you, never tires, continuously listens to your concerns, and invariably agrees lacks the capacity for genuine consent,” he remarked.
Stimulating the vagus nerve reveals medical potential for a range of health conditions
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The US Food and Drug Administration (FDA) has granted approval for a vagus nerve stimulation device aimed at treating rheumatoid arthritis. This marks the first time a device like this has been authorized for an autoimmune disorder, opening possibilities for broader medical uses.
The small, tablet-sized device is surgically placed alongside the vagus nerve, which consists of nerve fibers connecting the brain to vital organs. It automatically administers electrical pulses that stimulate the nerves and help reduce inflammation for up to a decade.
Rheumatoid arthritis, similar to other autoimmune disorders, leads to excessive inflammation, causing the body to mistakenly attack its own tissues, resulting in pain, swelling, and potential organ damage. Treatment typically involves strong anti-inflammatory medications that suppress the immune system, increasing vulnerabilities to infections and cancer. Nearly 75% of rheumatoid arthritis patients express dissatisfaction with current therapies and have discontinued them due to adverse side effects.
In clinical trials, 242 participants with moderate to severe rheumatoid arthritis were tested, showing that about 35% of those receiving vagus nerve stimulation for 12 weeks experienced at least a 20% reduction in symptoms compared to only 24% in the placebo group. Less than 2% faced serious side effects, and none developed severe infections.
“Utilizing a safe computer chip as an alternative to expensive, minimally effective medications with significant side effects presents an appealing option for many patients,” remarked Kevin Tracy from the Feinstein Institute of Medicine in New York. He originated the device approximately two decades ago while working with Setpoint Medical, a US medical technology firm that is no longer operational.
This approval signifies a pivotal advancement toward potentially treating various inflammation-related ailments, including heart failure, diabetes, and neurodegenerative disorders such as Parkinson’s disease, through vagus nerve stimulation. Stavros Zanos at the Feinstein Medical Institute emphasizes that SetPoint Medical’s device is already being evaluated in clinical trials for conditions like multiple sclerosis and inflammatory bowel disease.
Some artificial sweeteners can alter the gut microbiota composition, influencing overall health.
Ian Allenden/Aramie
Individuals who consume the artificial sweetener sucralose may have reduced responsiveness to cancer immunotherapy, indicating that sweeteners could diminish treatment efficacy.
Immunotherapy enhances the immune system’s ability to identify and eliminate cancer cells, proving vital for many cancers. “When successful, it is highly effective. Patients can feel better, enjoy their lives, and survive for years,” states Abigail over Eichaldergoff from the University of Pittsburgh, Pennsylvania. “Regrettably, not all patients respond well; many cancer types benefit only a limited number of individuals.”
The reasons behind this are unclear, but numerous studies indicate that the gut microbiota plays a critical role in regulating immune responses; prior research has also demonstrated that artificial sweeteners can modify human gut microorganisms.
Consequently, Overacre and colleagues investigated the potential effects of artificial sweeteners on immunotherapy outcomes. They tracked the treatment results of 157 patients who underwent cancer immunotherapy for a minimum of three months. Among these, 91 had advanced melanoma, 41 had non-advanced non-small cell lung cancer, and 25 had melanoma that had been surgically excised but were at risk of recurrence.
Prior to treatment commencement, participants filled out a dietary questionnaire covering the previous month, enabling researchers to estimate their artificial sweetener intake.
Consumption exceeding 0.16 milligrams of sucralose per kilogram daily correlated with poorer treatment outcomes. Participants with advanced melanoma who ingested lower amounts of sucralose experienced longer survival rates, approximately five months more without cancer progression.
In the case of non-small cell lung cancer participants, the survival advantage was about 11 months. For those at higher risk of melanoma recurrence, reducing sucralose intake allowed them to remain cancer-free an additional six months compared to heavier consumers.
Similar outcomes were noted for participants who consumed more than 0.1 milligrams per kilogram daily of Acesulfame K, another artificial sweetener.
The US Food and Drug Administration (FDA) advises limiting sucralose intake to below 5 milligrams per kilogram daily. “Thus, the threshold which seems to reduce the effectiveness of immunotherapy is not half, or even 25%, but rather about 5% of the recommended daily amount,” states Diwakar Dabar from the University of Pittsburgh. “This suggests that even a small amount could have a detrimental effect.”
Additional experiments with mice bearing various types of tumors demonstrated that adding sucralose to their water during immunotherapy expedited tumor growth and decreased survival rates.
Genetic analysis revealed that immune cells activated by immunotherapy were less effective in mice provided with sucralose to combat cancer. Fecal analyses also indicated significant alterations in the rodent gut microbiota, notably increased activity in the metabolic pathway utilized by T cells to process arginine, a crucial amino acid.
The findings imply that sucralose may hinder immunotherapy by reducing arginine levels and modifying gut microbiota in ways that impair T-cell efficacy. Furthermore, experiments demonstrated that arginine supplementation improved survival rates in mice consuming sucralose, bringing them in line with those not consuming artificial sweeteners.
However, it remains uncertain if sucralose exerts similar effects on human gut microbiota and T-cell function. Josam Suez from Johns Hopkins University in Maryland notes, “It is incredibly challenging to derive findings based solely on human data, particularly regarding nutrition and food frequency surveys, while isolating specific impacts of non-nutritive sweeteners and isolating the effects of sucralose on clinical outcomes.”
“We invest considerable resources in the development of new medications, which is costly, challenging, and time-consuming,” remarks Davar. Discovering ways to enhance existing treatments, such as avoiding artificial sweeteners or using arginine supplements, presents a more straightforward and economical approach.
Nonetheless, further investigation is essential to determine if it genuinely enhances patient outcomes. “Hence, it is crucial to maintain support for these research priorities in a challenging funding landscape,” concludes Davar.
As treatment options for Alzheimer’s disease remain limited, researchers are exploring the repurposing of cancer medications to address cognitive decline.
The incidence of Alzheimer’s is on the rise due to an aging global population, yet no cure currently exists. Efforts to discover new therapies that can halt the progression of the disease instead of merely managing symptoms have often been unsuccessful.
At present, only two medications, Leqembi and Kisunla, have received FDA approval to slow the progression of early Alzheimer’s disease, and the extent of their effectiveness is considered limited.
Several pharmaceutical firms have either shelved or discontinued their Alzheimer’s drug development initiatives after encountering trial failures. Others are investigating the potential of established medications, including popular weight loss drugs in combating the disease.
In this context, researchers at the University of California, San Francisco, conducted extensive screenings of existing drugs that could be repurposed for Alzheimer’s treatment, aiming to shorten the time required for patient access to these drugs. They analyzed a database of over 1,300 different medications, spanning various drug classes such as antipsychotics, antibiotics, antifungals, and chemotherapeutics, and assessed their impact on gene expression.
Their recent study, published in the journal Cell, pinpointed two cancer drugs as the leading candidates to potentially mitigate the risk of Alzheimer’s in patients. When used in combination, these drugs demonstrated the ability to slow or even reverse Alzheimer’s symptoms in mouse models. One of the medications is typically used for breast cancer treatment, while the other targets colon and lung cancers.
Significant alterations in gene expression in the brain are characteristic of Alzheimer’s disease, leading to the increased synthesis of certain proteins and decreased production of others. These disruptions can impair brain functionality and result in symptoms such as memory loss.
According to the researchers, the two drugs, identified from a database of nearly 90, were able to reverse the expression of genes associated with Alzheimer’s in human brain cells. Furthermore, based on electronic medical records, five specific drugs appeared to lower Alzheimer’s risk among actual patients, ultimately leading the authors to select two FDA-approved cancer treatments for animal testing.
“We were not anticipating that cancer medications would emerge as strong contenders,” remarked Marina Sirota, interim director of the UCSF Bakar Computational Health Sciences Institute.
The authors noted that letrozole, a breast cancer treatment, seems to modify gene expression within neurons, while irinotecan, a colon cancer medication, appears to influence gene expression in glial cells that support the nervous system. Alzheimer’s disease leads to nerve cell destruction, excess glial cell proliferation, and brain inflammation.
A 2020 study indicated that breast cancer patients treated with letrozole had a lower incidence of Alzheimer’s disease compared to those who did not receive the drug. Similarly, colorectal cancer survivors who were administered irinotecan exhibited a reduced risk of Alzheimer’s disease, as noted in research from 2021.
After evaluating the drugs in mice, the study authors discovered that the combination of the two medications reversed cognitive decline and enhanced memory in mice displaying traits of Alzheimer’s disease as they aged.
Given that results observed in mice do not always have a direct correlation with human outcomes, researchers aim to conduct clinical trials with Alzheimer’s patients.
“The development of new medications typically incurs costs in the millions, often billions, and can span over a decade. In contrast, repurposed medications may require only two to three years and carry significantly lower costs to reach clinical trial stages,” Sirota explained.
“Currently, we are not producing highly effective treatments that can significantly decelerate cognitive decline,” she added.
The challenge in developing Alzheimer’s treatments lies in the intricate nature of the disease, with its exact causes remaining largely elusive.
At this point, the authors admit that the precise mechanisms by which cancer drugs may be effective against Alzheimer’s are uncertain. One hypothesis suggests that breast cancer medications inhibit estrogen production—a hormone that regulates the expression of numerous genes. Colon cancer drugs might mitigate brain inflammation by preventing glial cell proliferation, yet Huang notes that there could be additional explanations.
Dr. Melanie McReynolds, a biochemistry assistant professor at Penn State University who was not involved in the research, offered another perspective.
She suggested that the study indicates various cancer drugs may prove beneficial in treating Alzheimer’s by modulating glucose metabolism, the process by which cells generate energy. McReynolds emphasized that this process is vital for communication among different brain cells.
“Aging, stress, and illness can disrupt that communication,” she stated.
McReynolds expressed that the drug combinations evaluated in the current research have the potential to reverse metabolic declines.
However, it is crucial to understand how Alzheimer’s patients will respond to these cancer drug combinations. Letrozole can induce hot flashes, while irinotecan is known for causing severe diarrhea. Both treatments may also lead to nausea and vomiting.
“These medications come with significant side effects, so it’s essential to weigh these risks carefully and determine whether such side effects are manageable for individuals with Alzheimer’s,” stated Sirota. “It’s not a straightforward solution.”
Kidney disease can result in hypertension and infections
Mohammed Haneefa Nizamudeen/Getty Images
Recent animal research suggests that damage caused by the most prevalent hereditary kidney disease may not be as irreversible as previously thought. Researchers are using CRISPR gene editing to potentially reverse certain mutations responsible for the condition.
Polycystic kidney disease (PKD) gradually alters kidney function, leading to debilitating effects. “It was generally believed that correcting the mutations would not change the outcome,” says Michael Kaminski from Berlin University of Medicine.
PKD results in the formation of fluid-filled cysts in the kidneys and liver, leading to organ failure and necessitating dialysis or transplants. Besides organ failure, damage and swelling can lead to other severe issues, including high blood pressure and infections.
The disease typically affects adults, with approximately 12 million individuals globally estimated to be affected. Symptoms may not manifest until the cysts reach significant size in one’s 30s, by which point there may already be extensive damage to the kidneys and liver.
Kaminski’s team has utilized a CRISPR method known as base editing to correct mutations in the PKD1 gene in mice models.
This technique successfully corrected mutations primarily in the liver, resulting in a reduction in both the size and number of cysts post-treatment. Kaminski noted potential improvements in the kidneys as well.
Specifically, Xiaogang Li’s team at Mayo Clinic conducted a similar study using more precise methods targeting the kidneys, indicating a reduction in cyst size and quantity, according to Li.
Both teams employed viral vectors to deliver gene-editing tools, which poses challenges with repeated doses due to immune responses might hinder treatment. “This is a legitimate concern,” Li notes. “However, we’ve observed limited immune responses in our animal models so far.”
Utilizing lipid nanoparticles instead of viral vectors, as seen in mRNA vaccines, could mitigate immune-related issues, but Kaminski warns that these particles may struggle to penetrate deeply into the kidneys through the bloodstream. “I believe that the delivery method using [lipid nanoparticles] might become more feasible through urinary pathways,” he says.
Another limitation is that base editing primarily addresses single-character mutations, rendering it ineffective for individuals with longer mutation sequences. However, Li reports successful outcomes using a technique known as Prime editing.
These findings are poised to be published shortly in scientific journals, with plans for human trials to follow. “After our publication, I aim to organize a small clinical trial,” he states.
The broader implications of this research suggest that if PKD can indeed be reversed, it could ignite more research into this potential therapeutic avenue. Currently, the only approved treatment is tolvaptan, which only slightly slows disease progression and requires significant fluid intake.
Dogs treated for fleas release chemicals into the water that kill insects when they swim
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Researchers advise against using spot-on flea and tick treatments if your dog has access to any water bodies.
Studies indicate that dogs submerged in water can release harmful levels of active ingredients into aquatic ecosystems, affecting wildlife and animals that consume them, including birds, for up to 28 days post-treatment.
“If your dog frequently swims, it’s best to avoid spot-on treatments,” says Rosemary Perkins from the University of Sussex, UK.
When spot-on treatments were introduced, it was mistakenly believed they posed no threat to the environment. It wasn’t until 2011 that a European Medicines Agency paper suggested protecting animals from water for 48 hours, yet Perkins notes that there was no substantial evidence backing this guideline. “It seems to be just a finger-in-the-air estimate,” she says.
Her concern grew after detecting fipronil, a pesticide used in these treatments, in a UK river. “We noted alarmingly high concentrations,” Perkins recalls.
Her team administered spot-on treatments containing either fipronil or neonicotinoids like imidacloprid to 25 and 24 dogs, respectively. After 5, 14, or 28 days, dogs were immersed up to their shoulders in a plastic tub for 5 minutes to measure pesticide levels in the water.
The findings revealed that even after 28 days, a single large dog’s chemical residue could exceed safe levels in 100 cubic meters of water—similar to the volume of a small pond. Regular swimming by treated dogs can further escalate contamination in larger bodies of water, warns Perkins.
She advocates for regulatory changes globally but fears that such adjustments may require significant time. In the meantime, dog owners should limit spot-on treatments to necessary occasions and keep their pets away from water for at least a month following treatment. “The key takeaway is that if your dog swims during this period, there are associated risks,” she emphasizes.
Currently, alternatives exist in the form of oral tablets, but Perkins remains uncertain of their environmental impact, noting that the active ingredients can persist in feces and contaminate soil. “We still lack clarity on their effects.”
Known as Verve-102, this treatment could revolutionize heart attack prevention and significantly lower LDL cholesterol levels (often referred to as “bad” cholesterol) with a single injection.
While statins can achieve similar cholesterol reductions, they typically require daily administration.
“This is the future,” stated Professor Riyaz Patel, an academic from the University of London and a doctor at Barts Health NHS Trust involved in the trial – BBC Science Focus.
“This is not a fantasy; it’s reality. We are actively implementing it. I was providing this treatment to my patient during the exam.”
Unlike statins, which gradually lower cholesterol, Verve-102 aims for a one-time alteration by “turning off” a specific gene called PCSK9 in the liver. This gene is crucial in managing the levels of LDL cholesterol that the liver can detect and eliminate from the bloodstream.
In simpler terms, a reduction in PCSK9 means less LDL in the bloodstream.
“The results are stunning,” Patel remarked. “This drug disables a small segment of your DNA, and your LDL cholesterol will be permanently 50% lower thereafter. That’s a game-changer!”
Cholesterol builds up in blood vessel walls, leading to plaque formation that can obstruct blood flow.
Elevated LDL cholesterol levels heighten the risk of this buildup, prompting millions (over 40 million in the US and over 7 million in the UK) to take daily medications like statins for cholesterol management.
The VERVE-102 clinical trial included 14 participants with familial hypercholesterolemia, a genetic disorder that heightens the risk of heart disease, heart attacks, and strokes due to extremely high LDL cholesterol levels.
Initial outcomes from Verve-102 injections show that all participants reacted positively to the treatment with no severe side effects.
Responses varied by dosage. The lowest dose group experienced an average LDL reduction of 21%, while the intermediate group showed a 41% reduction, and the high-dose group saw a 53% reduction.
Remarkably, one individual in the high-dose group achieved a 69% reduction in LDL cholesterol after receiving Verve-102.
Dr. Eugene Braunwald, a distinguished medical professor and Hershey’s professor of medicine at Harvard Medical School who did not take part in the study, noted that the preliminary data is “promising” and indicates “the potential for a new era in cardiovascular disease treatment.”
Verve is actively recruiting participants for further stages of clinical trials involving even higher Verve-102 doses in the UK, Canada, Israel, Australia, and New Zealand. The final results are expected to be revealed in the latter half of 2025.
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About our experts
Professor Riyaz Patel is a consultant cardiologist and clinical academic scholar at University College London (UCL) and Barts Health NHS Trust. He is a fully funded clinician scientist with the British Heart Foundation and serves as a professor of cardiology at UCL, where he investigates the causes of heart disease, focusing on cardiovascular risks and the genetics of coronary heart disease. He has established and led new cardiovascular prevention services at Barts Heart Center.
On Thursday, federal health authorities released a report stating there is no scientific basis for administering hormones or surgical interventions to young individuals experiencing gender dysphoria. This marks a significant shift from prior agency guidelines and the recommendations made by various US health organizations, highlighting concerns surrounding potential long-term harm.
The report emphasizes the importance of psychotherapy, which has sparked considerable debate, as some proponents equate it with conversion therapy aimed at altering gender identity.
Certain sections of the review appeared to challenge the very notion of a gender identity that diverges from one’s sex assigned at birth.
In January, President Trump issued an executive order titled “Protecting Children from Chemical and Surgical Amputations,” directing the Department of Health and Human Services to compile a report within 90 days on optimal treatment approaches for youth indicating a disconnect between their gender identity and their birth sex.
The executive order suggested that the administration had already drawn its conclusions about gender transition treatments for minors, denouncing the “blatant harm done to children” as a “stain on our country’s history.”
The report, spanning 400 pages, adopted a calmer tone yet arrived at a similar verdict. Notably, the report’s author remains unidentified as the post-publication review process is set to commence in “the coming days.”
Officials at HHS declined to comment on the review process, noting contributors included a diverse group of physicians, medical ethicists, and methodologists selected for their commitment to scientific integrity.
Importantly, the report clarified that it is not designed to establish standards for healthcare or formulate policy recommendations.
The findings were primarily based on analyses of scientific studies regarding adolescent blockers, hormone treatments, and surgical interventions conducted over the past few decades as these therapies became accessible to adolescents.
The assessment concluded that while the advantages of medical interventions remain unclear, potential harms, including erosion of birth rates, are deemed less substantial.
“Clinical practice in this area is exceptional and concerning,” the report asserts, pointing to American medical groups that advocate for an intolerant atmosphere where clinicians may feel compelled to self-censor.
The appropriateness of treatments such as adolescent blockers, hormone therapy, or surgery for young individuals remains a subject of vigorous debate.
Recently, several European nations have imposed restrictions on such treatments, with scientificreviews and discussions highlighting the insufficient quality of supporting evidence and uncertainties about long-term risks.
In the United States, 24 states have enacted legislation preventing doctors from offering such treatments to adolescents.
“This report misrepresents the prevailing healthcare consensus and fails to represent the realities of pediatric care,” stated Dr. Susan Cresley, chair of the Academy. “This document favors opinions over a rational examination of evidence.”
Advocates for transgender rights criticized the report for presenting ideological views disguised as scientific.
During Trump’s initial 100 days in office, his administration aimed to downplay transgender identities in public forums. The measures included cutting funding for hospitals that provide gender transition treatments to individuals under 19 and contemplating barring transgender individuals from military service.
The administration facilitated the transfer of transgender men and women from federal prisons to their homes and ceased recognizing the gender of transgender individuals on their passports.
“Is the administration’s animosity towards this healthcare grounded in genuine scientific insights or ideologically motivated by its disapproval of transgender individuals believing that transgender identity is fabricated?” questioned Shannon Minter, director of legal affairs at the National Center for Lesbian Rights.
The Center represents transgender plaintiffs in multiple lawsuits contesting the administration’s policies’ constitutionality.
“This is an ideological declaration, not a medical one,” stated Casey Pick, director of law and policy at the Trevor Project, an organization focused on suicide prevention among LGBTQ youth.
This report signifies a triumph for individuals who categorize this medical field within a broader agenda to deny the reality of biological gender.
Roger Severino, vice president of domestic policy at the Heritage Foundation, commended the HHS report while condemning “profit-driven physicians and ideological groups” for convincing families that “children’s sex aligns with everything they profess.”
According to government statistics, around 3% of high school students identify as transgender, a significant surge over the last decade. However, a much smaller percentage of these adolescents seek medical interventions.
Despite this, the topic of medical transition for minors has turned into a political battleground, with Trump making it a focal point of his campaign while some Democrats believe this strategy may aid his electoral prospects.
The new HHS report extends beyond similar assessments in Europe, which have initiated new limitations on gender-related treatments for adolescents.
Independent Clinical Services Reviews for UK youth reached a comparable conclusion, led by Dr. Hillary Cass, former president of the Royal College of Pediatrics. It noted the insufficient quality of evidence supporting the use of adolescent blockers and hormone treatments for minors, with surgery being unavailable to minors in the UK.
However, this review, conducted over four years, painted a broader picture of the medical landscape by consulting young patients, transgender adults, parents, and clinicians.
Dr. Kass concluded that evidence regarding the benefits and risks associated with treatment is “significantly weak,” but acknowledged that some young individuals are very likely to benefit from early interventions.
“Certain young people will undoubtedly reap the benefits of the medical pathway. As research evolves, we need to ensure that those individuals can access care under research protocols, but we mustn’t assume this is the right path for everyone,” Dr. Cass expressed in an interview last year.
This review concluded that the use of blockers in England should be restricted and that clinicians are encouraged to prescribe hormones to teenagers only with “extreme caution.”
Other clinicians who have expressed concern about the field of adolescent gender medicine are unclear about how the new report will be applied.
“We are pleased that in recent years US authorities are finally considering what is happening in Europe,” remarked Erica Anderson, a child psychologist and former president of the American Transgender Health Association.
She is outspoken about her concerns that adolescent gender clinics are shifting away from thorough mental health evaluations in light of the growing number of children seeking gender treatment.
However, Dr. Anderson supports the need for early intervention for certain young people, despite the inflammatory presidential order leading to the report.
“It’s akin to calling someone out on their rank and then expecting to engage in a meaningful conversation,” she stated. “This approach doesn’t work well with real individuals, who possess emotions and histories.”
Minter from the National Center for Lesbian Rights argued that by emphasizing psychotherapy over medical interventions, the HHS report effectively endorses conversion therapy aimed at altering minors’ gender identity or sexual orientation.
Variousmedicalassociations support prohibiting the practice, citing evidence that it leads to depression, anxiety, and feelings of self-loathing.
However, the Supreme Court has agreed to review a First Amendment challenge concerning Colorado’s conversion therapy regulations, initiated by a licensed professional counselor who contends that “individuals prosper when they align with God’s design, including their biological sex.”
Other therapists, including Dr. Anderson, advocate for what they term “exploratory therapy” which assists supportive clinicians in addressing mental health challenges related to adolescents’ gender identity.
Kellan Baker, a researcher focusing on transgender health policies at Whitman-Walker, a Washington-based nonprofit health center, remarked that the report signifies a divergence from customary health policy development in the US.
“It’s crucial that healthcare is administered by individuals with specialized knowledge, not dictated by the federal government, but by skilled clinicians operating according to the standards set by their respective healthcare fields,” Dr. Baker stated.
He voiced concerns that the report could be utilized by the government as a pretext for denying medical coverage for transgender youth.
The Centers for Medicare and Medicaid Services, a branch of HHS, issued a letter last month directing Medicaid agencies to refrain from using funds for gender transition care for minors.
West Texas doctors are seeing measles patients whose illness is complicated by alternative therapy approved by vaccine skeptics, including health secretary Robert F. Kennedy Jr.
Parents in Gaines County, Texas, are at the heart of the outbreak of turbulent measles, many of which have become increasingly repurposed and unproven treatments to protect children who have not been vaccinated against the virus.
One of those supplements is Vitamin A, which Kennedy advertises as a miraculous treatment for measles. Doctors at Covenant Children’s Hospital in Lubbock, Texas, say they have treated a small number of children who were given so much vitamin A, which has signs of liver damage.
Dr. Summer Davis, who cares for children with acute illnesses at the hospital, said some of them had been receiving unsafe supplements for weeks to prevent measles infection.
“We were sick for just a few days, five days, five days, and five days, but we had been taking it for about three weeks,” Dr. Davis said.
Doctors may manage severe measles by administering high doses of vitamin A in hospitals, but experts do not recommend taking it without supervision from a doctor. Vitamin A is not an effective way to prevent measles. However, two doses of measles, mumps and rubella vaccines are about 97% effective.
At high doses, vitamin A can cause liver damage. Dry skin peeled skin. Hair loss; and in rare cases, seizures and com sleep. So far, doctors at a hospital in West Texas have said they have seen patients with high yellow skin and liver enzymes in both blood tests for both liver signs.
Many of these patients were in hospital due to severe measles infection. The doctor only discovered liver damage after regular lab work.
As of Tuesday, the outbreak that began in January had spread to more than 320 cases in Texas. Forty patients were hospitalized and one child died.
Nearby New Mexico County, the virus has suffered 43 illnesses and two hospitalised. Seven confirmed cases in Oklahoma are also linked to outbreaks.
Local doctors and health officials are increasingly concerned about the growing popularity of unproven treatments to prevent and treat measles. They fear that people will delay serious treatment and refuse vaccination, the only proven way to prevent measles infection.
Alternative medicine has always been popular in Gaines County. Many of the large Mennonite communities in areas where most cases are clustered are avoiding interaction with the healthcare system and adhere to a long tradition of natural therapy.
Health officials said the popularity of Vitamin A’s recent use of measles could go back to a Fox News interview with Kennedy.
in Opinion essay In the Washington Post Tuesday afternoon, Kevin Griffith, who was the communications director for the Centers for Disease Control and Prevention until last week, wrote that he had resigned to handle Kennedy’s outbreak.
“In my last few weeks at the CDC, I saw a career infectious disease expert being tasked with spending valuable time wasting data searches to support Kennedy’s preferred treatment,” writes Griffith.
A few weeks after the interview with Fox News, Drugstore In West Texas, I had a hard time maintaining vitamin A and cod liver oil supplements on my shelf. “I didn’t hear anything about Vitamin A until he said that on TV,” said Katherine Wells, director of public health at Lubbock.
One local doctor, appointed as one of the doctors that Kennedy said in an interview with Fox News, opened a makeshift clinic in Gaines County, and began eliminating a variety of treatments, including vitamin A supplements, to treat active incorrect cases and prevent infection.
Dr. Davis said he suspected that the majority of the children she treated had taken vitamins at home.
Experts say Vitamin A can play an important role in the “advocacy care” provided by doctors to patients with severe measles infection.
Dr. William Schaffner, an infectious disease expert at Vanderbilt University Medical Center, works by replenishing physical reservoirs that have been depleted by viruses that strengthen the immune system.
In hospitals, doctors only give measles children two vitamins, usually over two days, and “adjusting very carefully” the amount according to their age and weight, he said.
Dr. Schaffner emphasized that it is not a miraculous treatment of the virus, and that measles does not have antiviral drugs. Also, there is no reliable evidence that vitamin A can help prevent infection in children in the US, with extremely rare vitamin A defects.
In fact, giving children high doses of repeated vitamins is dangerous. Unlike other vitamins that are washed away from the body via urine, excess vitamin A accumulates in adipose tissue and is more likely to reach dangerous levels over time.
“I think this type of preventative use is particularly concerning,” said Dr. Lara Johnson, another doctor at Lubbock Hospital.
“When you’ve been taking it on your kids for weeks or weeks, you can have a cumulative toxicity impact.,” she added.
Dr. Johnson added that local doctors don’t always accurately reflect the amount of vitamins the label contains and are particularly concerned about parents’ dependence on over-the-counter supplements that can accept dosage recommendations from unverified sources.
Viruses are infectious agents that affect all living things, including humans. A common human virus called herpes simplex virusor HSV, can cause cold sores, genital infections, fingertip and eye infections. When HSV infects the eye, it is called: HSV keratitis. This infection can cause visual impairment, blindness, and swelling of the brain.
The standard way for doctors to treat HSV infections is with an antiviral drug called . Acyclovir. However, the drug has been found to be no longer effective in treating some HSV infections. Persistent infections are called Acyclovir resistance infection. Therefore, scientists are searching for new ways to treat HSV infections.
Scientists at Nanjing University in China tested 502 natural compounds to determine if any could be used to treat HSV keratitis. The researchers took kidney cells from African green monkeys, treated them with these natural compounds, and infected them with the virus.
After two days, they examined the cells under a microscope to see how much damage each compound prevented compared to untreated infected cells. control sample. Out of all 502 compounds tested, the one that best protects cells from viral infection was found to be present in purple passionflower. The name of the compound is Harmol, And they reported that it inhibited more than 90% of HSV in kidney cells.
The scientists further investigated Harmol’s anti-HSV properties by testing whether it was toxic to host kidney cells. If Harmol is toxic to uninfected cells, it may kill the cells instead of protecting them from the virus. They tested nine concentrations of harmol on cells, ranging from 0 micromolar to 250 micromolar harmol. They found that the compound protected against viruses and did not kill host cells at a concentration of 12.5 micromolar.
They then studied how harmol affects virus replication and the production of viral proteins and particles within host cells. They found that Harmol inhibited HSV growth and reproduction, even in acyclovir-resistant infections. Scientists also reported that Harmol treatment inhibited HSV growth when added before, during, and after HSV infection. However, the sooner it is added, the more effective it is, and it is most effective when added before infection.
The scientists then tested whether Harmol worked in mice. They wanted to determine whether Harmol would be safe and effective for treating live animals with HSV keratitis, rather than cells alone. First, the scientists investigated whether Harmol solution could be safely applied to the eyes of mice. They applied Harmol to the eyes of one group of mice and saline to the eyes of a control group. They examined the mice’s body weight and cornea over a five-day period and found that Harmol had no effect on either compared to mice given saline. They suggested that this means Harmol is a safe treatment for mice with HSV keratitis.
Next, the scientists infected three groups of mice with HSV. They treated the first group with acyclovir, the second group with Harmol, but left the third group untreated. They administered Harmol eye drops daily for five days and observed the mice on the third and fifth day. They reported that Harmol-treated mice performed better than untreated mice in all the characteristics they observed. Harmol-treated mice fed HSV had less damage to the cornea, less eyelid inflammation, less weight loss, decreased corneal thickness, and central nervous density compared to untreated mice fed HSV. was high. They found that this was also true in mice infected with acyclovir-resistant HSV.
The researchers concluded that Harmol can reduce the early symptoms of HSV keratitis in mice. Therefore, they proposed that Harmol could be an antiviral agent against HSV. Because harmol and acyclovir interact differently with acyclovir-resistant HSV, scientists believe the two compounds must inhibit HSV in different ways. However, they noted that one limitation of the study was that the mice were only tested for five days after infection. They said further researchers should investigate the long-term safety and efficacy of Harmol treatment for HSV keratitis.
Measuring body fat more carefully may help treat obesity
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Rethinking how obesity is defined could help millions of people around the world, claims a team of researchers who want to introduce a new category of “preclinical” obesity.
The current definition of obesity as set by the World Health Organization (WHO) is having excess body fat that poses a risk to health. The WHO recommends that health professionals assess whether people are obese by calculating their body mass index (BMI), a measure of weight in relation to height. A BMI between 18.5 and 24.9 is considered healthy, while anything below or above that indicates being under or overweight. A BMI of more than 30 indicates obesity.
It’s true that having a lot of body fat can cause fat to infiltrate organs such as the liver and pancreas. impair function. It can also worsen inflammation and increase the risk of diseases such as cancer, liver disease, and heart disease.
However, BMI does not reflect a person’s body fat level very well. “BMI does not tell you whether that ‘excess’ weight is due to excess body fat or increased muscle and bone mass,” he says. Francesco Rubino He led research on obesity at King’s College, London.
Body fat levels, even when properly assessed by waist measurements or, in rare cases, X-ray scans, do not completely determine a person’s health status. “No two people react the same way to excess body fat. This is influenced by a person’s race/ethnicity, age, and the food they eat, with genetics playing a huge role.” says. stephen heimsfield at Louisiana State University.
That’s why Rubino and his colleagues want to introduce more nuance to the definition of obesity, separating cases into preclinical and clinical cases. Although both forms are characterized by excess body fat, only the clinical form is associated with symptoms caused by excess fat, such as difficulty breathing, heart problems, and difficulty with daily activities. Preclinical obesity, on the other hand, increases the risk of eventually developing such obesity-related symptoms, Rubino says.
This is similar to prediabetes, where blood sugar levels are higher than normal but not high enough to be diagnosed as full-blown type 2 diabetes, Rubino said.
Under the proposed changes, medical staff would use waist width and X-rays in addition to BMI calculations to directly measure people’s body fat levels, but people with a BMI over 40 would always be overweight. It will be considered fatty. Blood tests are then used to assess organ health and people are asked if they have symptoms. Blood tests are routinely done by many clinicians anyway, but directly measuring body fat would add some workload, Heimsfield says.
If the new definition is widely adopted by clinicians, it could mean people will receive more personalized advice and treatment, Rubino said. In general, people with pre-clinical obesity may only need to monitor their health and make lifestyle changes, while those with clinical obesity are more likely to need treatment with drugs or surgery, Rubino said. say.
“This allows us to better triage people and get them the right care,” he says. Adrian Brown At University College London.
Laura Gray Researchers at the University of Sheffield in the UK also welcomed the proposed changes. “It’s very necessary. These guidelines put what current research says into clinical practice,” she says. “Not all people who are obese according to their BMI are unhealthy, and not all people with a low BMI are healthy.”
This updated definition has already been endorsed by 76 health organizations around the world and may also help reduce the stigma surrounding the condition. “The hope is that by defining obesity in a more nuanced way, we will be able to show that it is a disease in itself. It is not just the result of behavior, but there are many risk factors, including environmental, psychological, and genetic. ” says Gray.
Wastewater treatment facilities are a major source of PFAS contamination in U.S. drinking water, estimated to contain enough “forever chemicals” to raise concentrations above safe levels for more than 15 million people. is being discharged. It also has the potential to release long-lasting prescription drugs into the water supply.
Although these plants purify wastewater, they do not destroy all the contaminants added upstream, and the remaining chemicals are released into the same waterways that provide drinking water. “This is a funnel into the environment,” he says bridger lyle at New York University. “We capture different things from different places and release them all in one place.”
Perfluoroalkyl and polyfluoroalkyl substances (PFAS) are of particular concern because they contain carbon-fluorine bonds and are highly persistent in the environment. Regular exposure to several types of PFAS is associated with an increased risk of many health problems, from liver damage to various forms of cancer. The U.S. Environmental Protection Agency (EPA) recently established strict drinking water limits for six of the most well-studied PFAS.
Wastewater treatment facilities are a known source of PFAS contamination worldwide. sewage sludge It is produced as a by-product and sometimes used as fertilizer. To determine whether similar contamination remained in treated water, Ruyle and his colleagues measured concentrations of PFAS and other molecules containing carbon-fluorine bonds in wastewater at eight large treatment facilities across the United States. .
Their findings suggest that wastewater treatment plants across the United States release tens of thousands of kilograms of fluorine-containing compounds into the environment each year, including significant amounts of PFAS. Once the treated wastewater leaves the facility, it mixes with natural water from rivers and lakes. “That would create downstream drinking water issues,” Lyle said.
Applying these numbers to a model of the U.S. drinking water system, the researchers estimated that wastewater could cause PFAS concentrations in the drinking water of approximately 15 million people to exceed EPA limits. In times of drought, as natural water for diluting wastewater decreases, models suggest that concentrations rise above the limit by up to 23 million people. And Ruyle says these may be conservative estimates. Their model assumes that natural water is already free of PFAS.
“This shows that wastewater treatment facilities are a very important source of these compounds,” he says. Carsten Plasse The professor at Johns Hopkins University in Maryland was not involved in the study. Although there are ways to remove or destroy PFAS in water, and more drinking water facilities are installing such systems, currently “our wastewater treatment plants are not set up to deal with this.” he says.
While chemicals alone will forever be a problem, researchers also found that PFAS only make up a small portion of the total amount of fluorinated chemicals emitted by facilities. Most were not PFAS, but other compounds used in common medications such as statins and SSRIs. These drugs are also a concern for ecosystems and people.
“Another person could be taking a cocktail of fluoridated prescription drugs,” Lyle says. But the effects of low doses and long-term exposure to such compounds are not well understood, he says.
“We need to start having a conversation about whether we should use more fluoride in medicines,” Ruiru says. Fluoridation is widely used in medicines to increase their effectiveness in the body, but “preventing widespread chemical contamination should also be important,” he says.
Drugs are rarely famous, and even more rarely superstars, but with his ability to grab headlines, Ozempic is the Taylor Swift of pharmaceuticals. So what exactly is behind that star power? Even as the drug and its derivatives become more widespread, researchers are racing to figure out how they work.
“We are seeing an incredible amount of benefits,” he says. Stephen Dayan at the University of Illinois. “It's early days, but these drugs look like they're going to change not only medicine, but the entire economy.”
Because most drugs treat only one or two symptoms, “cures” that promise to address all ailments are usually met with skepticism and suspicion. Ozempic seems to be bucking that trend. Wegovy, a version of Ozempic approved for weight loss last year. Reduce your risk of heart attack and stroke An increase of almost 20%. The emergence of “Ozempic pregnancy” suggests Fertility benefits. People started noticing its positive effects on depression and anxiety. In May, Results showed that it also reduced the risk of kidney failure Deaths in diabetic patients were also observed during the three-year trial. In July, another version of The drug was found to reduce brain atrophy Cognitive function declines slowly in Alzheimer's patients.
Wegovy and other weight loss drugs are widely promoted in the US
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Why this drug is effective against so many conditions remains a mystery, but researchers are beginning to unravel the mechanisms underlying its extraordinary abilities. Understanding everything from its impact on reward circuits in the brain to its impact on inflammation…
Stem cells are produced in the bone marrow and develop into different types of blood cells.
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Human blood stem cells have been grown in a laboratory for the first time, which could dramatically improve how certain types of cancer are treated.
The lab-grown cells have so far only been tested in mice, but when injected into the animals, they resulted in functional bone marrow similar to levels seen after umbilical cord blood cell transplants.
Treating cancers such as leukemia and lymphoma with radiation and chemotherapy can destroy blood-forming cells in the bone marrow. A stem cell transplant means new healthy bone marrow and blood cells can grow. The umbilical cord is a particularly rich source of stem cells, but there is a limited amount they can provide, and the transplant may be rejected by the body.
The new method allows researchers to create stem cells from actual patients, eliminating supply issues and reducing the risk that the patient's body will reject the stem cells.
First, they transformed human blood and skin cells into so-called pluripotent stem cells through a process called reprogramming. “This involves temporarily switching on four genes, so that the patient's cells revert to an earlier stage of development that can become any cell in the body,” he said. Andrew Elefanti At the Murdoch Children's Research Institute in Melbourne.
The second step is to turn the pluripotent cells into blood stem cells. “You start by making thousands of tiny, free-floating balls of cells, each containing a few hundred cells, and then you induce them to turn from stem cells to blood vessels to blood cells,” Elefanti says. This process, called differentiation, takes about two weeks and produces millions of blood cells, he says.
When these cells were then injected into mice that lack immune systems, they produced functional bone marrow in up to 50 percent of cases. That means they made the same cells that carry oxygen and fight infection as healthy human bone marrow, Elefanti says. “This unique ability to make all blood cell types over an extended period of time defines them as blood stem cells,” he says.
Abbas Shafi A researcher from the University of Queensland in Brisbane said the work was an “exciting step forward” towards new treatments for blood cancers. “It's never been done before and has great potential for the future.” But even once animal testing is complete, he said a lot of human research still needs to be done before the technique can be used in the clinic.
Simon Cohn Researchers at Flinders University in Adelaide, Australia, say a key advantage of their approach is that it can be scaled up to produce “an essentially limitless supply” of blood stem cells, but they add that the work is based on blood or skin cells, and success rates and blood cell diversity depend on the starting cell type.
“This suggests that treatments are inconsistent even at the preclinical stage in mice, and will need to be addressed before clinical trials in human patients,” he says.
Those who suffer from the throbbing pain of migraines know how challenging everyday tasks can become. However, a new medication, if taken at the first signs of a migraine, could potentially prevent the onset of debilitating symptoms.
The drug, ubrogepant, has recently been approved for use in the US and is available in the UK, although not covered by the NHS. A study funded by AbbVie, the manufacturer of ubrogepant, revealed that taking the pill at the first indication of a migraine can prevent severe headaches from developing.
Before a migraine attack, some individuals experience early warning signs like sensitivity to light and sound, fatigue, neck pain, stiffness, and dizziness. Identifying these symptoms can help in timely treatment with ubrogepant to inhibit the protein CGRP in the brain responsible for migraines.
The research conducted by Dr. Richard B. Lipton and his team involved 518 participants who were able to predict the onset of migraines within a few hours. Those who took ubrogepant reported being able to function normally two hours after ingestion, with fewer limitations on their activities even after 24 hours.
While promising, ubrogepant may not be effective for those who experience sudden migraine attacks without warning signs. Regardless, experts view this development as a positive step in migraine treatment, providing hope for those who suffer from this common but debilitating condition.
About our experts:
Dr. Steven Ross, a professor and vice chair of clinical affairs in the Department of Neurology at Pennsylvania State University College and Penn State Health, USA, has conducted extensive research in neurology, medicine, and pediatric emergency medicine.
Health workers assess a suspected case of MPOX in North Kivu province, Democratic Republic of Congo.
Arlette Basij/Bloomberg via Getty Images
Mpox, formerly known as monkeypox, is currently in the midst of an outbreak in the Democratic Republic of Congo (DRC) and neighboring countries, with the surge in cases being blamed in part on a new variant that is thought to be more deadly than the variant of the Mpox virus that caused a global outbreak in 2022. But there are treatments that may help.
How deadly is MPOX?
Studies have shown that the mortality rate among people infected with the currently circulating lineage I variant of MPOX is approximately 1-2%. 11 percentThe variation in reported mortality rates is probably due to differences in affected populations and problems with disease surveillance, Lilith Whittles At Imperial College London.
For example, infants and children with underdeveloped immune systems may be more likely than adults to develop serious, even fatal, infections, and people with suppressed immune systems, such as those with HIV, may also be more susceptible, she says.
Additionally, some areas have little access to health care and limited surveillance for MPOX. As a result, health care providers only catch the most severe cases and miss milder cases, making the death rate appear higher than it actually is. If MPOX symptoms are increasingly misdiagnosed as other illnesses, like measles or chickenpox, more cases will go undetected, Whittles says.
In fact, most deaths from MPOX occur due to complications such as sepsis, where infection enters the bloodstream and causes organ failure, and inflammation caused by the MPOX virus that damages the lungs, he said. Piero Oriaro At Oxford University.
What are the MPOX treatment options?
In the Democratic Republic of Congo and neighboring countries where the current outbreak is occurring, there are few treatments specific to MPOX. Instead, doctors focus on treating symptoms, which usually last two to four weeks, such as reducing fever and headaches with paracetamol (acetaminophen) and cleaning skin lesions to prevent bacterial infections, he said. Jean-Claude Udahemka At the University of Rwanda.
In the UK and US, doctors can use the antiviral drug Tecovirimat to treat people with severe smallpox. Originally developed to treat smallpox, its use against the disease was based on animal studies in which it improved survival rates compared to a placebo. Tecovirimat works by binding to a protein on the surface of both variola and smallpox that the virus uses to attack. Freeing itself from infected cells spreads to other cells.
Doctors in the United States and the United Kingdom can also treat MPOX with other antiviral drugs, such as brincidofovir and cidofovir. Protecting mice from lethal doses of the mpox virus. Both brincidofivir and cidofovir Interfering with enzymes Used by viruses to replicate their genome.
Another treatment, known as VIGIV, involves injecting people with smallpox with antibodies against smallpox taken from people who have had the smallpox vaccine, thus strengthening their immune response to the virus.
How effective is mpox treatment in humans?
Animal studies suggest that these treatments may be effective against MPOX, but their effectiveness in humans is unknown. Early results from a recent randomized controlled trial (best medical evidence) in the Democratic Republic of Congo suggest that tecovirimat does not accelerate the progression of MPOX. Healing of painful lesions in children and adults infected with lineage I variants of MPOX.
Nonetheless, the researchers found that the MPOX mortality rate for participants who received the antiviral drug was 1.7 percent, an improvement over the 3.6 percent mortality rate typically seen in the Democratic Republic of Congo. But this can be partly explained by the fact that participants in the trial received close care in hospital, Oriaro says.
Ultimately, better treatments and a better understanding of MPOX's lethality will be essential to protect people, especially in the Democratic Republic of Congo, from the ongoing epidemic. Lucille Blumberg She is a researcher at the University of Pretoria in South Africa. “There's a lot of work to be done,” she says.
ohRan Knowles, a British teenager with a severe form of epilepsy called Lennox-Gastaut syndrome, became the first person to try the new brain implant last October, with astonishing results: his daytime seizures reduced by 80 percent.
“The device has had a huge impact on my son's life as he no longer falls and injures himself like he used to,” said his mother, a consultant paediatric neurosurgeon at Great Ormond Street Hospital in London (Gosh), who implanted the device. She added that there has been a huge improvement in her son's quality of life as well as his cognitive abilities. He is more alert and outgoing.”
Oran's neurostimulator is implanted under the skull and sends constant electrical signals deep into the brain with the aim of blocking the abnormal impulses that cause seizures.The implant, called Picostim, is about the size of a cell phone battery, is charged through headphones and works differently during the day and at night.
“The device has the ability to record from the brain, to measure brain activity, and we can use that information to think about how to improve the effectiveness of the stimulation that children are receiving,” says Tisdall. “What we'd really like to do is to make this treatment available on the NHS.”
As part of the trial, three children with Lennox-Gastaut syndrome will be fitted with the implant in the coming weeks, with a full trial planned for 22 children early next year. If the trial is successful, academic sponsors Ghosh and University College London plan to apply for regulatory approval.
Tim Denison, a professor of engineering science at the University of Oxford and co-founder and chief engineer at Amber Therapeutics, a London-based company that developed the implant in collaboration with the university, hopes that the device will be available on the NHS and around the world within the next four to five years.
The technology is one of a number of neural implants being developed to treat a range of conditions, including brain tumors, chronic pain, rheumatoid arthritis, Parkinson's disease, incontinence and tinnitus. These devices are more sophisticated than traditional implants in that they not only decode the brain's electrical activity but also control it, and this is where Europe is racing against the US to develop life-changing technology.
The latest generation of brain implants can not only detect brain activity but also control it. Photo: UCL
Amber isn't the only company working on brain implants to treat epilepsy. California-based Neuropace has developed a device that responds to abnormal brain activity and has been cleared by US regulators for use by people aged 18 and over. But the battery is not rechargeable and must be surgically replaced after a few years. Other devices are implanted in the chest with wires running to the brain that must be reinserted as the child grows.
When most people think of brain chips, they think of Neuralink, another California-based startup from Elon Musk that just implanted a brain chip in a second patient with a spinal cord injury. The device uses tiny wires thinner than a human hair to capture signals from the brain and translate them into actions.
The first recipient, Noland Arbaugh, was in January and is paralyzed from the neck down. Some of the wires had shifted and the implant needed to be adjusted. The implant allows Arbaugh to control a mouse cursor on a computer screen with his mind, as if he were watching a movie. Star Wars A Jedi who “uses the Force.”
Other US companies, such as Syncron, backed by Bill Gates and Jeff Bezos, have also recently implanted brain-computer interfaces (BCIs) in people who cannot move or speak.
But scientists say these implants simply decode electrical signals. In contrast, a number of companies in the U.S., Britain and Europe, like Amber, are working on so-called “BCI therapy,” or modulating signals in deep brain stimulation to treat disease. Amber's implants are also being used in academic trials for Parkinson's disease, chronic pain and multiple system atrophy, a condition that gradually damages nerve cells in the brain. The company is also sponsoring an early trial in Belgium to treat incontinence, with promising results.
Professor Martin Tisdall led the team that gave Oran Noorsson, who suffers from severe epilepsy, the implant last October. Photo: UCL
A different kind of technology will be tested in humans in clinical trials starting in a few weeks, using the first brain implant made from graphene, a “miracle material” discovered 20 years ago at the University of Manchester.
Medical teams at Salford Royal Infirmary will implant a device with 64 graphene electrodes into the brains of patients with glioblastoma, a fast-growing form of brain cancer. The device will stimulate and read neural activity with high precision, to spare other parts of the brain while removing the cancer. The implant will be removed after surgery.
“We use this interface to map out where the glioblastoma is and then remove it. [cut it out] “Without affecting areas of function such as language or cognition,” says Carolina Aguilar, co-founder and CEO of InBrain Neuroelectronics, the Barcelona-based company that developed the implant in collaboration with the Catalan Institute of Nanoscience and Nanotechnology and the University of Manchester.
Traditionally, platinum and iridium have been used in implants, but graphene, made from carbon, is ultra-thin, harmless to human tissue, and can be decoded and modulated very selectively.
InBrain plans to conduct clinical trials of similar artificial intelligence-powered implants in people with speech disorders caused by Parkinson's disease, epilepsy and stroke.
Professor Costas Kostarellos, head of nanomedicine at the University of Manchester, co-founder of InBrain and principal investigator on the glioblastoma trial, says the company's goal is to “develop more intelligent implantable systems”.
Equipped with AI, the device, with 1,024 electrical contacts, “will help provide optimal treatment for each patient without the neurologist having to program all those contacts individually, as they do today,” he says.
InBrain has partnered with German pharmaceutical company Merck to use its graphene device to stimulate the vagus nerve, which controls many bodily functions including digestion, heart rate and breathing, to treat severe chronic inflammatory, metabolic and endocrine diseases such as rheumatoid arthritis.
Galvani Bioelectronics, founded in 2016 by the UK's second-largest pharmaceutical company GSK and Alphabet's Verily Life Sciences, has a pioneering treatment that treats rheumatoid arthritis by stimulating the splenic nerve. Galvani has begun clinical trials with patients in the UK, US and the Netherlands, with first results expected within the next 6-12 months.
Bioelectronics, which combines biological sciences and electrical engineering, is a market worth $8.7 billion today and is predicted to reach more than $20 billion (£15 billion) by 2031. According to Verified Market Research:The field focuses on the peripheral nervous system, which transmits signals from the brain to organs and from organs to the brain. When brain-focused neuromodulation and BCIs are added, Aguilar believes the overall market could be worth more than $25 billion.
While U.S. neuromodulation companies are making waves with devices targeting chronic pain and sleep apnea, a growing number of European startups are also working on the technology. MintNeuro, a spinout from Imperial College London, Working on developing next-generation chips The company is developing an implant that can be combined into a smaller implant and has partnered with Amber. With the support of an Innovate UK grant, its first project will be to develop an implant to treat mixed urinary incontinence.
Geneva-based Neurosoft has developed a device that uses a thin metal film attached to stretchy silicon – soft enough to put less pressure on the brain and blood vessels – to target severe tinnitus, which affects 120 million people worldwide.
“Tinnitus begins with ear damage, typically caused by loud noise, but it can also cause changes in the wiring of the brain, making it effectively a neurological disorder,” said Nicholas Batsikouras, the company's chief executive officer.
Founded in 2009 by 13 neurosurgeons, neurologists, engineers and other scientists from the Policlinico Research Center and the University of Milan, Neuronica has developed a rechargeable deep brain neurostimulator that can be used to treat Parkinson's disease. The device provides closed-loop stimulation and adapts moment-to-moment to the patient's condition, and is currently being tested on patients.
“Europe and the UK can compete head-to-head with the US when it comes to getting treatments onto the NHS and distributing them around the world,” Denison said. “It's a fair competition and we're going to give it our all.”
Green and gold bell frogs in an artificial hotspot shelter
Anthony Waddle
One of Australia’s most endangered amphibians can fight off a deadly fungal infection with the help of a naturally heated shelter that researchers are calling a “frog sauna.”
The disease, chytridiomycosis, has wiped out about 100 species of frogs, toads and salamanders worldwide.
Green and gold bell frog (Litoria aureaThe fungus was once widespread along the south-eastern coast of Australia, but its range has shrunk by 90 percent, and although other factors such as habitat loss are also at play, chytrid is thought to be the greatest threat to the endangered species.
It has long been known that warm temperatures suppress fungal infections, and many frog species, including the Japanese bush frog, are susceptible to the disease in winter when it’s hard for them to stay warm, especially when it’s hard to find a warm place.
To learn more, Anthony Waddle The researcher, from Macquarie University in Sydney, and his colleagues studied two groups of captive frogs that were intentionally infected with chytridiomycosis over the winter.
The first group was provided with bricks with holes in them in an unshaded greenhouse shelter where temperatures rose to nearly 40°C (104°F), while the second group was provided with bricks in a shaded greenhouse shelter where temperatures rose to 35°C (95°F).
Frogs that were given warmer shelter had 100 times fewer chytrid spores on their skin than other groups.
Although chytrid has difficulty growing above 28°C (82°F), warmer temperatures appear to activate the frogs’ immune systems, Waddle said.
“Using shelter to survive is like a vaccination for the frogs,” Waddle says, “and we’ve shown that firefly frogs can develop resistance after heat has cleared their infection, potentially making them 22 times more likely to survive future infections in cold environments.”
Although the researchers have only tested the shelter on one species at this stage, they believe the technology could be used with other animals threatened by chytrid fungus, as long as they seek out natural warmth when it’s cold. Waddle says there are at least six Australian animal species that could benefit from the technology.
Importantly, these thermal shelters are easy and inexpensive to set up: “All you need is a small vegetable greenhouse from the hardware store and a few bricks, and it will only cost about $60-70. [Australian] “It will cost a few hundred dollars to build,” Waddle said, “and I can envision people putting them in their backyards to help the frogs through the winter.”
Antibodies are proteins that can target and attack specific cells.
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An experimental treatment rejuvenates the immune systems of older mice and improves the animals’ ability to fight infections. If this treatment is effective in humans, it could reverse the age-related decline in immunity that makes older people more susceptible to illness.
These reductions may be due to changes in blood stem cells, which can develop into all types of blood cells, including important components of the immune system. As we age, a greater proportion of these stem cells tend to produce some immune cells than others. Jason Ross at Stanford University in California. This imbalance impairs the immune system’s ability to fight infection. It also promotes chronic inflammation, which accelerates aging and increases the risk of age-related diseases such as heart disease, cancer, and type 2 diabetes.
Ross and his colleagues have developed a treatment that uses antibodies, proteins that recognize and attack specific cells, to target these biased stem cells. Next, they tested the treatment on six mice aged 18 to 24 months. This is roughly equivalent to a human being between 56 and 70 years old.
One week after receiving the antibody injection, these abnormal stem cells in the mice had decreased by about 38 percent compared to six rodents of the same age who did not receive treatment. They also had significantly higher amounts of two types of white blood cells important for recognizing and fighting pathogens, and lower levels of inflammation.
“You can think of this as turning back the clock,” says Ross. “We are adjusting these percentages [immune] more similar cells [those of] A young adult mouse. ”
To test whether these changes result in a stronger immune system, the researchers vaccinated 17 older mice with a mouse virus. Nine of these mice had received antibody treatment eight weeks earlier. The researchers then infected rodents with the virus. After two weeks, the number of infected cells in the animals was measured and it was found that almost half of the treated mice (4 out of 9) had completely cleared the infection, compared to 1 out of 8 of the untreated mice. It turned out that there was only one.
Taken together, these findings demonstrate that antibody treatment rejuvenates the immune system of aged mice. Humans, like rodents, have more abnormal blood stem cells as they age, so a similar antibody treatment could also boost their immune systems, Ross said.
Such a possibility is still far away, robert signer at the University of California, San Diego. First, we need a better understanding of the potential side effects of treatments. In an accompanying article, Signer and his colleagues write: Yasar al-Fat KassResearchers, also at the University of California, San Diego, suggest that depletion of stem cells, even abnormal stem cells, may increase cancer risk. On the other hand, “if you have a better immune system, you’ll be better at investigating cancer, so we don’t know exactly what will happen yet,” Signer says.
Still, Ross says these findings are a promising advance in understanding age-related immune decline and how to reduce it.
Aging is the biggest risk factor for various diseases. “Rejuvenating or improving immune function in older adults could really help fight infectious diseases,” Signer says. “It may also have an impact on different types of chronic inflammatory diseases. That’s what’s so exciting here.”
Hemophilia B is a blood disorder that affects about 30,000 people in the United States. Individuals with hemophilia B have a deficiency in factor IX, a protein essential for proper blood clotting. This condition is hereditary, leading to the inability to clot blood effectively.. Treatment for hemophilia B involves injecting factor IX into the veins, but this method is costly and time-consuming. Therefore, scientists are exploring alternative treatments for hemophilia B.
A team of British researchers conducted a gene therapy trial using adenovirus FLT180a to increase factor IX levels in the livers of hemophilia B patients. They administered this gene therapy to a total of 10 male patients with severe or moderate hemophilia B, aged between 25 to 67 years. The patients were given different doses of the adenovirus and monitored for 26 weeks.
The results of the trial showed that some patients reached normal factor IX levels, while others experienced dangerously high levels. Patients who received lower doses had factor IX levels ranging from 40% to 60% of normal, while those who received higher doses had levels up to 300% of normal. The researchers noted varying responses in different patients and observed side effects like unexpected bleeding.
Despite the variability in patient response, the researchers believe that gene therapy could be a potential treatment for increasing factor IX levels in hemophilia B patients and improving blood clotting. They caution about the need to monitor and manage side effects effectively and suggest that this therapy could provide benefits for up to a year before requiring further intervention.
It is estimated that one in two people will develop cancer during their lifetime. However, advancements in diagnosis and treatment have led to more people surviving cancer than ever before. The question now is, will this trend of increasing survival rates continue, and how close are we to finding a cure?
The trend of improving survival rates is likely to continue, but the discovery of a cure for cancer is complicated due to the fact that cancer is not a single disease, but rather a group of over 200 diseases, each with its own unique characteristics. Despite this complexity, all cancers originate from mutant cells that divide uncontrollably.
While cancer cells evade normal controls on cell division, targeted cancer therapy has emerged as a promising treatment approach. This therapy focuses on inhibiting genetic mutations that drive cancer growth and has shown to be more effective with fewer side effects than traditional treatments like chemotherapy and radiation therapy.
Targeted therapies like hormone therapy and drugs such as imatinib have revolutionized the treatment of certain types of cancer, improving survival rates significantly. The development of new drugs and the repurposing of existing ones have been accelerated by genetic technologies that utilize big data to understand genetic changes driving cancer.
The power of big data
Advances in cancer treatment have been further propelled by genetic technologies and clinical trials that utilize big data to develop new drugs and repurpose existing ones. The Cancer Genome Atlas Project, for example, provides valuable genetic information for various types of cancer, allowing for targeted treatments based on individual genetic profiles.
Credit: Getty Images
While drug treatments have seen significant advancements, immunotherapy has also emerged as a promising approach in cancer treatment. Immunotherapy aims to boost the patient’s immune system to detect and destroy cancer cells more effectively. This field is rapidly evolving, with treatments like immune checkpoint inhibitors and adoptive cell therapy showing promising results.
Vax is on track
Developments in cancer immunization, including mRNA-based vaccines, are changing the landscape of cancer treatment by utilizing the body’s immune system to target cancer cells. Early diagnosis remains crucial in cancer treatment, with advancements in AI technology offering improved diagnostic capabilities.
Prevention is also a key focus in the fight against cancer, with vaccines against infectious causes of cancer such as HPV and HBV showing promising results. Additionally, cancer prevention strategies using drugs or vaccines to eliminate cancer cells before they form detectable tumors are gaining traction.
While a single “cure” for cancer may be unlikely, ongoing advancements in diagnosis, treatment, and prevention offer new hope to cancer patients worldwide. The future of cancer treatment holds the promise of personalized medicine, targeted therapies, and innovative approaches to combat this complex disease.
Crohn’s disease can cause abdominal pain, diarrhea, and weight loss
Jacob Wackerhausen/iStockphoto/Getty Images/www.peopleimages.com
A one-year study of 386 people found that receiving advanced treatment soon after diagnosis of Crohn’s disease improves outcomes for patients.
This disease is a lifelong inflammatory bowel disease; impact millions of peopleIn the world. Symptoms include abdominal pain, diarrhea, fatigue, and weight loss.
“These symptoms have a huge impact on people’s quality of life, education, relationships, and ability to work,” he says. Miles Parks at Cambridge University. “While there is no cure, there are ways to reduce some of these negative outcomes.”
Treatment often includes dietary changes, immunosuppressants, and steroids. In the UK, a drug called infliximab (an antibody that targets a specific protein in the body that is thought to contribute to intestinal inflammation) is given to people who regularly experience flare-ups of Crohn’s disease, or other mild symptoms. It can be prescribed to people who are not responding to. Treatment.
“This is a ‘step-up’ approach where treatment is progressively escalated in a reactive manner as the disease returns,” he says. Nurlaminnuralso at the University of Cambridge.
To see what happens if this more powerful treatment is used as early as possible, Parkes and Noor et al. studied 386 newly diagnosed Crohn’s disease patients aged 16 to 80 in the UK. Recruited people.
They were divided into two groups. One patient received infliximab immediately regardless of symptoms, and the other was treated with other Crohn’s disease drugs. If symptoms persist or continue to worsen, participants in the second group will also be prescribed infliximab, in line with a “step-up” approach.
After one year, 80 percent of patients who initially received infliximab had their symptoms under control over time, compared with only 15 percent of those who did not receive treatment immediately.
Additionally, only 0.5% of people in the group who received infliximab immediately required abdominal surgery for Crohn’s disease, compared to 4.5% in the second group.
The results of this study suggest that giving patients with Crohn’s disease intensive treatment as soon as they are diagnosed may be more effective in improving their lives, Dr. Noor said.
Parks said the extra money spent on medication would be balanced out by not having to pay for subsequent scans, colonoscopies and surgeries for people with repeated relapses.
“People with Crohn’s disease don’t want to be hospitalized or undergo surgery. They want to go out into the outside world and live their lives. Anything that speeds the path to remission. It can only be a good thing,” says Ruth Wakeman of the charity Crohn’s & Colitis UK.
Sperm that don't move fast enough have a hard time reaching the egg and can cause fertility problems.
Alexei Kotelnikov / Alamy
Laboratory research has revealed that applying ultrasound to immobile sperm causes it to move. If sperm does not move properly, it becomes difficult for them to reach the egg, which is a major cause of infertility. With further research, this technology could help improve the success rate of in vitro fertilization (IVF).
Previous research suggests that: High frequency ultrasound increases sperm motility. However, the study did not involve isolating the sperm to assess which individual cells would be beneficial, allowing doctors to find the best cells to use in fertility treatments.
In the latest research, Ali Vafaie The researchers, from Monash University in Melbourne, Australia, classified 50 semen samples into three groups (fast, slow, and stationary) according to sperm motility, based on guidelines for assessing swimming speed.
After separating individual sperm cells from semen samples, the researchers measured the motility of the cells before and after exposure to ultrasound waves with a power of 800 megawatts and a frequency of 40 megahertz.
After 20 seconds of ultrasound, 59 percent of the immobile sperm slowed down, and some started swimming rapidly. Changes in sperm motility peaked at an increase of 266%.
Overall, immotile sperm made up 36% of the samples at the start of the study, but this decreased to just 10% after treatment. It is unclear how long the increase in migration lasted.
Researchers believe that exposure to ultrasound improves dysfunction in sperm's mitochondria, the cells' powerhouses, contributing to increased motility.
This approach could increase the success rate of in vitro fertilization, which requires motile sperm for conception, and could avoid the need for multiple costly surgeries.
But first, Vafai says, the research group will need to test the effectiveness of the approach on sperm, particularly in people experiencing infertility due to reduced sperm mobility. Scientists also need to assess whether it is safe to create embryos from sperm exposed to ultrasound, he says.
Despite being a phenomenon known for hundreds of years, there is still much to learn about the placebo effect, which improves health after receiving dummy treatments like sugar pills. It is thought that behind this is the expectation of a positive outcome, and that negative expectations are responsible for the opposite undesirable phenomenon, the nocebo effect, which worsens symptoms. But questions remain about how the mind influences the body in this way and why some people feel its influence more strongly than others.
Luana Colloca are among those grappling with such questions. Colloka, a neuroscientist and director of the Placebo Beyond Opinion Center at the University of Maryland School of Nursing in Baltimore, and colleagues have shown how certain genetic variations shape the extent to which a person responds to a placebo. Ta. They are now studying how best to harness such effects to relieve pain, which could reduce the use of prescription opioid drugs and the risk of addiction to them. They are also exploring the use of virtual reality, with results published last year showing that virtual reality can effectively reduce levels of perceived pain and anxiety.
Colloca shares all of the latest discoveries about the placebo and nocebo effects in her book, which she co-edited. Placebo effect from a translational research perspective.she spoke new scientist About her research on pain relief, whether the placebo effect can help treat mental health conditions, and how it affects our lives…
A recent study led by Indiana University and the University of Chicago Medicine suggests that a drug called alpha-difluoromethylornithine (DFMO) could revolutionize the treatment of type 1 diabetes. Based on a decade of research, DFMO has demonstrated the potential to reduce insulin dependence, and larger clinical trials are currently underway to evaluate DFMO’s impact on beta cell preservation and disease amelioration. Tested.
A recent study led by Indiana University School of Medicine in collaboration with the University of Chicago School of Medicine shows exciting future possibilities for the management of type 1 diabetes and its potential reduction. insulin dependence. The researchers’ findings are cell report medicine, They suggest that repurposing the drug alpha-difluoromethylornithine (DFMO) could open the door to innovative treatments in the future.
Type 1 diabetes is a chronic disease in which the body’s immune system mistakenly attacks and destroys insulin-producing beta cells in the pancreas, resulting in high blood sugar levels and currently requires lifelong insulin treatment to keep patients alive. Is required. Many people living with type 1 diabetes find current treatments, such as daily insulin injections and frequent blood sugar monitoring, inconvenient and difficult to manage.
A 10-year research journey
These latest translation results represent more than a decade of research. Raghu Mirmila, M.D., Ph.D., co-corresponding author of the study, said in 2010 that while running his lab at IU School of Medicine, his team was able to develop beta It was discovered for the first time that cells can be protected from infection. Environmental factors suggest that type 1 diabetes may be preserved. The team then validated these findings in mice.
From 2015 to 2019, Linda DiMeglio, MD, MPH, Edwin Letzter Professor of Pediatrics at IU School of Medicine, Pediatric Endocrinologist and Division Director at Riley Children’s Health, provided guidance to people with newly diagnosed type 1 diabetes. He led a clinical trial to confirm the safety of DFMO. They also suggested that it may stabilize insulin levels by protecting beta cells. The trial was funded by the Juvenile Diabetes Research Foundation (JDRF) and used the drug provided by Panvera Therapeutics.
Dr. Emily K. Sims. Credit: Chapital Photography, provided by Emily K. Sims
“After several years of bench-to-bedside research, Drs. [Sarah] “We are pleased to finally share the promising results from the Tercy mouse model, a pilot study in humans,” said DiMeglio, senior author of the study. “Having established the preliminary safety of DFMO in patients with type 1 diabetes, we are excited to collaborate to further explore its potential benefits in a larger clinical trial.”
Regulatory benefits of DFMO and new formulations
Since 1990, DFMO has been approved by the FDA as a high-dose injectable to treat African sleeping sickness, and in 2020 received breakthrough therapy designation as a post-remission neuroblastoma maintenance therapy. Ta. This prior regulatory approval could streamline adoption as a treatment for type 1 diabetes and shorten the approval process from decades to just a few years.
“Using the new formulation of DFMO as a pill allows patients to take it by mouth rather than needing regular injections, and it has a very favorable side effect profile,” said Dr. said one Milmira. Chicago Medicine. “We are very happy to be able to say that we have developed a drug that works differently than other treatments for this disease.”
Current and future clinical research
Researchers have already begun the next steps to investigate the potential of DFMO. The study’s lead author and co-corresponding author, Emily K. Sims, M.D., associate professor of pediatrics at IU School of Medicine and pediatric endocrinologist at Riley Children’s Health, recently studied the effects of infectious diseases in more detail. To define it, we have begun a large-scale clinical study involving six institutions. His DFMO treatment to preserve beta cell function in type 1 diabetes. This new research was also funded by JDRF and supported by Panbela Therapeutics.
Sims, who is also a physician and scientist at the IU School of Medicine’s Herman B. Wells Pediatric Research Center and Center for Diabetes and Metabolic Diseases, hopes that DFMO, perhaps as part of a combination therapy, will not only help patients; There is. People who have recently been diagnosed with type 1 diabetes but are at risk of developing the condition may also be tested.
“As we embark on this new multicenter clinical trial to further investigate the efficacy of DFMO, we are confident that the encouraging results obtained to date will allow us to modify the underlying disease process of type 1 diabetes. ”Sims said. “We invite even more participants to this pioneering study. With their help, the knowledge we gain today has the potential to shape a brighter future for people affected by type 1 diabetes. Masu.”
Reference: “Inhibition of polyamine biosynthesis preserves beta cell function in type 1 diabetes” Emily K. Sims, Abhishek Kulkarni, Audrey Hull, Stephanie E. Werner, Suzanne Cabrera, Lucy D. Mastrandrea, Batur Hammoud, Soumyadeep Sarkar, Ernesto S. Nakayas, Teresa L. Mastracci, Susan M. Perkins, Ouyang Fangqian, Bobbie Jo Webb Robertson, Jacob R. Enriquez, Sarah A. Turcy, Carmela Evans. · Molina, S. Alice Long, Lori Blanchfield, Eugene W. Garner, Raghavendra G. Mirmila and Linda A. Dimeglio, November 1, 2023; cell report medicine. DOI: 10.1016/j.xcrm.2023.101261
A breakthrough assay to detect acute myeloid leukemia (AML) through a KMT2A gene fusion promises to enhance diagnosis and treatment and represents a major advance in leukemia research.
The researchers Accuracy
Detecting specific molecular markers within leukemia cells has the potential to significantly improve the assessment of measurable residual disease. This advancement will enable better-informed treatment decisions and ultimately improve patient outcomes.
A new assay that detects unique molecular markers in patients with acute myeloid leukemia (AML) could revolutionize how the disease is detected and treated, according to a recently published new report. Molecular Diagnostic Journal Published by Elsevier. This assay may improve the detection of AML due to factors such as: Kuomintang 2A Gene fusions can impact treatment decision-making, assessment of response to treatment, and long-term monitoring.
AML is a rare, aggressive blood cancer that is diagnosed in approximately 120,000 people worldwide each year. Detecting residual disease during treatment is essential to determine prognosis and guide treatment decisions.Currently, methods to detect measurable residual disease (MRD) during treatment of AML include bone marrow morphology, multiparameter flow cytometry (MPFC), and DNA Sequencing.
Morphological evaluation detects leukemic cells only with a detection limit of 5%. Although MPFC has a more sensitive detection limit of 0.01% to 0.001%, it is difficult to implement and interpret and is not standardized across laboratories. DNA sequencing approaches can identify leukemic cells by somatic mutation profiles, but are expensive and can be confounded by clonal hematopoiesis in nonleukemic blood cells.
Breakthrough progress in leukemia research
“We’ve seen a lot of research in this field,” explained lead researcher Dr. Grant A. Challen, of the Department of Oncology at Washington University School of Medicine in St. Louis. Normally absent in healthy cells. Other diseases such as chronic myeloid leukemia (CML) can already be tracked by standard BCR-ABL fusions, and sensitive detection of these fusions has revolutionized the way CML is treated. . For AML patients whose disease is caused by oncogenic fusions, the KMT2A fusion is a molecular marker that can be exploited for sensitive MRD detection. Therefore, we wanted to develop a platform for sensitive KMT2A fusion detection to improve detection and treatment methods for this disease. ”
Researchers have developed a new droplet digital PCR assay that allows for high sensitivity. Kuomintang 2A Fusion detection with the five most common fusion partners.At least 80 are known Kuomintang 2A There are fusion partners, but approximately 80% of fusions involve only 5 partners – AF9, AF6, AF4, Elleand English. They benchmarked the assay in human cell lines and patient samples and demonstrated sensitivity and specificity. Kuomintang 2A Fusion detection.
This assay detects these fusions by splitting cDNA molecules into microfluidic droplets and assaying them using primers and probes that generate a positive signal only when the fused transcript is present. Researchers were able to combine multiple primer/probe sets targeting different fusions into a pooled fusion detection reagent. they again, Kuomintang 2A Fusions in patient samples are known to be present Kuomintang 2A fusion.
Implications for AML treatment and future research
Dr. Challen said: This assay can be easily extended to include additional oncogenic fusions. This has potential implications for treatment decision-making and assessment of response to treatment. Knowing whether treatment is effective is critical to deciding when to escalate treatment or perform a hematopoietic stem cell transplant. ”
“This is a powerful new tool for highly sensitive KMT2A fusion detection and can be directly applied to disease detection in leukemia patients caused by these fusions. This fills a void in oncogenic fusion detection. , we offer several technical improvements. This assay is also highly scalable, and additional fusions can be easily added to the assay to expand coverage of other oncogenic fusions. We is improving blood cancer detection one drop at a time.”
Reference: “Droplet Digital PCR for Oncogenic KMT2A Fusion Detection” by Andrew L. Young, Hannah C. Davis, and Grant A. Challen, October 7, 2023. Molecular Diagnostic Journal. DOI: 10.1016/j.jmoldx.2023.09.006
This research was funded by: National Institutes of Health and the Leukemia and Lymphoma Society.
Stanford University engineers have created an injectable hydrogel depot technology that allows GLP-1 drugs to be administered once every four months, rather than requiring daily injections. This new hydrogel has the potential to revolutionize treatment for type 2 diabetes and weight management by significantly reducing the burden of daily injections.
The hydrogel drug delivery system was developed by materials engineers at Stanford University and turns daily or weekly injections of drugs like Ozempic, Maunjaro, Trulicity, and Victoza into a single injection every four months. This new system could greatly improve patient compliance and health outcomes for people with type 2 diabetes, as well as providing a more manageable treatment regimen.
The hydrogel contains GLP-1 drug molecules and slowly releases them over time, eliminating the need for frequent injections. This novel nanocomposite hydrogel is made of polymers and nanoparticles that dissolve over the course of several months, similar to how a sugar cube dissolves in water. Once the hydrogel is injected under the skin, it gradually releases the drug as it dissolves, providing sustained delivery over a four-month period.
Initial testing in laboratory rats has shown promising results, and future trials will be conducted on pigs to further validate the system’s effectiveness. The ultimate goal is to conduct human clinical trials within the next two years to evaluate the long-term administration of GLP-1-based treatments.
This research was supported by a grant from the National Institute of Diabetes and Digestive and Kidney Diseases and a seed grant from the Stanford Diabetes Research Center.
Texas A&M University researchers are co-leading a $20 million project to develop a $1 cancer treatment.
What if a single dose of $1 could cure cancer?
A multi-university research team is receiving federal funding to develop a highly efficient bacterial therapy that targets cancer more precisely and makes treatment safer at a cost of $1 per dose.
Traditionally, cancer treatments have had limited effectiveness in treating patients. Some treatments, such as radiation therapy and chemotherapy, can cause harmful side effects, while others tend to have poor patient response, not to mention the high cost of treatment.Survey results from American Cancer Society Cancer Action Network reports that 73% of cancer survivors and patients are concerned about how they will pay for their cancer treatment, and 51% say they have medical debt from their treatment. For example, cutting-edge cancer treatment can cost up to $1,000,000.
Texas A&M University and the University of Missouri are leading efforts to develop low-cost, safe and controlled cancer treatments. Researchers received a $20 million grant from the Advanced Research Projects Agency for Health (ARPA-H) to fight cancer. The four-year project is part of the current administration’s cancer moonshot plan to boost cancer research and increase funding. It is funded by a newly established agency that aims to accelerate improved health outcomes for all by supporting the development of highly effective solutions to society’s most challenging health problems. It was one of my first projects.
Rapid analysis of cells
$12 million of the grant will go to the Texas A&M Engineering Experiment Station/Texas A&M. Alam Han, Jim Song, and Chelsea Hu are developing programmable synthetic bacteria for immune-induced killing in the tumor environment (SPIKE). The idea is to engineer the bacteria so that the T cells kill the cancerous tissue, and once the cancer is gone, they destroy themselves and are safely excreted out of the body as human waste.
“SPIKE can specifically target tumor cells,” said Han, a professor in Texas Instruments’ Department of Electrical and Computer Engineering. “And because we only target the cancerous tissue and not the surrounding healthy cells, patient safety is dramatically increased. I’m excited to be part of this team tackling a critical health issue that affects so many people. I am very honored.”
Han’s lab is developing high-throughput microfluidic systems that can rapidly process and screen large bacterial therapeutic libraries one cell at a time to rapidly identify the most promising treatments. By fusing microfabrication techniques and biotechnology, these systems create picoliter-scale liquid handling systems that can accurately analyze single cells with high precision and speed, and devices that rapidly analyze individual cells. Realize.
“The big challenge is figuring out how to actually develop these sophisticated microdevices that can run millions of fully automated tests with very little manual or human intervention,” Han said. said. “That’s the engineering challenge.”
Rescue anti-tumor immune cells
While Han innovates and designs microdevices, Song, an immunologist with a background in microbial pathogenesis, T-cell biology, and T-cell-based immunotherapy, has spent the past five years working in bacterial immunotherapy. We are working on this.certain bacteria known as Brucella melitensis At least four types of cancer can be treated by manipulating the human body’s microenvironment and promoting T cell-mediated antitumor immunity.
“We are working on improving Brucella melitensis We can more effectively prevent or suppress tumor growth,” said Song, a professor at Texas A&M School of Medicine. “Our current approach involves finding ways to manipulate bacteria to rescue anti-tumor immune cells and make them more effective at killing tumor cells.
“According to the data so far, BrucellaThe efficiency is dramatically higher than other cancer treatments such as chimeric antigen receptor T-cell therapy and T-cell receptor therapy, with a response rate of over 70%,” said Song.
Safe and controllable treatment
While Professor Song continues to test the effectiveness of bacteria using cancer models, Professor Hu, an assistant professor in Artie McFerrin’s Department of Chemical Engineering and a synthetic biologist, has demonstrated that live bacterial treatments are safe and controllable. We are working to confirm.
” Brucella The strain we are using is attenuated and has been shown to be safe for the host as it lacks key genes required for bacterial virulence,” Hu said. Told. “Ultimately, we want to control the rate at which bacteria multiply within the tumor environment and their ability to self-destruct when their mission is completed.”
To control the rate of growth, the bacteria’s genes are modified to regulate its population, which fluctuates around a certain set point. Hu also plans to incorporate biosensors into the bacteria, allowing them to distinguish between healthy and tumor tissue, allowing them to grow only within the tumor microenvironment.
The bacteria are engineered with receptors that allow patients to take antibiotics after the cancer has gone away. This sends a signal to the bacteria to essentially shred itself and safely remove it from the patient’s body.
“We humans are actually covered in bacteria, and many diseases are caused by imbalances in these bacterial communities,” Hu said. “For example, some people have incredibly fragile stomachs, while others have strong stomachs. The science behind this is that people with strong immune and digestive systems have a healthy gut. It means that it has a population of bacterial cells. There are many possibilities for biological therapy.”
“It’s a really great opportunity to have a great team with the expertise and the ability to push this technology to the forefront,” Hu said. “So the goal is to go into the clinic and provide patients with effective cancer treatment for less than $1 per treatment.”
Tackling difficult problems with unconventional approaches
Other collaborators include Dr. Zhilei Chen of Texas A&M Health Science Center, Dr. Xiaoning Qian of the Department of Electrical and Computer Engineering, and Principal Investigator Dr. Paul de Figueiredo of the University of Missouri.
“The three important advantages of this study are high safety, low cost, and specific targeting of cancerous tumors,” Han said. “We are very excited to be one of the first teams to receive support from ARPA-H, a brand new agency supported by Congress. We take an unconventional approach to tackling difficult problems. High risk, high impact is the hallmark of our approach.”
And the future applications of bacterial engineering that this research unlocks are limitless.
“For our next big project, we will work together to develop bacteria that fight autoimmune diseases such as type 1 diabetes and rheumatoid arthritis,” Song said. Bacteria-based immunotherapy is an exciting frontier in medicine and offers the potential to revolutionize the treatment of autoimmune diseases. With the power of beneficial microorganisms harnessed to modulate the immune system, we are changing the future of medicine. Our research and expertise promises to transform the lives of millions of people, giving them new hope and a healthier tomorrow. ”
Researchers from Indiana University and the University of Notre Dame focused on reversing vascular remodeling through an epigenetic pathway involving the protein SPHK2, as reported in the journal Circulation Research. A potential treatment has been discovered. This innovative approach could transform the treatment of this serious lung disease.
Researchers at Indiana University School of Medicine’s South Bend Regional Campus and colleagues at the University of Notre Dame have discovered a new therapeutic target to treat pulmonary hypertension. This form of hypertension particularly affects blood vessels in the lungs. The team’s research and findings were recently published in the journal circulation research.
Pulmonary hypertension is a complex and often fatal condition in which the heart works harder than normal to pump blood to the lungs. The exact cause of pulmonary hypertension is unknown, but one of its hallmarks is thickening of pulmonary blood vessels caused by cell overgrowth, also known as vascular remodeling.
Discovery of new treatments
Margaret A. Schwartz, M.D., professor of pediatrics at IU School of Medicine and senior author of the study, said there are few treatments for pulmonary hypertension, and they usually treat the symptoms of vascular remodeling rather than the remodeling itself. Ta.
Dr. Schwartz said that an interesting finding of her team was the discovery of an epigenetic pathway through the protein SPHK2 that can reduce and potentially reverse vascular remodeling in pulmonary hypertension.
Dr. Dushani Ranasinghe (left) and Margaret Schwartz, MD, attend the University of Notre Dame Ranasinghe graduation ceremony.Credit: Provided by Margaret Schwartz
“This is one of the first reversible mechanisms of pulmonary hypertension identified,” she says. “Patients with pulmonary hypertension are usually given drugs to lower blood vessel pressure in the lungs or to help the heart contract to pump blood, both of which are symptoms of vascular remodeling. Our research focuses on targeting an epigenetic reversal of this mechanism. Ultimately, stopping the vascular remodeling process entirely may be the answer.”
Schwartz said the concept is similar to cancer treatment.
“In the case of cancer, we don’t just treat the symptoms, we stop the tumor from growing,” she says. “Vascular remodeling is a different mechanism, but the idea is that treatments target the mechanism rather than the symptoms.”
Main findings and future directions
Other key findings from the study include:
SPHK2 promotes the development of pulmonary hypertension through hyperacetylation of histone H3K9 and contributes to vascular remodeling in pulmonary artery smooth muscle cells (PASMCs).
SPHK2 deficiency results in decreased pulmonary vascular resistance, right ventricular hypertension, and thickened distal vessel walls.
EMAP (endothelial monocyte activation polypeptide) II plays an important role in stimulating the nuclear SPHK2/S1P epigenetic regulatory axis, suggesting cooperation between SPHK2 and S1P.
EMAPII may be a major driving force of epigenetic-mediated vascular PASMC reprogramming and remodeling in pulmonary hypertension.
Pulmonary endothelial cells are priming factors for the EMAPII/SPHK2/S1P axis that alters PASMC-specific acetylome through histone H3K9 hyperacetylation.
Schwartz and the study’s lead author, Dr. Dushani Ranasinghe, who was a member of Schwartz’s lab when Schwartz was a graduate student at Notre Dame, also thanked Dr. Schwartz for this episode. were interviewed about their findings. Podcast “Discover CircRes”It is produced by. circulation research.
Dr. Schwartz said the next steps in her research include collaborating with Brian Bragg, director of the Warren Center for Drug Discovery and Development at the University of Notre Dame, to further explore the SPHK2 protein as a therapeutic target for pulmonary hypertension. Stated.
Reference: “Changes in smooth muscle cell histone acetylome through the SPHK2/S1P axis promote pulmonary hypertension” A. Dushani CU Ranasinghe, Maggie Holohan, Kalyn M. Borger, Deborah L. Donahue, Rafael D. Kuc, Martin Gerig, Andrew Kim, Victoria A. Propris, Frances J. Castelino, Margaret A. Schwartz, September 12, 2023. circulation research. DOI: 10.1161/CIRCRESAHA.123.322740
Other IU authors on the study include Maggie Holohan and Martin Gerrig.
This research was made possible in part through funding from the following institutions: National Institutes of HealthLilly Endowment, O’Brien Family Excellence Fund, National Science Foundation, Buckner Family Scholarship.
Scientists have developed a promising treatment for sepsis, and clinical trials using sodium ascorbate, a vitamin C preparation, have shown effective results. The treatment has progressed into extensive clinical trials across Australia and demonstrated significant improvements in sepsis patients, including improved kidney function and reduced dependence on other drugs. This breakthrough, the result of decades of research, brings hope to a disease that is the leading cause of death in intensive care units around the world.
Flory Institute researchers, in collaboration with hospital intensivists, have demonstrated that sodium ascorbate, a pH-balanced formulation of vitamin C, is effective in treating sepsis.
Researchers at the Florey Institute have demonstrated that the formulation they have developed reduces deadly sepsis, and the next phase of clinical trials is set to begin across Australia next month.
Promising results from early clinical trial conducted at Melbourne’s Austin Hospital published in journal Critical carehave shown that sodium ascorbate, a pH-balanced formulation of vitamin C, is effective in treating sepsis.
Lead researcher Associate Professor Yugish Lankadeva said sepsis is notoriously difficult to treat and is often fatal.
LR Florey Professor Clive May, Austin Health Intensivist Professor Rinaldo Bellomo and Florey Associate Professor Yugish Rankadeva discovered that sodium ascorbate can be used to treat sepsis.Credit: Flory
Challenges in sepsis treatment
“Sepsis accounts for 35 to 50 percent of all hospital deaths. It is when the immune system is unable to fight the underlying infection, causing a life-threatening drop in blood pressure, multiple organ failure, and death. ,” said Associate Professor Lankadeva. In our clinical trial at Austin Hospital, sodium ascorbate was administered into patients’ bloodstreams, resulting in promising improvements in multiple organs. ”
Associate Professor Lankadeva, Florey’s research director for Systems Neuroscience, said of the next steps: $4.9 million government-funded research project Delivered in intensive care units in Adelaide, Melbourne, Perth, Brisbane, Alice Springs and Sydney.
“We will recruit 300 adult sepsis patients who will receive either our formulation or a placebo in addition to their usual hospital care. These results will provide additional data to determine the efficacy of the formulation. It will help in collection,” said Associate Professor Lankadeva.
Flory scientists have created a special formulation of sodium ascorbate to treat sepsis.Credit: Flory
Insights into previous trials
Professor Rinaldo Bellomo, director of intensive care research at Austin Hospital, said the first part of the trial at his department involved 30 adult sepsis patients between October 2020 and November 2022.
While in intensive care in the hospital, half of the patients were randomly assigned to receive sodium ascorbate, and the other half received a placebo.
This study found that patients with sepsis treated with sodium ascorbate:
Signs that more urine is produced and kidney function has improved
Less need for noradrenaline, a drug used clinically to restore blood pressure
He showed signs of improved function in multiple organs.
“Sepsis is the number one cause of death in intensive care units in Australia and around the world,” Professor Bellomo said. “In many cases, the disease progresses so rapidly that by the time patients reach us, they are already seriously ill. It will be a huge change.”
Decades of research bear fruit
Professor Clive May, Florey Senior Research Fellow on the project, has been researching how sepsis causes organ failure, particularly damage to the brain and kidneys, for more than 20 years.
“By showing decreased oxygen levels in the tissues of sepsis, we found that sodium ascorbate was a possible treatment.
“We have seen dramatic results in preclinical studies, where extremely high doses of sodium ascorbate caused complete recovery within just three hours with no side effects. It’s heartening to see that it’s paying off and bringing treatments into the hands of patients,” said Professor Clive May.
Surviving sepsis: The patient’s perspective
Longtime Flory staffer Brett Purcell serves as the consumer representative for the MEGASCORES research program, providing a valuable perspective from sepsis survivors.
“In 2011 I was taken to the hospital by ambulance with high fever and delirium. I was suffering from the early stages of sepsis. My condition gradually worsened and I was transferred to a larger hospital after 12 days. By that time My heart was severely infected and I was in septic shock. Six months ago I had a successful aortic valve replacement. Unfortunately the valve was infected.
“The surgical team repaired the damage in a six-hour operation, but my condition deteriorated to critical condition. I was told it would be an hour. It was the good decision-making of the surgical team and ICU intensivist that saved me. I was put on life support with an ECMO machine and dialysis, and my symptoms rapidly worsened. Improved.
“After almost eight weeks in the hospital, I’m home. I’m really lucky to be alive and hope this new research using sodium ascorbate is less invasive, faster, and extremely effective in fighting sepsis.” We hope to provide hospitals with a new and effective life-saving tool.”
Reference: “Ultra-dose sodium ascorbate: pilot, single-dose, physiological effects, double-blind, randomized, controlled trial” Fumitaka Yanase, Sofia Spano, Akinori Maeda, Anis Chaba, Thummaporn Naorungroj, Connie Pei Chen Ow , Yugeesh R. Rankadeva, Clive N. May, Ashenafi H. Betley, Darius JR Lane, Glenn M. Eastwood, Mark P. Plummer, Rinaldo Bellomo, October 12, 2023. Critical care. DOI: 10.1186/s13054-023-04644-x
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