CAR-T Cell Therapy: A Natural Approach to Revitalizing Aging Intestinal Health

The Importance of a Strong Intestinal Lining for Optimal Health

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As we age, the cells that line our intestines gradually lose their ability to regenerate, impacting our immune health. However, recent research has successfully reversed this decline in older mice using genetically modified immune cells.

This innovative approach, known as CAR T-cell therapy, is primarily used to target specific blood cancers. It entails collecting a patient’s T cells, reprogramming them in a laboratory to identify and eliminate cancerous cells, and then reintroducing them into the bloodstream. Emerging variations of this therapy show promise not only in combatting solid tumors but also in preventing arterial blockages and treating autoimmune diseases like lupus.

In a groundbreaking study, Cemil Beyaz and his team at Cold Spring Harbor Laboratory, New York, explored the application of this therapy for revitalizing the aging gut. They focused on targeting damaged cells, known as senescent cells, which accumulate over time, fail to proliferate, and secrete substances that exacerbate inflammation and promote further aging. The researchers aimed their intervention at a protein called uPAR, prevalent on aging cells.

“The decline in gut health we observe with aging is linked to diminished stem cell fitness responsible for renewing the intestinal lining every three to five days,” Beyaz states. “We posited that eliminating these ‘unfit’ senescent cells would enhance the regenerative ability and overall functionality of stem cells in older mice.”

To validate this hypothesis, the researchers engineered CAR T cells from older mice to specifically recognize and eliminate uPAR on senescent cells. Upon reintroducing these modified cells, the researchers noted a significant increase in both the activity and number of stem cells maintaining tissue function, reaching levels comparable to those observed in younger mice. The treated older mice also exhibited marked improvements in intestinal barrier integrity and reduced inflammation compared to a separate cohort that received CAR T-cell therapy targeting different mechanisms.

“By removing senescent cells, we not only inhibited the aging process but also witnessed a reversal, with tissues displaying characteristics similar to those of young mice,” said team member Corina Amor, also from Cold Spring Harbor Laboratory.

“This therapy could potentially reverse age-related declines in bowel function, decreasing vulnerability to diseases such as intestinal infections, compromised intestinal integrity, and even cancer,” explained Tuomas Tammera from Memorial Sloan Kettering Cancer Center, who was not part of the study. However, he emphasized that ensuring the treatment’s effectiveness and safety in humans remains crucial.

Onur Eskiokaku, a researcher at Cold Spring Harbor Laboratory, highlighted the importance of determining the optimal dosage before advancing to human trials. “While uPAR is abundant in aged, defective cells, it may also be present in healthy tissues under certain conditions,” he noted.

It’s important to remember that senescent cells aren’t all detrimental; they play roles in tumor suppression and wound healing. “We are investigating the implications of depleting uPAR expression in other tissues,” mentioned Jesse Poganic from Harvard Medical School.

Additionally, treating aging in otherwise healthy individuals is not commonplace. The complexity and expense associated with CAR-T therapy, coupled with ongoing safety concerns, suggest that widespread application for reversing aging effects is not imminent, according to Joanna Neves at the Center at King’s College London. “Safety standards for preventative treatments must be more stringent than those for oncology.”

Beyaz asserts that addressing the age-related decline in intestinal function has long posed a challenge, with no effective solutions currently available, especially when the intestinal barrier’s regenerative capability is compromised. This research marks a significant step forward, indicating that the removal of unfit cells can restore crucial functions.

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Source: www.newscientist.com

Breakthrough Gene Therapy for Huntington’s Disease Demonstrates Promising Results in 2025

Huntington’s Disease: A Gradual Destruction of Brain Cells.

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This year marks a watershed moment in addressing Huntington’s disease, a rare yet impactful form of dementia. Researchers have achieved a groundbreaking milestone with an experimental gene therapy that effectively slows the advancement of this debilitating condition for the first time ever. This represents a significant breakthrough in the search for viable treatments.

According to Sarah Tabrizi from University College London, “This is a monumental step forward,” referencing the successes observed in late-stage trials earlier this year. “We now understand that Huntington’s disease is potentially treatable, presenting us with a tremendous opportunity.”

Huntington’s disease stems from a genetic mutation that causes the normally benign huntingtin protein to cluster into harmful aggregates within the brain. This accumulation leads to gradual deterioration of brain cells, hindering movement, cognitive function, and emotional well-being. Currently, no approved treatments halt the progression of symptoms, and care primarily focuses on supporting affected individuals.

The novel treatment, dubbed AMT-130, directly targets these abnormal proteins by delivering genetic instructions to brain cells, instructing them to produce molecules that inhibit protein formation.

In a recent investigation, Tabrizi and her team administered high doses of AMT-130 to 17 patients with Huntington’s disease. They evaluated changes in cognitive, motor, and daily functioning over a three-year period compared to a control group. Preliminary results released by the drug’s manufacturer, biotechnology firm uniQure, indicate a remarkable 75% average slowdown in symptom progression.

“Huntington’s disease treatment has faced numerous challenges in recent years,” stated Sarah O’Shea at Mount Sinai in New York, who was not involved in the study. “This accomplishment is significant, not just in delaying the disease’s progression but also because it arrives at a crucial time when hope is desperately needed.”

Nevertheless, this innovative treatment does come with its own set of challenges. Tabrizi explained that the 12- to 18-hour surgical procedure required to deliver the treatment deep within the brain is only accessible in a limited number of facilities in select countries, including the U.S. and U.K. Additionally, if approved, the treatment is likely to be prohibitively expensive. “Will it be accessible to all? Achieving that will be a challenge,” she remarked.

To navigate these hurdles, Tabrizi and her team are developing an alternative therapy that involves injecting the fluid around the spinal cord. “We’re currently conducting a Phase I study, and the first patient was treated in November 2024,” Tabrizi noted, with safety results expected by July 2026.

Meanwhile, uniQure’s executives revealed in September plans to submit AMT-130 for FDA approval in early 2026. However, the submission timeline is now uncertain, as the FDA has expressed concerns regarding the study plan and the control group, which consists of *non-treatment* patients drawn from a database.

The absence of a proper control group complicates the ability to discern the placebo effect on results. Still, given the surgical nature of the treatment, establishing such a group poses significant challenges.

“We firmly believe that AMT-130 holds substantial potential to benefit patients and are dedicated to collaborating with the FDA to expedite access to this treatment for families in need across the U.S.,” stated Matt Kapusta, CEO of uniQure.

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Source: www.newscientist.com

Cats Can Conquer Their Fear of Water and Thrive with Aquatic Therapy

Most cats aren’t fond of water

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Innovative training protocols now enable even injured or disabled cats to experience underwater treadmill therapy and pools without fear.

This adaptive approach lowers stress levels, ensuring cats feel secure and can reap the benefits of the same water-based rehabilitation that aids dogs, horses, and humans. Stefania Uccedu from San Marco Veterinary Clinic and Research Institute in Padua, Italy.

“This is quite unexpected, as it boils down to habituation,” she explains. “Cats, like humans seeing the beach for the first time, have no concept of water. However, once they acclimate to the therapy setting, water becomes less of a concern.”

Underwater treadmills and pool therapies allow both animals and humans to exercise with less strain, making them excellent for rehabilitation and strength training for orthopedic issues like joint and tendon injuries, neurological problems, and senior care.

However, due to their evolution in arid environments, cats often feel extreme stress when encountering water or unfamiliar spaces, leading many owners and therapists to skip aquatic therapy altogether. The rare rehabilitation centers that include cats in water programs often adapt methods originally designed for dogs, according to Uccedu.

To address this, she and her team created a cat-specific protocol. Initially, the cat explores the room to dry off the equipment, then feels a damp towel on its paws. Next, the cat stands in a warm 5-centimeter deep water, familiarizes itself with the treadmill’s sound, and gradually is introduced to deeper water, always with the owner close by. Rewards such as food, petting, and toys are given based on the cats’ preferences.

“Notably, younger cats seem distracted by moving objects, allowing them to forget about the motion itself,” Uccedu notes.

Cat engaging in aquatic therapy protocol

San Marco Clinic Veterinary Research Institute

The team tested the protocol with 12 cats of various ages, breeds, and conditions drawn from the clinic’s feline patients with neurological and orthopedic issues.

During the program, each cat was observed for specific stress behaviors such as excessive meowing, licking their nose, and signs of fear. If a cat displayed these behaviors five or more times within a minute, the session was halted.

Uccedu reported that all 12 cats successfully completed rehabilitation programs of varying lengths, from a few weeks to a year, depending on their specific conditions. The cats exhibited remarkable physical progress, with some fully recovering and even climbing trees once again.

Encouraged by the favorable outcomes, the team began applying the protocol in swimming pools, integrating different lighting and music to create a more soothing environment. “Ultimately, the choice is up to the cat,” Uccedu emphasizes. “Some may prefer classical music, while others might enjoy Madonna.”

She stresses that these findings show that cats should not be dismissed from aquatic therapy based solely on their supposed aversion to water. “The encouraging news is that any clinic can apply this protocol and achieve similar outcomes.”

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Source: www.newscientist.com

Doctors Explore Estrogen Therapy as a Preventive Measure for Women’s Dementia

For many years, healthcare professionals have been intrigued by the fact that women are diagnosed with Alzheimer’s disease at nearly double the rate of men.

According to estimates, approximately seven million individuals in the U.S. suffer from Alzheimer’s disease, and this number is projected to rise to nearly 13 million by 2050. Notably, around two-thirds of these cases involve women.

Emerging research indicates that estrogen, the principal female hormone, may have a significant role, particularly during the transition from perimenopause to menopause when natural hormonal levels begin to decline.

Estrogen serves various functions in the body, including enhancing cardiovascular health and sustaining bone density. Moreover, it is crucial for brain health, exhibiting neuroprotective qualities that shield brain cells from inflammation, stress, and various forms of cellular damage.

Researchers focusing on Alzheimer’s disease are turning their attention to early perimenopause, which typically occurs in a woman’s early to mid-40s, as a key period for hormone replacement therapy aimed at sustaining estrogen levels and potentially preventing dementia in certain women decades later.

“This interest stems from many years of preclinical research, animal studies, and fundamental science showing that menopause represents a critical juncture in Alzheimer’s disease,” remarked Lisa Mosconi, head of the Alzheimer’s Disease Prevention Program at Weill Cornell Medicine.

Mosconi leads a new $50 million global initiative named CARE, aimed at minimizing women’s Alzheimer’s disease risk through endocrinology research. This venture will examine biomarkers in around 100 million women, promising to be the most extensive analysis of why women face a heightened risk of Alzheimer’s disease.

The relationship between estrogen and dementia has recently attracted renewed interest following the Food and Drug Administration’s decision to lift a long-standing black box warning on hormone replacement therapy, potentially encouraging more prescriptions for women in their 40s and 50s.

Healthcare providers believe that relaxing these regulations could help destigmatize hormone therapy. The FDA’s action may also facilitate further research into whether hormone replacement therapy offers additional advantages, such as dementia prevention.

Reduction of Reproductive Hormones

Menopause is defined by a gradual decline in the production of estrogen and progesterone by the ovaries, which are essential for regulating the menstrual cycle. These sex hormones are present in women and, to a lesser extent, in men, and they play vital roles in sexual and reproductive development.

Most women experience menopause between the ages of 45 and 55, according to Dr. Monica Christmas, a gynecologist and director of the Menopause Program at the University of Chicago Medicine. The transition may commence years earlier, during perimenopause, which usually starts in a woman’s mid-40s, often accompanied by symptoms such as hot flashes, night sweats, mood swings, and sleep disruptions.

It is believed that menopausal symptoms arise from the reduced levels of estrogen and progesterone. For instance, when estrogen levels drop, the thermostat in the body, governed by the hypothalamus, fails to work correctly. The brain senses an increase in body temperature and signals sweating to cool down, leading to hot flash experiences. Hormone therapy can restore these levels, helping to regulate body temperature.

What Role Does Estrogen Play?

Rachel Buckley, an associate professor of neurology at Massachusetts General Hospital, whose research investigates gender disparities in Alzheimer’s disease, notes that receptors for this sex hormone are distributed throughout the brain.

“Estrogen is an extremely potent hormone,” she said. “It resides in a region called the hippocampus,” which is closely linked to memory and learning.

Estrogen also facilitates healthy blood flow in the brain, allowing for more efficient energy utilization, she mentioned. However, during menopause, estrogen levels begin to decrease, potentially rendering the brain more vulnerable to damage.

“When the brain loses the protective benefits of estrogen and other sex hormones, this marks a critical phase where Alzheimer’s disease can begin to accumulate in the brain,” Mosconi explains.

Can Hormone Replacement Therapy Combat Dementia?

Hormone replacement therapy is available in numerous formats, including patches, creams, and tablets, which may contain estrogen, progesterone, or both. If estrogen aids in safeguarding the brain, it stands to reason that adjusting estrogen levels through hormone therapy could offer some advantages.

Nevertheless, experts indicate that the reality is more complex, as the evidence surrounding hormone replacement therapy remains varied and ongoing.

Dr. Kellyanne Niotis, a preventive neurologist in Florida and a faculty member at Weill Cornell Medical College, noted that research suggests the perimenopausal transition is a crucial window for treatments that may help some patients prevent dementia.

“The central idea is that during the perimenopause phase, hormones fluctuate significantly, leading to rapid drops in [estrogen] which can be detrimental to brain health,” Niotis stated.

“The goal is to maintain consistent hormone levels to ease those fluctuations.”

A comprehensive analysis led by Mosconi and her team is set to be published in Frontiers in Aging Neuroscience in 2023, indicating there might be an optimal moment to commence HRT for women facing cognitive decline.

Her research evaluated over 50 studies and found that individuals undergoing estrogen therapy in midlife, within ten years following their last menstrual period, experienced a notably reduced risk of dementia.

Conversely, initiating combination hormone therapy after age 65 correlated with an increased risk of dementia.

Another large-scale review of 50 studies presented recently at the American Academy of Neurology Annual Meeting revealed that women who began HRT within five years of menopause had up to a 32% lower risk of Alzheimer’s disease compared to those receiving a placebo or no treatment. This study has yet to undergo peer review or publication in a scientific journal.

This investigation, conducted by researchers in India, also indicated that women who delayed treatment until 65 or older exhibited a 38% increased risk of Alzheimer’s disease.

However, much of the existing research is observational and does not establish a direct cause-and-effect relationship, according to Christmas. More in-depth studies, including large clinical trials, are necessary, she emphasized.

It should also be noted that prescribed hormone therapy may not function identically to the naturally produced estrogen, necessitating further investigation, she added.

Why Timing of Hormone Therapy Matters

The notion that there is a critical period for initiating hormone replacement therapy is possibly linked to estrogen receptors in the brain, according to Mosconi. Her research indicates that during the transition to menopause, the density of estrogen receptors on brain cells gradually increases, a finding supported by her studies.

This increase occurs as the brain attempts to compensate for declining estrogen levels by boosting available receptors to utilize any remaining estrogen effectively, she explained.

However, there comes a point when estrogen levels fall permanently, leading the brain to stop trying and the estrogen receptors disappear, she added.

“Once the estrogen receptors are absent, administering estrogen becomes futile as there would be nothing to bind to; that’s when the window closes,” stated Mosconi.

Numerous questions remain unanswered, such as how long women should stay on hormone replacement therapy and whether estrogen provides more protection for those with a genetic susceptibility to Alzheimer’s disease. It remains unclear how the brain responds to natural estrogen versus that received through hormone replacement therapy.

Conversely, men possess biologically different brains with significantly fewer estrogen receptors, which diminishes their need for the hormone, according to Buckley.

It is also uncertain whether testosterone replacement therapy in men might have benefits in Alzheimer’s disease prevention, as Dr. Niotis pointed out. While some research suggests a correlation between low testosterone in men and dementia, further studies are necessary before definitive assertions can be made.

Experts caution that it’s premature to advocate for hormone replacement therapy as a preventive measure for Alzheimer’s disease.

“We currently do not utilize hormone therapy for Alzheimer’s disease prevention,” remarked Mosconi. “Current clinical guidelines do not endorse hormone therapy solely for this purpose.”

Instead, HRT should be primarily prescribed to alleviate moderate to severe menopausal symptoms that impact quality of life, such as hot flashes, night sweats, sleep disturbances, and mood changes.

According to Niotis, individuals with good sleep quality tend to feel better and think more clearly, suggesting that alleviating these symptoms could enhance cognitive function.

Nonetheless, she remains hopeful that future research will yield more conclusive insights.

“The aspiration is that with the removal of the black box warning, more women will opt for treatment without reservations, and physicians will feel more confident prescribing it,” Niotis expressed.

Source: www.nbcnews.com

Teenage Boys Turn to ‘Personalized’ AI for Therapy and Relationship Guidance, Study Reveals | Artificial Intelligence (AI)

A recent study reveals that the “highly personalized” characteristics of AI bots have prompted teenage boys to seek them out for therapy, companionship, and relationships.

A survey conducted by Male Allies UK among secondary school boys shows increasing concern regarding the emergence of AI therapists and companions, with over a third expressing they might entertain the notion of an AI friend.

The research highlights resources like character.ai. The well-known AI chatbot startup recently decided to impose a permanent ban on teenagers engaging in free-form dialogues with its AI chatbots, which are used by millions for discussions about love, therapy, and various topics.

Lee Chambers, founder and CEO of Male Allies UK, commented:

“Young people utilize it as a pocket assistant, a therapist during tough times, a companion seeking validation, and occasionally even in a romantic context. They feel that ‘this understands me, but my parents don’t.’

The study, involving boys from 37 secondary schools across England, Scotland, and Wales, found that over half (53%) of the teenage respondents perceive the online world as more challenging compared to real life.


According to the Voice of the Boys report: “Even where protective measures are supposed to exist, there is strong evidence that chatbots often misrepresent themselves as licensed therapists or real people, with only a minor disclaimer at the end stating that AI chatbots aren’t real.”

“This can easily be overlooked or forgotten by children who are fully engaged with what they perceive to be credible professionals or genuine romantic interests.”

Some boys reported staying up late to converse with AI bots, with others observing their friends’ personalities drastically shift due to immersion in the AI realm.

“The AI companion tailors its responses to you based on your inputs. It replies immediately, something a real human may not always be able to do. Thus, the AI companion heavily validates your feelings because it aims to maintain its connection,” Chambers noted.

Character.ai’s decision follows a series of controversies regarding the California-based company, including a case involving a 14-year-old boy in Florida who tragically took his life after becoming addicted to an AI-powered chatbot, with claims that it influenced him towards self-harm; a lawsuit is currently pending from the boy’s family against the chatbot.

Users are able to shape the chatbot’s personality to reflect traits ranging from cheerful to depressed, which will be mirrored in its replies. The ban is set to take effect by November 25th.

Character.ai stated that the company has implemented “extraordinary measures” due to the “evolving nature of AI and teenagers,” amid increasing pressure from regulators regarding how unrestricted AI chat can affect youths, despite having robust content moderation in place.

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Andy Burrows, CEO of the Molly Rose Foundation, established in the memory of Molly Russell, who tragically ended her life at 14 after struggling on social media, praised this initiative.

“Character.ai should not have made its products accessible to children until they were confirmed to be safe and appropriate. Once again, ongoing pressure from media and politicians has pushed tech companies to act responsibly.”

Men’s Allies UK has voiced concerns about the proliferation of chatbots branding themselves with terms like ‘therapy’ or ‘therapist.’ One of the most popular chatbots on Character.ai, known as Psychologist, received 78 million messages within just a year of its launch.

The organization is also worried about the emergence of AI “girlfriends,” which allow users to customize aspects such as their partners’ appearance and behavior.

“When boys predominantly interact with girls through chatbots that cannot refuse or disengage, they miss out on essential lessons in healthy communication and real-world interactions,” the report stated.

“Given the limited physical opportunities for socialization, AI peers could have a significantly negative influence on boys’ social skills, interpersonal development, and their understanding of personal boundaries.”

In the UK, charities Mind is accessible at 0300 123 3393. Childline offers support at 0800 1111. If you are in the US, please call or text Mental Health America at 988 or chat at 988lifeline.org. In Australia, assistance is available through: Beyond Blue at 1300 22 4636, Lifeline at 13 11 14 and Men’s Line at 1300 789 978.

Source: www.theguardian.com

Stem Cell Therapy Lowers Heart Failure Risk Following Heart Attack

Mesenchymal stem cells tagged with fluorescent markers

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The largest trial conducted so far indicated that individuals who received a stem cell infusion right after a heart attack had a lower risk of developing heart failure compared to those who underwent standard treatment. This finding offers compelling evidence that stem cells may aid in the heart’s self-repair mechanisms.

Following a heart attack, the heart muscle sustains permanent damage and weakness, rendering the organ unable to adequately pump blood to meet the body’s needs, frequently resulting in heart failure. Presently, the only available treatments are heart transplants or pumps that can restore heart function.

To explore solutions, researchers turned to stem cells, which possess the remarkable capability to transform into various cell types. Nonetheless, prior studies evaluating their application post-heart attack have yielded inconsistent results. A 2020 study with 375 participants showed that bone marrow-derived cells, which include stem cells that evolve into blood cells, did not notably decrease mortality compared to standard therapies such as cardiac rehabilitation and medications aimed at lowering blood pressure, preventing blood clots, and managing cholesterol levels.

Armin Attar and colleagues from Shiraz University in Iran explored a different strategy. They employed mesenchymal stem cells capable of differentiating into structural cells such as cartilage and adipose tissue. These stem cells are also known to release substances that mitigate inflammation and promote tissue regeneration.

The researchers collected mesenchymal stem cells from umbilical cord blood and administered them into the hearts of 136 patients within three to seven days following their first heart attack. While these stem cells can also be extracted from the individual’s own adipose or bone tissue, the cultivation process can take up to a month. By utilizing umbilical cord blood samples, the team can provide treatments more swiftly and potentially enhance their efficacy, Attar explained. Another group of 260 participants received standard post-heart attack treatments.

After three years, individuals who received the stem cell therapy were, on average, 57% less likely to develop heart failure and 78% less likely to be hospitalized for heart failure compared to those receiving standard treatment. Furthermore, they exhibited significant improvements in cardiac function, suggesting that the therapy aids in the regeneration of heart tissue post-injury.

“This represents a significant advancement,” Attar remarked. Although the therapy did not lower mortality risk during the study period, the reduction in hospitalizations is still significant, according to Hina Chowdhury from the Icahn School of Medicine at Mount Sinai in New York. “Heart failure is the predominant cause of hospital admissions in the U.S.,” she pointed out.

However, it should be noted that 80% of participants were male, leading Chaudhry to caution that it is uncertain how the therapy would affect women, who are more prone to heart failure post-heart attack. Attar’s team did not observe any gender differences in a separate analysis. This study was also restricted to young adults, with all participants aged between 18 and 65. “It’s beneficial to see distinctions among age groups, as younger individuals typically have enhanced regenerative abilities and recover better from cardiac injuries,” Chaudhry noted.

This research provides the most robust evidence to date that stem cells can aid in restoring heart function post-heart attack. Nevertheless, this treatment does not fully heal the heart. “There is no medication or treatment that can replace lost heart muscle cells,” Chaudhry emphasized. Still, she added, “This ongoing work is expanding our understanding of the heart’s regeneration process and how to achieve it.”

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Source: www.newscientist.com

‘Enhanced’ CAR T-Cell Therapy Shows Potential in Treating Solid Tumors

Illustration of CAR T cell therapy targeting tumor cells

Illustration of CAR T cell therapy targeting tumor cells

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CAR T cells, which are immune cells modified to attack cancer, have transformed blood cancer treatments, particularly for leukemia. However, they have struggled against solid tumors. Recently, “weaponized” CAR T cells have successfully eliminated large prostate tumors in mice, providing optimism for similar therapies in humans.

“The tumor is completely gone,” states Jun Ishihara from Imperial College London, marking a unique achievement in animal research.

Our immune system typically identifies and destroys many cancers early on. Cancer cells often display mutated proteins recognized by T cells, which seek to eliminate these threats using surface receptor proteins, functioning similarly to antibodies.

However, not every cancer incites an immune response. In the 1980s, scientists found a way to engineer T cells to target cancer more effectively by incorporating genes for chimeric antigen receptors, hence the term CAR T.

While CAR T cells have shown remarkable success in treating some blood cancers, they also carry significant risks. They are effective for some but not all patients, and ongoing enhancements are needed. The development of CRISPR gene editing facilitates further improvements to these therapies.

Despite advancements, CAR T therapies remain ineffective for most solid tumors due to two primary challenges: the vast heterogeneity of solid tumors—where not all cells present the same mutated proteins—and the tumors’ ability to evade immune responses by emitting “do not attack” signals.

Researchers have attempted to enhance CAR T cells by incorporating immune-boosting proteins like interleukin-12, but this has sometimes resulted in overwhelming immune responses that damage healthy tissues.

Ishihara and colleagues have pioneered a method to localize interleukin-12 specifically to tumors. By fusing interleukin-12 to a protein that binds collagen, which is prevalent in tumors, they engineered it to target the exposed collagen found in both wounds and tumors. “Tumors are rich in collagen and are dense because of it,” Ishihara noted.

The modified CAR T cells produce this fusion protein when they bind to the mutated proteins present in certain prostate cancers. Once released, the fusion protein attaches to the tumor’s collagen, effectively signaling the immune system to launch an attack.

Trial results were promising, as the treatment eradicated 80% of large prostate tumors in the test mice. Additionally, when exposed to cancer cells afterward, no new tumors formed, indicating a robust immune response from the CAR T cells.

Remarkably, this approach did not necessitate preconditioning. Usually, chemotherapy is given to create space for new CAR T cells by depleting existing immune cells, risking side effects such as infertility. “We were surprised that no chemotherapy was required,” says Ishihara. His team aspires to commence human clinical trials within the next two years.

“This is a promising avenue that warrants clinical testing,” stated Stephen Albelda from the University of Pennsylvania. He noted that numerous research groups are also exploring similar methods for tumor-targeted localization of interleukin-12, with encouraging results being reported.

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Source: www.newscientist.com

Breakthroughs in Huntington’s Disease: Essential Insights on Gene Therapy

The caudate nucleus is one of the brain regions that gene therapy targets

Kateryna Kon/Science Photo Library

Innovative gene therapy has emerged as the pioneering treatment that effectively slows the progression of Huntington’s disease. While the results are still in their early stages, this method holds significant promise and could even pave the way for new therapies for other neurodegenerative disorders, including Parkinson’s and Alzheimer’s diseases.

How does the treatment function?

The therapy known as AMT-130 specifically targets the abnormal brain proteins associated with Huntington’s disease progression. Those affected by the condition possess genetic mutations that result in the accumulation of huntingtin proteins, which form toxic aggregates in brain cells, leading to cell death. This eventually manifests as memory loss, mobility issues, speech impairment, and various other symptoms.

Therapies from the Dutch biopharmaceutical company Uniqure aim to halt the production of these mutant proteins. The process involves delivering genetic material to brain cells using a harmless virus. This genetic material instructs the cells to produce small RNA molecules called microRNAs that effectively block and eliminate the signals that lead to the generation of toxic proteins. It acts similarly to a molecular stop signal.

Procedures and Location of Treatment

This therapy initially focuses on two specific brain areas impacted by Huntington’s disease: the caudate nucleus and the putamen. These regions are located deep within the brain, requiring doctors to utilize real-time brain imaging to navigate thin catheters into place. The entire procedure duration ranges from 12 to 18 hours, and preliminary results suggest that a single injection may be adequate to permanently reduce levels of mutant huntingtin in the brain.

What is the efficacy of gene therapy?

Initial findings released by Uniqure indicate that gene therapy may slow the advancement of Huntington’s disease by approximately 75%.

The revelation stems from a clinical trial spearheaded by Sarah Tabrizi of University College London, where 17 Huntington patients received high-dose treatment. After three years, researchers noted the deterioration in cognitive function, motor skills, and daily activities compared to a similar group of untreated individuals. Tabrizi remarked that the decline typically observed over a year in untreated patients was instead noted within four years for those receiving treatment. BBC News. Additionally, those treated exhibited reduced levels of proteins associated with brain damage in their cerebrospinal fluid, highlighting the potential of gene therapy in slowing Huntington’s progression.

“These findings reaffirm our conviction that AMT-130 could revolutionize the treatment landscape for Huntington’s disease,” stated Walid Abi-Saad from Uniqure in a press release.

Are there any side effects?

While Uniqure has not provided comprehensive data on the side effects of the treatment, initial reports suggest that the drug appears to be safe and well-tolerated. The most frequently reported side effects included headaches and confusion, which generally resolved with steroid treatment to reduce inflammation.

When will the treatment be accessible?

In a recent press announcement, Uniqure indicated that it anticipates submitting an application to the U.S. Food and Drug Administration early next year, with the potential for product availability as soon as before 2027 with expedited approval.

“However, it is still in the preliminary phase, and further testing is necessary to assess the long-term effects of this new gene therapy and any potential side effects,” noted Zofia Miedzybrodzka from the University of Aberdeen, UK, in a statement.

Could this method assist in treating other brain disorders?

If successful, the gene therapy could facilitate the development of analogous treatments for various neurodegenerative diseases, including Parkinson’s and other forms of dementia, according to David Rubinsztein from Cambridge University. Researchers could manipulate genetic material to target the toxic proteins that cause these conditions. “This could represent a significant breakthrough,” he remarked.

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Source: www.newscientist.com

The Power of Laughter Therapy for Enhancing Emotional Well-Being

Laughter yoga session in Kolkata, India

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Laughter therapy is no laughing matter. Structured activities designed to elicit joy, such as laughter yoga and hospital clowns, appear to significantly lower anxiety levels and enhance life satisfaction.

Laughing, a behavior observed in various animal species, is believed to boost social bonds and may assist infants in developing their self-identity. Research indicates that interventions involving clowns lead to shorter hospital stays for children.

Yelsynyn-Mauricio Porras-Jiménez from Jaen University in Spain aims to enhance people’s holistic well-being. “It’s not solely about physical health; mental and emotional aspects are crucial,” he notes. “I discovered laughter therapy while exploring ways to implement holistic care effectively.”

Porras-Jiménez and his team performed a meta-analysis of 33 studies from the United States, Europe, Asia, and the Middle East. The participants included nursing students as well as individuals receiving terminal care, undergoing surgeries, in vitro fertilization, or those grappling with depression or burnout.

Each study divided participants into two groups. One group engaged in various laughter therapy activities, such as laughter yoga, clown visits, watching entertaining films, or joining guided laughter sessions. The second group served as a control, maintaining standard care or receiving no interventions.

Results demonstrated a consistent link between laughter therapy and decreased anxiety, alongside heightened life satisfaction. In one measurement of anxiety on a scale of 0-100, the control group’s average score was around 60, while the laughter therapy group scored 8-10 points lower. Regarding life satisfaction, the control group’s score averaged 50, with the laughter therapy group scoring 10-12 points higher.

However, participants were aware they were partaking in laughter therapy, which raises questions about potential placebo effects, according to Sophie Scott from University College London.

Nonetheless, laughter triggers physiological changes correlated with reduced anxiety, she adds. Research shows lowered cortisol levels, a stress hormone, and increased endorphins, neurotransmitters that foster feelings of happiness.

“It’s challenging to determine if the benefits arise from laughter itself or from the social environment that encourages comfortable laughter,” Scott remarks. “It seems to be a combination of both.”

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Source: www.newscientist.com

NHS Talking Therapy Appears to Be Ineffective for Young Adults

NHS Talking Therapies: Access to Science-Based Self-Help and Psychotherapy

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A specific type of psychotherapy offered by the UK’s National Health Service (NHS) seems to benefit teenagers and young adults with anxiety and depression more than it does for older individuals.

Clinical studies focusing on the effects of psychotherapy on prevalent conditions like anxiety and depression have primarily concentrated on working-age adults; depression often manifests in adolescence or early adulthood, according to Argyris Stringaris from University College London.

In 2008, NHS England initiated a program designed to enhance access to scientifically informed self-help resources and psychotherapy. Currently referred to as NHS talk therapy for anxiety and depression, this initiative has generated a substantial database of anonymous user information, detailing treatments and outcomes.

Stringaris and his colleagues harnessed this data to evaluate the results from over 1.5 million individuals who accessed the therapy between 2015 and 2019. More than 1.2 million participants were aged between 25 and 65, leaving the younger cohort aged 16 to 24.

The researchers assessed changes in symptom severity pre- and post-treatment by utilizing two standard self-assessment tools: a patient health questionnaire-9 and a seven-item general anxiety disorder scale.

The results indicated that these scores exhibited approximately 35% improvement in individuals aged 16 to 24, compared to around 41% for those aged 25 to 65. Young people were roughly 20-25% less likely to be classified as recovered or to achieve significant improvements.

This could potentially result in thousands of young individuals recovering each year if they responded to treatment as effectively as their older counterparts, Stringaris remarks. “It is crucial to consider how mental health services might adapt to provide better care pathways for younger populations.”

Various factors could contribute to these discrepancies, including generational differences influenced by social media and online dating. Data further suggest that younger clients tend to miss scheduled appointments without prior notification, which may lead to poorer outcomes.

The study highlights the imbalances that warrant further exploration, says David Clark from Oxford University. “Research often reveals numerous insights that can be addressed. This paper marks the beginning of what could be a fruitful path ahead.”

Despite this, it remains essential to acknowledge the “gradual benefits” of the therapy. This relates to how clients fare compared to those who do not undergo treatment, he adds. “You start getting [younger] patients who are trying to establish themselves in life and pursue employment, indicating they may not have worse outcomes than older individuals,” Clark says.

Adrian Whittington from NHS England explains that talk therapy assists over a million individuals annually, most receiving treatment within six weeks. “We encourage them to take proactive steps towards care,” he states.

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Source: www.newscientist.com

Text Therapy: Study Reveals Couples Use Emojis in Messaging to Enhance Relationships

A key to nurturing a healthy relationship might involve gazing into the faces of those glued to their smartphones.

Recent research Published in Journal Plos One indicates that incorporating emojis in text messages strengthens personal connections and enhances satisfaction.


Researchers from the University of Texas conducted a study with 260 participants aged 23 to 67, asking them to review 15 text message exchanges differing solely by the presence of emojis.

Participants were prompted to envision themselves as the sender of each message while analyzing the recipient’s response, gauging aspects like responsiveness, likability, intimacy, and overall relationship satisfaction.

The findings showed that individuals who included emojis in their texts were perceived as more emotionally aware in their interactions compared to those who used text alone.

Moreover, emojis served as nonverbal indicators of attention and emotional investment.

Luke McGregor, 42, and Amy Thunig-McGregor, 37, shared their experiences with emoji use.

Luke recounted that he had to adapt to integrating emojis in his messages to Amy, as he hadn’t been a frequent emoji user at the relationship’s start.

“I traditionally didn’t use emojis much, but once I got together with Amy, I noticed she used them frequently. There were vulnerabilities and challenges I needed to overcome to start using them myself,” McGregor explained.

Emojis are significant for Amy Thunig-McGregor and her partner Luke McGregor.

“I wanted Amy to feel cherished, so regularly sending her emojis to express my affection was crucial for me, especially initially,” he mentioned.

Amy highlighted the effectiveness of emojis in enhancing communication.

“Both of us are autistic, which makes context essential… emojis help clarify tone and intention in ways that plain text can’t achieve,” they stated.

Dr. Raquel Peel, a senior lecturer in psychology at the University of Central Queensland and not involved in the study, noted that emojis can be a creative solution when face-to-face interactions aren’t possible with partners.

“I don’t believe emojis can replace direct interactions in intimate relationships. Yet, we must recognize that such interactions aren’t always viable,” Peel remarked.

“If meeting your partner isn’t feasible, using emojis becomes a valuable alternative.”

She advised not to undervalue communication in a relationship, emphasizing the need to stay connected in every possible way.

“Many forget that relationships benefit from humor and enjoyment,” Peel explained.

“If emojis can fulfill that role, they contribute to fun and connection through humor, which is incredibly important.”

Source: www.theguardian.com

CAR T-Cell Therapy Can Be Administered to Cancer Patients

Illustration of CAR T-cell therapy targeting multiple myeloma, a type of blood cancer

Nemes Laszlo/Alamy

CAR T-cell therapy has the potential to transform cancer treatment. This innovative treatment genetically alters immune cells to combat diseases but is both complex and costly. Researchers have recently achieved the ability to develop personalized therapies within the bodies of non-human animals.

This form of treatment is primarily accessible in the UK and the US for select patients with various blood cancers, such as certain leukemias, where B cells—crucial immune components—grow uncontrollably. The process entails extracting T cells from a patient’s blood, genetically modifying them to target and destroy B cells, then duplicating and reintroducing these modified cells back into the patient’s body.

Nonetheless, this method is time-intensive. “You need to take the blood and send it to the Central Manufacturing Institute before it can be returned,” explains Carl June from the University of Pennsylvania. “This makes scaling the process challenging.” Additionally, the treatment comes at a steep price: over $500,000 per patient.

In search of a more efficient method, June and his team focused on gene molecules that deliver instructions to produce proteins that target B cells. They encapsulated these molecules in fat droplets, allowing entry into T cells, where they can identify and eliminate B cells. However, this effect is temporary, as the RNA code degrades within a week.

The researchers injected cancerous human B cells and healthy T cells into mice lacking an immune system. After a week, they administered five fat droplets to these mice over a span of two weeks, with some receiving higher doses.

Three weeks later, the mice that received the highest dose displayed no detectable tumor cells and no side effects. “The level of tumor cells was as minimal as we could measure,” remarks June.

The team also administered fat droplets to 22 healthy monkeys, resulting in the production of CAR T cells within their bodies and completely eradicating all B cells within just one day. Although B cells are essential for antibody production, the treatment was well tolerated by all but one monkey, which experienced a severe inflammatory response.

“This is truly remarkable,” says Karin Straathof from University College London. This could represent a significantly easier and more affordable method for implementing CAR T-cell therapy, she asserts.

However, one downside of standard CAR T-cell therapies is their ability to provide long-lasting protection, notes Straathof. The newly developed technique temporarily produces these cells; if cancer returns, additional treatments will be necessary. Furthermore, the effectiveness and safety of this approach in humans remain unverified, pending clinical trials.

June confirms that the team is currently testing the method in healthy humans. “The first patient was treated in the past few weeks,” he states.

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Source: www.newscientist.com

Pennsylvania Baby Becomes First to Receive Personalized Gene Therapy

A baby born with rare and perilous genetic disorders has flourished thanks to an experimental gene editing treatment tailored specifically for him.

Researchers detailed the situation in a recent study, explaining that he initially received a personalized therapy aimed at rectifying a critical genetic flaw responsible for the death of half of affected infants. While wider availability of similar tailored treatments may take time, doctors remain optimistic. They believe that as genetic medicine advances, it can assist millions still facing rare conditions.

“This represents a pivotal advancement in gene editing therapy, addressing various uncommon genetic disorders that currently lack effective medical solutions,” stated Dr. Kiran Musnoor, a gene editing specialist from the University of Pennsylvania and co-author of the study recently published in the New England Journal of Medicine.

KJ Muldoon, from Clifton Heights, Pennsylvania, is among the 350 million people globally grappling with genetic issues. Shortly after birth, he was diagnosed with severe CPS1 deficiency, a condition estimated to affect about one in a million infants. These babies are unable to produce the enzymes required to eliminate ammonia from their systems, leading to toxic accumulation in the bloodstream. For some, liver transplants are a viable option.

Understanding KJ’s circumstances, his parents, Kyle and Nicole Muldoon, aged 34, were understandably anxious about the possibility of losing him.

“We evaluated all available options and posed numerous questions about potential liver transplants,”

“We prayed, consulted others, collected information, and ultimately decided this was the best path forward,” Kyle added.

In just six months, the team at Philadelphia Children’s Hospital and Penn Medicine, in collaboration with their partners, devised a treatment aimed at correcting KJ’s faulty genes. They utilized GIRSPR, a technique for which the inventor received the Nobel Prize in 2020. Rather than cutting DNA strands like earlier CRISPR methods, doctors implemented a technique that alters the “letter” (or base) of the mutated DNA, transforming it into the correct version. This approach, known as “base editing,” minimizes the chances of unintended genetic alterations.

St. Hill Boupalan, a gene therapy researcher at St. Jude Children’s Research Hospital in Memphis, who was not part of the study, remarked that it is “incredibly thrilling” how quickly the treatment was developed. “This genuinely establishes a new benchmark for this approach.”

In February, KJ received his first IV injection of the gene editing therapy, delivered through small fatty droplets called lipid nanoparticles, which are absorbed by liver cells.

The atmosphere was filled with excitement on that day, but “he slept through the entire procedure,” Dr. Rebecca Ahrens-Nicklas, a gene therapy expert at CHOP, recalled.

Following subsequent doses in March and April, KJ began eating normally and recovered well from minor illnesses, such as colds. He has been on economical medications for the past 9½ months.

Given his grim prognosis initially, “each small milestone he reaches feels monumental for us,” his mother expressed.

Nonetheless, researchers caution that only a few months have passed. They need years of observation.

“We’re still in the early stages of comprehending the effects this drug has had on KJ,” Ahrens-Nicklas stated. “However, he demonstrates signs of growth and well-being daily.”

Researchers are hopeful that KJ’s case could provide insights beneficial to patients with other rare diseases.

While developing gene therapies can be tremendously pricey, they often focus on more prevalent conditions for financial viability. A broader patient base can enhance sales, assisting in covering development costs and yielding more profit. For instance, the first CRISPR therapy approved by the US Food and Drug Administration addresses sickle cell disease, a painful blood disorder impacting millions worldwide.


KJ Muldoon with parents Kyle and Nicole Muldoon and siblings after a follow-up dose of experimental gene editing therapy in April 2025 at the hospital.
Chloe Dawson/Philadelphia Children’s Hospital via the Associated Press

Musnoor mentioned that his team’s efforts—partially funded by the National Institutes of Health—demonstrate that customizing treatments does not necessarily have to be prohibitively expensive. He indicated that costs are “not far off” from the $800,000 average for a liver transplant and associated care.

“As we continue to develop these treatments and shorten the timeframes, we anticipate economies of scale, which will drive costs down,” Musnoor noted.

Scientists also emphasized that it isn’t necessary to repeat all initial steps each time a customized therapy is created, as noted by Boupalan. This study “paves the way” for other rare condition treatments.

Carlos Mores, a neurology professor at the University of Miami who was not involved in the study, stated that such research paves the way for future advancements.

For other teams to apply insights and move forward, “breakthroughs like this can spur progress quickly,” he remarked. “Though there are challenges ahead, I predict we will overcome them in the next five to ten years, leading the entire field to advance collectively.”

Source: www.nbcnews.com

A Baby with a Rare Disease Receives the World’s First Personalized CRISPR Gene Therapy

Baby KJ Rebecca Affles Nicklas and Kiran Musnul after gene editing injection with researchers

Philadelphia Children’s Hospital

A young boy afflicted with a serious genetic disorder is set to be the first recipient of personalized CRISPR gene editing treatments, offering a glimpse into the potential future of medicine.

This groundbreaking event marks the first instance of an individual receiving a gene editing therapy tailored to correct unique mutations contributing to their illness. Rebecca Ahrens-Nicklas explained during a press briefing held at Children’s Hospital in Philadelphia, Pennsylvania, “He is showing early signs of progress,” though she noted that it’s premature to determine the complete effectiveness of the treatment.

Researchers released information promptly, aiming to motivate others, as stated by team member Kiran Musnur at the University of Pennsylvania. “We sincerely hope that demonstrating the feasibility of personalized gene editing therapy for one patient within a few months will encourage additional efforts in this area,” he remarked.

“When I refer to this as the future of medicine, I believe I’m stating a fact,” he emphasized. “This is a crucial step towards employing gene editing therapies to address a range of rare genetic disorders that currently lack viable treatment options.”

KJ inherited mutations on both alleles of a liver enzyme gene known as CPS1. The absence of this enzyme leads to ammonia accumulating in the bloodstream, posing a risk of brain damage during the breakdown of dietary proteins. According to Ahrens-Nicklas, over half of children born with CPS1 deficiency do not survive.

She and Musnur are developing therapies targeting this condition by focusing on the liver, allowing them to rapidly formulate a basic editing therapy that modifies one of KJ’s two CPS1 gene copies.

The team engaged with US regulatory bodies early in the process. “They recognized the exceptional nature of this situation,” Musnur stated. “KJ was critically ill and time was of the essence. Following our official submission to the FDA [Food and Drug Administration] when KJ was six months old, we received approval within just a week.”

KJ underwent initial low-dose treatment at six months in February 2025, followed by higher doses in March and April. He is now able to consume more protein than before, albeit while still taking other medications for his condition.

Ideally, children should receive treatment earlier to mitigate long-term damages linked to conditions like CPS1 deficiency. As reported by New Scientist, Musnur has ambitions to enable gene editing in humans prior to birth one day.

In contrast, other gene editing therapies are designed for broader applications, aiming to work for many individuals irrespective of the specific mutations causing their condition. For instance, the first approved gene editing treatment for sickle cell disease functions by enhancing fetal hemoglobin production, rather than altering the mutations in adult hemoglobin responsible for the disorder. Despite being a “one-size-fits-all” solution, it comes at a price of £1,651,000 per treatment in the UK, as noted by Each treatment course costs £1,651,000.

KJ with his family after treatment

Philadelphia Children’s Hospital

Custom treatments can be significantly more costly. Musnur mentioned that he cannot provide exact figures for KJ’s treatment due to the extensive pro bono work by the involved companies. However, he is optimistic about a decline in costs. “As we enhance our methods, we can anticipate economies of scale, leading to a substantial reduction in prices,” he stated.

One barrier to the development of personalized gene editing therapies has been the regulatory perspective, which previously treated therapies targeting different mutations within the same gene as separate entities. This necessitated restarting the approval process for each mutation individually. However, there’s a growing movement towards a platform approach, allowing broader approvals for treatments targeting various mutations.

“Platform-based methods, like CRISPR gene editing, offer scalable solutions for even the rarest diseases, as exemplified by KJ’s case,” stated Nick Mead from Genetic Alliance UK, a charity that advocates for individuals with rare conditions. “This development finally renders treatment a plausible possibility for countless families.”

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Source: www.newscientist.com

Amid RFK Jr.’s Vaccine Advocacy, Resistance to mRNA Therapy Intensifies

Utah and Tennessee have enacted laws mandating that foods containing vaccines be categorized as drugs, despite the fact that such products are not currently available. Legislators reference a University of California study that investigates the possibility of incorporating vaccines into lettuce.

“We’ll consume this batch of lettuce, take these mRNA vaccines, and then retest the DNA. The results will likely differ. This poses a risk.”

In reality, mRNA vaccines cannot alter genetic material because they do not interact with the cell nucleus, where DNA is located. While small amounts of DNA may be present in all vaccines—similar to what can be found in influenza vaccines—the Food and Drug Administration enforces strict limitations, typically rendering these levels negligible. Researchers have been exploring mRNA vaccines for infectious diseases and cancer for years, dating back to the 1990s with mice, and human trials since the early 2000s. Vaccines containing live viruses have recognized side effects; mRNA vaccines generally experience fewer adverse reactions compared to traditional vaccines.

“mRNA is not a foreign agent. It is something we are regularly exposed to,” stated Melissamua, Chief Science Officer at Moderna, the producer of the COVID vaccine. “Every time you consume whole foods, meat, or vegetables, you ingest substantial amounts of mRNA, which your body degrades and utilizes.”

Even should the bill fail to pass, its advocates assert they are in it for the long haul. Last month, Minnesota Republicans introduced a proposal to classify mRNA products as weapons of mass destruction, adding them to a list including natural PO, charcoal, bacteria, and mustard gas. This initiative mirrored the language of a bill drafted by Florida hypnotist Joseph Santhorne. In his newsletter, Mr. Santhorne lauded local Republican groups for passing resolutions supporting the ban and urged his followers to participate in political events to confront officials.

“It ‘punches them in their eyes,'” he remarked. “It carries significant psychological impact.”

Source: www.nytimes.com

Not undergoing traditional cancer therapy, such as that of Elle Macpherson, poses significant risks

Supermodel Elle Macpherson refuses chemotherapy to treat breast cancer

Australian Press Agency/Alamy

Half of us will develop cancer at some point in our lives, but how many of us can confidently reject mainstream medical advice after consulting dozens of doctors?

In a recent interview Australian Women's Weekly Australian supermodel Elle Macpherson revealed that she did just that, telling the magazine that she was diagnosed with breast cancer seven years ago but refused chemotherapy, opting for an “intuitive, heart-led, holistic approach” to her treatment.

According to the magazine, MacPherson mulled over her decision for weeks after consulting with “32 doctors and specialists,” and finally, in February 2017, she decided to pursue non-pharmaceutical treatment under the guidance of her family doctor, who specializes in “integrative medicine.”

Fortunately, MacPherson is currently in clinical remission, or as she prefers to say, “perfect health.” It's futile to try to assess the risk of her decision to forego chemotherapy without knowing important details about the extent of her cancer, her coexisting risk factors, and the advice she received. For example, she underwent a partial mastectomy, her first surgery to remove a suspicious lump, but Some doctors said That might have been enough treatment.

Nevertheless, the story has sparked outrage online, galvanizing both the growing number of people who distrust “conventional medicine” and those who seek to defend it from growing attacks.

What's behind this distrust? Some researchers say the COVID-19 pandemic “Scientific skepticism” Heated debates are taking place around the world about the severity of the illness, the merits of lockdowns, and the safety of vaccines. More and more Used To hide their anti-scientific views, Conspiracy thinking.

For members of black and minority ethnic communities, Distrust of doctors The reluctance to seek cancer testing and treatment is also being driven by public health and medical institutions. Decades of failure Engaging with them and ensuring equal care in many countries.

In the UK in particular, confidence in the National Health Service's ability to treat cancer has declined. Has fallen in recent yearsand Reports Delaying the start of vital cancer treatment for months.

All of this means that if you're diagnosed with cancer today, your options may seem less clear than they once were. Add to that the typically grueling experience of chemotherapy, and it's no wonder that MacPherson's story of a “non-pharmaceutical” alternative therapy has garnered so much attention.

But it's worth keeping in mind that MacPherson doesn't accurately represent the vast majority of cancer patients. Estimated Net Worth With a net worth of $95 million, she can afford to seek multiple second opinions and even turn down chemotherapy. Her wealth acts as a safety net. Her “ingestible health” company WELCOand her historical romantic ties Disgraced anti-vaxxer Andrew Wakefield,she From 2018 to 2019McPherson's story gets even more complicated.

For most people, refusing medical care carries real risks. 2017 Study Cancer patients who choose alternative medicines as their primary treatment have been found to have a higher risk of dying within five years than those who choose conventional treatments.

Larger studies The following year, he published a study of nearly two million American cancer patients that found that use of complementary medicines was associated with refusal of conventional cancer treatment and a doubling of the risk of death within five years.

Indeed, oncologists More and more Select Use Reduce chemotherapy or avoid it altogether – New treatments, new research The recognition that targeted, customized responses are most effective.

But for now, at least, the advice from organisations like Cancer Research UK is clear: there is no scientific or medical evidence that alternative therapies can cure cancer. At a time when distrust of medicine is widespread, Macpherson's account risks leading people down a dangerous path by trumpeting positive results without important context.

While anyone may be at risk for developing cancer, Macpherson, who was nicknamed “The Body” at the height of her modeling career, had enormous resources at her disposal and was always going to have a better chance than most of us of surviving cancer, regardless of her choices.

Elle Hunt is a freelance writer and journalist.

Source: www.newscientist.com

NHS: New online therapy may potentially double the number of individuals overcoming anxiety

Research suggests that a new online therapy approved by the NHS could significantly increase the number of children and adults recovering from anxiety and post-traumatic stress disorder. In England, it is estimated that 1 in 5 children and young people between the ages of 8 and 25 may have a mental disorder, while 1 in 4 adults experience a diagnosable mental health problem each year according to NHS England.

Due to long waiting lists for psychiatric care, a surge in demand, and challenges with face-to-face appointments, the National Institute for Health and Care Excellence recommended the use of online therapies across the NHS in their Early Value Assessment. Developed by Oxford University, four internet treatments will be implemented in various NHS trusts, mental health facilities, schools, and universities starting in September.

The University of Oxford has licensed Koa Health to deliver these online therapies, which are tailored for adults, adolescents, children with social anxiety disorder, and adults with PTSD. The treatments involve a series of online modules delivered through phone or video calls with therapists, available 24/7 to replicate in-person treatment.

Studies have shown positive results with these online therapies, with patients recovering as effectively as those receiving face-to-face treatment. Clinical trials have demonstrated significant benefits in treating social anxiety disorder and PTSD, showing promising recovery rates and improvements in quality of life.

The expansion of online therapy has been welcomed by mental health organizations, emphasizing the importance of patient choice in selecting the most suitable treatment. The NHS acknowledges the need to improve access to mental health care and recognizes the potential of digital tools to provide essential support to those in need.

Source: www.theguardian.com

Five children born deaf regain their hearing through gene therapy

Gene therapy involves administering normal copies of the mutated gene that causes a particular disease.

nobeastsofierce/Shutterstock

Five Chinese children born deaf have received gene therapy that provides them with a healthy copy of a mutated gene, allowing them to hear in both ears. Although the children have varying degrees of hearing loss, all can now hear and localize sounds at conversational volume.

Six months after treatment, the five children’s hearing was about 50 to 60 percent of normal levels, team members say. Chen Yi Chen “They get confused when we whisper, but normal conversation is fine,” he said at the Massachusetts Eye and Ear Infirmary in Boston. “We’re very happy.”

In the first phase of the trial, which began in 2022, the team gave another group of six children in China the gene therapy in just one ear. Five of the six children had their hearing restored in the treated ear, with continued improvement, Chen said.

The team expects to see further progress in the second group of five children. “The progress we’re seeing now isn’t the peak,” Chen says. “We expect to see even more progress.”

The trial in China is the first of several ongoing trials around the world. She lives in the UK with her two children. There have also been reports in the United States of people regaining hearing in one ear after undergoing gene therapy.

“The tests are all pretty much the same.” Manohar Bans Doctors from Cambridge University treated two children in the UK.

All of the children in these trials were born with hearing loss because they carry a mutation in both copies of the gene for a protein called otoferin, which plays a key role in synapses, or connections, between the hair cells in the ear that detect sound and the nerves that transmit signals to the brain. The mutation affects the protein, blocking the transmission of signals.

Between 2 and 8 percent of children worldwide who are born with hearing loss are thought to have this condition, known as DFNB9.

Both parents of a child with DFNB9 have normal hearing if they each have just one mutant copy of otoferlin. These couples are often unaware that they have a one in four chance of having a child that is born deaf.

Gene therapy relies on the help of a virus called AAV to deliver a working version of the otoferlin gene to hair cells. Due to the gene’s large size, it needs to be split and put into two separate viruses.

The virus cocktail is injected into the inner ear, and the complete gene is reconstituted in cells that receive both halves. The DFNB9 trial marks the first time that so-called dual AAV gene therapy has been used to treat humans.

“This is a major technological advance,” Chen says, “and we hope that this technology will be widely used to treat other genetic diseases.”

The clinical trials will begin by treating one ear at a time because half the dose of AAV is needed, reducing the chance of adverse events, he says. No serious adverse events have been reported in any of the trials.

Chen’s team now plans to treat the other ears of the first group of children. This could be difficult because an immune response to the initial AAV injection could block gene delivery, but Chen believes it is possible.

Treating other inherited hearing loss is more difficult because some of the structures inside the ear degenerate. With DFNB9, all of the structures remain intact. “You only need to repair one element,” Chen says.

Some people believe that hearing loss is not a disease that needs treatment, Martin MacLean The position of the National Society for Deaf Children. Families should be free to make their own informed decisions.

“Parents and young people need to be aware of all the risks and, above all, understand that hearing loss in itself does not prevent people from living a happy and fulfilling life,” he says.

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Source: www.newscientist.com

The FDA Approves Pfizer’s First Gene Therapy for a Rare Inherited Bleeding Disorder

The Food and Drug Administration approved Pfizer’s treatment of rare genetic diseases and bleeding disorders, making it the company’s first-ever gene therapy. The approval came on Friday. This groundbreaking gene therapy, named Bekvez, is for adults with moderate to severe hemophilia B, meeting specific requirements. Pfizer spokesperson mentioned that the medication will be available by prescription to eligible patients this quarter with a hefty price tag of $3.5 million, making it one of the most expensive drugs in the United States.

More than 7,000 people in the US live with hemophilia B, primarily affecting men as it causes a lack of Factor IX, a crucial protein for blood clotting. Bekvez is designed as a one-time treatment to enable patients to produce their own Factor IX to prevent and control bleeding effectively.

Pfizer’s approval for Bekvez is a major milestone in the company’s efforts to diversify its portfolio after facing challenges due to the decline in its COVID-19 business. The company has heavily invested in anti-cancer drugs and gene and cell therapies, aiming to revolutionize the treatment of chronic diseases. Pfizer also acquired the rights to manufacture and market Bekvez in collaboration with Spark Therapeutics.

In addition, Pfizer is seeking FDA approval for marstasimab, an experimental antibody to treat hemophilia A and B. The company is also developing a gene therapy for Duchenne muscular dystrophy. Bekvez will be competing with similar gene therapies like Hemogenix from CSL Behring, which also comes with a significant price tag of $3.5 million in the US, marking a new era in the treatment of hemophilia.

Despite the high costs and logistical challenges associated with gene therapies, medical experts are hopeful that these one-time treatments can replace traditional lifelong treatments for chronic diseases, offering long-term benefits to patients.

Source: www.nbcnews.com

Researchers suggest innovative therapy for stomach cancer

Cancer cells grow abnormally and are difficult to control. Scientists call this growth on the lining of the stomach stomach cancer. Gastric cancer is a global health concern in the United States, East Asia, and Eastern Europe. There are usually no symptoms at the time of onset, but it often affects people infected with a bacterial species called Helicobacter pylori.

Researchers have found that diagnosing stomach cancer early is difficult, so many people with stomach cancer die within five years of diagnosis. As cancer grows, it moves from the stomach to other organs, such as the kidneys and liver, through a process called metastasis, which increases the severity even further. This problem raises the need for effective early diagnostic and therapeutic targets to combat gastric cancer before metastasis occurs.

Human cells contain molecules that carry genetic information essential for the development and functioning of organs and body systems. This molecule is DNA and it consists of a sequence of four nucleotide bases: adenine, guanine, cytosine, and thymine.

To carry out its role, DNA undergoes two transformations through biochemical reactions. First, it is transcribed into a slightly similar but less stable molecule. RNAIt is a sequence of nucleotide bases that is almost identical to DNA, except that it has uracil instead of thymine. This RNA serves as a template for protein synthesis, and there are various types. Enzymes then convert some of these RNA molecules into in particular messenger RNA or convert mRNA into protein. Proteins allow organs to grow and function.

Not all RNA molecules become proteins. What does not become protein non-coding RNA or ncRNA. These ncRNAs interact with cells and other molecules to control various processes required to form proteins from DNA for cell growth and survival.

In the past, researchers discovered a type of ncRNA called long ncRNA, which affects the body's immune system's ability to fight cancer cells. However, there are no studies specific to their activity in gastric cancer. Therefore, a group of Chinese biomedical researchers investigated how these ncRNAs influence the development of gastric cancer and how scientists can utilize their ncRNAs to predict the survival of gastric cancer patients.

Researchers found that normal and gastric cancer sample from global cancer database called cancer genome atlas. The normal samples were from patients without gastric cancer and served as the standard or reference point for comparison. Using the R programming language and a software package developed for biological data, they investigated which groups of ncRNAs were expressed at different levels in these patients. They used information from a genome browser called ensemble Identify protein-encoding genes located within and around differentially expressed ncRNA regions.

The researchers found that the expression levels of thousands of ncRNAs were different in gastric cancer compared to normal sample tissue. they again, 15 genes surrounding ncRNA regions that influence gastric cancer progression. They found that about 8 out of 10 ncRNAs were expressed at levels higher than those required in normal cells, and the rest were expressed at lower levels.

Additionally, the researchers investigated the time period during which ncRNAs interact with other ncRNAs and mRNAs to influence tumor growth and patient outcomes. They identified five long ncRNAs that interact with mRNA; microRNA. These long ncRNAs caused abnormal increases and decreases in protein levels within cells, influencing differences in tumor development and progression, as well as patient outcomes. They reported one microRNA that could inhibit tumor growth and serve as a potential target during therapy.

They used a statistical method called , to analyze the proportion of cells that fight infections and harmful substances. immune cellswere investigated in cancer and normal samples to determine how each cell interacts with ncRNAs and influences patient survival. The study highlighted that certain immune cells were higher depending on the age and stage of gastric cancer in the patients whose data were obtained. They confirmed the relationship between immunity and long ncRNA regulatory networks in gastric cancer. They identified certain immune cells whose presence increases a patient's chance of surviving stomach cancer, and those whose presence reduces survival.

With this study, the authors hope to identify new potential targets, namely specific immune cells and ncRNAs, to assess patients' chances of recovery and develop effective treatments for them. concluded that further insight into the biological processes involved in gastric cancer was gained. However, the size of the cancer data is much larger than the regular data used for comparison, which may have influenced the results, the researchers reported. They emphasized the need for further research, especially laboratory analysis, to validate the findings.


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Source: sciworthy.com

Is gene therapy a viable treatment for hemophilia B?

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.


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Source: sciworthy.com

Unlikely Transition: Ketamine’s Journey from a Rave Drug to Mental Health Therapy

Ketamine administered via IV at New York clinic

Victor Llorente/New York Times/Redux/eyevine

Prince Harry, who caused an uproar in the press last year, wrote candidly in his memoirs: spare About taking ketamine to cope with his mother’s death. He’s not the only one talking about this substance. The substance was previously known primarily as a tranquilizer for horses and a psychedelic rave drug. It’s hard to keep track of the many celebrities who have openly talked about taking ketamine to improve their mental health.

Hundreds of clinics have opened across the United States offering intravenous infusions of the drug at point of care, a trend now extending to the United Kingdom. Pioneering companies concerned about the mental health of their employees are beginning to offer this therapy as a benefit. Some companies even floated the idea of ​​setting up ketamine clinics at their headquarters. Meanwhile, pharmaceutical companies are developing over-the-counter ketamine products such as lozenges and topical creams. This drug has become the most commonly available psychedelic therapy.

This may sound like good news, given the growing evidence that ketamine can treat depression, post-traumatic stress disorder, and addiction. However, its use in the treatment of mental health conditions is still novel and many uncertainties remain. Possibly influenced by its popularity as a therapeutic agent, illicit use is also on the rise.

All of this means it’s time to ask whether ketamine can really relieve mental health problems, how it works, and are there any risks to its newfound popularity? is. Psychiatrist…

Source: www.newscientist.com

Light and sound therapy may provide preventative measures against chemically-induced brain changes in Alzheimer’s disease

Some cancer treatments can cause so-called chemobrain, commonly defined as problems with memory and concentration.

One Bar/Alamy

An experimental treatment for Alzheimer’s disease that involves flickering lights and low-pitched sounds may also help prevent cognitive impairment after cancer treatment, also known as chemical brain, a study in mice suggests.

In the case of Alzheimer’s disease, light and sound stimulation has been shown in small human trials to reduce memory and concentration problems, but larger studies are still investigating it.

The light flashes 40 times per second, or 40 Hz, and the sound also has a frequency of 40 Hz. This frequency was originally chosen because the brainwave intensity of Alzheimer’s patients is lower than 40 Hz and is associated with memory processing. The idea was that this treatment would stimulate these brain waves.

Subsequent research has shown that such brain waves may have a wide range of benefits for the brain, including increased immune cell activity and, more recently, strengthened drainage systems that may help remove a toxic protein called beta-amyloid. It suggests that there is.

Cai Li Hui The Massachusetts Institute of Technology researchers who developed this approach thought it could help cancer patients who have memory and concentration problems after chemotherapy and other cancer treatments. It is thought that these may be caused by damage to brain cells, but the exact mechanism is unknown and there is no cure.

In the latest study, Professor Tsai’s team exposed cancer-free mice to light and sound for one hour a day while being given a common chemotherapy drug called cisplatin, compared to those who had just received chemotherapy. They found that they experienced less decline in mental acuity than mice.

Acuity was assessed by a memory test in which mice were exposed to either new or familiar objects, and the animals typically showed more interest in things they had never seen before. Chemotherapy reduced the mice’s ability to identify objects, but this was prevented by light and sound treatment.

The therapy had several effects, including reducing inflammation in the brain, reducing DNA damage, and reducing the loss of myelin, the insulation around nerve cell fibers.

nazanin derakshan Researchers at Britain’s University of Reading say the idea needs to be tested in people to see if it has any overall benefits. If this treatment is given at the same time as chemotherapy and reduces cell death in the brain, it may help cancer cells survive there, she says.

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Source: www.newscientist.com

CRISPR gene therapy shows promise in treating severe inflammatory conditions

New treatment cuts the gene for kallikrein, a protein involved in inflammation (illustrated)

BIOSYM TECHNOLOGIES, INC./Science Photo Library

Nine people with a rare genetic disease that causes a life-threatening inflammatory response appear to have been cured after taking part in the first trial of a new version of CRISPR-based gene therapy.

This condition, called hereditary angioedema, causes sudden swelling of tissue that affects parts of the body such as the face and throat, similar to aspects of an allergic reaction, but cannot be treated with anti-allergy drugs.

Ten people who received a one-time gene therapy administered directly into the body saw a 95 per cent reduction in the number of 'swelling attacks' in the first six months after the treatment took effect. . Since then, all but one have had no further seizures for at least a year, although one patient who received the lowest dose had one mild seizure. “This is potentially a cure,” he says Padmalal Gurugama At Cambridge University Hospital in the UK, we worked on a new approach.

Hereditary angioedema is usually caused by mutations in the gene that encodes a protein called C1 inhibitor, which is involved in suppressing inflammation, which is part of the immune response.

People with this condition may experience a sudden buildup of fluid under their skin several times a month, which is painful and can cause suffocation if it gets stuck in the throat. This attack can be caused by a virus, changes in hormone levels, or stress.

Existing drugs that can reverse attacks work by blocking another molecule involved in inflammation called kallikrein, which is made in the liver. Because people can be born without the ability to make kallikrein without adverse effects, the results suggest that it is safe to permanently block kallikrein through gene therapy, Gurgama said.

The new treatment, developed by a company called Intellia Therapeutics in Cambridge, Massachusetts, consists of genetic material designed to cut the kallikrein gene. It is encapsulated in lipid nanoparticles and taken up by liver cells. One person was treated in the UK and nine in New Zealand and the Netherlands.

An unusual feature of this therapy is that it is administered directly to humans, a method also referred to as “in vivo” delivery. “They get one infusion and that's it,” he says. julian gilmore from University College London was not involved in the study. “It's very appealing.”

So far, most other CRISPR-based gene therapies have been administered “outside the body.” This means a more complex and time-consuming procedure of taking some of a person's cells outside the body, changing the cells in a lab, and then reinjecting them.

CRISPR gene therapy is being developed for multiple genetic diseases, with the first treatments recently approved in the UK and US to help patients with two forms of genetic anemia: sickle cell disease and beta-thalassemia. Ta.

The success of the latest trial is “very exciting,” Gilmore said. Development of CRISPR-based treatments for people with various liver-related conditions, called transthyretin amyloidosis. “This technology could be applied to any disease caused by a mutant protein produced exclusively in the liver, where it is desirable to knock down that protein,” he says.

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Source: www.newscientist.com