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
People who want to know more about New clinical trials can be accessed on the research website.
Source: scitechdaily.com
