A national clinical trial is underway to evaluate the potential of benfotiamine, a synthetic form of vitamin B1, in slowing the progression of early Alzheimer's disease and mild cognitive impairment (MCI). The BenfoTeam trial, a Phase 2A-2B study, is being conducted at 50 sites across the United States and is funded by the National Institute on Aging (NIA), a part of the National Institutes of Health (NIH).
The trial focuses on individuals aged 50 to 89 with mild Alzheimer's disease or MCI. Participants will receive either benfotiamine or a placebo over an 18-month period. The primary endpoint is to assess the impact of benfotiamine on cognitive function and daily living activities. Researchers aim to determine if the drug can improve memory, concentration, and overall cognitive performance.
Rationale Behind Benfotiamine
Benfotiamine can elevate blood thiamine levels up to 100 times higher than what can be achieved with thiamine alone. Studies have indicated that individuals with Alzheimer's disease often exhibit thiamine deficiencies in brain tissue, even when blood thiamine levels appear normal. The trial is designed to investigate whether benfotiamine can effectively deliver thiamine to the brain, potentially slowing cognitive decline.
Gary E. Gibson, Ph.D., Professor of Neurosciences at Burke Neurological Institute (BNI), explained that existing Alzheimer's treatments often target symptoms like amyloid plaques but may not address the underlying causes of the disease. Benfotiamine has demonstrated promise in animal models by improving memory and reducing plaques, tangles, inflammation, and oxidative stress.
Expert Perspectives
Rajiv Ratan, M.D., Ph.D., Executive Director at Burke Neurological Institute, emphasized the potential of benfotiamine, stating, "The approach of using benfotiamine in patients with mild Alzheimer's is very promising. This clinical trial builds on years of hard work to address a disease that affects millions of patients."
Howard Feldman, Director of the Alzheimer's Disease Cooperative Study (ADCS) at UC San Diego, highlighted the accessibility and cost-effectiveness of benfotiamine as a potential Alzheimer's therapy. "The launch of the BenfoTeam clinical trial represents a fresh approach with a potential treatment that is readily accessible, scientifically compelling, and cost-effective – that's a trifecta for a potential AD therapy in a Phase 2 trial – and benfotiamine has a strong safety profile."
Trial Design and Enrollment
The BenfoTeam trial plans to enroll 406 participants. Jose Luchsinger, M.D., MPH, Vice-Chair for Clinical and Epidemiologic Research at Columbia University, noted the trial's inclusive design, stating, "While Alzheimer's disease impacts all aging populations, it is not clear that recently approved Alzheimer's therapies benefit underrepresented racial and ethnic groups. The BenfoTeam trial is designed for all patient populations."
With nearly 7 million Americans currently living with Alzheimer's, and projections estimating a rise to almost 14 million by 2050, the need for effective and accessible treatments is critical. The BenfoTeam trial represents a significant step in exploring a novel approach to combatting this devastating disease.
