WHITE PLAINS, N.Y.--(BUSINESS WIRE)--A small exploratory clinical trial, carried out by Dr. Gary E. Gibson’s laboratory at the Burke Neurological Institute in collaboration with researchers at Weill Cornell Medicine and Columbia University Irving Medical Center, suggests that benfotiamine, a synthetic precursor of thiamine (vitamin B1), has the potential to be an effective treatment or preventive measure for Alzheimer’s disease (AD). The study found the drug both safe and effective in slowing the rate of functional decline in participants with mild cognitive impairment (MCI) or early AD. The research was published in the Journal of Alzheimer's Disease and paves the way for a larger clinical trial.
Dysfunction in the brain’s ability to metabolize glucose is a known marker of AD and other dementias and is impaired decades before a person has memory loss or other symptoms. Animal models show that benfotiamine improves cognitive function, reduce several biological markers of AD, including the amyloid-β (Aβ) plaques and neurofibrillary (tau) tangles that are the hallmarks of AD in the brain.
In the current study, Dr. Gibson and his team evaluated the use of benfotiamine over a year-long period in individuals with existing MCI or mild AD. Half of the group of 70 participants took 300-mg benfotiamine pills twice daily, while the other half took placebo (inert) pills. To measure cognitive function, including memory, language, and attention, the team used several tools, including the Alzheimer’s Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) and the clinical dementia rating (CDR). Glucose metabolism was captured via PET scans of the brain and biomarkers in the blood. Genetic testing was also done to determine which participants carried the APOE ε4 gene variant, which puts one at higher risk for AD.
Benfotiamine was very safe as shown by the observation that there were no benfotiamine related adverse events. After a year, the increase in ADAS-Cog score was 43% lower in people who’d taken benfotiamine, compared to placebo, which indicates less cognitive decline in the treatment group. Though the finding wasn’t statistically significant (meaning it could technically have been due to chance), it suggests an effect that may be better detected with a larger study. Among those who took benfotiamine, CDR scores were significantly reduced (by 77%), relative to placebo, again indicating a slower rate of functional decline. Select measures of glucose metabolism improved significantly in the benfotiamine group. The effects of benfotiamine were more robust in participants who did not have the APOE ε4 gene variant, though those subgroups were small.
The results of the study are encouraging, as they suggest a potential new avenue for treating or preventing AD. Currently, more than six million Americans are living with AD, a number that is forecasted to rise to nearly 13 million by 2050. Few effective treatments exist, and recent clinical trials have either failed or produced mixed results. Last month, the FDA approved the first new therapy in 18 years, a decision that sparked considerable controversy over whether the drug’s benefits outweigh the risks. There is enormous global need to develop new methods, not only to treat AD but also to prevent it in a growing population.
“Our next step is to propose a larger clinical trial appropriately powered to replicate our findings,” writes Dr. Gibson. “We believe that further studies would be very valuable to determine whether benfotiamine may be helpful in delaying onset or treating AD.”
The complete clinical trial and research were published in the November 2020 issue of Journal of Alzheimer’s Disease. The full text can also be found on PubMed.
Coauthors on the study were affiliated with Lenox Hill Hospital; University of Liege, Belgium; Georgetown University; Rancho Los Amigos National Rehabilitation Center, University of Southern California, Los Angeles; Einstein College of Medicine; and Westmed Medical Group.
About Burke Neurological Institute
The Burke Neurological Institute is based in White Plains, NY, and was established in 1978 by Dr. Fletcher McDowell as a research institute dedicated to finding cures for chronic neurological disabilities. The Institute transforms groundbreaking research into clinical treatments so that people can see, talk, and walk again. Their goal is to combine the most rigorous, contemporary brain science with heartfelt compassionate care to innovate and develop novel cures for stable disability in those afflicted with neurological conditions such as stroke, traumatic brain injury or spinal cord injury. Burke Neurological Institute is an academic affiliate of Weill Cornell Medicine.