NIH grants $45 million to study potential Alzheimer’s therapy in clinical trial

The NIH awarded a $45 million grant over 5 years to Burke Neurological Institute to evaluate benfotiamine, a synthetic precursor of thiamine, as a potential therapy for mild Alzheimer’s disease or mild cognitive impairment.

In a release, Burke Neurological Institute (BNI) stated the phase 2 multicenter randomized controlled clinical trial will expand on a pilot study that suggested 300 mg doses of benfotiamine given twice daily significantly slowed the rate of functional decline in 35 participants with mild cognitive impairment or early AD.

“I am particularly excited about this trial because it will determine how relevant these decades of research are to the treatment of Alzheimer’s disease,” Gary E. Gibson, PhD, researcher at Weill Cornell Medicine and leader of the upcoming clinical trial, said in the release. “If our hypothesis is correct, we will advance an exciting investigative clinical treatment pathway relevant for millions of patients, and with potential advantages in safety and value.”

Planned to last 18 months, the study is expected to enroll approximately 400 participants in up to 50 United States-based sites and will be carried out in collaboration with leading researchers in AD and related dementias — Howard Feldman, MD, director of the Alzheimer’s Disease Cooperative Study at University of California, San Diego, and Jose Luchsinger, MD, investigator at Columbia University Irving Medical Center.

Additional academic institutions partnering with BNI to support this trial include University of Gothenburg in Sweden, University of Cambridge in the United Kingdom, and Georgetown University. BNI will also join forces with brain health company C2N Diagnostics.

Enrollment of participants is expected to begin in the first quarter of 2023.