Irina Pikuleva, PhD: Activating CYP46A1 with Efavirenz

“If I don’t do it, nobody else will because it is extremely difficult to organize and get funding for a clinical trial.”

Irina Pikuleva, PhD, and her colleagues are testing a well-known anti-HIV therapy, efavirenz, to see if it could work as a preventive treatment for Alzheimer disease. The study of it is currently in early phases.

A biochemist by training, Pikuleva recounted the beginning of this road to clinical testing, more than a decade ago when CYP46A1 was crystallized. She spoke about her and her colleagues’ efforts to see which currently available therapeutics would activate the enzyme. Although, as the therapy is being repurposed, the group has had difficulty getting funding to explore efavirenz’s potential in Alzheimer—which is where the Alzheimer’s Drug Discovery Foundation (ADDF) came in to help.

The ADDF’s 19th Annual Meeting in Jersey City, New Jersey, the Carl F. Asseff Professor of Ophthalmology and the Director of the Visual Sciences Research Center at Case Western Reserve University sat with NeurologyLive to discuss their reasoning for the research, and how it all started.