Alzheimer’s Drug Discovery Foundation (ADDF) and Harrington Discovery Institute Partnership Helps Move Promising Alzheimer’s Research from Bench toward Bedside

A recently announced licensing agreement between drug maker Acadia Pharmaceuticals and Vanderbilt University represents a major milestone for the ADDF-Harrington Scholar Award Program, which provided funding and pharmaceutical expertise to support the research in the early phases. Acadia and Vanderbilt will collaborate to develop and commercialize novel drug candidates targeting synaptic receptors in the brain, long thought to play a key role in Alzheimer’s disease.

“This type of licensing agreement is precisely the goal of the ADDF-Harrington partnership,” said Dr. Andrew A. Pieper, Harrington Discovery Institute Director of Neurotherapeutic Discovery. “We bridge the gap between academia, where many great medical ideas are born, and industry, where these ideas can be guided through the costly and complex process of transforming them into new medicines for patients.”

Vanderbilt University principal investigator Jerri Rook, Ph.D. and her Vanderbilt University Medical Center physician collaborator Dr. Paul Newhouse received the 2015 ADDF-Harrington Scholar Award. The award provided funding and expertise in formulation and interpretation of safety pharmacology and toxicology data from experienced drug development professionals.

The compounds covered by the agreement work by activating muscarinic M1 receptors in the brain in a unique way that increases their responsiveness to a neurotransmitter called acetylcholine, which plays a critical role in regulating memory and cognition.

“We are excited about the commercial support for the important work of Dr. Rook and her colleagues that we hope will lead to an effective and safe treatment for people with Alzheimer’s disease,” said Dr. Howard Fillit, ADDF Founding Executive Director and Chief Science Officer.

As explained by Dr. Rook, researchers have long theorized that this mechanism could effectively treat memory loss in Alzheimer’s disease and other brain disorders, but intolerable side effects have barred their use—at least so far. “Our focus has been on discovering and developing compounds that have the desired treatment benefits without the unwanted side effects. We have now optimized this in a mouse model of Alzheimer’s disease and are very much looking forward to seeing how they perform in human studies,” said Dr. Rook. The lead compound has entered Phase I clinical trials with support from the ADDF.