Paul Aisen, MD, director of the Alzheimer’s Therapeutic Research Institute (ATRI) of the Keck School of Medicine at USC, will oversee a study of a new drug in patients with early Alzheimer’s disease.

Dr. Paul Aisen

The study will be funded, in part, through a $75.8 million grant awarded by the National Institute on Aging (NIA) of the National Institutes of Health (NIH) to Cognition Therapeutics, Inc., a New York-based neuroscience company whose labs are based in Pittsburgh.  (The NIA grant number is R01AG065248.)

Alzheimer’s disease is characterized by the accumulation of a substance called amyloid (or Aβ) in the brain.  The most toxic forms of amyloid are small aggregates of amyloid peptide called oligomers.  

This study will investigate whether CT1812, a novel and promising therapeutic candidate which targets the Aβ oligomer, can halt or slow the disease process. “The drug, called CT1812, is designed to protect the brain from synapse damage by a substance called toxic amyloid beta oligomers. CT1812, a small-molecule drug, penetrates the brain and targets a receptor that’s a key regulator of cellular damage response,” the company said in a release. “By doing so, CT1812 displaces toxic amyloid beta oligomers from synapses and protects against further oligomer binding, potentially stopping the synapse damage and destruction that is characteristic of neurodegenerative diseases such as Alzheimer’s.”

The study will be conducted by the Alzheimer’s Clinical Trials Consortium (ACTC), an NIH-funded clinical trial network of 35 academic sites. The ACTC will also be providing the infrastructure to accelerate the recruitment of participants through APT Webstudy and the use of centralized resources and shared expertise.

“CT1812 is a novel and promising therapeutic candidate that targets the most toxic form of amyloid, the oligomer,” said Aisen, a professor of neurology at the Keck School who’s also a member of the ACTC leadership team. “We are excited to be able to test this idea in a nationwide randomized controlled trial.”

About 540 subjects are expected to be enrolled in the five-year Phase 2 study sometime in 2021.  (Sign up for information from the Alzheimer’s Therapeutic Research Institute.) Susan Catalano, PhD, co-founder and chief science officer at Cognition Therapeutics; Christopher van Dyck, MD, director of the Yale Alzheimer’s Disease Research Center; and Aisen will serve as co-investigators.