Research News

Feixiong Cheng, PhD, Genomic Medicine Institute, has been awarded a 5-year, $3.3M grant from the National Institute on Aging, part of the National Institutes of Health, to develop computational tools to identify novel repurposable drugs for Alzheimer’s disease (AD).

Currently the leading cause of dementia and sixth leading cause of death in the United States, AD is set to affect approximately 16 million Americans by 2050 if effective treatments are not developed. Numerous clinical trials for AD treatments have been conducted; however, because the attrition rate remains high and many studies adhere to the outdated “one gene, one drug, one disease” paradigm, drug discovery for AD remains stunted.

Recent studies suggest that AD often shares common underlying mechanisms (termed “endophenotypes”) with other neurodegenerative diseases. The presence of these endophenotypes presents an opportunity to apply drug repurposing, or the repositioning of existing drugs for new therapeutic purposes, to pinpoint effective AD treatments. In preliminary studies, Dr. Cheng found that the underlying AD-related endophenotype modules, shared by amyloidosis and tauopathy, will serve as models for in silico drug repurposing and rational design of combination therapy in AD. With this new grant, Dr. Cheng aims to develop intelligent computer-based network medicine and systems pharmacology tools (https://alzgps.lerner.ccf.org) that can search human genome sequencing findings and existing drug-target databases as well as test the most promising network-predicted repurposable drugs and drug combinations.

“These tools and potential prevention and therapeutic approaches will help improve outcomes in long-term dementia care and quality of life for the rapidly growing AD population,” says Dr. Cheng.