Vanderbilt University Medical Center and Acorda Therapeutics Inc. released findings from a Phase 1 clinical trial of glial growth factor 2 (GGF2) last week at the American College of Cardiology Annual Scientific Session in San Francisco.
The study was designed to evaluate safety, tolerability and exploratory efficacy in people with heart failure who were already receiving optimized standard therapy.
Trends of long-lasting and dose-related improvement in cardiac function were seen following a single dose in patients; however, GGF2 warrants further investigation as a treatment for heart failure, said Daniel Lenihan, M.D., professor of Medicine and director of Clinical Research.
A single dose of GGF2 in patients with heart failure was generally well tolerated up to 0.75 mg/kg. Among participants receiving GGF2, the most commonly observed adverse events were headache, site injection reaction and gastrointestinal symptoms.
GGF2 is the leading development candidate from Acorda’s neuregulin program. Neuregulins are a class of naturally occurring protein growth factors that have multiple effects on the nervous and cardiovascular systems.
“In pre-clinical studies, GGF2 appeared to make the endogenous muscle stronger. If you can make the heart muscle stronger, you might prevent some people from getting worse or needing a heart transplant,” said Doug Sawyer, M.D., Ph.D., co-investigator on the study who first began working with GGF2 more than 15 years ago.
No currently available therapies do this, and GGF2 may therefore offer an important new treatment option for people with heart failure, Sawyer said.
The study enrolled 40 patients with advanced heart failure at Vanderbilt and St. Joseph’s Hospital in Atlanta, and each participant received a single dose or placebo and was monitored for 90 days.
“We have completed the first in-human trial with GGF2 in patients with heart failure, and especially want to thank our patients who volunteered for this important study. We are very encouraged by the results,” Lenihan said.