The University of Alabama at Birmingham has received a grant from the National Institutes of Health totaling $8.9 million to fund a multiyear program evaluating the role of a new medication in a clinical trial for patients suffering from idiopathic pulmonary fibrosis (IPF).
Victor Thannickal, M.D., the Ben Vaughan Branscomb Chair of Medicine in Respiratory Disease and division director of Pulmonary, Allergy and Critical Care, says UAB was selected among a highly competitive group of academic institutions to move this translational program project grant into the clinical testing phase.
"UAB has one of the largest interstitial lung disease programs in the country," he said. "It is important that we receive grants like this so we can continue to develop better treatments for fibrotic lung diseases."
While there currently is no cure for IPF, the drug GKT831 is designed to improve on current drugs that do not improve survival or quality of life, and Thannickal says it may be better tolerated than current medications.
The phase II clinical trial will be a placebo controlled, double blind, randomized, parallel group study to evaluate the safety and efficacy of GKT831. A total of 60 patients will be allocated to a 48-week treatment with the drug or matching placebo. The clinical trial is the centerpiece of the program project grant that will be led by Steven Duncan, M.D., professor in the Division of Pulmonary, Allergy and Critical Care Medicine. Other key UAB investigators in the program include Brent Carter, M.D., Veena Antony, M.D., and Rui-Ming Liu, Ph.D.
IPF is a disease in which the gas-exchanging regions of the lungs known as alveoli become thickened and scarred over time. As alveoli scar, the capacity of the lungs to properly move oxygen into the bloodstream becomes limited. This results in progressive shortness of breath and eventually leads to right heart failure and death.
Thannickal says approximately 150,000 Americans and 5 million people worldwide suffer from IPF, and 40,000 die each year. He cites that genetic risks as well as environmental risks - smoking - can increase the likelihood of developing the disease.
He adds that being able to test drugs like GKT831 in both early and late phase clinical trials makes UAB a unique place to advance novel therapies for IPF.
"The science that allowed us to develop this therapeutic strategy was actually based on a biochemical activity I discovered almost 20 years ago," he said. "IPF has been my lifelong passion, not only to understand the disease process, but to be able to devise more effective treatments. Is this going to be the answer? We don't know, but we think it has more promise than what is already out there."
Recruiting for the trial will begin as early as April 2019. UAB is the primary center, and other participating institutions include the University of Michigan, University of Minnesota, Temple University and Tulane University.