2018 New Therapeutic Uses for Experimental Assets

In April 2018, NCATS issued funding for three cooperative agreements designed to match academic research groups with selected compounds from industry as part of the New Therapeutic Uses program. The goal is to use molecules that already have undergone significant research and development by the pharmaceutical industry to more quickly advance new treatments for patients.

AZD9668: A First in Class Disease Modifying Therapy to Treat Alpha-1 Antitrypsin Deficiency, a Genetically Linked Orphan Disease

University of Alabama at Birmingham

Principal Investigators: Mark T. Dransfield, M.D., and Gary R. Cutter, Ph.D.
Grant Number: 1-UG3-TR-002450-01

Alpha-1 antitrypsin deficiency (AATD) is the most common genetic cause of chronic obstructive pulmonary disease and emphysema. The disorder affects between 70,000 and 100,000 individuals in the United States. Individuals with AATD have extremely low plasma and lung levels of AAT, a protein that helps lung tissue remain elastic and flexible. Current treatment for AATD is invasive, expensive and does not permanently slow the development of lung damage. This project team will study the safety, tolerability and effectiveness of AZD9668 as an improved noninvasive treatment for patients with AATD.

Learn more about this project in the NIH RePORTER

AZD9668 and Neutrophil Elastase Inhibition to Prevent Graft-versus-Host Disease

Duke University and NIH’s National Cancer Institute

Principal Investigators: Nelson J. Chao, M.D., M.B.A. (Duke), and Steven Z. Pavletic, M.D. (NCI)
Grant Number: 1-UG3TR002448-01 

Graft-versus-host disease (GVHD) is the major cause of treatment-related death for patients who undergo stem cell transplants. The enzyme neutrophil elastase, which makes cells less elastic and flexible, may be a key trigger of GVHD. This project team will test a new strategy to prevent GVHD using AZD9668, which blocks neutrophil elastase, in a series of milestone-driven preclinical and clinical trials.

Use of the Src Family Kinase Inhibitor Saracatinib in the Treatment of Pulmonary Fibrosis

National Jewish Health, Icahn School of Medicine at Mount Sinai and Yale University

Principal Investigators: Gregory P. Downey, M.D. (NJH), Joel Thomas Dudley, Ph.D. (Mt. Sinai), and Naftali Kaminski, M.D. (Yale)
Grant Number: 1-UG3TR002445-01

Scarring of the lung, called pulmonary fibrosis, is a chronic, progressive and usually deadly disorder. Although two drugs were recently approved for the treatment of pulmonary fibrosis, neither cures the disease, and nearly 40 percent of patients stop taking their medication within one year because of side effects. This project will study the use of saracatinib, a drug originally developed to treat certain types of cancers, in the treatment of pulmonary fibrosis in both preclinical models and a clinical trial.

Learn more about this project in the NIH RePORTER