A $7.5 million research award will go to Southern Research to identify potential new drugs for people with rare cystic fibrosis mutations, known as nonsense mutations, from Cystic Fibrosis Foundation Therapeutics Inc.
The University of Alabama at Birmingham is collaborating with Southern Research on this five-year project.
Southern Research will use a sophisticated screening process to identify compounds in its chemical library that could promote the production of functional CFTR protein. In individuals with CF, a defective CFTR protein leads to a buildup of thick mucus, which in turn leads to persistent lung infections and complications in other parts of the body.
CF is a life-threatening, genetic disease that affects approximately 30,000 people in the United States and nearly 70,000 people worldwide. About 10 percent of individuals with CF have nonsense mutations. Cystic Fibrosis Foundation Therapeutics has ramped up its efforts to pursue cutting-edge strategies that target these and other mutations by addressing earlier cellular processes that generate the malfunctioning CFTR protein.
| CF is a life-threatening, genetic disease that affects approximately 30,000 people in the United States and nearly 70,000 people worldwide. About 10 percent of individuals with CF have nonsense mutations. |
Research has shown that nonsense mutations in the CF gene cause messenger RNA molecules to transmit a signal to stop production of the CFTR protein too early. This premature stop signal results in a shortened, and often nonfunctional, CFTR protein.
Using special tests developed by UAB, Southern Research will look for compounds that would override that premature stop signal, allowing fully functional protein to be made. Southern Research also will look for compounds that would address other problematic cellular processes.
Initial funding through the Alabama Drug Discovery Alliance provided the groundwork for this drug discovery project, which originated with UAB’s David Bedwell, Ph.D., and Steven M. Rowe, M.D., working with Bob Bostwick, Ph.D., at Southern Research. UAB’s Venkateshwar Mutyam, Ph.D., is a co-principal investigator.