When statins aren’t enough, there’s a new class of drugs called PCSK9 inhibitors that dramatically lower LDL cholesterol levels. The story of this class of drugs also stems from research success at UT Southwestern.
In 2000, UT Southwestern physician-scientist Dr. Helen Hobbs, a protégé of Drs. Brown and Goldstein, launched the Dallas Heart Study. Researchers collected extensive health information and genetic profiles from several thousand individuals of different ethnic backgrounds in Dallas County to study cardiovascular disease.
The database has proven invaluable in cardiovascular research and allowed Dr. Hobbs and her UT Southwestern colleague Dr. Jonathan Cohen to identify individuals with mutations in a gene that produces PCSK9 proteins. They were further able to show that individuals who had one mutation in the PCSK9 gene had low cholesterol levels. Drs. Hobbs and Cohen eventually identified a woman who had two PCSK9 mutations and extremely low cholesterol levels, but was otherwise healthy. Their findings helped spur further research leading to the production of PCSK9 inhibitors, two of which were approved in 2015 by the FDA. In clinical studies, one of the inhibitors, alirocumab, reduced LDL cholesterol in individuals taking statins an additional 44 to 58 percent on average.
Photo: Dr. Helen Hobbs