(PHILADELPHIA) -- Researchers at the University of Pennsylvania School of Medicine have demonstrated the potential of a new type of therapy for patients who suffer from high cholesterol levels. The findings are in the January 11 issue of the New England Journal of Medicine (NEJM). In this study, patients with homozygous familial hypercholesterolemia (FH), a high-risk condition refractory to conventional therapy, had a remarkable 51% reduction in low-density lipoprotein (LDL) or "bad cholesterol" levels.
"Our study shows that targeted inhibition of the microsomal triglyceride transfer protein (MTP) is highly effective in reducing cholesterol levels in these very high risk patients," stated Daniel J. Rader, MD, Director of Preventive Cardiology and the Clinical and Translational Research Center at Penn, and principal investigator of this study. "Furthermore, there are many other patients who have cholesterol levels that are difficult to treat or who are not tolerant to treatment with statins. New therapies are required for these patients as well, and it is possible that after further research MTP inhibition could eventually be used for such patients."
Genetic defects in MTP lead to profoundly low levels of LDL. Using this information, Bristol-Myers Squibb began to search for inhibitors of this protein and discovered the study drug, originally known as BMS-201038. Bristol-Myers Squibb then donated it to Penn for use in clinical trials in patients with severe cholesterol problems. Rader and his team at Penn designed and carried out the current study in homozygous FH patients with support from the Doris Duke Charitable Foundation. Due to the success in this study, Penn has licensed the drug to Aegerion Pharmaceuticals Inc for further development as AEGR-733.
Patients who suffer from homozygous FH typically respond poorly to standard drug therapy and have a very high risk of premature cardiovascular disease. Homozygous FH is caused by los
Contact: Susanne Hartman
University of Pennsylvania School of Medicine