Researchers at the University of Pennsylvania School of Medicine have reported significant progress in demonstrating the utility of a new type of cholesterol therapy. In a new study, published in the New England Journal of Medicine, patients with homozygous familial hypercholesterolemia, a high-risk and hard-to-treat condition, demonstrated a 51 percent reduction in low-density lipoprotein or "bad cholesterol" levels. Researchers began with the knowledge that genetic defects in MTP lead to profoundly low levels of LDL. 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 after reporting troubling side effects. Rader and his team at Penn designed and carried out the current study in homozygous FH patients. Due to the success in this study, Penn has licensed the drug to Aegerion Pharmaceuticals for further development as AEGR-733.
"Our study shows that targeted inhibition of the microsomal triglyceride transfer protein 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."
- here's the release on the new therapy