Promising Therapy for Treatment of High Cholesterol in Statin Intolerant Pts.

statin intolerance Skimming through the April 2013 Cardiology Today publication, I came upon an article addressing a fairly new-found protein known as PCSK9 and its long-term potential for helping people with History of  High Cholesterol (particularly those with elevated LDL’s) and an intolerance to statin therapy. The article, ‘PCSK9 inhibitors poised for breakthrough as new cholesterol-lowering therapy’ states the “research in biologics is getting closer to finding an alternative to statins. At the forefront are these drugs that inhibit proprotein subtilisin/kexin type 9, or PCSK9.”

According to the article, “Protein PCSK9 was accidentally discovered in 2003 by a group of doctors in France who were studying high cholesterol in families. These doctors noted that when there was an increase in this unknown protein (at the time), it stopped the LDL receptors from functioning. In another study of family members with low LDL levels and no known cardiovascular disease, there were findings of defective PCSK9. The data led those in the research arena to hypothesize that if they could inhibit PCSK9 from binding to the LDL receptor, the receptor would work better in lowering the LDL levels. Since then, many studies are now underway

Most people diagnosed with History of High Cholesterol are not uncommonly managed with statin therapy, unless found to be intolerant of the side effects. There are a few other non statin agents in the market yet generally not as effective as statin therapy in lowering cholesterol. Dr. Sergio Fazio MD, PhD, Chief of the section of cardiovascular disease prevention at Vanderbilt University, states that  “although it is possible that some untoward effects may eventually be identified (re: PCSK9), today there are no obvious symptomatic or biochemical adverse events within the context of short clinical trials.”

The article points out that “PCSK9 inhibitors provide the most promising alternative to lowering cholesterol since the development of statins 30 years ago.”  Dr. Steven E Nissen MD, Chairman of the Dept. of Cardiovascular medicine at Cleveland Clinic, states “the effects are long-lasting. In one case, the PCSK9 inhibitor can be given once a month (as an injection).” Dr. Sergio Fazio MD, PhD also added that “if this therapeutic concept gets FDA approval (still a few years out from FDA approval) and reaches the market, it will transform the way we manage CV (cardiovascular) Risk.”

Dr. Peter Libby, Chief of the division of Cardiovascular medicine at Brigham and Women’s Hospital, states “the patients thus far appear to be satisfied with antibody therapies such as PCSK9 inhibitors.”  “They don’t have to take pills which alleviates the worry about missing or skipping a dose.”Lastly, Dr. Libby noted that the favorable response will most likely continue with these biologic injections. Next step, phase 3 and future studies, will focus on testing these PCSK9 inhibitors and others in large patient populations.

In my 20+ yrs working in Cardiology, this is a big breakthrough in managing Cardiovascular disease risk. For those of you currently being treated for High cholesterol with known intolerance to statin therapy, don’t be afraid to ask and/or discuss this with your practitioners. Many people have suffered with statin intolerance and because of it, are in a catch 22 situation; possess risk factors for cardiovascular disease yet can’t tolerate side effects secondary to therapy. As the experts foresee, I do hope PCSK9 inhibitors will be the other option for those in need.





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