UTSW docs’ discoveries at heart of new cholesterol-lowering drugs


You may have seen the article in The Wall Street Journal noting that powerful means of controlling blood cholesterol are on the horizon. What you may not realize is that these discoveries are emerging directly from scientific investigation initiated and performed at UT Southwestern.

Working from the platform of the Dallas Heart Study, the first large-scale cardiovascular study ever done in North Texas, UT Southwestern's Drs. Helen Hobbs and Jonathan Cohen identified a person with serum cholesterol levels near zero. Intriguingly, this person was found to be active and healthy. Rather than ignoring this as a medical curiosity, Drs. Hobbs and Cohen went on to identify a mutation in a protein called PCSK9 that rendered the protein inactive. Low levels of PCSK9 were tied to very low levels of cholesterol, one of the major causes of heart disease.

Immediately, the pharmaceutical industry seized upon this breakthrough to develop novel drugs that target PCSK9, lowering serum cholesterol levels and likely diminishing risk of atherosclerosis and heart attack. Now, three drug companies are testing new medicines that target PCSK9, and preliminary results are highly promising: Cholesterol levels drop precipitously and stay low when the drugs are delivered once or sometimes twice monthly.

Time will tell whether this novel strategy proves effective in reducing the risk of heart attack and stroke. Following on the heels of previous work to lower cholesterol – also performed at UT Southwestern – that ultimately led to the development of statins, these exciting developments are likely to provide robust benefit to patients around the globe.