"A yearold heart patient with dangerously high levels of cholesterol that could not be adequately reduced by statin drugs now has nearnormal cholesterol levels thanks to a new class of drugs that grew out of work done by UT Southwestern Medical Center researchers.  Two of these drugs in a category known as PCSK inhibitors were approved by the Food and Drug Administration last summer for use by some individuals with extremely high cholesterol levels.  If you take the core patients who are at highest risk it makes you appreciate how important this drug class is said Dr. Amit Khera httphttpprofiles.utsouthwestern.eduprofile Director of the Preventive Cardiology Program and Associate Professor of Internal Medicine at UT Southwestern.  Frank Brown of Dallas grandfather of six and the owner of Franks Wrecker Service in Dallas has familial hypercholesterolemia an inherited condition that causes high levels of cholesterol especially lowdensity lipoprotein LDL cholesterol or bad cholesterol. High levels of LDL cholesterol are strongly associated with heart disease. Mr. Brown with a history of two heart attacks had been aggressively treated with multiple drugs to reduce his cholesterol levels but they remained stubbornly high. When I first met Mr. Brown he had a strong family history of heart disease he had a cholesterol level that was ridiculously high with an LDL of  and he was having chest pains said Dr. Amit Khera who is Mr. Browns cardiologist. Dr. Khera who holds the Dallas Heart Ball Chair in Hypertension and Heart Disease at UT Southwestern was treating Mr. Brown with three cholesterollowering medications a statin which is a class of drugs that works by blocking a substance the body needs to make cholesterol ezetimibe a drug that blocks absorption of cholesterol in the intestine and colesevelam which sequesters bile acids. Even with this trio of medicines Mr. Browns LDL cholesterol level hovered around .  The two PCSK inhibitors approved by the FDA last year were developed as a result of research done by UT Southwestern geneticists Dr. Helen Hobbs httphttpprofiles.utsouthwestern.eduprofile and Dr. Jonathan Cohen httphttpprofiles.utsouthwestern.eduprofile. Using data from the Dallas Heart Study a populationbased study that gathered extensive medical data on  Dallas residents the two researchers showed that certain mutations to the gene that codes for the protein PCSK lead to low levels of cholesterol in the blood. FurtherDr. Hobbs and Dr. Cohen found a woman who had inherited not one but two of these mutations in the PCSK gene  one mutation from each parent. This woman had stunningly low levels of LDL cholesterol. While anything below  is considered good her LDL cholesterol level was just . Crucially this woman was in good health suggesting that therapies aimed at blocking PCSK would not only be effective but also safe.  Their research led to the development by drug companies of evolocumab and alirocumab the two approved PCSK inhibitors which are delivered by monthly or semimonthly injections. Dr. Hobbs Director of the Eugene McDermott Center for Human Growth and Development holds the Eugene McDermott Distinguished Chair for the Study of Human Growth and Development the Philip OBryan Montgomery Jr. M.D. Distinguished Chair in Developmental Biology and the  Dallas Heart Ball Chair in Cardiology Research. Dr. Cohen Professor of Internal Medicine and with the McDermott Center holds the C. Vincent Prothro Distinguished Chair in Human Nutrition Research. Mr. Brown had been part of a clinical trial testing the safety and effectiveness of one of the new PCSK inhibitors and when the drugs were approved Dr. Khera began working to get Mr. Brown approved by his insurance to be on the new regimen.  In the world of cholesterol patients most are wellcontrolled with statins and they should stay on those said Dr. Khera. But Mr. Brown is the perfect patient for PCSK inhibitors. We knew he could tolerate it because hed been in a clinical trial he knew how to administer the injections and he was very highrisk. Wed exhausted the other options in trying to control his cholesterol. Two months after he started his biweekly injections of the new drug Mr. Browns LDL cholesterol level was down to   not quite normal which is  or less  but an almost  percent reduction from the level it had been for years.  Its a good sign for Mr. Brown who looks forward to many more years of running his business watching Dallas Cowboys games and spending time with his grandchildren.  There are many more Frank Browns out there  patients who cant control their cholesterol with the standard drugs said Dr. Khera. Its wonderful to have this option to offer this special set of patients. About UT Southwestern Medical CenterUT Southwestern one of the premier academic medical centers in the nation integrates pioneering biomedical research with exceptional clinical care and education. The institutions faculty includes many distinguished members including six who have been awarded Nobel Prizes since . The faculty of almost  is responsible for groundbreaking medical advances and is committed to translating sciencedriven research quickly to new clinical treatments. UT Southwestern physicians provide medical care in about  specialties to more than  hospitalized patients and oversee approximately . million outpatient visits a year.  This news release is available on our home page at http To automatically receive news releases from UT Southwestern via email subscribe at  http"