Texas Heart Institute Recruiting Patients for Clinical Trial
to Address a Common Genetic Heart Disease

Incurable Hypertrophic Cardiomyopathy
a Leading Cause of Sudden Death in Young 

Houston, Texas (October 8, 2013)  – Doctors and researchers at the Texas Heart Institute (THI) are recruiting patients to participate in a clinical trial they hope will lead to a way to prevent and reverse hypertrophic cardiomyopathy (HCM), the most common genetic heart disease, and a common cause of sudden cardiac death (SCD) in young people, especially young athletes.  

HCM, which is genetically transmitted, causes a thickening of the heart muscle and causes the heart muscle to function abnormally. It can affect people of any age. Often, there are no symptoms in young people and the first sign of the undiagnosed disease can be sudden cardiac death (SCD). It can also cause disabling cardiac symptoms such as shortness of breath, chest pains, palpitations, and fainting. It is also an important cause  of cardiac complications in the elderly.   

Current medicines used to treat patients with HCM treat symptoms and reduce risk of SCD, but none reverse the disease.   

THI researchers, in a small randomized group of 75 patients over 12 months, will study use of the drug, N-Acetylcysteine (NAC), commonly used as an anti-oxidant and to treat cystic fibrosis patients because of its ability to break down mucus in the body. Previous animal models showed NAC helped reduce thickening of the heart muscle and fibrosis in heart muscle tissue, features of HCM, and improve cardiac dysfunction (diastolic dysfunction) which causes shortness of breath and exercise intolerance.   

"We believe this drug offers promise because it addresses at the molecular level certain changes in the cardiac proteins that are the consequences of the causal mutations present in patients with HCM," said the trial's principal investigator, Dr. A. J. Marian, a THI researcher and professor at The University of Texas Health Science Center in Houston.  

"It's a safe drug and we also hope to prove that it is also effective as a potential therapy. Given that there is no effective pharmacological therapy for HCM, we are excited and hope that our colleagues will share our enthusiasm in testing the beneficial effects of this safe medication in patients with HCM." 

For more information about the trial, see http://www.stlukeshouston.com/OurServices/Research/StudyDetail.cfm?PNO=HSC-MS-10-0533.

Director of Public Affairs
Texas Heart Institute
Frank Michel  ♦  832-355-9510  ♦  fmichel@texasheart.org 

For THI media profile, see Public Affairs.