Safe New Therapy For Genetic Heart Disease

Main Category: Heart Disease
Also Included In: Genetics
Article Date: 02 Jan 2009 - 3:00 PDT

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A new clinical trial suggests that long-term use of candesartan, a drug currently used to treat hypertension, may significantly reduce the symptoms of genetic heart disease. The related report by Penicka et al, "The effects of candesartan on left ventricular hypertrophy and function in non-obstructive hypertrophic cardiomyopathy: a pilot, randomized study," appears in the January issue of The Journal of Molecular Diagnostics.

Hypertrophic cardiomyopathy, or HCM, is a genetic heart disease where the heart muscle is thickened, especially in the left ventricle. Although people with HCM usually display mild symptoms or are completely asymptomatic, up to 1% of affected people succumb to sudden cardiac death (SCD), often with no previous signs of illness. HCM can be caused by mutations in a number of different genes, and different gene mutations may result in more or less severe symptoms.

Researchers lead by Dr. Jiri Krupicka of Na Homolce Hospital, Prague conducted a double-blind, placebo-controlled, randomized study on the long-term administration of candesartan in patients with HCM. Dr. Krupicka's group found that candesartan reduced the symptoms of HCM, including decreasing the thickening of the left ventricle. This effect was found to be dependent on the underlying gene mutations in each patient. They did not observe any adverse effects of candesartan use over the course of their study.

These data suggest that effectiveness of angiotensin II receptor blockers, such as candesartan, on HCM may vary depending on the nature of the causative mutation and that the treatment protocol, therefore, should be customized to individual patients. Future studies in Dr. Penicka's group will extend this pilot trial to more patients to confirm the current findings and to identify the mechanism by which candesartan improves left ventricle thickening.

This study was supported partly by the Charles University Prague Research Project MSM and partly by the Czech Ministry of Health.

Penicka M, Gregor P, Kerekes R, Marek D, Curila K, Krupicka J: The effects of candesartan on left ventricular hypertrophy and function in non-obstructive hypertrophic cardiomyopathy: a pilot, randomized study. J Mol Diagn 2009 11:35-41

The Journal of Molecular Diagnostics, the official publication of the Association for Molecular Pathology, co-published by the American Society for Investigative Pathology, seeks to publish high quality original papers on scientific advances in the translation and validation of molecular discoveries in medicine into the clinical diagnostic setting, and the description and application of technological advances in the field of molecular diagnostic medicine. The editors welcome for review articles that contain: novel discoveries or clinicopathologic correlations including studies in oncology, infectious diseases, inherited diseases, predisposition to disease, or the description or polymorphisms linked to disease states or normal variations; the application of diagnostic methodologies in clinical trials; or the development of new or improved molecular methods for diagnosis or monitoring of disease or disease predisposition.

The Journal of Molecular Diagnostics