Testing new treatments for hypertrophic cardiomyopathy

Defining therapeutic targets in hypertrophic cardiomyopathy (renewal)

Professor Hugh Watkins and his team at the University of Oxford have had a key role in understanding how certain gene faults can lead to hypertrophic cardiomyopathy (HCM). HCM is a disease of the heart muscle where the muscle wall becomes thickened and stiff. It is caused by an alteration in one or more genes and can be passed on through families. Each child of a person with HCM has a 50% chance of inheriting a genetic fault that means they will develop the condition. The BHF has awarded the Oxford researchers a grant to study three aspects of the disease process in mice and test new treatment approaches for each. Firstly, they will test a drug called DCA to see if it can correct abnormal energy supply. Secondly, they will test a class of molecule derived from green tea to see if it can correct the way the heart muscle cells use calcium. Thirdly, they will try to remove or correct the underlying faulty gene alteration itself. This work may reveal completely new types of treatment for HCM or other types of cardiac hypertrophy, either on their own or in combination with existing drugs. If they are shown to be effective in animals, two of the approaches they are exploring could quickly be tested in people. Ultimately they may be used to prevent the disease developing in family members who carry alterations in genes that cause HCM.