Treating Hearts at Risk for Cardiomyopathy: Leveraging Human iPSC Disease Models

Lay Summary: Hypertrophic cardiomyopathy (HCM) is the most common inherited heart disease. What makes this disease particularly scary is that it is impossible to predict how the disease will affect the lives of those born with an HCM mutation. Some carriers lead “normal” lives, with only mild symptoms later in life, while others are at risk of developing life-threatening complications during childhood. Camzyos^® is the first therapy that improves heart function and partially reverses cardiac remodeling associated with HCM. Unfortunately, Camzyos^® is currently only approved for adults with severe disease. We will use the funds provided by the Saving tiny Hearts Society to determine whether long-term low-dose Camzyos^® can correct contractility in human stem cell models of HCM. These results will lay the groundwork for future studies aimed at establishing if Camzyos^® can be used in children to prevent the development of life-threatening consequences of HCM.