A new study has found that people with obstructive cardiomyopathy, a common heart condition, were able to use significantly more oxygen while exercising after taking an investigational drug. The study was published today in the New England Journal of Medicine and the findings were presented at the European Society of Cardiology’s Heart Failure 2024 meeting in Lisbon, Portugal.
Oregon Hea rnrnlth & Science University is taking part in a randomized, double-blind Phase 3 trial to assess the effectiveness of the experimental drug aficamten, developed by Cytokinetics, in treating the obstructive form of hypertrophic cardiomyopathy (HCM). Out of the 282 adults in the trial, 19 were enrolled through OHSU, the highest number from any trial center.
“Patients with obstructive hypertrophic cardiomyopathy may find it easier to walk, perform daily household chores, and engage in other activities with the increased availability of oxygen during exercise,” explained cardiologist Ahmad Masri, M.D., M.S., who co-authored the paper and heads the OHSU Knight Cardi department.of the participants in the clinical trial conducted by the Cardiovascular Institute’s Hypertrophic Cardiomyopathy Center were given a new drug called aficamten, while the other half took a placebo. The results of the trial showed that aficamten could be a promising treatment for HCM, a condition that affects approximately 1 in 500 people and is a leading cause of sudden death in young and healthy athletes. HCM is often the result of inherited gene mutations, which cause the heart muscles to thicken and impede the heart’s ability to function properly. This can lead to symptoms such as shortness of breath and reduced exercise tolerance, while the obstructive form of HCM can also decrease blood flow out of the heart.In a study, participants were given a placebo and acted as the control group. Their oxygen levels were measured while they used treadmills or bicycles. Those who took aficamten showed a significant increase in their maximum oxygen use, with 1.7 milliliters per kilogram per minute more than those in the control group. Having a higher peak oxygen uptake can improve a patient’s ability to be physically active, while a lower oxygen uptake can increase the risk of heart failure, the need for a heart transplant, and mortality. Surgical options for obstructive HCM include removing excess heart muscle. In 2022, the Food and Drug Administration approved aficamten as the first drug treatment for obstructive hypertrophic cardiomyopathy.The Food and Drug Administration has recently approved mavacamten as the first drug specifically designed to address the root cause of obstructive HCM. However, it is important to note that mavacamten may potentially increase the risk of heart failure and can have interactions with commonly used medications. As a result, patients who are prescribed mavacamten must be closely monitored.
Over the past ten years, OSHU has actively been involved in various research studies aimed at discovering new treatment options for HCM. It has served as a key research center for numerous mavacamten studies and is also participating in gene therapy research. Additionally, the university is currently engaged in four other trials involving aficamten to assess its potential as a treatment option.The treatment for various types of HCM and in different patients, including children, is an exciting time, according to Masri. While traditional surgical and procedural therapies are still offered, there are now other treatment options available for patients. These include therapies recently approved by the FDA and investigational therapies available through clinical trials.
