0
0
In a groundbreaking development, individuals suffering from obstructive hypertrophic cardiomyopathy (HCM) may soon find relief in a novel investigational drug, according to findings published today in the New England Journal of Medicine and presented at the European Society of Cardiology’s Heart Failure 2024 meeting in Lisbon, Portugal.
Oregon Health & Science University (OHSU) played a pivotal role in this international clinical trial evaluating the efficacy of the experimental drug aficamten, developed by Cytokinetics. Dr. Ahmad Masri, co-author of the study and director of the OHSU Knight Cardiovascular Institute’s Hypertrophic Cardiomyopathy Center, expressed optimism about the drug’s potential. “By having more oxygen available during exercise, patients with obstructive hypertrophic cardiomyopathy can more easily walk, perform household chores, and do other everyday tasks,” Dr. Masri stated.
HCM, affecting approximately 1 in 500 individuals, is notorious for thickening heart muscles, impeding normal heart function, and limiting physical activity. The obstructive form of HCM further diminishes blood flow out of the heart, exacerbating symptoms such as shortness of breath and reduced exercise tolerance.
The randomized, double-blind Phase 3 trial enrolled 282 adults, with OHSU contributing the largest cohort of 19 participants. Half of the participants received aficamten, while the remaining half served as the control group receiving a placebo. Utilizing treadmills or bicycles, scientists measured oxygen levels during exercise. The results revealed a significant increase in maximum oxygen use among those administered aficamten, indicating a potential boon in physical activity.
Enhanced peak oxygen uptake not only augments patients’ ability to engage in physical endeavors but also mitigates risks associated with reduced oxygen uptake, such as heart failure and mortality.
While surgical intervention remains a conventional treatment option for obstructive HCM, recent years have witnessed the emergence of pharmaceutical solutions. In 2022, the FDA approved mavacamten as the first drug targeting the underlying cause of obstructive HCM. However, concerns regarding its potential side effects and interactions with other medications persist, necessitating vigilant monitoring.
OHSU has been at the forefront of HCM research, participating in various studies exploring innovative treatment modalities. The university’s involvement spans mavacamten trials, gene therapy research, and ongoing investigations into aficamten across different patient demographics.
Dr. Masri emphasized the transformative impact of these advancements, stating, “This is an exciting time for treating HCM. While we continue to offer traditional surgical and procedural therapies, we are now also able to offer patients other treatment options: therapies that were recently approved by the FDA and investigational therapies that are available by participating in clinical trials.”
With promising results from the aficamten trial, the landscape of HCM treatment appears poised for significant evolution, offering hope to countless individuals grappling with this debilitating condition.
