China Achieves Breakthrough in Hypertrophic Heart Disease Treatment

Chinese researchers have achieved major advancements in the treatment of hypertrophic cardiomyopathy (HCM), a global health issue since the late 1950s. Their pioneering work has attracted international acclaim.

China Achieves Breakthrough in Hypertrophic Heart Disease Treatment
Chinese scientists have achieved notable progress in tackling hypertrophic cardiomyopathy (HCM), a global health issue since the late 1950s. Their advancements have drawn significant international attention and acclaim.

For nearly forty years, the medical community has lacked specific drug treatments, with available HCM medications only managing symptoms instead of addressing the underlying cause of myocardial hypertrophy, according to professor Xie Xiaojie from the Department of Cardiology at the Second Affiliated Hospital, School of Medicine, Zhejiang University.

Adolescents and young patients often experience more severe HCM symptoms and face a higher risk of sudden cardiac death. This condition is a leading cause of fatal cardiac events in individuals under 35. As the disease progresses, it can lead to complications such as heart failure, arrhythmia, and stroke, severely impacting the well-being of patients and their families, Xie added.

Xie stated that although invasive procedures like surgery or interventional therapy can be effective, they are complex, carry risks, and are suitable for only a limited patient population. The development of new drug therapies has been a critical unmet need in the field, Xie added.

A major breakthrough occurred this year with the approval of Mavaket capsules by China's State Food and Drug Administration. This approval marks a global first, as Mavaket is the world's first selective cardiac myosin allosteric inhibitor. The drug provides new hope for adult patients in China suffering from obstructive hypertrophic cardiomyopathy.

Xie noted that Mavaket's mechanism of action is similar to targeted cancer therapies. It selectively reduces the ATPase activity of cardiac myosin heavy chain, effectively decreasing excessive myosin-actin cross-bridge formation.

This mechanism promotes a shift in the myosin group to an energy-conserving super-relaxed state, ultimately inhibiting excessive myocardial contraction and improving diastolic compliance and energy metabolism. The approval of this innovative drug is regarded as a significant advancement in the treatment of hypertrophic cardiomyopathy.

Xie further explained that HCM can be categorized into obstructive (HOCM) and non-obstructive types. Common symptoms include shortness of breath, chest pain, and fainting, which can often resemble other heart conditions, leading to overlooked or misdiagnosed cases.

Recognizing its significance, Mavaketel has been included in several major clinical guidelines, such as the "Chinese Hypertrophic Cardiomyopathy Guidelines 2022," "2023 Guideline for Diagnosis and Treatment for Chinese Adult Patients with Hypertrophic Cardiomyopathy," "2023 ESC Guidelines for the Management of Cardiomyopathies," and "2024 AHA/ACC Guideline for the Management of Hypertrophic Cardiomyopathy."

This broad endorsement has solidified Mavaketel's position as a preferred treatment option for patients with HOCM.

Additionally, Chinese researchers have introduced a beating-heart myectomy system and have developed a minimally invasive procedure to treat HOCM.

The research was conducted by a team led by Wei Xiang, a professor at the Tongji Hospital in Wuhan, central China's Hubei Province, and was published in the Journal of the American College of Cardiology.

It is estimated that there are about 2 to 5 million HOCM patients in China, with the global population ranging from approximately 15 to 20 million, posing a serious threat to human health.

Currently, the standard treatment for HOCM involves open-chest surgery to remove the hypertrophic myocardium. This method is highly invasive, technically challenging, and associated with suboptimal outcomes and high mortality rates in hospitals with less experience.

The novel transapical beating-heart septal myectomy procedure has overcome the limitations of traditional open-chest methods. It allows for the precise resection of hypertrophic myocardium without the need for chest opening, cessation of the heartbeat, or blood transfusion.

Wei was recently invited to the Mayo Clinic, a prestigious cardiovascular treatment center in the United States, to discuss this groundbreaking medical advancement. Cardiac surgeons from countries such as the United States, Switzerland, and Italy have also visited China to learn about the technology.

"In the field of HOCM surgery, we used to follow the world, but now we are keeping pace with it or even leading," Wei said.

(With input from Xinhua)

Thomas Evans contributed to this report for TROIB News