Several years ago, researchers from the University of Virginia (UVA) and the University of Oxford announced a joint project involving a large international registry of hypertrophic cardiomyopathy (HCM) patients to facilitate research into HCM. Backed with funding from the National Heart Lung and Blood Institute, this project, known as the HCM Registry, includes data from 2,750 patients with HCM at 44 sites in six countries.
This week, researchers from UVA announced their first findings from this registry which suggest that HCM patients can be separated into two basic groups:
- Patients with a known genetic mutation who are not obstructed but have scarring of the heart muscle;
- Patients who do not have a known genetic mutation and do not exhibit scarring, but who do have a significant amount of obstruction to blood flow.
According to Dr. Christopher Kramer of UVA, this information should provide doctors with information that allows them to better assess the degree of risk to any particular patient, and to help inform a treatment strategy for each patient based on his or her unique profile.
This Medscape article highlights the extraordinary efforts of Dr. Harry Lever, Director of the Cleveland Clinic’s Hypertrophic Cardiomyopathy Center, in educating patients and physicians alike about quality issues with generic drugs. Dr. Lever has been instrumental in publicizing the fact that generic drugs are NOT always the same as their name brand counterparts, and that foreign generics are not put through the same level of scrutiny as drugs in the U.S.
Continue reading “Dr. Harry Lever Speaks Out About Problems With Generic Drugs”
A recent study by Canadian researchers published in the American Heart Association journal Circulation found a much lower incidence of HCM related sudden death than expected. The study included deaths in individuals that occurred between the ages of 10 and 45 in the Canadian province of Ontario between 2005 and 2016. According to lead author Dr. Paul Dorian of the University of Toronto, the expectation was that 1 incident of sudden cardiac death would be identified in every 100 to 200 people who had HCM. Instead, researchers found that the likelihood of sudden death of HCM patients was instead only about 1 in 3,000 people/year.
The study also found that found 7 in 10 HCM-related sudden deaths occurred in people not previously diagnosed with the condition. Men had more than 5 times the risk of sudden cardiac death than women, and most deaths occurred during rest or light activity: only about 17% happened during or immediately after exercise.
As a result of the study, according to Dr. Dorian, “our findings allow us to lower the temperature on our degree of worry about the condition.”
Another clinical take away from the study, according to the researchers, is that current exercise guidelines for HCM patients may be too restrictive.
NEWS FROM MYOKARDIA
MyoKardia recently announced a new clinical trial of its drug, mavacamten (formerly known as MYK-461) which will compare the clinical results of mavacamten with septal reduction therapies currently used in clinical practice, i.e. the open heart surgical procedure known as septal myectomy and the catheter based procedure known as alcohol septal ablation.
The study will be run by the Cleveland Clinic with Dr. Milind Desai serving as principal investigator. MyoKardia expect to begin enrolling patients in early 2020.
Read the press release here.
NEWS FROM CYTOKINETICS:
Cytokinetics released positive data from its Phase 1 study of the drug currently known as CK-274 in a poster session at the HFSA 23rd Annual Scientific Meeting in Philadelphia. The study found that CK-274 was safe and well tolerated, while no serious adverse events or negative changes to vital signs, ECGs or laboratory tests were observed.
The company will now be moving into a Phase 2 clinical trial of CK-274 in patients with obstructive hypertrophic cardiomyopathy, expected to begin in late 2019.
Read their press release here.
DISCLOSURES: HCMBEAT HAS RECEIVED PAST UNRESTRICTED EDUCATIONAL GRANTS FROM MYOKARDIA. ADDITIONALLY, CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVES AS A PATIENT ADVISOR ON THE STEERING COMMITTEE FOR MYOKARDIA’S EXPLORER TRIAL.
Recently, Cynthia Waldman of HCMBeat corresponded with Dr. Srihari S. Naidu of Westchester Medical Center the second edition of an HCM textbook he recently edited, as well as about medical education surrounding hypertrophic cardiomyopathy in general. What follows is a transcript of their correspondence (which has been slightly edited for readability).
Continue reading “Dr. Srihari Naidu Talks About HCM Medical Education”
This informative blog article written by a social worker for the University of Michigan’s Health Blog has some great tips on how to deal with anxiety when living with an ICD.
I recommend it.
Two San Francisco based companies are now conducting clinical trials for three drugs specifically targeting HCM.
Continue reading “2 Companies Testing Drugs for HCM”
A recent study by doctors at Toronto’s Hospital for Sick Children suggests that current screening guidelines for children from HCM families are inadequate and should instead recommend earlier screening exams. In the U.S., screening begins at age 12 pursuant to American College of Cardiology (ACC)/American Heart Association (AHA) guidelines. In Europe, screening begins at age 10 pursuant to the European Society of Cardiology (ESC) guidelines.
Continue reading “Should Children from HCM Families be Screened Earlier?”
A group of scientists led by Stanford University’s Dr. James Spudich, working together with researchers from the University of California-Santa Barbara, the University of Washington and the Institut Curie in Paris, has recently been awarded a $10 million grant by the National Institute of General Medical Sciences to develop novel treatments for hypertrophic cardiomyopathy (HCM).
The researchers hope that the added resources from this grant will help them find ways to correct pathological heart protein changes they believe to be at the root of HCM. The team then plans to partner with pharmaceutical companies to develop more personalized approaches to HCM treatment.
Dr. Spudich has long been involved in HCM research and has been a founder of two separate companies which are currently engaged in drug trials for potential HCM treatments: MyoKardia and Cytokinetics.
A story about Dr. Spudich and the inspiration for his work was featured in this recent post on HCMBeat.