In November at the American Heart Association’s Scientific Sessions held in Philadelphia, AHA announced that it would be starting a three-year initiative focused on hypertrophic cardiomyopathy, also known as HCM. The initiative is sponsored by MyoKardia, a San Francisco based company that is currently in clinical trials for mavacamten, the first drug specifically intended to treat HCM.
Last week, Cynthia Waldman of HCMBeat had the opportunity to speak with Amy Schmitz, AHA’s National Corporate Relations Director and Alexson Calahan, a Communication Manager for AHA.
What follows is a summary of their conversation about the forthcoming HCM initiative that has been edited for clarity.
Continue reading “AHA Seeking Patient Input for New HCM Initiative”
A small study of 29 patients conducted recently in the U.K. found that the use of a biventricular pacing in patients with non-obstructive hypertrophic cardiomyopathy improved symptoms of breathlessness and improved exercise capacity as demonstrated during oxygen consumption testing.
Medications are the only treatments currently available to non-obstructed patients. The authors of this study hypothesized that biventricular pacing could be a viable way to address exercise limitations in non-obstructed patients if medications have been ineffective.
Larger trials may establish biventricular pacing as a viable treatment for non-obstructed patients in the future.
A recent retrospective study conducted by doctors at Yale -New Haven Health System found that patients with hypertrophic cardiomyopathy fare best when treated at a specialty center using a team approach to HCM. The study found that this was especially true for patients coming from disadvantaged backgrounds who often fare worse outside of a HCM specialty setting.
The findings of this study suggest that patients with HCM are best served when referred to HCM specialty care instead of receiving care solely from general cardiologists.
Dr. Stephen Heitner, together with his colleagues at Oregon Health & Sciences University, published an article last week in the European Journal of Heart Failure which gives a glimpse into the treatment of hypertrophic cardiomyopathy (HCM) in the future. Although recent publications have stated that the majority of HCM patients today have a favorable prognosis when receiving appropriate treatment, a heavy disease burden continues to be placed upon patients. Hence, better and more effective treatments for HCM are still needed in order to lessen this burden.
The article provides a very thorough overview of both current and future HCM therapies and divides them into a few different categories listed below.
- Potential for New Drugs: No drugs have been specifically approved for the treatment of HCM by the Federal Drug Administration. Up until now, all drugs used to treat HCM patients (with the limited exception of propanolol) have been off-label uses. However, this is likely to soon change. Current trials of myosin modulators like MyoKardia’s mavacamten and Cytokinetic’s CK-274 have so far yielded promising results and have the potential to change the treatment paradigm for HCM.
- Subcutaneous ICDs (S-ICDs) are sometimes a reasonable choice for preventing sudden cardiac death in HCM patients when pacing is not required. This allows the patient to avoid risks associated with lead malfunction.
Septal Reduction Therapy:
Gene Editing and Silencing:
- Pre-implantation Genetic Diagnosis (PGD): Current medical technology allows patients with known HCM gene(s) to use pre-implantation genetic diagnosis of embryos together with in-vitro fertilization in order to avoid passing along the HCM gene to the embryo. The future may soon see these methods being used in tandem with gene repair, using CRISPR/Cas9 in order to edit out the errant gene and replace it with a normal one.
- Gene Silencing: Allele specific gene silencing may also prove to be a technique used in the future for preventing HCM. This technique involves the introduction of an adenovirus containing a short RNA segment designed to turn off the HCM gene.
In conclusion, this paper highlights the many things that HCM patients have to be optimistic about going forward. Perhaps one day soon one of these methods will lead to the ultimate goal – a cure!
Cytokinetics today announced that its Phase 2 double-blind study of its experimental drug CK-274 entitled “REDWOOD-HCM” (Randomized Evaluation of Dosing With CK-274 in Obstructive Outflow Disease in HCM (hypertrophic cardiomyopathy) has begun enrollment. The trial will enroll patients with symptomatic, obstructive HCM.
CK-274 is a next-generation cardiac myosin inhibitor which the company hopes will prove to be beneficial for the treatment of hypertrophic cardiomyopathy (HCM).
There are currently two companies in clinical trials for HCM: Cytokinetics and MyoKardia. You can read more about their efforts here and here.
A recent study in Europe found that HCM patients’ risk of death continues to exceed the risk in the general population.
This study looked at 4893 patients with hypertrophic cardiomyopathy treated at 7 different European HCM centers between 1980 and 2013. Although the statistics improved for those who were treated more recently, this study makes it clear that there is still much room for improvement in risk stratification and treatment for patients with HCM.
Here is an informative new video from our friend Doug Rachac that nicely explains the safety of MRIs for patients with implantable defibrillators and pacemakers.
I wrote a blog piece about this same issue a few years back. Here it is:
Yes We Scan! ICDs and MRIs
And a few other relevant blog entries here on HCMBeat:
Study Shows MRIs Safe for Pacemaker & ICD Patients
Chapter 3: MRI Safety for ICD & Pacemaker Patients
Safety of MRIs With Abandoned Leads
Last year, Doug wrote this blog entry for HCMBeat specifically about magnets and airports. Read that here:
Blogger Doug Rachac – Magnets and Airports: Should ICD Patients Be Afraid?
And, you can find more about ICDs from Doug on his YouTube Channel.
This week, researchers from the eight HCM centers comprising the Sarcomeric Human Cardiomyopathy Registry [SHARE Registry] published a paper that every HCM patient should take to heart.
The sobering findings are that overweight HCM patients have a higher incidence of obstruction, heart failure and atrial fibrillation than their normal weight counterparts. As a result of this study, the researchers suggest heightened attention to weight management and exercise in order to prevent disease-related progression and complications.
Continue reading “Overweight HCM Patients Fare Worse”