AHA Seeking Patient Input for New HCM Initiative

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.

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Biventricular Pacemaker For Non-Obstructed HCM Patients?

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.

Patients Fare Best at HCM Specialty Centers

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.

The Future of HCM Care

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.

Drugs:

  • 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.

 

Arrhythmia Management:

 

  • 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 Announces its Phase 2 Clinical Trial – “Redwood-HCM”

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.

 

Racial Differences in HCM

A recent study  found that African-American patients with HCM tend to be younger and more symptomatic than their white counterparts. Additionally, these patients are less likely to undergo septal reduction therapies or have genetic testing.

The implications, according to Dr. Neal Lakdawala of Brigham and Women’s Hospital who is an author of the study, is that doctors should always consider the possibility of hypertrophic cardiomyopathy in patients with left ventricular hypertrophy.  If diagnosed with HCM, these patients should be referred to specialists with experience in treating HCM.  This could potentially help these patients avoid the two most devastating complications of HCM:  sudden death and stroke.

 

More HCM Research is Needed

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.

Dr. Harry Lever Speaks Out About Problems With Generic Drugs

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.

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Dr. Srihari Naidu Talks About HCM Medical Education

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).

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2 Companies Testing Drugs for HCM

Two San Francisco based companies are now conducting clinical trials for three drugs specifically targeting HCM.

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