Tag: cleveland clinic

First Patient Undergoes Gene Therapy for HCM

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Tenaya Therapeutics and the Cleveland Clinic today announced that the first patient in Tenaya’s groundbreaking gene therapy clinical trial for hypertrophic cardiomyopathy has been dosed. This trial is testing a groundbreaking therapy intended to treat HCM caused by the MYBPC3 gene which is one of the most common HCM genes.

The Phase 1b trial is looking at the investigational drug TN-201.  Preliminary results are expected in 2024.

You can find much more information about the clinical trial and Tenaya’s plans in this HCMBeat interview with Tenaya’s Executive Medical Director Laura Robertson M.D.

Myectomy Sooner?

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An article by doctors at the Cleveland Clinic recently published in the Journal of the American Heart Association advocates for earlier surgical intervention for patients with obstructive hypertrophic cardiomyopathy (HCM).   

According to this article, obstructed HCM patients who undergo myectomy earlier have better long term survival. Therefore, these doctors take the position that patients should not wait until they become severely symptomatic and/or have run out of medical options to undergo myectomy surgery. 

Meanwhile, an accompanying editorial by Dr. Mark Sherrid of NYU Langone Health is to the contrary.  Dr. Sherrid argues that medications like disopyramide (Norpace) are effective in reducing symptoms and that the inherent risks from open heart surgery are not outweighed by a theoretical improvement in longevity.

Regardless of the timing of surgery, Dr. Sherrid points out that with multiple companies now developing novel treatments for HCM, visibility of the disease will increase which will ultimately result in better patient outcomes for all with HCM. 

 

 

 

Dr. Harry Lever Speaks Out About Problems With Generic Drugs

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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” →

Cardiac MRI Helps Assess Sudden Death Risk

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A recent study by doctors at the Cleveland Clinic suggests that the presence of late gadolinium enhancement  (LGE) should be added to the various risk factors currently used to assess patients who are at low or intermediate risk of sudden death.  The presence and balancing of these risk factors are used by patients and doctors to determine the need for implantable cardiac defibrillators (ICDs).   LGE is an indication of cardiac scar tissue and can be seen on cardiac MRI scans.  This study recommended that LGE comprising a total of 15% or more of left ventricular mass be used as an additional risk factor. The study found that this indicator worked equally well when applied to both obstructed and non-obstructive HCM patients.

Interestingly, an earlier but recent study published by Cleveland Clinic doctors found that the risk factors currently in use to determine the need for an ICD fall short as applied to patients with the obstructive form of HCM.

Risk factors in common use today have been propounded by the American College of CardiologyAmerican Heart Association (ACC/AHA) in the U.S., while a different set of guideline and a mathematical risk calculator was promulgated more recently in Europe by the European Society of Cardiology (ESC).  You can find more about the ACC/AHA and ESC guidelines here.

A second and related finding of this study by the Cleveland Clinic, known for its large HCM program and high volume of myectomies, was that patients who undego  myectomy appear to experience a protective effect from their surgeries.  Even when found to have 25% or more LGE, patients in this study who previously underwent myectomy experienced a lower than expected rate of adverse events.