Category: News

Lisa Marie Presley Dies of Cardiac Arrest at Age 54

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Very sad update to the news about Lisa Marie Presley. RIP. This family has had far too much grief.

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All major news outlets reporting that Lisa Marie Presley has died this afternoon after suffering cardiac arrest at her home this morning.  She was resuscitated at home, and then transported to a local hospital where she passed away.

It has previously been reported that Elvis himself carried a HCM causing gene.   Elvis’ genetic analysis was featured in a program by U.K.’s Channel 4 Television in 2014.  Elvis’ mother also died before age 50 of a “heart attack.”

Sending deepest condolences to Priscilla Presley, daughters Riley Keough, Harper Lockwood and Finley Lockwood.

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Tenaya Therapeutics Gets Go-Ahead for HCM Gene Therapy Trial

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Tenaya Therapeutics announced on Monday that they have received FDA clearance to begin a Phase 1 clinical trial of targeted gene therapy for HCM.

Tenaya is developing TN-201, a first in class adeno-associated virus based therapy being developed to treat HCM caused by mutation(s) in the MYBPC3 gene. They anticipate that the trial will begin in the third quarter of 2023. The therapy delivers one fully functional MYBPC3 gene to the patient via injection with a deactivated virus. Tenaya hopes that this therapy will restore normal levels of the MYBPC3 protein, thereby halting disease progression, and even potentially reversing the course of the disease, after just a single treatment.

The TN-201 Phase 1b clinical trial will be a multi-center, open-label study designed to assess the safety of an intravenous infusion of TN-201. They hope to enroll at least 6 symptomatic, non-obstructive HCM patients who carry the MYBPC3 gene and who already have received an automatic implantable cardioverter defibrillator (ICD) as part of their treatment plan to date.

You can read the full  press release here.

Stay tuned to HCMBeat for updates!

Results Published from REDWOOD-HCM Phase 2 Trial

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Results from Cytokinetics’ REDWOOD-HCM Phase 2 clinical trial of the experimental drug aficamten were just published in the Journal of the American College of Cardiology. 

Aficamten, a second generation myosin inhibitor, was administered to a small group of 28 patients as part of a phase 2 trial to determine safety of the drug of the drug in obstructive HCM over a 10 week period. 

No safety issues were found, while most trial participants experienced substantial reductions in left ventricular outflow tract gradients. Study participants also reported improvement to their symptoms, while blood tests showed improvement to certain biomarkers of heart stress.   

You can read more about the drug aficamten and Cytokinetics, the company behind it, in the below posts on HCMBeat. Next for aficamten is the Phase 3 trial entitled SEQUOIA-HCM.

Promising Data about Aficamten Presented at Meetings

2 Companies Testing Drugs for HCM

HCM Clinical Trials – the Latest News

The Future of HCM Care

Positive Signs from REDWOOD-HCM

Cytokinetic’s Drug Aficamten & Upcoming HCM Summit – Interview with Dr. Martin Maron

Aficamten Gets “Breakthrough Drug” Status from FDA

More on Aficamten & Mavacamten from ACC 2022

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

Cytokinetics Moves Forward with HCM Drug Trial

Cytokinetics Announces Positive Results from REDWOOD-HCM Phase 2 Clinical Trial

LGE Seen on MRI Top Risk Factor for HCM

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To date, risk assessment in patients with hypertrophic cardiomyopathy (HCM) has largely focused on whether there is a need for an implantable defibrillator by evaluating an individual patient’s short term risk of sudden death, but has not looked at the long term risks of heart failure and other adverse outcomes over time.

A recent retrospective study by European HCM specialists asked the question of whether the addition of advanced imaging could assist in pinpointing patients at high risk of heart failure and/or other serious HCM complications over time. 

Continue reading “LGE Seen on MRI Top Risk Factor for HCM” →

HCM Genes Common in General Population

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A recent study sought to find how common genetic cardiomyopathies are in the general population. 

By studying United Kingdom Biobank samples from 200,643 people who had undergone complete genetic sequencing, researchers established that genes linked to HCM are found in one out of every 149 people! 

More than half of those identified as having a HCM gene had a mutation in the MYBPC3 gene, making it the most common HCM gene in this sampling.

Mavacamten Approved in Canada

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Bristol Myers Squibb’s first in class drug  mavacamten (brand name Camzyos) has been approved by Health Canada for the treatment of obstructive hypertrophic cardiomyopathy.

Longer Term Results for Mavacamten

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The VALOR-HCM trial  was a Phase 3 clinical trial which compared the Bristol Myers Squibb drug mavacamten (brand name Camzyos) to septal reduction therapy (myectomy and septal alcohol ablation) over a 16 week period.  At the end of the 16 week period, the patients originally randomized to the placebo were prescribed mavacamten, while those originally prescribed mavacamten continued taking the drug for an additional 16 weeks for a total of 32 weeks

At last weekend’s American Heart Association meeting in Chicago and simultaneously published in Circulation, lead investigator Dr. Milind Desai of the Cleveland Clinic announced that after 32 weeks of mavacamten treatment, patients continued to see positive cardiac remodeling, as well as improvement to left ventricular outflow tract gradient, diastolic function and quality of life. This ultimately allowed a whopping 88% of patients enrolled in the trial to avoid a septal reduction procedure. 

Similar results were noted in those patients who began mavacamten after an initial 16 weeks on a placebo.

This is good news for HCM patients!

The Future of HCM Treatment

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Doctors from the University of Pennsylvania this week published an overview of current therapies available for the treatment of HCM.  While this article mentions all of the old standards – beta blockers, calcium channel blockers, myectomy and alcohol ablation, the real focus is on the future of HCM treatment. In particular, the article describes several new and/or experimental therapies which look promising for the future.

Continue reading “The Future of HCM Treatment” →

Ranolazine Improves Chest Pain in HCM

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A recent paper by Italian HCM specialists found that chest pain completely resolved for 73% of patients who took the drug ranolazine. The researchers also found that the drug was safe and well tolerated.  In addition, it reduced arrhythmias for those whose arrhythmias were triggered by ischemia and it improved biomarkers in HCM patients.

This study is a follow up to the 2018 RESTYLE-HCM study, which found that while ranolazine did not improve heart failure symptoms in HCM, it was useful for treating chest pain.

Apical Aneurysms in HCM

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A recent paper published by doctors at Canada’s Peter Munk Cardiac Center looked at the implications of left ventricular apical aneurysms in hypertrophic cardiomyopathy,  This paper recommends that patients with an apical aneurysm of at least 2 centimeters consider prophylactic anticoagulant therapy to prevent stroke, as well as considering surgical placement of an implantable defibrillator to protect against sudden cardiac death.

A companion editorial suggests caution in basing aggressive measures on an aneurysm alone and says that all risk factors be considered together.

In 2017, HCMBeat covered a retrospective study of patients treated at Minneapolis Heart Institute and Tufts which discussed the risks for patients with apical aneurysms.  That can be found here.