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!
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.
While competitive sports used to be frowned upon in the HCM literature, there is now some evidence that a patient’s risk from exercise is low when they have been implanted with an implantable cardioverter defibrillator (ICD). This feature from Medpage Today gives an overview of contemporary thinking about exercise in HCM patients.
Continue reading “Sports and HCM – Moving Toward Shared Decision Making”
Berritt Haynes, a 19 year old with hypertrophic cardiomyopathy, was chosen for Team Blake Shelton last night on the MGM Television/NBC show THE VOICE. After Berritt’s mother submitted a tape, Berritt was chosen to audition on stage in front of the coaches.
Berritt had hoped to attend a taping of the show last season through Make-A -Wish Foundation which grants wishes to kids with serious health issues. However, due to COVID, he was not able to make that happen. Instead, this year his mother helped make his dream come true by making it possible for him to actually perform.
Berritt was diagnosed with HCM when he was 8 and received an implantable defibrillator when he was 14. Clearly, Berritt’s HCM has not interfered with his performing talents. Watch him performing on The Voice here.
Good luck Berritt. All of the HCM world will be rooting for you to advance to the next round!
Read more at:
THE VOICE can be seen on NBC on Monday and Tuesday nights. Check your local schedule for times.
A recent study published in the European Journal of Preventative Cardiology found that a 12-lead electrocardiogram (EKG) was not useful as a screening tool to determine which children were at increased risk of sudden death and therefore, a candidate to receive an implantable defibrillator.
The full article can be found here.
In this study just published in the Heart Rhythm journal of the Heart Rhythm Society, doctors demonstrated that an Apple iPhone 12 (which is built with more magnets than previous iPhone models due to the Mag Safe charging technology) can disable the ability of an implantable defibrillator to deliver therapy.
So, this is a reminder for ICD patients that according to all device manufacturers, in order to avoid a problem, keep your phone at least 6 inches away from your device, and remember that you should NEVER PUT YOUR PHONE (ANY PHONE, NOT JUST AN IPHONE 12) IN YOUR SHIRT POCKET!
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 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.
According to a paper published last week in JAMA Cardiology, doctors at Tufts University’s HCM Center have been able to identify 95% of their patients at high risk of sudden cardiac death (SCD) from HCM. Tufts applied an updated and modified version of the risk factors enumerated in the American College of Cardiology/American Heart Association Guidelines promulgated in 2011.
Continue reading “Docs Reliably Identify HCM Patients in Need of ICDs”