According to this recent study looking at exercise capacity in patients with HCM, women with HCM demonstrated reduced exercise capacity when compared to men. This paper theorizes that the differences are likely attributable to passive diastolic properties and that these could aid in the development of interventions specifically targeted for women.
Cytokinetics has announced that its experimental drug aficamten, currently in trials as a potential treatment for obstructive hypertrophic cardiomyopathy, has received breakthrough therapy designation from the FDA. This designation is awarded by the FDA to certain drugs which may offer substantial improvement to patients over available therapies. The designation could shorten the FDA approval process for the drug by about 4 months.
You can read more about Cytokinetics and aficamten in these older posts from HCMBeat:
Bristol Myers Squibb has launched a new hypertrophic cardiomyopathy awareness campaign and website entitled “Could it be HCM?” The campaign launch is in connection with the expected early 2022 FDA approval for the first-in-class cardiac myosin inhibitor drug mavacamten,
A video made for the campaign features professional basketball player Jared Butler of the Utah Jazz. In the video, Butler shares his surprise and dismay when he learned of his HCM diagnosis. Butler was fortunate that he was cleared to play basketball by his doctors at the Mayo Clinic who continue to follow him closely. He was even featured in People Magazine talking about his HCM. See also this article in the Salt Lake Tribune.
Check it out!
On October 22, patients, physicians, and other interested parties will have the opportunity to provide input on the value and cost of mavacamten – the first drug specifically designed to treat hypertrophic cardiomyopathy. Last year, Bristol Myers Squibb paid $13.1 billion to acquire MyoKardia, the San Francisco biotech company that developed the drug and brought it through clinical trials.
At a virtual public meeting, The Institute for Clinical and Economic Review or ICER will listen to further testimony in order to evaluate mavacamten’s value and potential benefits. ICER is a non-profit organization that evaluates the cost effectiveness of drugs and medical procedures. Many insurance companies rely on ICER’s findings when deciding how much to pay for a certain treatment or test.
In an Effectiveness Report which was published today, ICER valued the benefit that mavacamten would bring to a patient at between $12,000 to $15,000 a year. By contrast, some analysts have suggested that mavacamten could carry a price tag as high as $75,000 per patient per year.
If you would like to share your thoughts at the online public meeting click here to sign up.
You can find a press release from ICER about their review of mavacamten here.
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.
Editor’s note: You have probably noticed a distinct uptick in clinical trials of potential treatments for hypertrophic cardiomyopathy. HCMBeat has been following this trend and has previously published a host of stories about such trials, including this story about the positive results from the REDWOOD-HCM Phase 2 clinical trial, as well as past stories discussing the biopharmaceutical company Cytokinetics, its drug aficamten (previously known as CK-274), and the REDWOOD-HCM trial.
Some of these earlier stories are as follows:
Recently, Cynthia Waldman of HCMBeat had the opportunity to speak over Zoom with Dr. Martin Maron, who recently served as the principal investigator of Cytokinetics’ REDWOOD trial. The conversation focused both on Cytokinetic’s drug aficamten (previously known as CK-274), and the new class of drugs known as “myosin inhibitors.” What follows is a transcript of their conversation (which has been edited for readability).
Recent HCM research has taken a much needed pivot away from a focus on adult men to the exclusion of others. Women and babies get HCM too, yet until this point, there has been far less attention and research on their issues. HCM is not just a disease of adult men, though they do make up the majority of participants in research studies which inform current treatment protocols.
Finally that may be changing. It seems that people are finally starting to pay some attention to other folks who have HCM. Here is a recent European article looking at HCM in infants and here is a recent article looking at HCM in women.
Let’s hope the trend continues since previous research has shown that HCM is often more severe in these two groups.
A new technology called “virtual native enhancement” (VNE) may soon eliminate the need for gadolinium as a contrast agent for patients with hypertrophic cardiomyopathy undergoing cardiac MRI. Gadolinium, a heavy metal contrast agent which is injected intravenously, has long been used in cardiac MRI scans to spot cardiac scar tissue in patients with HCM. In 2017, the FDA issued a safety communication relating to gadolinium because it was found that gadolinium remains in the body for months to years after the use of the drug.
The new VNE technology, recently described in the journal Circulation, uses artificial intelligence (AI) to virtually enhance the standard MRI image. The technology was developed using data taken from 1348 HCM patients and was validated in the HCM population, but the technology may have uses extending beyond HCM.
By avoiding the use of the contrast agent, this technology avoids side effects and long term consequences from the use of gadolinium. Additionally, it will make cardiac MRI available to patients who are allergic to gadolinium. VNE is also faster and cheaper that current technology used for cardiac MRI, which may make more frequent MRI monitoring of patients feasible.
A common cardiac drug called valsartan has shown positive results in those who carry the gene for hypertrophic cardiomyopathy but who do not have overt disease.
As reported by Dr. Carolyn Ho of Brigham and Women’s Hospital and Harvard Medical School at the online European Society of Cardiology meeting, the Phase 2 double blind VANISH trial looked at 178 pre-symptomatic young people who carried a sarcomere gene mutation known to cause HCM. These patients were randomly assigned to take either valsartan, an angiotensin II receptor blocker (ARB), or a placebo.
At the time of trial enrollment, these patients showed no or only mild signs of the disease. At the end of the two year trial, the individuals who took valsartan had a better overall cardiac picture compared to the group taking only the placebo.
Dr. Ho had this to say in a story about the trial results in Mirage News:
“Valsartan improved cardiac structure/function and remodeling in patients with early stage sarcomeric HCM, suggesting that this strategy may help prevent disease progression among those who have received a genetic diagnosis of HCM.”
“Our results suggest that valsartan may not only stabilize disease progression but may also promote improvement.”
You can also read this summary of the presentation on MedPage Today.
A study by researchers from Mayo Clinic recently published in the International Journal of Cardiology found that a deep learning artificial intelligence algorithm using a standard 12 lead electrocardiogram was able to detect hypertrophic cardiomyopathy in young people with impressive accuracy. This accuracy was particularly strong among adolescents aged 15 – 18.
Mayo has been looking at artificial intelligence for its potential to screen populations for HCM for some time now. Here is a previous HCMBeat story about Mayo’s work on artificial intelligence from February 2020.