When Stanford biochemist Jim Spudich settled down in bed with a book recommended by his wife, he had no idea that the book would inspire one of the biggest discoveries of his career. Spudich drifted off to sleep while reading The Haunted Mesa, a science fiction novel by Louis L’Amour. His scientific discovery was based on an image he saw in his dreams when the image of a mesa morphed into a myosin molecule.
Myosins are proteins that make the contraction of heart muscle possible. In hypertrophic cardiomyopathy, these proteins work too well, causing a heartbeat that is too strong.
The mesa in the book inspired Spudich to look at the head of the myosin. His previous research had focused on random locations along the entire length of the myosin.
Spudich’s groundbreaking work ultimately led to current clinical trials of the drug mavacamten which is being developed by the San Francisco biotech company MyoKardia.
This article tells the whole story of the beginnings of this groundbreaking research.
This week, MyoKardia announced positive data on its experimental drug for HCM, mavacamten (formerly known as MYK-461), for obstructive HCM.
After 6 months on the drug, of the 13 patients enrolled in the open label extension to the Pioneer study, NYHA Class improved in 8 of 10 patients, while 7 of the 8 patients were considered asymptomatic by the end of the study.
In addition to seeing an improvement in symptoms, patients also showed improvement to certain markers of cardiac function including left ventricular filling pressure,
NT-proBNP, a marker of cardiac wall stress, and left atrial volume index. Ejection fractions remained within normal levels for all patients in the study.
The complete results of this study will be presented by Oregon Health & Science University’s Dr. Steven Heitner at next weekend’s meeting of the American College of Cardiology in New Orleans.
DISCLOSURES: HCMBEAT HAS RECEIVED UNRESTRICTED EDUCATIONAL GRANTS FROM MYOKARDIA. ADDITIONALLY, CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVES AS A PATIENT ADVISOR ON THE STEERING COMMITTEE FOR MYOKARDIA’S EXPLORER TRIAL.
According to this press release, MyoKardia expects to dose the first patient in the EXPLORER-HCM trial of mavacamten (formerly known as MYK-461) for obstructive HCM in the second quarter of 2018.
MyoKardia says that it expects 220 patients to enroll in the 30 week long trial. These patients will be randomly assigned to receive either mavacamten or a placebo. Participants will also be able to continue on their normal beta blockers or calcium channel blockers.
MyoKardia, a San Francisco biotech company currently in clinical trials on a HCM drug called Mavacamten, has come up with a way to spot HOCM simply by using a wristband fitness monitor. The wristband works through the use of optical biosensors which monitor arterial pulse waves.
During MyoKardia’s trials, the bracelet biosensors were used on HOCM patients and non-affected controls. The algorithm was able to distinguish HOCM patients from unaffected individuals more than 95% of the time, suggesting that a non-invasive way to screen for HOCM may not be too far in the future.
The linked article at Medgadget contains an interview with MyoKardia’s Dr. Robert McDowell, MyoKardia’s Chief Scientific Officer, and Dr. Eric Green, MyoKardia’s Senior Director of Translational Research with more on the happenings at MyoKardia.
Editor’s Note: This is the 4th of 4 blog entries which summarizes the presentations given at the recent International HCM Summit VI in Boston. The presenter and their hospital affiliation are noted below, along with the topic of their presentation. When possible, you may access the presenters’ slides via hyperlink by clicking on the name. (Note that not all presenters made their slides available).
Continue reading “HIGHLIGHTS FROM THE INTERNATIONAL HCM SUMMIT, PART IV”