Just in time for today’s HCM Awareness Day, on Sunday the Times of London ran a feature story about the work that U.K. professor Hugh Watkins and his team are doing to find a cure for HCM using gene therapy.
The story begins with an interview with Massachusetts resident Wendy Borsari, who shared the tale of her family’s extensive and tragic history with HCM. Over the past several generations, 27 members of Wendy’s family have suffered from HCM, and family members have endured 7 heart transplants and 18 deaths.
On a personal note, Wendy has worked tirelessly for many years to improve things for other HCM patients, and she was integral to the creation of HCMBeat. You can read her guest blog about her daughter Ashlan’s cardiac arrest right here.
Going back to the Times feature, the focus of the story is on the work that a team led by Dr. Hugh Watkins is doing under the name the Cure Heart Project. The project was funded by a £30 million grant ($36 million) from the British Heart Foundation which the team won last year. Over the 5 years of the grant, Dr. Watkins and his team are testing multiple gene therapies intended to treat genetic cardiomyopathies. The team plans to have their technology ready for human trials by the end of the study. The hope is that these treatments will stop progression of disease, possibly reverse some of the damage, and if given early enough, the scientists hope that the treatments can prevent the disease from developing at all.
Clinical trials would start on the sickest patients, such as those who are waiting for a heart transplant. Once they demonstrate that the treatments work, they hope to administer the treatment to gene positive patients who have not yet developed the disease with the hope that in these patients, it would be a cure.
With so much work being done around gene therapy for HCM, sooner or later, someone is going to be successful. We just hope it is sooner rather than later!
HCMBeat has written several previous blog entries describing the work being in hopes of finding a a genetic cure for HCM. They are linked below.
Gene Therapy – Is a Cure for HCM Around the Corner?
Tenaya Therapeutics Gets Go-Ahead for HCM Gene Therapy Trial
Gene Therapy – Is HCM Cure Possible?
HCM Genetics Discovery by British Researchers