Scientists believe they will soon be able to effectively treat inherited, life-threatening heart muscle disorders to prevent sudden cardiac death in more young individuals.
The British Heart Foundation has offered £30 million to gene researchers who believe they can repair the damage caused by defective DNA.
Cardiomyopathies can kill unexpectedly.
Due to their DNA, many members of the same family are often in danger.
Cardiomyopathies are frequently in the news when they cause sudden cardiac mortality or cardiac problems in young, elite athletes.
Sport can aggravate a preexisting condition.
In 2012, 23-year-old Fabrice Muamba fainted on the field during a game for Bolton due to cardiac difficulties. Others, such as the 28-year-old Cameroonian soccer sensation Marc-Vivien Foe, were unable to be resuscitated by doctors.
The grant-awarded research team is convinced that their gene therapy could be ready for human clinical trials within five years, now that the genes and precise genetic flaws responsible for various cardiomyopathies and how they function have been identified.
They have already conducted preliminary research on animal and human cells.
Injected into the arm, the treatment could arrest the progression of hereditary cardiomyopathies, according to the researchers.
And it could prevent the disease from developing in family members who carry a mutated gene but have not yet manifested the disorder, thereby resolving the issue.
A few years ago, the 27-year-old Max Jarmey was informed that he had inherited a form of hereditary cardiomyopathy. Max’s father died of arrhythmogenic right ventricular cardiomyopathy (ARVC) when he was 13 years old.
Tom, the younger sibling of Max, also has it.
Max says he focuses on what he can do rather than what his illness prevents him from doing, but he has given up certain sports as a precaution.
He has been implanted with a defibrillator that can shock his heart back into a normal rhythm, thereby protecting him from cardiac arrest.
James Taylor of England required the same treatment for his ARVC.
Max stated, “I’m fairly mentally robust, but the first six months after my diagnosis were extremely challenging.
“I believe the only way to cope with my diagnosis is to accept it and acknowledge that I have no control over it.”
He hopes to participate in the CureHeart clinical trial.
“When I consider my future, the decision to have children, and their future, CureHeart could assist me in making this decision. My children may never have to go through what I have with this illness. That is absolutely life-altering.
This endeavor gives me hope.
Cardiomyopathy is a condition in which the heart muscle is often weaker, deformed, and impaired in function. This can result in symptoms such as chest pain, shortness of breath, and palpitations, and often progresses to heart failure.
While medications, devices such as pacemakers, and surgery can often alleviate the symptoms of cardiomyopathy, there is presently no cure for heart failure.
Prof. Sir Nilesh Samani, medical director of the British Heart Foundation, stated, “This is a watershed moment in cardiovascular medicine. Not only may CureHeart be the first to develop a cure for inherited heart muscle illnesses by targeting killer genes in family trees, but it could also usher in a new era of precision cardiology.
Once effective, the same gene editing technologies could be applied to treat a variety of common heart ailments in which genetic defects play a significant role. This would have a transformative effect and provide hope to the tens of thousands of families stricken by these debilitating diseases around the world.
