Inventors: Dr Jin Seok Lee & Dr Catherine Wilson, Gurdon Institute
Unlike other organs, the adult heart cannot heal itself because its cells lose the ability to multiply. When someone suffers a heart attack, up to 1 billion heart cells die. Instead of growing new muscle, the body repairs the damage with tough scar tissue. This weakens the heart and leads to heart failure, a progressive and fatal condition that currently affects over 64 million people worldwide.
Current treatments only manage the symptoms rather than fixing the damaged muscle. For patients in the final stages of heart failure, a heart transplant is the only permanent solution – but donor hearts are incredibly scarce. As our population ages and lifestyle risks rise, the number of people suffering from heart failure is continuing to increase.
The inventor has developed a novel mRNA therapy that temporarily instructs heart cells to divide and multiply. This unlocks the heart’s natural, hidden ability to repair and regrow its own healthy tissue.
The initial testing in animals shows a significant increase in healthy heart muscle and a clear improvement in how well the heart pumps. The treatment can be delivered through a commonly-used type of catheter, avoiding the need for risky, open-heart surgery.
The next stage for the development is to carry out further small animal studies, which would then be followed by large animal studies in pigs.
While the next stages of the clinical development are clearly mapped-out, the questions for the i-Teams are threefold.
Firstly, what is the current landscape for new mRNA therapies, and for the LMP packaging needed to deliver the mRNA safely into the cells.
Secondly, what is the view of clinicians who work in this area? What problems would a therapy of this type solve for them, and what are their current stress points? What evidence would clinicians needs to see before they would be willing to consider adopting a new treatment of this kind?
Thirdly, the i-Teams will also need to investigate the regulatory hurdles that the treatment would need to address, as well as the pathways into the NHS.