New Gene Editing Treatment on Horizon for Heart Failure
Trial results of a pioneering drug to counter a type of heart disease have shown the first ever successful gene editing to occur within the body. Gene editing is a process by which DNA in cells is altered, including by deletion and insertion of genes.
ATTR amyloidosis is a life threatening disease that causes a build-up of fibrous proteins in the heart, weakening its muscle and leading to heart failure. Current treatments rely on reducing the formation of these tissues, resulting in ongoing treatment to extend life expectancy by two to six years. Patients are often hospitalised due to the severity of their symptoms.
NTLA-2001 is an experimental drug that offers a new treatment for this disease - it utilises the Nobel Prize winning CRISPR CAS-9 method to facilitate gene editing, without needing to remove any tissue from the patient. Unlike current methods, the drug targets the gene responsible for the formation of the fibrous amyloid protein and replaces it.
The results of the phase 1 clinical trials carried out by NHS cardiologists were positive, showing reductions in the presence of the amyloid proteins in the blood at varying doses. However, this was only phase 1 stage and so the sample size was small and it is still too early to tell how long this reduction will last, or indeed if the treatment may provide a cure for the disease altogether.
Even while these questions remain unanswered, the early signs from this trial show promise that other similar diseases might be treated by using similar methods.
More detail of the trial results to date can be found in the research article.
