GENE THERAPY – A CURE FOR SICKLE CELL DISORDER

Sickle Cell Disease (SCD) is a blood disorder that has plagued many for many years and has been the subject of research for decades.

Blood transfusion and several drugs have been in use over several years to contend with pains associated with crisis periods but have not provided a permanent solution for patients.

Bone Marrow Transplantation has been used to provide a cure for this disorder. However, the success of this approach depends on having a suitable bone marrow donor. And there is an age limitation in which success can be achieved.

Research in several European countries and the United States of America has led to the consideration of gene therapy as a permanent cure.

The National Institute for Health and Care Excellence (NICE) has finally approved CASGEVY or EXA-CEL as a treatment for SCD following extensive clinical tests over a period of more than three years.

As reported by Sky News, “Casgevy earned its inventors the Nobel Prize for chemistry in 2020. It works by editing the faulty gene in a patient’s own stem cells. It is an option for patients when a stem cell transplant is suitable but no donor can be found.”