Advances in medical practice might soon see the treatment of sickle cell disease, a devastating, incurable disorder that afflicts millions of people around the world
Doctors are reporting the first evidence that genetically edited ‘Supercells’ could offer a safe way to treat the incurable disease that affects the hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body.
Billions of cells that were genetically modified with the powerful gene-editing technique called CRISPR have started working, as doctors had hoped, inside the body of the first sickle cell patient to receive the experimental treatment, according to highly anticipated data released Tuesday.
The edited cells are producing a crucial protein at levels that have already exceeded what doctors thought would be needed to alleviate the excruciating, life-threatening complications of the genetic blood disorder, the early data show. Moreover, the cells appear to have already started to spare the patient from the agonizing attacks of pain that are the hallmark of the disorder.
“We are very, very excited,” says Dr. Haydar Frangoul of the Sarah Cannon Research Institute in Nashville, Tennessee- USA, who is treating the patient. “This preliminary data shows for the first time that gene editing has actually helped a patient with sickle cell disease. This is definitely a huge deal.
Despite the progress recorded, Frangoul and other researchers caution, that the results involve just one patient who was only recently treated, they insist there is still some work to be done.
“We are hoping it is” a success, Frangoul says. But “it is still too early to celebrate.”