Sickle Cell Gene Therapy

One man's story may offer hope to many. Evie Junior is a 27 year old with sickle cell disease complications. He had to have his gall bladder and spleen removed. Then he developed pericarditis. Sometimes he couldn't walk because of the pain. Sometimes he ended up at the emergency room and stayed in the hospital for a few days. In 2019, he was part of an experimental stem cell gene therapy trial at UCLA to correct the sickle cell mutation and produce healthy red blood cells. Although a similar therapy worked for other immune system deficiencies, it didn't work as well for sickle cell disease. During the summer of this year, Junior "received an infusion of his own blood-forming stem cells that had been genetically modified to overcome the mutation that causes his disease." So far, so good: 70% of the man’s blood stem cells had the new corrected gene, while researchers report that even a 20% correction could prevent future sickle cell complications.

"The goal of this treatment is to give him a future, let him plan for college, family or whatever he wants without worrying about getting hospitalized because of another pain crisis,” said Dr. Donald Kohn, a distinguished professor of microbiology, immunology and molecular genetics, pediatrics, and molecular and medical pharmacology at the David Geffen School of Medicine at UCLA. The treatment has given Junior a better quality of life: more energy and no pain crises. Dr. Kohn stated, "It’s too early to declare victory, but it’s looking quite promising at this point. Once we’re at six months to a year, if it looks like it does now, I’ll feel very comfortable that he’s likely to have a permanent benefit.”

 

Read the full press release on Newswise.