UAB says new gene therapy could one day be cure for sickle cell disease

BIRMINGHAM, Ala. (WBRC) - New hope for those living with sickle cell disease. Research from UAB shows a new gene therapy could one day be the cure for sickle cell disease. Dr. Julie Kanter explained sickle cell disease is a group of disorders that defines people who make abnormal hemoglobin, or the protein in our red blood cells that carries oxygen to our tissues.

In the U.S., sickle cell disease is most common among African Americans. The CDC said for every 365 births, one black child will have the disease, and about one in 13 black babies is born with the sickle cell trait. The disease can cause the cells to be misshapen and stick to other cells, which often leads severe pain in patients sending them to the hospital. Sickle cell can also cause organ and bone problems. But clinical trials using a new gene therapy called Lenti-Globin are showing promise at UAB and offer immense hope in the field

“That’s when we take out someone’s blood stem cells and we actually put in a new gene that makes healthy hemoglobin, and once the cells are ready, we have to bring that person to the hospital, give them chemotherapy, just like for any other transplant, and give them the cells back. We say it is awesome, right? It is life changing, but to be truly curative, we have to measure it over time and on many different levels,” said Dr. Kanter.

Dr. Kanter said right now, the gene therapy is being studied in adults and adolescents. She said researchers are still working to learn how long the therapy will last, and whether it will prevent or reverse organ damage.

Dr. Kanter hopes to see it more widely available within the next few years.