New Cure For Sickle Cell Disease Awaits FDA Approval

This is great news!

Sickle Cell disease, a health condition that affects millions of people across the globe, may have a cure in the coming months.

The U.S. Food and Drug Administration is reviewing the effects of gene therapy for sickle cell disease. According to NBC News, the gene therapy will be based on CRISPR, and the first gene therapy will be based on the gene editing tool to hit the U.S. market if it receives approval. The FDA will discuss the potential unplanned side effects of the treatment, and a decision for the treatment will be made in December before the focus shifts to a different sickle cell therapy.

Dr. Allison King, a professor at Washington University School of Medicine in St. Louis, is optimistic about the treatment. King, who works with children and young adults with sickle cell disease, said, “Anything that can help relieve somebody with this condition of the pain and the multiple health complications is amazing.” She added, “It’s horribly painful. Some people will say it’s like being stabbed all over.”

The new gene therapy will not require a donor, according to the outlet. Exa-cel is a one-time gene therapy made by Vertex Pharmaceuticals and CRISPR Therapeutics.

Only a few patients have been tested using the treatment, according to a report from the nonprofit Institute for Clinical and Economic Review.

Victoria Gray, a resident of Mississippi, was the first recipient of the gene therapy treatment test. She described the results as positive and felt like she “was being reborn” on the day she received the treatment. “My children no longer have a fear of losing their mom to sickle cell disease,” Gray said.

Sickle cell disease is an inherited blood disorder that affects Black people at higher rates. According to the Centers for Disease Control, approximately 100,000 Americans have sickle cell disease, and it occurs in 1 out of 365 Black or African American births. Scientists discovered that being a carrier of sickle cell trait protects people from contracting severe malaria. It would take a sickle cell gene from both parents for a child to develop the disease. The trait is more commonly seen in ethnic groups such as people of African, Middle Eastern, and Indian descent, where malaria is or was previously prevalent.

According to NBC News, sickle cell disease affects the shape of red blood cells, which carry oxygen throughout the body. However, the misshaping of the blood cells can interfere with blood flow and cause severe pain, organ damage, and other health complications.

Although results have been positive, the FDA is considering whether the unwanted consequences of gene therapy need to be studied more. Prices for the treatment have not been released yet; however, the institute report estimates costs to be around $2 million. Current treatment costs from birth to 65 years old are about $1.6 million for women and $1.7 million for men. Before the year ends, the FDA will also discuss an additional gene therapy made by Bluebird Bio.