Promising Sickle Cell Cure Faces Cost and Access Challenges

By Milcah Tanimu

A potential lifelong cure for sickle cell disease is on the horizon as Vertex Pharmaceuticals utilizes CRISPR technology to modify patients’ DNA. Clinical trials show positive outcomes, but concerns over cost and accessibility loom large.

The U.S. FDA advisory committee will review the treatment’s safety data on October 31, 2023, with a potential approval decision on December 8.

The treatment involves extracting stem cells, gene editing, and intensive chemotherapy. Challenges include scaling production and high costs, posing accessibility issues, particularly in developing countries with a high prevalence of sickle cell disease.

The success of the Vertex treatment may open doors for similar therapies, offering hope to those with the condition. Other companies and academic institutions are also exploring gene therapy treatments, with Vertex pioneering a pill-based gene editing approach.