The Gene Therapy Curing Sickle Cell Disease | Rickey’s Story

This is the story of a new gene therapy curing sickle cell disease. One morning, Dr. Lametra Scott nudged her husband awake and said, Look at his eyes. For the first time in years, the whites of her son Rickeys eyes were actually white the sign she had been watching for every morning, before any test could confirm what she already felt in her heart.

Sickle cell disease has been documented in medical textbooks for more than a hundred years, and it disproportionately affects Black and Hispanic Americans. For most of that history, doctors could manage the pain but not change the disease. That changed when the FDA approved a new gene-editing therapy, first for patients 12 and older, and this July for children as young as 2.

In this episode, Rickeys mother, pharmacist Dr. Lametra Scott, and Dr. Haydar Frangoul, who directs the Pediatric Hematology, Oncology, and Stem Cell Transplant program at TriStar Centennial Childrens Hospital in Nashville, take us through what it actually took: the diagnosis, the daily fear of a fever or a pain crisis, the month in the hospital, the chemotherapy, and the moment they knew it had worked. Dr. Frangoul also explains how the therapy uses CRISPR gene-editing technology, and why one state Alabama still isnt offering it to its residents.

FDA approval announcement

Casgevy patient information (Vertex Pharmaceuticals)

Sickle Cell Disease Association of America

CDC Sickle Cell Disease

NIH / NHLBI Sickle Cell Disease

CMS Cell and Gene Therapy Access Model (state Medicaid coverage)

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