Deshawn “DJ” Chow He waited a year to receive treatment that could change his life. The 19-year-old was born with sickle cell disease, which makes his red blood cells crescent-shaped and sticky. The misshapen cells build up and block blood vessels, cutting off oxygen to parts of the body and causing episodes of excruciating pain. The disease affects about 100,000 people in the United States, most of them black.
Pain became increasingly common for Chow in high school, frequently landing him in the hospital. I missed school, birthday parties, and sleepovers with friends. Sometimes the pain lasted for days. “It’s like my body is on fire,” he says.
A year ago, she discovered a new treatment called Casgevy that could end her long battle with pain. It is the first approved drug to use the Nobel Prize-winning technology known as Crispr, a type of gene editing. Chow welcomed Casgevy on December 5 at the City of Hope Cancer Center in Los Angeles. He is among the first patients in the U.S. to receive the treatment since its approval in December 2023. It was also approved for beta thalassemia, a related blood disorder, in January.
Because of manufacturing complexities, insurance delays and the extensive preparation involved for patients, few people in the U.S. have received a dose of Casgevy since it became commercially available. The slow rollout underscores the complicated nature of commercializing cutting-edge medical treatments and getting them to patients. Another genetic treatment for sickle cell anemia, Lyfgenia, won approval last December and the first patient was treated in September. Manufactured by Bluebird Bio, it uses an older technology that introduces a new gene to treat the disease.
Vertex Pharmaceuticals and Crispr Therapeutics, which developed Casgevy, have not publicly said how many patients have received the therapy so far. WIRED reached everyone 34 US hospitals approved to administer it starting in December. Of the 26 that provided responses, only City of Hope and Children’s National Hospital in Washington, D.C., said they had administered Casgevy. (Three hospitals declined to comment and five others did not respond to multiple inquiries.) Chow is City of Hope’s first sickle cell anemia patient, while a beta thalassemia patient has been treated at Children’s National. Several authorized centers told WIRED they will begin Casgevy infusions in early 2025.
“The process of getting this medication is very different than just taking a pill,” says Leo Wang, Chow hematologist-oncologist at City of Hope. It is a unique therapy that involves collecting and editing a person’s stem cells. For the patient, it means a tough round of chemotherapy before receiving the cells and a month in the hospital afterward.