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. This disease causes red blood cells to become sticky and shaped like a crescent moon. Malformed cells form and block blood vessels, cutting off oxygen to parts of the body and causing extreme pain. The condition affects approximately 100,000 people in the United States, most of whom are black.
During Chow’s high school years, pain became more and more frequent and she was frequently admitted to the hospital. He missed school, birthday parties and sleepovers with friends. Sometimes the pain lasted for several days. “I feel like my body is on fire,” he says.
A year ago, he learned about a new treatment called Casgevy that could end his years-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 received Casgevy on December 5 at the City of Hope Cancer Center in Los Angeles. He is one of the first patients to receive this treatment in the United States since its approval in December 2023. It was also approved for beta thalassemia, a related blood disease, in January of this year.
Because of manufacturing complexity, insurance delays and extensive preparation for patients, few people in the United States have received Casgevy since it was commercialized. The slow rollout highlights the complex nature of the process of commercializing cutting-edge medical treatments and making them available to patients. Lifgenia, another gene therapy for sickle cell, was approved in December last year, and the first patient was treated in September. The product, made by Bluebird Bio, uses an old technology to introduce new genes to treat diseases.
Vertex Pharmaceuticals and Crispr Therapeutics, which developed Casgevy, have not publicly disclosed how many patients have received the treatment so far. WIRED reached out to everyone. Approved to administer it at 34 hospitals in the United States As of December. Of the 26 hospitals that responded, 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 was City of Hope’s first sickle cell patient and beta thalassemia patient treated at Children’s National. Several approved centers told WIRED they will begin administering Casgevy infusions as early as 2025.
“The process of taking this medication is very different than just taking a pill,” says Leo Wang, Chow’s hematologist-oncologist at City of Hope. This is a one-time treatment that collects and edits an individual’s stem cells. From the patient’s perspective, this means that they must undergo harsh chemotherapy before the cells are collected, and must be hospitalized for a month after the cells are collected.
