Deshawn “DJ” Chau Tinh Tri waited a year to receive a treatment that could change his life. The 19-year-old was born with sickle cell disease, which causes red blood cells to become sickle-shaped and sticky. Deformed cells build up and block blood vessels, cutting off oxygen to parts of the body and causing severe pain. The condition affects about 100,000 people in the United States, most of whom are black.
The pain increasingly plagued Chow while in high school, causing him to be frequently hospitalized. He missed school, birthday parties, and sleepovers with friends. Sometimes the pain lasts for many days. “My body felt like it was on fire,” he said.
A year ago, he discovered a new treatment called Casgevy that could end his years-long battle with pain. That is first approved drug using Nobel Prize winning technology called Crispr, a type of gene editing. Chow hosts Casgevy on December 5 at City of Hope Cancer Center in Los Angeles. He was one of the first patients in the US to receive treatment since approval in December 2023. It was also approved to treat beta thalassemia, a related blood disorder, this January.
Due to manufacturing complications, insurance delays, and extensive patient preparation, few individuals in the United States have used Casgevy since it was marketed. The slow rollout underscores the complex nature of commercializing cutting-edge medical treatments and getting them into the hands of patients. Another genetic treatment for sickle cell disease, Lyfgenia, was approved last December and the first patient was treated in September. Manufactured by Bluebird Bio, it uses older technology to introduce a new gene to treat the disease.
Vertex Pharmaceuticals and Crispr Therapeutics, the company that developed Casgevy, have not publicly said how many patients have been treated with the therapy so far. WIRED reached out to them all 34 US hospitals are approved to administer it as of December. Of the 26 people who responded, only Children's Hospital and City of Hope in Washington, D.C., said they had received the Casgevy shot. (Three hospitals declined to comment and five others did not respond to multiple inquiries.) Chow is City of Hope's first sickle cell disease patient, while a beta thalassemia patient has been treated. treatment at Children's National. Some authorized centers told WIRED they will treat the first patients as early as 2025.
“The process of receiving this drug is very different from just taking a pill,” said Leo Wang, Chow's hematologist-oncologist at City of Hope. This is a one-time therapy that involves collecting and editing a person's stem cells. For patients, that means a grueling round of chemotherapy before the cell retrieval and a month in the hospital afterward.