Deshawn “DJ” Chow I waited a year to receive potentially life-changing treatment. The 19-year-old was born with sickle cell disease, which causes his red blood cells to become crescent-shaped and sticky. The misshapen cells build up and block blood vessels, cutting off oxygen supply to parts of the body and causing excruciating pain. The condition affects approximately 100,000 people in the United States, most of whom are Black.
As he entered high school, Chow’s pain became more frequent, and he was often taken to the hospital. He missed school, birthday parties, and sleepovers with friends. Sometimes the pain lasted for days. “I feel like my body is on fire,” he says.
A year ago, he learned about a new treatment called Kasugebi that could end his years-long struggle with pain. It is the first approved drug to use the Nobel Prize-winning technology known as Crispr, a type of gene editing. Ms. Chow underwent Kasugebi treatment on December 5th at City of Hope Cancer Center in Los Angeles. He is one of the first patients to receive the treatment in the United States since its approval in December 2023. In January, it was also approved for beta-thalassemia, a related blood disorder.
Because of manufacturing complexities, insurance delays, and the extensive preparation required for patients, few people have received Casgevy since it became commercially available in the United States. This slow rollout highlights the complex nature of commercializing and delivering cutting-edge medical care to patients. Another sickle cell gene therapy, Lifgenia, was approved last December and the first patient was treated in September. The treatment, made by Bluebird Bio, uses an old technology to introduce new genes to treat the disease.
Vertex Pharmaceuticals and CRISPR Therapeutics, which developed Kasugevy, have not publicly disclosed how many patients have received the therapy. WIRED contacted all 34 U.S. hospitals approved to perform the treatment 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 is City of Hope’s first sickle cell patient, while the beta-thalassemia patient is at Children’s・Receiving treatment at National Hospital. Several certified centers told WIRED they expect to treat their first patients in early 2025.
“The process of obtaining this drug is very different from just taking a pill,” says Leo Wang, Chau’s hematology-oncologist at City of Hope. This is a one-time treatment that involves collecting and editing a person’s stem cells. For patients, that means grueling chemotherapy before the cells can be harvested, followed by a month in the hospital.
(Tag Translation) Science
