Deshawn “DJ” Chau Waited for a year for a treatment that could have changed his life. The 19-year-old was born with sickle cell disease, which makes his red blood cells crescent-shaped and sticky. The deformed cells form and block blood vessels, cutting off oxygen supply to certain parts of the body and causing excruciating pain. The condition affects about 100,000 people in the United States, the majority of whom are black.
In high school, Chow began experiencing recurring pain, resulting in frequent hospitalizations. He missed school, birthday parties, and sleepovers with friends. Sometimes the pain persists for several days. “It feels like my body is on fire,” he says.
A year ago, he learned about a new treatment called Casgravi that could end his years-long battle with pain. it is first approved drug Nobel laureates known to use technology CRISPR, a type of gene editingChow received Casgravi on December 5 at the City of Hope Cancer Center in Los Angeles. He is among the first patients to receive treatment in the US Since its approval in December 2023It was also approved for the related blood disorder beta thalassemia this January.
Due to manufacturing complications, insurance delays, and extensive preparation for patients, doses of CasGevi have been administered to few individuals in the US since it became commercially available. The slow implementation underlines the complex nature of commercializing cutting-edge medical treatments and delivering them to patients. Another genetic treatment for sickle cell, Lyfgenia, was approved last December, and the first patient was treated in September. Made by Bluebird Bio, it uses an older technology that introduces a new gene to treat the disease.
Vertex Pharmaceuticals and Crispr Therapeutics, which developed Casgevi, have not publicly disclosed how many patients have received the therapy so far. WIRED reaches out to everyone 34 US hospitals approved to administer it until December. Of the 26 that provided responses, only City of Hope and Children's National Hospital in Washington, D.C., said they had administered Kasegavi. (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 a beta thalassemia patient has been treated at Children's National. Several authorized centers told WIRED that they will treat their first patients as early as 2025.
“The process of taking this drug is very different from just taking a pill,” says 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 the patient, this means a rigorous round of chemotherapy and then a month in the hospital before receiving the cells.