Kendric Cromer, a 12-Year-Old boy, residing in a suburb of Washington, made history on Wednesday (May 1) as the first person in the world to undergo commercially approved gene therapy for sickle cell disease.
According to a report by the NYT, Kendric's successful treatment offers hope to approximately 20,000 individuals in the United States suffering from sickle cell disease. However, it also highlights the challenges patients face in accessing new sickle cell treatments.
Last December, the US Food and Drug Administration granted authorization to two companies to sell gene therapy to individuals with sickle cell disease—a genetic disorder of red blood cells causing debilitating pain and other medical complications.
With over 100,000 people in the US affected by sickle cell, most of whom are Black, individuals inherit the mutated gene for the condition from both parents. While the treatment has undergone numerous clinical trials to cure the disease, Kendric marks the first commercial patient for Bluebird Bio, a company based in Somerville, Mass. Another company, Vertex Therapeutics of Boston, did not disclose whether it had initiated treatment for any patients with its approved CRISPR gene-editing-based remedy.
Bluebird lists a price of $3.1 million (approximately Rs 258,478,620) for its gene therapy, known as Lyfgenia, making it one of the highest-priced treatments ever.
At Children's National Hospital in Washington, doctors extracted bone marrow stem cells from Kendric, which Bluebird will genetically modify in a specialized lab for his treatment. However, doctors require hundreds of millions of stem cells from the patient, and if the initial collection—taking six to eight hours—is insufficient, the company will attempt one or two more extractions. Kendric will have to undergo another month of preparation for additional stem cell extraction.
The entire process is extensive and time-consuming, with Bluebird estimating that it can treat the cells of only 85 to 105 patients each year—a figure that includes not only sickle cell patients but also individuals with a much rarer disease, beta thalassemia, who can receive a similar gene therapy, as per the NYT report.
Moreover, medical centers have limited capacity to handle gene therapy patients, with each person requiring expert and intensive care. After the patient's stem cells have been treated, they must remain in the hospital for a month, during which time they are severely ill from powerful chemotherapy.