sickle cell

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The first patient to undergo a new and innovative treatment for sickle cell disease is a 12-year-old Black child from Washington D.C.
A new commercially approved gene therapy designed to treat and possibly cure sickle therapy had its first patient last Wednesday (May 1). Kendric Cromer, a Black 12-year-old who is from a suburb of Washington, D.C., began his first treatment administered by the Bluebird Bio company at Children’s National Hospital in the D.C. metro area. The treatment is significant as Black people make up the majority of the 100,000 people in the United States that are dealing with the affliction passed on through genetics from both parents that can cause debilitating and constant pain in addition to other problems such as low oxygen and jaundice.

The new treatment, known as Lyfgenia, is an exhausting one, requiring Kendric to remain in the hospital for a month afterward just to recover from the extraction component of the treatment. He became the first to qualify after meeting the two major requirements – who was the sickest and whose insurance came through. “We always prayed this day would come,” Kendric’s mother, Deborah Cromer, said during the interview with the New York Times.
“We’re nervous reading through the consents and what he will have to go through.” But for Kendric who has suffered with the affliction since he was 3 years old resulting in five-day crises and multiple emergency room visits, he was ready to undergo the rigorous procedure. “Sickle cell always steals my dreams and interrupts all the things I want to do,” he said. “I want to be cured.”

The first step involved doctors removing Kendric’s bone marrow stem cells after administering a drug to pry them loose known as plerixafor. The extraction’s desired objective is to obtain millions of stem cells over a six to eight-hour period then transport them to a laboratory in Allendale, New Jersey, to have a healthy hemoglobin gene added to them to correct the mutation causing the sickle cell disease. The modified cells would then be sent back and reintroduced into Kendric’s bone marrow three months later.
The Lyfgenia gene therapy treatment has been touted as a revolutionary procedure and ranks near the top of the most costly treatments in healthcare at $3.1 million. Bluebird Bio has said that due to the amount of time that’s needed for the entirety of the treatment, it can only treat 85 to 100 patients a year.