May 6, 2024 – Twelve-year-old Kendric Cromer’s sickle cell disease has wreaked havoc on his young body, including causing a painful condition known as bone death in his hips, back, and shoulders.
Last week, he became the first patient outside of a clinical trial to start a new genetic treatment that was shown in clinical trials to cure 88% of people of the hallmark symptoms of sickle cell disease, which is a genetic condition that can affect blood flow. The CDC estimates 100,0000 people in the U.S. have sickle cell, and it’s more common among Black people – about 1 in every 365 Black newborns in the U.S. have it.
The FDA approved the new therapy, called Lyfgenia, in December. Until then, the only options for people with sickle cell were treatments to manage symptoms or donor stem cell transplants that carry serious risks.
The months-long treatment process for Cromer began with the collection of bone marrow stem cells at Children’s National Hospital in Washington, DC, according to The New York Times and an announcement by Bluebird Bio, the company that developed the treatment.
“We are thrilled to be the first center in the country to commercially collect cells from a person living with sickle cell disease and are proud to be the trailblazers in using this new approach,” David Jacobsohn, MD, chief of the Division of Blood and Marrow Transplantation at Children’s National Hospital, said in a statement. “The recent approval of gene therapies to treat patients with sickle cell is an enormous breakthrough in patient care and a silver lining to families witnessing their children’s struggles with this condition. In the face of immense burden, gene therapy is a groundbreaking treatment alternative and a beacon of hope for a better future.”
Bluebird’s price tag for Lyfgenia therapy is $3.1 million, making it one of the most expensive medical treatments of all time, the Times reported, adding that Bluebird’s current annual capacity is to treat the cells of between 85 and 105 patients. There are currently waiting lists of people seeking the life-changing treatment.
Once enough stem cells are collected from a patient, they are sent to Bluebird’s lab in New Jersey and modified to function like those of people not affected by sickle cell. Cromer will have chemotherapy before the modified stem cells are transplanted.
The process does not cure sickle cell itself. Instead, newly modified genes instruct the body to produce more of a type of red blood cell protein that can counteract the effects of mutated proteins that result in red blood cells taking on a C-shape (hence the name, sickle cell). Sickle-shaped red blood cells restrict blood flow and limit oxygen delivery, but the new treatment helps the body produce enough non-mutated red blood cell proteins to keep red blood cells from forming the sickle shape.