May 6, 2024 – Twelve-year-old Kendric Cromer Sickle cell anemia has wreaked havoc on his young body, causing, amongst other things, a painful condition often called bone death in his hips, back and shoulders.
Last week, he became the primary patient outside of a clinical trial to start a brand new genetic treatment that had been shown in clinical trials to cure 88% of individuals of the standard symptoms of sickle cell anemia, a genetic disorder that may affect blood flow. CDC It is estimated that 100,000 people within the United States are affected by sickle cell anemia, with the disease being more prevalent amongst blacks: roughly 1 in 365 black newborns within the United States are affected.
The FDA approved the brand new therapy called Lyfgenia in December. Until then, the one options for individuals with sickle cell anemia were treatments to alleviate symptoms or transplants of donated stem cells, which include serious risks.
The months-long treatment process for Cromer began with the removal of bone marrow stem cells at Children's National Hospital in Washington, DC, based on The New York Times and a notice from Bluebird Bio, the corporate that developed the treatment.
“We are very excited 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 pioneers in the use of this new approach,” said Dr. David Jacobsohn, chief of the Division of Blood and Marrow Transplantation at Children's National Hospital, in an announcement. “The recent approval of gene therapies to treat patients with sickle cell disease is a tremendous breakthrough in patient care and a ray of hope for families witnessing their children's struggle with this disease. Given the enormous burden, gene therapy is a groundbreaking treatment alternative and a glimmer of hope for a better future.”
Bluebird's price tag for Lyfgenia therapy is $3.1 million, making it one of the crucial expensive medical treatments ever. Just reported, adding that Bluebird can currently treat the cells of 85 to 105 patients annually. There are currently waiting lists for individuals who need to receive this life-changing treatment.
Once enough stem cells are collected from a patient, they're sent to Bluebird's lab in New Jersey and modified to operate like those of people that would not have sickle cell anemia. Cromer will undergo chemotherapy before the modified stem cells are transplanted.
The process doesn't cure sickle cell anemia. Instead, newly modified genes instruct the body to provide more of a selected form of red blood cell protein that counteract the consequences of mutated proteins that cause red blood cells to tackle a C shape (hence the name sickle cell). Sickle-shaped red blood cells restrict blood flow and limit oxygen delivery, but the brand new treatment helps the body produce enough non-mutated red blood cell proteins to forestall red blood cells from taking over the sickle shape.