Kendric Cromer, a 12-year-old boy, made a significant milestone on Wednesday when he left Children’s National Hospital in a wheelchair after 44 days of treatment, becoming the first patient to receive an approved gene therapy for sickle cell disease. Celebrated by hospital staff who lined the hallways with cheers and noisemakers, Kendric’s journey marks a hopeful turning point in the fight against this debilitating condition.
Wearing a T-shirt and cap adorned with designs from the anime series “Naruto,” Kendric expressed his relief, stating, “I thought I would have sickle cell for the rest of my life.” The disease, which had robbed him of his childhood by rendering activities like playing basketball or riding a bike impossible due to the risk of severe pain and hospitalization, has left an indelible mark on his young life.
However, the celebratory mood is tempered by the challenges Kendric and his family faced during his hospital stay. They struggled to fully comprehend the gravity of the situation, despite extensive discussions with medical professionals and reviewing a daunting 13-page consent form that outlined potential risks, including organ damage and death.
Sickle cell disease affects about 100,000 people in the U.S., with approximately 20,000 experiencing the most severe symptoms. The condition arises from a mutation in hemoglobin genes, resulting in crescent-shaped red blood cells that can obstruct blood vessels and cause excruciating pain, organ damage, strokes, and reduced life expectancy.
Until recently, options for patients were limited. However, the landscape began to shift last December when the Food and Drug Administration (FDA) approved two gene therapies: a $3.1 million treatment from Bluebird Bio and a $2.2 million option from Vertex Pharmaceuticals. For patients like Kendric, these therapies offer a potential path to a life unencumbered by the disease—provided their insurance covers the cost.
Despite the promise these treatments hold, access remains limited. As of September 24, Bluebird reported that only 10 patients had started their therapies since receiving FDA approval, while Vertex announced 20 patients had begun treatment
Kendric Cromer’s Remarkable Recovery: First Patient to Receive Approved Gene Therapy for Sickle Cell Disease
