Twelve-year-old Kendric Cromer became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may offer a potential cure for the condition. The treatment, authorized by the FDA, holds promise for the estimated 20,000 people in the U.S. with sickle cell disease, most of whom are Black. Sickle cell disease is a genetic disorder that causes debilitating pain and other medical issues. Kendric, a solemn and shy adolescent, had his life interrupted by episodes of excruciating pain preventing him from enjoying everyday activities. However, with the start of his gene therapy treatment, he now has hope for a normal and pain-free life.
The gene therapy process involves removing bone marrow stem cells from the patient, genetically modifying them in a specialized lab, and then reintroducing them back into the patient. This intricate process is time-consuming, with the company estimating that only 85 to 105 patients can be treated each year due to the complexity and limited capacity of medical centers to handle gene therapy patients. Patients require expert and intensive care during the treatment process, which includes staying in the hospital for a month post-treatment. Kendric started his treatment at Children’s National Hospital in Washington, with the hope that gene therapy will improve his quality of life.
Kendric’s journey towards a cure began with the collection of his bone marrow stem cells, which were genetically modified to correct the mutated hemoglobin gene causing his sickle cell disease. The modified cells will be returned to Kendric three months later, with the aim of providing him with healthy red blood cells that do not turn into fragile crescent shapes that can become trapped in blood vessels and organs. Despite the grueling process and the high cost of the gene therapy, Kendric’s family and many other families affected by sickle cell disease are hopeful for relief from the debilitating symptoms and complications of the condition.
Kendric’s parents, Deborah and Keith, were devastated when they found out that he had sickle cell disease. The family had to cope with frequent E.R. visits, pain crises, and severe complications, including avascular necrosis in Kendric’s hips, back, and shoulders. However, they never left his side, providing unwavering support and care throughout his medical journey. Despite the challenges, Kendric always maintained good grades in school and aspires to become a geneticist and play basketball once his treatment is complete. With the gene therapy now underway, the Cromer family is hopeful for a brighter future for Kendric without the constant pain and limitations imposed by sickle cell disease.
The high cost of gene therapy, estimated at $3.1 million, poses a financial burden to families like the Cromers, despite insurance approval for the treatment. Medical centers have waiting lists of eligible patients hoping for relief from the disease’s debilitating symptoms, including strokes, organ damage, bone damage, and shortened lifespans. Kendric’s eligibility for treatment was based on the severity of his condition and the prompt approval from his insurance provider. While the road to a cure is challenging and uncertain, the Cromers remain optimistic about the potential benefits of gene therapy for Kendric’s quality of life and future aspirations.
Kendric’s gene therapy journey offers hope not only for him but for the thousands of individuals with sickle cell disease in the U.S. The commercial approval of gene therapy treatments represents a significant advancement in the management of genetic disorders, providing potential cures for conditions that were once considered untreatable. As Kendric undergoes his treatment at Children’s National Hospital, his story serves as a beacon of hope for others affected by sickle cell disease, showcasing the possibilities of modern medical advancements in offering relief and a chance at a normal life for those living with genetic disorders. Despite the challenges that lie ahead, Kendric’s determination and perseverance in seeking a cure inspire others to remain hopeful for breakthroughs in treating genetic diseases and improving the lives of patients worldwide.
