A Breakthrough in Sickle Cell Treatment: Kendric Cromer’s Journey
Kendric Cromer, a spirited 12-year-old, left Children’s National Hospital in a wheelchair on a joyous Wednesday, his attire reflecting his passions—he wore a T-shirt and cap adorned with vibrant designs from the beloved anime series “Naruto,” complemented by a sleek black face mask. As he made his way down the hallway, hospital staff gathered to celebrate, cheering and waving colorful noisemakers in a heartwarming send-off. This moment marked a significant milestone: Kendric had just become the very first patient to receive an FDA-approved gene therapy for sickle cell disease, a groundbreaking treatment that holds the promise of liberating him from the relentless grip of this debilitating condition.
After spending an arduous 44 days in the hospital, Kendric felt somewhat dazed yet hopeful. “I thought I would have sickle cell for the rest of my life,” he confessed, his voice tinged with emotion. The disease had overshadowed his childhood, robbing him of simple pleasures such as playing basketball or riding a bike, as these activities often triggered excruciating pain that led to frequent hospital visits.
However, amid the celebratory atmosphere, Kendric and his parents could not shake the memories of the challenges they faced during his hospital stay. Despite their extensive preparations—discussions with medical professionals, thorough readings, and highlighting crucial sections of a daunting 13-page consent form that outlined potential outcomes including organ damage and even death—nothing could truly prepare them for the trials he encountered during treatment.
Currently, around 100,000 individuals in the United States are affected by sickle cell disease, with approximately 20,000 experiencing the most severe form of the condition. For these patients, gene therapy represents a beacon of hope, potentially offering a pathway to a life free from the burdens of the disease. Sickle cell disease arises from a mutation in the hemoglobin genes, leading to the formation of crescent-shaped red blood cells. These misshapen cells often become lodged in blood vessels, resulting in episodes of excruciating pain that can cause significant organ damage, lead to strokes, and ultimately shorten lifespans.
Until recently, many felt trapped with no viable options for relief. However, a major shift occurred last December when the Food and Drug Administration (FDA) approved two innovative gene therapies: a $3.1 million treatment developed by Bluebird Bio, based in Somerville, Massachusetts, and a $2.2 million therapy from Vertex Pharmaceuticals in Boston. These advancements now offer patients like Kendric a potential lifeline, provided their insurance covers the cost, paving the way for a future less overshadowed by the devastating effects of sickle cell disease.
