The approval of two gene therapies by the Food and Drug Administration over a year ago raised hope among sickle cell patients nationwide. However, access to these potential cures has been limited, with only a few dozen patients able to receive the treatment so far. Challenges such as paperwork delays, high costs, and concerns over side effects have hindered widespread adoption by hospitals.
Sickle cell disease affects over 100,000 individuals in the United States, with the majority being Black. This inherited condition causes red blood cells to take on a sickle shape, leading to severe pain, strokes, organ damage, and shortened lifespans. The gene therapies offered by Vertex Pharmaceuticals and Bluebird Bio hold promise for a cure.
Despite initial skepticism, patients like Wedam have pursued these treatments, undergoing insurance approvals and medical consultations. For Wedam and his family, the prospect of a cure is both hopeful and surreal. The process involves mobilizing stem cells, modifying them in a lab using CRISPR technology, and reintroducing them back into the bloodstream.
As Wedam embarks on this groundbreaking therapy, medical professionals commend his bravery in embracing a new frontier in treatment. The potential benefits include reduced pain crises, prolonged life expectancy, and fewer hospital visits. Dr. David Jacobsohn from Children’s National Hospital emphasizes the transformative impact of these advancements, highlighting the progress made in providing effective cures compared to previous years.
Few patients have begun gene therapy treatment, with only 10 individuals at Children’s National having initiated or completed the process out of a pool of approximately 1,500 sickle cell disease patients treated in the Washington area. Initially, the hospital leadership was enthusiastic, anticipating a higher patient turnout. However, the process has been a learning curve for both hospitals and insurance companies. Dr. Jacobsohn mentioned that his team plans to start one or two patients on the treatment per month, emphasizing the high-risk nature of the therapy and the need for careful consideration before scaling up.
The treatment from Vertex Pharmaceuticals and Bluebird Bio comes with significant price tags, prompting insurers to establish strict pre-authorization procedures. Hospitals authorized to offer the treatment face resource-intensive processes, including stem cell collection, chemotherapy, and genetic alteration of cells. Patients undergoing the treatment endure intense chemotherapy, leading to severe side effects such as infections and mouth sores. Despite the challenges, Dr. Wang from City of Hope Children’s Cancer Center remains optimistic about increasing patient uptake as success stories emerge.
One patient, Wedam, hopes to inspire others by sharing his positive experience with the treatment. Although battling the disease has limited his activities, he envisions a future as a filmmaker and looks forward to attending college classes in person. By sharing his journey, Wedam aims to instill hope in others seeking a cure.
“I would be able to create new ones,” he said. “I would be able to grasp the material better if I am in person, asking the teacher real questions. I just believe that, being an ordinary person, doing what an ordinary person would, I believe that is what truly excites me.”
The Minyila family endured the ravages of the disease for nearly two decades. Along with Wedam, his 14-year-old brother, Wekem, also suffers from sickle cell.
Wedam’s younger brother, Wekem, managed to learn how to ride a bike after a stem cell transplant cured his sickle cell disease.
“It has taken almost everything,” Sylvia Minyila lamented. “It has taken our happiness.”
Wedam, she recalled, was once a cheerful boy who adored school and dancing. However, when his pain crises began around middle school, she observed a change in him.
“He became withdrawn. He experienced pain in any bone; he could feel pain everywhere,” she shared. “From any part of the house, you could hear his moans. And this is something that haunted me often: ‘Am I going to lose him?’ Because he was in so much pain.”
Before the advent of gene therapy, the sole cure for sickle cell disease was a stem cell transplant from a donor. This method succeeded for Wekem after Sylvia found out she was a match for her younger son. Despite encountering frightening complications, the procedure was ultimately successful.
However, antibodies in Wedam’s bloodstream obstructed the same cure for him.
“I was overjoyed. I said, ‘God, you did this for me. But what about my other son?'” Sylvia expressed tearfully. “I desired the same for Wedam.”
When the doctors at Children’s Hospital contacted her just months later to inform her that Wedam might be a suitable candidate for the new gene therapy, it felt like a double miracle — an answer to her prayers.
“All I kept asking was: ‘Is it a cure? Is this something that he’ll no longer need medications for? Will he still experience pain crises?’ They replied, ‘No.’ And I replied, ‘We’re in. We are in.'”
Wedam has witnessed his younger brother’s transformation since his treatment — even utilizing his newfound energy to teach himself how to ride a bike, a task that was previously impossible due to the excruciating pain Wekem endured before his stem cell transplant.
Wekem expressed anticipation in seeing his older brother healed.
“I feel like it altered the life he is going to lead,” Wekem remarked. “I never really believed that there would be another option available to us for him to be treated and cured.”
Nonetheless, Wedam remains doubtful. “Perhaps 60%,” he stated regarding his chances of being cured.
Nevertheless, despite the potentially life-threatening complications that may arise, he affirmed that if there exists any opportunity for a future with less
