At the end of last year, the FDA approved two gene therapies based on CRISPR technology for sickle cell disease, the first cell-based treatments for the disease.
“Sickle cell disease is a rare, debilitating, and life-threatening blood disorder with significant unmet need, and we are excited to advance the field, especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” Nicole Verdun, director of the Office of Therapeutic Products with the FDA’s Center for Biologics Evaluation and Research, said at the time, as reported by The Dallas Express.
“Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”
Cells are collected from a patient, modified using CRISPR, and then infused back into the patient’s system in a one-time dose as a “hematopoietic [blood] stem cell transplant.” These modified cells will then multiply in a patient’s bone marrow and increase production of fetal hemoglobin which will aid oxygen delivery and prevent the spread of red blood cells affected by the disease.
Only 10 months after the approval was announced, The New York Times reports on one of the first children ever to be treated with the new gene therapy. Here’s the start of the story:
There was supposed to be a special party for Kendric Cromer, 12, last Wednesday, but it had to be postponed because he was too groggy to celebrate.
It was meant to mark the first day of his new life — the day he became one of the first children ever to be treated with a newly approved gene therapy that will free him from the sickle cell disease that has stolen his childhood.
On Sept. 11, despite the excitement of the moment, Kendric was unable to keep his eyes open as he lay in his hospital bed at Children’s National Hospital in Washington because of the drugs he had been given in preparation for his treatment.
His life with the disease has been punctuated by episodes of excruciating pain, requiring days in the hospital as doctors tried to control it. Sickle cell eroded his hip bones. It prevented him from riding a bike or playing soccer or even going outside when the temperature was below 55 degrees Fahrenheit because cold often brought on intense pain.
Now he could see a future — in a month or so — without pain from sickle cell.
“I can’t wait to start my new life,” he told his mother, Deborah Cromer.