Sickle Cell Gene Therapy , Cure

12-Year-Old Begins Newly Approved Sickle Cell Gene Therapy That Could Provide Cure

Kendric Cromer is the first commercial patient to receive the groundbreaking treatment.

Kendric Cromer, 12, is the first patient in the United States to undergo a newly approved gene therapy for sickle cell disease.

Kendric is being treated at Children’s National Hospital in Washington, D.C. By next year, he may become the first person cured of sickle cell disease with gene therapy. Kendrick suffers daily debilitating pain, which leads to frequent hospital stays.

“We see the light at the end of the tunnel and know that our child is the first to experience it outside of research—to know that it’s safe and the doctors here believe in it,” Deb Cromer, Kendric’s mother, told NBC News Washington.  “I would go to the end of the earth to make sure he was cured. There’s nothing I wouldn’t do for my son, but this makes me proud.”

According to the U.S. Centers For Disease Control and Prevention, approximately 100,000 people live with sickle cell disease in the U.S. Ninety percent of them are African American.

The FDA approved Bluebird Bio’s Lovo-Cel Gene Therapy last year. Before the gene therapy, Kendric had to have his bone marrow stem cells extracted, a process that took six to eight hours. If they didn’t retrieve enough cells in the first collection, the company had to try again. Those cells will be genetically modified and, after three months, infused back into his body.

The process is so cumbersome that Bluebird estimates it can only treat 85 to 105 patients each year.

“This is a big effort,” Dr. David Jacobsohn, chief of the medical center’s division of blood and marrow transplantation, told The New York Times.

Jacobsohn said at least 20 patients were eligible and interested at Children’s National. The decision of who would be treated first was based on who was the sickest and whose insurance would cover the costs.