First person with sickle cell disease starts gene therapy treatment

In a historic milestone, 12-year-old Kendric Cromer from a suburb of Washington has become the first person in the world with sickle cell disease to undergo a commercially approved gene therapy treatment.

The groundbreaking procedure, which offers hope to an estimated 20,000 sickle cell patients in the United States, marks a significant advancement in medical science.

Living with sickle cell disease, Kendric has endured debilitating pain and limitations in his daily life, unable to engage in ordinary activities without experiencing episodes of severe pain.

With the gene therapy treatment now underway, Kendric and others like him may finally have the opportunity for a life free from the constraints imposed by their condition.

Last year, the Food and Drug Administration granted authorization to two companies to provide gene therapy treatments for sickle cell disease. Kendric’s treatment, administered by Bluebird Bio, represents the first commercial application of this revolutionary therapy.

Another company, Vertex Therapeutics, has also received approval for a CRISPR gene-editing-based remedy but has not disclosed whether the treatment has commenced for any patients.

At Children’s National Hospital in Washington, Kendric began his journey towards potential recovery. The process involves the extraction of his bone marrow stem cells, which will then undergo genetic modification in a specialized lab at Bluebird Bio.

This intricate procedure, spanning several months, aims to replace Kendric’s defective cells with healthy ones, offering the possibility of a cure for his illness.

However, the road ahead is not without challenges. Bluebird Bio requires hundreds of millions of stem cells from Kendric for the treatment, a process that may necessitate multiple collections if the initial extraction is insufficient.

Despite the complexities involved, Kendric’s groundbreaking treatment represents a beacon of hope for individuals living with sickle cell disease. As medical science continues to advance, it offers the promise of a brighter future for those affected by this debilitating condition.