Gene therapy for sickle cell disease: A patient’s story

During Sickle Cell Awareness Month, Alabamian Lynndrick Holmes, 29, shared his experience of being free of the sickle cell disease two years after receiving genetic treatment as part of an NIH clinical trial that might hold the key to a cure of this disease.

Holmes was diagnosed at birth but sickle cell disease took the greatest toll on him as an adult. Now, he says he feels amazing, and encourages others to participate in clinical trials.

He enrolled in the gene therapy trial at NIH with guarded hopes after exhausting many avenues of treatment. His participation in the trial, he says, was “soul-awakening”—a stark contrast from his experiences back home in Mobile, Alabama. “I experienced burgeoning humanity—compassion, empathy, and a genuine concern for my disease,” he says. “I thought I was going to have to justify my circumstances, validate my disease, and prove that sickle cell exists all over again. But I didn’t.”

This clinical trial, which is still recruiting participants, is testing a novel gene replacement therapy in people with severe sickle cell disease. Preliminary findings suggest that the approach has an acceptable level of safety and might help patients consistently produce normal red blood cells instead of the sickle-shaped ones that mark this painful, life-threatening disease.

People interested in participating should contact the Office of Patient Recruitment by phone 1-800-411-1222 or email prpl@cc.nih.gov.

The experimental treatment involves removing hematopoietic stem cells from the patients’ bone marrow or blood and adding a therapeutic beta globin gene, which is defective in people with sickle cell disease. The cells are then returned to the patients, leading to the production of anti-sickling hemoglobin (T87Q).

Based on preliminary findings, which were presented at the latest meeting of the American Society of Hematology, researchers believe the new gene replacement therapy will enable the patients’ bone marrow to produce normal red blood cells consistently.

Learn more about this trial:

Visit the Office of Patient Recruitment
Call 1-800-411-1222
Email prpl@cc.nih.gov