An Alabama man is free of sickle cell disease after receiving gene therapy for 2 years as part of a clinical trial.
from the patient’s bone marrow and tweaking the gene that causes cells to become misshapen, says Julie Kanter, MD, director of the Adult Sickle Cell Clinic at the University of Alabama at Birmingham, one of the trial sites. The modified gene is then put back in using deactivated HIV. The trial is sponsored by biotech company bluebird bio and has test sites across the country.
Until now, the only way to treat someone was with a matched sibling transplant, Kanter says. But many patients do not have a full sibling, and even if they do, there is only a 15% chance of a match.But to call it a cure would be jumping the gun, she says. Holmes, who is being monitored every 3 months, must be sickle cell-free for 5 years before it can be classified as a cure.
“We hope it will be curative, but we can’t say that yet,” Kanter says. But, she says, “He’s having frequent follow-ups from the NIH, and it looks promising.” Sickle cell, which is present at birth, causes symptoms including painful swelling of the hands and feet, extreme
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