Experimental gene therapy could produce cure for Sickle Cell
Sickle Cell disease has been a silent killer in the Black community for decades, but it appears that new gene therapy could hold the key to curing the disease.
In a half-dozen clinical trials, researchers are testing strategies for correcting the problem at the genetic level according to the New York Times. Sickle cell disease is caused by a mutation in one errant gene that is found mostly in people of African descent.
An estimated 100,000 people in the United States and about 300,000 infants worldwide are born with the condition each year. Sickle Cell is most common in sub-Saharan Africa, where an estimated 70 percent of children with it die.
The treatments are being conducted by Bluebird Bio, a biotech company in Cambridge, Mass. Currently, the only true chance for clearing sickle cell disease is a bone marrow transplant.
“This would be the first genetic cure of a common genetic disease,” Dr. Edward Benz, professor of medicine at Harvard Medical School, said to the Times.
People with Sickle Cell have blood cells that are distorted into sickle shapes. The misshapen cells get stuck in blood vessels, causing strokes, organ damage, and severe pain called crises. Children usually return to normal between crises, but teenagers and adults may suffer chronic pain.
Patients may be severely anemic and prone to infections.
In the new treatments, subjects must have stem cells removed from their bone marrow. The stem cells are then genetically modified and infused back into the patient’s bloodstream. The goal is for the modified cells to take up residence in the bone marrow and form healthy red blood cells.
The results have been life-altering. Carmen Duncan of Charleston, S.C., had her spleen removed when she was 2-years-old due to sickle-cell. She spent much of her childhood in and out of hospitals.
“Sometimes I would stay two weeks,” she said told the Times. “A simple touch really hurt.”
Monthly blood transfusions helped and after gene therapy trial her doctors say, she no longer has signs of sickle-cell.
20-year-old Brandon Williams of Chicago, who had suffered multiple strokes in recent years and lost a sister to sickle cell, joined the experimental Bluebird study. Like Duncan, the treatment created enough normal cells to send his sickle cell into remission.
“I was so overwhelmed,” Leuteresa Roberts, Williams mother, said. “I cried tears of joy.
The therapy treatments are currently in the early stages and it will be at least three years before one is approved by the Food and Drug Administration.