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Stem Cells Stem Sickle Cell

Stem Cell Research
AP
A group of French doctors said Sunday they believe they can cure children with severe sickle cell disease through stem cell transplants without risking serious complications or death.

The researchers, who have performed 69 transplants since 1988, reported an 85 percent disease-free survival rate — and even better results since revising their mix of anti-rejection drugs in 1992.

Six patients died in the earlier phase — four after rejecting the new stem cells — but there were no deaths in the last 30 transplants, said lead researcher, Dr. Francoise Bernaudin of The Hopital Intercommunal de Creteil outside Paris.

The findings, which have not yet been published, were presented Sunday at the annual meeting of the American Society of Hematology in Philadelphia.

"It's going to change the way we treat individuals with this disorder," said Dr. Ronald Hoffman of the University of Illinois, the society's president. "If they're truly cured ... they're going to be freed of the consequences of a chronic disease."

Sickle cell disease is an inherited blood disorder that affects about 70,000 Americans, most of them black.

Researchers are pursuing several treatments for sickle cell disease, including gene therapy, drug therapies and stem cell transplants.

The disease prevents the blood from carrying enough oxygen and nutrients to the heart, brain and other organs. Patients often suffer chronic, painful conditions including kidney and lung damage and are prone to heart attacks and disabling strokes.

Most sickle cell patients used to die in childhood, but better treatments now enable them to live into their 40s and 50s. However, doctors have been frustrated in their attempts to improve the patients' quality of life.

"Maybe if a new drug appears which seems efficacious, we will stop again, but for the moment, stem cell transplant remains the only treatment offering the cure," Bernaudin said.

By Maryclaire Dale