Bluebird Bio declined to comment on the agreement or speculate on the price of the therapy.įour weeks after the infusion of stem cells, Helen was strong enough to be discharged. But the hospital has licensed patents it develops to the biotech firm Bluebird Bio, giving the company the option to sell the treatment and pay royalties to the hospital. If approved, such cures would almost certainly cost $1 million or more, experts say, raising questions about affordability.įor now, Helen’s therapy is covered by federal research grants. One also aims to flip the genetic switch, while the other adds a new gene. Two other gene therapy trials for sickle cell, using different methods, are also underway in the United States. Collins, the director of the National Institutes of Health. “It’s an exhilarating success story for those of us who have waited and hoped for this day,” said Dr. But if it is, sickle cell disease could be the first common genetic disorder to be cured by manipulating human DNA. And it will be years before they know if this new procedure is effective in the long term. Scientists have been experimenting with gene therapy for two decades, with mixed success. But then they heard about a potential breakthrough: a complex procedure to flip a genetic switch so the body produces healthy blood. They lived with one hope for a cure, a dangerous and sometimes fatal bone marrow transplant usually reserved for those with a healthy sibling as a match. Like many others affected by sickle cell, the Obando family faced a double whammy: not one but two children with the disease, Helen and her older sister, Haylee. Stuart Orkin, 73, who has labored against the disease since he was a medical resident in the 1970s. This is the story of two quests for a sickle cell cure - one by the Obando family, and one by a determined scientist at Boston Children’s Hospital, Dr. The disease also affects people with southern European, Middle Eastern or Asian backgrounds, or those who are Hispanic, like Helen.