Boston Gene Therapy Cuts Sickle Cell Wait to 7 Weeks

For people living with sickle cell disease, getting gene therapy has meant months of hospital visits and waiting up to half a year for treatment. Now, researchers at Boston Children's Hospital have cut that time down to seven weeks while making the process simpler and more effective.

The new approach successfully collected enough blood stem cells in just one hospital session for 10 out of 11 patients. Once collected, the team genetically modified these cells to reactivate fetal hemoglobin (which doesn't cause sickling) while turning off the problem gene causing the disease.

Dr. David Williams and his colleagues at the Dana-Farber/Boston Children's Cancer and Blood Disorders Center figured out how to speed up every step. After the modified stem cells were frozen and safety tested, patients received their personalized treatment in an average of seven weeks compared to industry standards of six months or longer.

The technique is based on fundamental research by Dr. Stuart Orkin, who discovered how fetal hemoglobin naturally shuts off during a baby's first year of life. Williams' team essentially found a way to turn it back on using the patient's own cells, creating a treatment without the complications of donor transplants.

The Ripple Effect

The results are already spreading far beyond Boston. The FDA and National Institutes of Health approved and funded a larger multisite trial that enrolled 25 additional participants based on the pilot study's safety success.

The FDA recently granted approval for even more patients to receive the therapy through its Expanded Access Program before formal drug approval. This means people with sickle cell disease don't have to wait years for help that's already proven safe and effective.

Perhaps most exciting is where this therapy is heading next. The team licensed their technology to Caring Cross, a nonprofit opening trials in Brazil and India, where large populations suffer from sickle cell disease but have limited access to advanced treatments.

Patients in the trial have been followed for up to seven years after treatment with no unexpected safety problems related to collecting, manufacturing, or transplanting their modified stem cells. The researchers say 36 people have now been treated using this faster, more efficient approach.

Williams says his team has figured out how to overcome the barriers that kept gene therapy out of reach for many people with sickle cell disease. What once required countless hospital visits and months of uncertainty now happens in weeks, transforming lives across the country and soon around the world.