The key to this trial is its gentleness. A patient undergoes a course of drugs and radiation to kill every bit of marrow, then the transplant and another course of drugs to prevent infection. Recovery can take more than a year, if a patient survives. Because people with FA are extremely sensitive to radiation and often develop tumors and leukemia as a result of treatment, transplants are often viewed as last resort therapy. This transplant protocol is different.
Patients skip the radiation and drugs, giving their immune systems a huge break.
All they have to donate is a bit of bone marrow, leaving the tricky part up to Walsh and his team. To make the fix, the research team plans to track down a few stem cells from each patient's marrow by extracting blood and then spinning it through a centrifuge. To those stem cells, they'll introduce a retrovirus carefully engineered to transport the repaired genetic material back into the cells. Once the vector inserts itself and its payload into the stem cells, it should swap out the old gene with the new establishing the correctly functioning gene into the stem cells. These cells will leave their lab life and get transplanted back into each patient.