While other research labs are working on isolating the remaining five, Walsh has focused his research efforts on FA-A, the gene that affects more than 70 percent of FA patients. In April, Walsh and 10 FA patients will start their trial testing the therapeutic effects of transplanting a corrected gene back into patients' bone marrow. He'll treat both adults and children, and it doesn't matter how poorly or how well they are doing. 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.