Gene therapy
Gene therapy is a new approach to stem cell transplantation. Instead of finding a donor who is enough of a match to your child to donate bone marrow stem cells or umbilical cord blood stem cells, the transplant is done using the childs own cells. The appropriate cells are removed from the people with ALD, the correct genetic sequence is inserted into those cells, and they are put back into the patient The repaired cells will then produce the protein that had been missing or defective prior to treatment, and once successfully engrafted, the disease process will halt or reverse. This is a very promising area of treatment, but there are many scientific challenges to conquer and risks to be considered. At present, it is undergoing clinical trial in France, with a very small group of patients, and is not generally available.
