A therapy using a child’s own stem cells has shown promise in fighting a deadly neurological illness made famous in the movie “Lorenzo’s Oil.”
Researchers from the Harvard Medical School found that genetically altering a patient’s stem cells, then reintroducing them to the patient’s body, has produce positive results in the battle against adrenoleukodystrophy.
Adrenoleukodystrophy affects about one in 21,000 boys, and it occurs when those boys lack one copy of the X chromosome. The degenerative neurological condition leaves patients unable to make a protein that helps break down specific fatty acids. A buildup of those acids affects the nerves, muscles and brain.
The treatment involved extracting stem cells from the boys, modifying them to carry the missing gene, chemotherapy to eliminate diseased cells, then the transplant of the altered stem cells.