NAC improves outcome after bone marrow transplant for ALD

Researchers at the University of Minnesota, Minneapolis, investigated the use of N-acetyl-L-cysteine (NAC) before and after stem cell (bone marrow) transplant in three boys with advanced adrenoleukodystrophy (ALD), a neurological disorder (the same disorder Lorenzo Odone had in the movie, Lorenzo's Oil). The researchers found the boys' medical conditions were stable six months after the transplant. Other children in a comparison group who did not receive NAC died within the first year after the transplant (even though the transplant was successful). The researchers are working to understand how NAC works in ALD, and whether NAC will benefit children with less severe forms of the disease. The research was published in the February 2007 issue of Bone Marrow Transplantation.