Bone marrow transplantation can prevent progressive neurological deterioration if performed early in life. The rationale behind cell therapy for ALD is to provide the patient with healthy stem cells that produce a functioning ABCD1 protein. Umbilical cord blood and bone marrow are commonly used stem cell sources for transplantation in ALD children patients. The healthy donated cells migrate into the brain, express the ABCD1 protein, and halt or even reverse brain damage.
Transplantations have been successful to some extent; some patients demonstrated stabilized condition and a few demonstrated slight improvements. There is a very narrow window for stem cell therapy intervention: between brain lesion detection by MRI and first appearance of symptoms.
EMBRYONIC DEVELOPMENT & STEM CELL COMPENDIUM
