What is the most effective treatment for ALD?
At present, there are only two treatments for ALD: Lorenzos oil and stem cell transplantation using either bone marrow stem cells or umbilical cord blood stem cells. These treatments appear to be most effective if they are used before the onset of symptoms. Can you tell me more about transplants (bone marrow and cord blood) There are currently two sources for transplanted stem cells: umbilical cord blood (UCBT) or bone marrow (BMT). In both cases, the goal is to provide the patient with healthy stem cells that produce a functioning ALD proteinthe protein that is lacking in people with ALD. Among the small number of patients who have had transplants, some have had their condition stabilize, and a few even made slight improvements. But there is a very narrow window in which stem cell transplantation appears to be effectivethe time between when the brain lesions appear on an MRI, and when the boy experiences only mild clinical symptoms. This treatment also has serious risks. Both types of
