Currently, the only form of treatment for a person diagnosed with MLD is a bone marrow or stem cell transplant, and these are typically only performed on people who do not display any significant symptoms of MLD. Gene therapy research is ongoing, but less-toxic transplants are currently showing the most promise. For more information on MLD research efforts, please click here.
The advantage that stem cell transplants have over bone marrow transplants is that stem cells do not have to match as closely as bone marrow, and therefore are much easier to obtain. In addition, doctors have found that patients who receive stem cell transplants seem to show more improvement than patients who receive bone marrow transplants. Click here to read a paper on the effectiveness of umbilical cord stem cell transplants for children with inherited metabolic disorders such as MLD.
Click here to view a video of Dr. Joanne Kurtzberg discussing stem cell transplants (click on "Dr. Joanne Kurtzberg" in the lower right-hand corner of the page that appears). Click on "Cord Blood Transplants" to hear MLD mother Jane Trimper discuss the benefits of using cord blood for stem cell transplant.
If you know of a child or loved one who was recently diagnosed with MLD and are looking for information regarding umbilical cord stem cell transplants, please contact Dr. Joanne Kurtzberg at Duke University by e-mail at firstname.lastname@example.org. You may also view the Pediatric Blood and Marrow Transplantation site.
Transplant Outcomes in Leukodystophies was published in January 2010 and authored by Paul Orchard and Jakub Tolar. It discusses the effectiveness of stem cell transplants for Krabbe disease, ALD, and MLD at various points of disease progression.