MLD Foundation

Is there a cure? - Treatments & Therapies

Today there is no cure for Metachromatic Leukodystrophy, MLD. Research is underway pursuing several new therapies, but there is no cure on the near-term horizon.

The only treatment (therapy) at present anywhere for anyone is a stem cell (SCT) or bone marrow transplant(BMT). But today's successful stem cell or bone marrow transplant (BMT) is not a cure for MLD - at best it slows further deterioration by replacing the bad marrow with "good" marrow that can produce the missing enzyme.

Today's transplants do not reverse the damage that the brain has already experienced so they are most successful when there is little or no damage. Often this means that an older sibling is diagnosed, a younger sibling is identified to have MLD but be pre-symptomatic and the younger sibling is transplanted.

The procedure is very hard on the MLD sufferer and the chance of death is significant. The transplant teams are working hard to try to minimize the conditioning, which has traditionally be some sort of chemotherapy to kill off the existing bone marrow. You need to weigh the potential benefits and risks for your personal situation. Transplants for MLD patients are still classified as experimental by many in the medical and insurance fields. Some insurance policies will not cover the $250,000+ price tag for these procedures and there can be a lot of expenses related to post-transplant follow-up. BMT's are in phase 2 of human clinical trials.