Drug therapy may include antiepileptics, or agents to help control spasticity. Attempts at enzyme replacement therapy have thus far been unsuccessful. The transplant process takes about a year to produce beneficial changes in the central nervous system. Thus, ideally a presymptomatic child should be transplanted at least one year before the age at which his sibling developed symptoms. The results of BMT in infantile MLD are conflicting, but in one case clinical stabilization occurred, and it was demonstrated that donor cells penetrated the central nervous system.
Transplantation is indicated in juvenile forms of the disease.
A recent report detailed the case of a child who had been symptomatic since age 9 and was transplanted at age 16. During 8 years of follow-up, he remained stable, with no further deterioration. It is unclear how BMT may work. Transplant-derived lymphoid cells may enter the brain and liberate enough arylsulfatase A extracellularly to stop further neuronal death. Alternatively, other factors, including the medications used for maintenance of transplants, could also contribute to modification of the disease process.