Special aspects of stem cell therapy in patients with MLD

Metachromatic leukodystrophy (MLD) is caused by mutations in the arylsulfatase A (ASA) gene and leads to demyelination in the CNS. In MLD, transplantation of hematopoietic stem cells is thought to establish a continuous endogenous source of ASA, which can be taken up by somatic cells of the recipient to correct their own lysosomal metabolism. We analysed 8 patients with MLD after transplantation regarding (i) toxicity of a reduced intensity condition regimen; (ii) the potential benefit of co-transplantation of MSC; and (iii) GvHD prophylaxis. i) All patients were transplanted successfully and reached complete do…