Study of bone marrow transplantation for the patient with mucopolysaccharidosis.

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1.Studies of bone marrow transplantation (BMT)Two basic studies were carried out in vitro for establishment of BMT.(1) Immunological properties of bone marrow cells treated with soybeen agglutinin and sheep red blood cell rosette methods. Bone marrow cells were bractionated by this method described by Reisner et al. for prevention of GVH, and then immunological properties of this fraction were investigated. In results, the bone marrow fraction obtained by this method had very little mature T cells and had low alloreactivity. These results suggest that the bone marrow fraction obtained by this method is able to be used for BMT.(2) Effects of cyclosporin A (CyA) and methotoxesate (MTX) for mixed lymphocyte reaction (MLR). CyA or MTX was usual used for prevention of BVH in vivo. MLR was one of the parameters for GVH. In this study, suppressive effects of CyA or MTX for MLR were investigated. In results, CyA or MTX greatly suppressed MLR. These results suggested that CyA or MTX is able to be use for prevention of GVH in BMT.2.Clinical trial of fibroblast transplantationOne patients with Mucopolysaccharidosis IH/S and one patient with Gaucher disease were treated by fibroblast transplantation. After therapy, it was recognized that both patients' liver size reduced transiently, but there was no effect on the other clinical picture. We conclude that the fibroblast transplantation was not therapeutically useful in these patients.