Allogeneic hematopoietic stem cell transplantation (TUMS)

This investigational cell therapy involves allogeneic hematopoietic stem cell transplantation (HSCT) for the treatment of Mucopolysaccharidosis (MPS) types 1, 6, and 7, developed by the Tehran University of Medical Sciences. The therapy utilizes hematopoietic stem cells derived from healthy donors (related, unrelated cord blood, or haploidentical) to provide a continuous endogenous source of deficient lysosomal enzymes. Following a non-total body irradiation (non-TBI) myeloablative conditioning regimen, the transplanted cells engraft and secrete functional enzymes—specifically alpha-L-iduronidase, arylsulfatase B, or beta-glucuronidase—which are then taken up by the recipient's tissues through a process known as cross-correction. This mechanism allows for the degradation of accumulated glycosaminoglycans (GAGs) in various organs, potentially halting or reversing the progressive systemic damage associated with these lysosomal storage disorders.

Other names

HSCT for MucopolysaccharidosisHematopoietic Stem Cell Transplantation for Mucopolysaccharidosis