Multi-Institutional Assessments of Transplantation for Metabolic Disorders

The rational for allogeneic transplantation as therapy for inherited metabolic disease was provided by the fundamentally important work done by Neufeld’s group over 40 years ago, confirming that normal cells could “cross-correct” cells with storage diseases such as Hurler syndrome. Initial reports in the early 1980s confirmed that these patients could be transplanted, resulting in improvement in visceral disease in patients with mucopolysaccharide accumulation. Later investigations suggested that transplantation arrested neurologic deterioration in some disorders, such as Hurler syndrome (mucopolysaccharidosis type IH [MPS IH]), but is less effective in achieving stabilization of cognitive function in MPS II (Hunter) and MPS III (Sanfilippo).