Monoclonal Antibody Conditioning for Allogeneic Stem Cell Transplantation of Patients With Inherited Metabolic Storage Diseases

Inclusion criteria: Patients with inherited metabolic storage diseases of all ages are eligible. Diagnosis of inherited metabolic storage disease confirmed by standard biochemical and genetic studies in consultation with the Department of Genetics at the Baylor College of Medicine Inherited metabolic storage diseases which may be stabilized or improved by stem cell transplantation include: Hurler, Hunter, Maroteaux-Lamy, Sly, Wolman, Gaucher, Farber, Nieman-Pick, Mannosidosis, Aspartylglucosaminuria, Fucosidosis, Neuronal Ceroid-Lipofuscinosis, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, and Adrenoleukodystrophy Availability of an HLA matched or mismatched (up to one haplotype) donor who is not an obligate carrier for the inherited condition or an unrelated HLA matched stem cell donor. Fully matched is defined as 6/6 match by high resolution DR based DNA typing. Female patients of childbearing age must have a negative pregnancy test and be willing to use an effective means of birth control. Exclusion criteria: Patients with a life expectancy (<6 weeks) limited by diseases other than inherited metabolic storage disease Patients with advanced inherited metabolic storage disease, which has not been stabilized or improved by hematopoietic stem cell transplantation. Patients with symptomatic cardiac disease, or evidence of significant cardiac disease by echocardiogram (i.e., shortening fraction <25%) Patients with severe renal disease (Creatinine >2 x normal for age) Patients with known allergy to rat serum products Patients with a Karnofsky or Lansky score <50%. Patients with a severe infection that on evaluation by the Principal Investigator precludes ablative chemotherapy or successful transplantation Patients with severe personality disorder or mental illness or neuropsychological evaluation indicating too much damage for the transplant to be of benefit. Patients with documented HIV positivity. Patients with grade III-IV liver toxicity not related to metabolic storage disease.