Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)

Inclusion criteria

• ✓. Patients must have a confirmed diagnosis of inherited metabolic disorder / inborn error of metabolism. Diagnosis should be confirmed by appropriate test(s) (enzyme and/or mutation analysis) before study entry. Patients must not be eligible for myeloablative chemotherapy as a preparative regimen for transplant due to age, co-morbidities or organ dysfunction.
• ✓. Patients must be ≥ 3 years of age
• ✓. Patients must have Lansky or Karnofsky performance status ≥40
• ✓. Patients must have adequate function of other organ systems as measured by:
• ✓. Patient must have a related donor \[identical or mismatched for 1, 2 or 3 histocompatibility leukocyte antigen (HLA)-A, -B or -DR loci\].
• ✓. Patient, and parent, or legal guardian must have given written informed consent according to FDA guidelines.
• ✓. Patients must have a minimum life expectancy of at least 6 months.
• ✓. Female patients of childbearing potential cannot be pregnant or lactating/breast-feeding and must be either surgically sterile, postmenopausal (no menses for the previous 12 months), or must be practicing an effective method of birth control as determined by the investigator (e.g., oral contraceptives, double barrier methods, hormonal injectable or implanted contraceptives, tubal ligation, or partner with vasectomy).