UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

This study provides a stem cell transplant combined with a new treatment involving specialized cells called oligodendrocyte-like cells (derived from umbilical cord blood) delivered directly into the spinal fluid. It targets children with serious inherited metabolic diseases that destroy myelin — the protective coating around nerve fibers in the brain — with the aim of slowing or stopping disease progression. You may be eligible if: - You are between 1 week and 21 years old - You have been confirmed (by enzyme or genetic testing) to have one of the following diseases: Adrenoleukodystrophy, Batten Disease, Hunter Syndrome, Krabbe Disease, Metachromatic Leukodystrophy, Niemann-Pick A or B, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Alpha Mannosidosis, or Sanfilippo Syndrome - You have neurological evidence of disease (abnormal EEG, brain MRI changes, or clinical symptoms) - You have adequate organ function and a performance status of at least 40% - You have a suitable matched umbilical cord blood unit available - Your life expectancy is at least 6 months You may NOT be eligible if: - You had a prior organ, tissue, or stem cell transplant within the last 3 years - You previously participated in a gene or regenerative cell therapy study - You cannot have an MRI or lumbar puncture (spinal tap) - You have intractable seizures, chronic aspiration, or a bleeding disorder - You are HIV positive - You have an uncontrolled infection - You are pregnant or breastfeeding - You have an active cancer or are receiving radiotherapy, immunosuppressants, or chemotherapy Talk to your doctor to see if this trial is right for you.