MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD)

Inclusion Criteria: * Age ≥ 4 years at time of study enrollment * Diagnosis of ALD - the diagnosis of ALD can be made by either biochemical or molecular/genetic evidence (plasma VLCFA or ABCD1 mutation analysis) supportive of ALD as the cause of cerebral demyelination * Evidence of active cerebral disease - defined as the presence of gadolinium enhancement on a single brain MRI study - MRI used for eligibility determination may be performed at an outside institution; the most recent MRI used to determine eligibility must be within 2 calendar months of the date of enrollment on this study * ALD MRI (Loes) score ≥ 10 * Off of N-acetylcysteine, systemic immunosuppressive drugs or any other therapeutic intervention (except hydrocortisone and/or fludracortisone for the treatment of adrenal insufficiency) for ≥ 10 days prior to the first IT-MSC dose * Life expectancy of \>6 months as determined by the enrolling researcher and documented in the medical record * Voluntary written consent provided by parent(s)/guardian(s) Exclusion Criteria: * A candidate for allogeneic hematopoietic stem cell transplantation as determined by the University of Minnesota Inherited Metabolic and Storage Disease group * Inability to undergo sedation, lumbar puncture or MRI studies for any reason * Inability to stay in Minnesota for therapy through the day 28 evaluation