Mucopolysaccharidosis IIIA (MPS IIIA), also known as Sanfilippo type A syndrome - Healing Genes

Mucopolysaccharidosis IIIA (MPS IIIA), also known as Sanfilippo type A syndrome

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UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (DUOC-01)

Augmentation of Umbilical Cord Blood Transplantation for Inherited Metabolic Diseases With Intrathecal Administration of Human Umbilical Cord Blood-Derived Oligodendrocyte-Like Cells

Phase 1


Doctors at Duke University Medical Center are recruiting participants with an inherited metabolic disorder for a trial of an augmented cord blood transplant directly through the spinal cord, with the goal of achieving remyelination of neurons and decreased gliosis, as well as production of the needed enzymes to correct the disorder. This cell therapy treatment will be evaluated through brain MRIs and other neurocognitive tests. The therapy, DUOC-01, will be administered through a spinal cord (intrathecal) injection between 26 to 28 days after a cord blood transplant. DUOC-01 is a portion of the cord blood cells that are grown into a type of cell that will produce the lysosomal enzymes relevant to these disorders:

  • Adrenoleukodystrophy (ALD)
  • Batten Disease Hunter Syndrome (MPS II)
  • Krabbe disease (Globoid Leukodystrophy)
  • Metachromatic Leukodystrophy (MLD)
  • Niemann Pick disease type A or B
  • Pelizaeus-Merzbacher disease (PMD)
  • Sandhoff disease
  • Tay Sachs disease
  • Alpha Mannosidosis Sanfilippo (MPS III)

The researchers hope that the treatment will cause engraftment, or permanent growth, of the transplanted cells and a constant replacement source of the enzymes needed for a reduction or resolution of the disease.


  • ≥ 1 week to <22 years of age
  • a life expectancy of ≥ 6 months
  • Have neurologic evidence of their disease (via MRI, EEG, or clinical assessment, etc.)
  • Not be pregnant or breastfeeding
  • Be HIV-negative
  • Able to have an MRI scan or lumbar puncture
  • Have an available, matched unrelated cord blood


  1. Prescreening to confirm eligibility of the patient to participate.
  2. A cord blood transplant
  3. A single spinal injection of the DUOC-01 therapy 26 to 28 days after step (2)
  4. The patient will be closely observed for adverse reactions for ~24 hours
  5. 1 month after therapy, a neurotoxicity screening will be required
  6. Standard follow-up evaluations including MRI, EEG, nerve conduction and other neurocognitive testing will be conducted for 1 to 5 years’ time.


Duke University Medical Center in Durham, NC is recruiting participants for this trial. Map

Contact: Jennifer Baker, RN  |  [email protected]



Joanne Kurtzberg, MD & The Marcus Foundation

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