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Table 1 Overview of HuCNS-SC clinical translation programs for central nervous system disorders

From: Clinical translation of human neural stem cells

CNS area Disease/injury Stage Outcome
Brain Neuronal ceroid lipofuscinosis (Batten disease): infantile and late-infantile Phase I completed Safety, feasibility and tolerability of HuCNS-SC transplants. Post-mortem evidence of long-term donor cell survival in post-mortem 3/6 subjects alive 5 years post-transplant
   Phase Ib suspended No accrual of eligible subjects
  Pelizaeus-Merzbacher myelin disorder Phase I completed MRI evidence of donor-derived myelin and modest gains in neurological function
  Alzheimer’s disease Preclinical Enhanced synaptic function and restored memory in two AD relevant models
Spinal cord Thoracic spinal cord injury Phase I/II in progress Sensory gains observed in first cohort.
  Cervical spinal cord injury Preclinical Improve motor function in SCI mice
Eye Age related macular degeneration Phase I/II in progress Subject accrual ongoing
  1. AD, Alzheimer’s disease; CNS, central nervous system; SCI, spinal cord injury.