Clinical translation of human neural stem cells

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

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

ISSN: 1757-6512