Lysosomal Storage Disorders / NCL: Pre-clinical Studies

Published pre-clinical data demonstrates the therapeutic potential of the StemCells HuCNS-SC® (purified human neural stem cells) platform technology to treat neurodegenerative diseases such as neuronal ceroid lipofuscinosis (NCL), a lysosomal storage disorder also known as Batten disease.

Download PDF of published research; see page 316, Figure 6…

NCL Before Image
Absence of neurons in control group
NCL After Image
Presence of neurons with transplanted stem cells
Time on Rotorod (seconds) 60  NCL Chart Data
  Age (weeks)  
  • ■  Non-diseased (normal)
  • ■  Diseased: No neural stem cells
  • ■  Diseased: With neural stem cells transplanted

Pre-clinical studies have shown that when transplanted in a mouse model of infantile NCL (INCL), our human neural stem cells engraft, migrate throughout the brain and continuously secrete the missing lysosomal enzyme characteristic of NCL, which is needed to process cellular waste and keep neurons functioning and healthy. When compared with a control (non-transplanted) group, the mice that received the transplanted neural stem cells showed a statistically significant reduction in cellular waste build-up, protection of critical host neurons and delayed loss of motor function.

We have further demonstrated that human neural stem cells also produce the enzyme missing in late infantile NCL (LINCL), thereby providing the scientific rationale for enzyme replacement via transplantation of these cells in this subtype, as well as in INCL.

Learn about our completed NCL clinical trials…

View our publications on NCL…

Learn more about NCL…

About NCL

Neuronal ceroid lipofuscinosis (NCL), also known as Batten disease, is a fatal neurodegenerative disorder in children that results in a progressive loss of neurological function. Today there is no cure or effective treatment.

Learn more about NCL…

Clinical Program

The Company completed a Phase I clinical trial in NCL in January 2009.

Learn about our completed NCL clinical trials…


Findings from studies performed by StemCells, Inc. and its collaborators on the use of human neural stem cells for the treatment of lysosomal storage disorders have been published.

View our publications on NCL…

  • Pre-clinical proof of concept in Batten disease

    Irving Weissman, MD
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