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.
- Absence of neurons in control group
- Presence of neurons with transplanted stem cells
|Time on Rotorod (seconds)||60 –||
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…