Neuronal Ceroid Lipofuscinosis (NCL) Clinical Program

Our clinical study in NCL produced the first evidence of human safety involving direct implantation of up to a total of one billion HuCNS-SC cells into multiple sites within each cerebral hemisphere. The long-term follow-up study has further confirmed long-term safety of HuCNS-SC cells out to five years post-transplant, as well as providing post-mortem evidence of donor cell survival and migration within recipient brains. Overall, the NCL study results represent the first, and thus far only, multi-year data set following transplantation of neural stem cells into human subjects. This long-term data supports the feasibility of the Company's approach in other potential neurological and neurodegenerative disorders affecting the brain.

Completed:  Phase I Clinical Trial in NCL

The Phase I clinical trial, conducted at Doernbecher Children's Hospital and Oregon Health & Science University (OHSU), transplanted a total of six patients and was completed in January 2009. All six patients were followed for 12 months after transplantation in the Phase I trial and five patients were enrolled in the separate, four-year, long-term, follow-up study. The long-term study has completed and three of the five patients survived through the four-year study.

The Phase I and long-term follow-up studies assessed cognitive and neurological function. Outcomes were generally consistent with the expected course of impairment associated with this neurodegenerative disease. It is noteworthy that each patient maintained stable quality-of-life measures.

For a neuroprotective strategy to show meaningful clinical outcomes, sufficient numbers of functional neurons must exist at the time of intervention, hence the need to transplant subjects earlier in their disease course. With this premise, a Phase Ib clinical trial was initiated to examine safety and signs of efficacy in subjects with early and less pronounced disease. The ultra-rare prevalence of NCL and the difficulties inherent in identifying patients in the early stage of the disease made Phase Ib enrollment extremely challenging. Of 22 patients considered for screening and enrollment, none met the inclusion criteria for the trial. No patients were enrolled and the study was terminated for lack of timely accrual.

About NCL

Neuronal ceroid lipofuscinosis (NCL), also sometimes referred to as Batten disease, is a fatal lysosomal storage disorder in children that results in severe progressive neurodegeneration. There are currently no effective treatments for lysosomal diseases that affect the central nervous system.

Learn more about NCL…


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

View our publications on neuronal ceroid lipofuscinosis…