HuCNS-SC® (human neural stem cells)
Our HuCNS-SC® human neural stem cells are manufactured by qualified personnel working in a clean room production environment, according to current Good Manufacturing Practices (cGMP). In 1999, StemCells scientists were the first to isolate and expand human neural stem cells. This groundbreaking research was based on using monoclonal antibodies against specific cell surface markers to prospectively isolate the population of neural stem cells from human brain tissue, and then purify and expand these cells into cryopreserved cell banks. These unmodified and highly purified cells are then prepared for direct transplantation.
Pre-Clinical Studies: Proof of Concept
The results of rigorous pre-clinical studies, including the transplantation of these cells in thousands of immunodeficient mice, have shown that our cells engraft, migrate, differentiate into the three major CNS cell types (neurons, astrocytes and oligodendrocytes), and possess the ability to survive long-term with no evidence of tumor formation or adverse effects. The engraftment and long-term survival of HuCNS-SC in multiple disease relevant animal models indicates the potential for a similar effect in human patients.
Differentiation of HuCNS-SC cells in transplanted mouse brain.
Clinical Studies: Proof of Safety and Initial Efficacy
The first clinical trial of our HuCNS-SC product candidate was completed in 2009. This Phase I trial in neuronal ceroid lipofuscinosis (NCL), a rare childhood disease, demonstrated the first clinical safety and tolerability of HuCNS-SC cells and the transplantation procedure. Data gathered in 2011 from subjects who participated in this first trial showed that transplanted HuCNS-SC cells can persist even after immunosuppression has been discontinued. In 2014, we reported long-term evidence of safety, up to seven years post-transplantation, for the surgical transplantation of the HuCNS-SC cells into multiple sites in the brain and at doses of up to one billion cells. The study results represent the first, and thus far only, multi-year data set following transplantation of neural stem cells into human subjects, and supports the feasibility of the Company's approach in multiple neurological disorders.
Our Phase I trial in PMD and the Phase I/II clinical trials in spinal cord injury (SCI) and age-related macular degeneration (AMD) reported continued emerging safety data as well as signs of preliminary efficacy.