StemCells, Inc. has announced that it will present evidence of engraftment, migration and the long-term survival of its proprietary HuCNS-SC neural stem cells following transplantation into patients with a severe neurological disorder.
Importantly, the results show that the cells can persist following the completion of the planned year-long immunosuppression regimen.
The data, which supports the Company’s premise regarding the viability and utility of neural stem cell therapy as a potential treatment for a wide range of debilitating and lethal central nervous system (CNS) disorders, will be reported at the International Society for Stem Cell Research (ISSCR) 9th Annual Meeting, June 15-18 in Toronto, Ontario, by Nobuko Uchida, Vice President, Stem Cell Biology at StemCells, Inc.
“These findings support our vision of a one-time transplantation of stem cells with a lasting clinical benefit,” said Stephen Huhn, MD, FACS, FAAP, Vice President and Head of the CNS Program at StemCells, Inc. “In a field of promises, we now have confirmation that we are on the right path. Ultimately, we hope to show that our HuCNS-SC human neural stem cells can dramatically impact a broad spectrum of neurological disorders.”
The durability of the Company’s HuCNS-SC human neural stem cells within the hostile inflammatory environment of the diseased brain is only one important aspect of the results being presented. The authors also report the persistence of the transplanted donor cells long after immunosuppressive treatment has been discontinued.
This finding is significant because other allogeneic tissue and organ transplants usually require life-long immunosuppression, which carries increased risk for cancer and opportunistic infections.
The data to be presented by StemCells, Inc. also support the belief that the central nervous system is “immune-privileged” and that a relatively brief period of immunosuppression may be all that is required to avoid the risk of transplant rejection within the CNS.
Additional studies will determine whether immunosuppression protocols can be further optimized.
The data to be presented was derived from postmortem examinations of three patients in the Company’s Phase I clinical trial of HuCNS-SC cells in neuronal ceroid lipofuscinosis (NCL, also referred to as Batten disease), a rare and fatal neurodegenerative disorder in children.
The patients expired from causes related to the underlying disease. Analysis of the brain tissue of two of the patients at autopsy revealed the presence of the donor cells at the sites of transplantation as well as evidence of migration into deeper structures of the brain, confirming similar observations made in animal models.
In one of these patients, the persistence of the donor cells was evident 2.5 years post transplant and 1.5 years after completion of the immunosuppression regimen.