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CNS Stem Cell Transplantation for Neuronal Ceroid Lipofuscinoses: Summary of Long-term Follow-up Study Results.
Selden NR, Al-Uzri A, Steiner R, Huhn SL
Abstract:
INTRODUCTION:
An open-label dose-escalation Phase I trial of a highly purified, expandable population of human central nervous system stem cells (HuCNS-SC, StemCells, Inc., Newark, CA) was conducted in subjects with advanced stage infantile and late-infantile neuronal ceroid lipofuscinoses (NCL). This investigation was the first US FDA authorized use of human neural stem cells for clinical testing. The current report summarizes the outcome of the separate 4 year long-term follow-up (LTFU) study which was conducted for post-transplantation surveillance.
METHODS:
Six subjects underwent bilateral intracerebral and intraventricular transplantation of HuCNS-SC as part of the Phase I study and surviving subjects enrolled in the LTFU for 4 years of observation. Subjects were routinely assessed with a comprehensive battery of tests and brain MR imaging.
RESULTS:
HuCNS-SC transplantation in combination with immunosuppression was well tolerated by all subjects. Neurological and neuropsychological outcomes were consistent with NCL progression during the Phase I study. One subject expired during the Phase I study at 11 months post-transplant and 2 subjects expired during the LTFU study at 30 and 41 months post-transplant, respectively. DNA PCR testing of post-mortem brain tissue from 2 of these subjects provided evidence of donor cell engraftment. There were no safety concerns attributed to the cell transplant during the conduct of the LTFU study, and analysis of the preliminary efficacy data will be presented.....
An open-label dose-escalation Phase I trial of a highly purified, expandable population of human central nervous system stem cells (HuCNS-SC, StemCells, Inc., Newark, CA) was conducted in subjects with advanced stage infantile and late-infantile neuronal ceroid lipofuscinoses (NCL). This investigation was the first US FDA authorized use of human neural stem cells for clinical testing. The current report summarizes the outcome of the separate 4 year long-term follow-up (LTFU) study which was conducted for post-transplantation surveillance.
METHODS:
Six subjects underwent bilateral intracerebral and intraventricular transplantation of HuCNS-SC as part of the Phase I study and surviving subjects enrolled in the LTFU for 4 years of observation. Subjects were routinely assessed with a comprehensive battery of tests and brain MR imaging.
RESULTS:
HuCNS-SC transplantation in combination with immunosuppression was well tolerated by all subjects. Neurological and neuropsychological outcomes were consistent with NCL progression during the Phase I study. One subject expired during the Phase I study at 11 months post-transplant and 2 subjects expired during the LTFU study at 30 and 41 months post-transplant, respectively. DNA PCR testing of post-mortem brain tissue from 2 of these subjects provided evidence of donor cell engraftment. There were no safety concerns attributed to the cell transplant during the conduct of the LTFU study, and analysis of the preliminary efficacy data will be presented.....
