INSTrUCT: INdependent observational STUdy of Cell Transplantation in SCI
Funded in: 2017, 2018, 2019
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HuCNS-SC are self-renewing, multi-potent adult stem cells purified, expanded, and banked from human brain tissue. The safety and preliminary efficacy of the HuCNS-SC has been evaluated in phase I/II clinical trials of thoracic and cervical SCI, sponsored by Stem Cell Inc.
In regards to these trials, the human data gathered to date from transplantation of HuCNS-SC into the spinal cord have shown 1) a favorable safety profile 2) no safety concern directly attributed to the HuCNS-SC; 3) no evidence of tumor formation or abnormal cell or tissue reaction; and 4) preliminary efficacy observations in the Phase I/II thoracic SCI trial.
The major risks posed by the preceding phase I/II trial involve those related to the surgical procedure, the HuCNS-SC cell transplant, and the immunosuppression regimen. The first category involves the risk of the surgical intervention and specifically the injection of human cells into the spinal cord. The second category involves the risk of an adverse reaction to the implanted HuCNS-SC cells. The third category relates to the potential complications of immunosuppression, which include both infection and systemic reactions to the specific agents. Although the onset of complications or evidence of preliminary efficacy related to the phase I/II trial are most likely during the first year following transplantation, long-term observation of the subject is appropriate in order to continue monitoring for safety and preliminary efficacy.
The long-term follow-up of the phase I/II safety and preliminary efficacy investigation of intramedullary spinal cord transplantation of HuCNS-SC in subjects with thoracic spinal cord trauma was formally early terminated by the sponsor (Stemcell Inc. investor.stemcellsinc.com/phoenix.zhtml. Wings for Life decided to fund the long term follow-up of the transplanted patients, for the following reasons: a) There is an ethical commitment to the enrolled patients to care about their safety and well-being, b) The goal of this clinical research is to develop the HuCNS-SC as a safe and effective therapy for SCI. Such development will include the advancement to appropriately controlled clinical trials that demonstrate the efficacy and continued safety of the HuCNS-SC in relevant patient populations. The scientific quality of a clinical trial is dependent on the completeness of data. Only based on solid clinical trials the development of a treatment for SCI can move on.
The follow–up study will start in 2017.
Here you'll find a published statement to the premature termination of clinical studies: Challenges to Translation and the Hippocratic Oath by Premature Termination of Spinal Cord Stem Cell-Based Trials.