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immunosuppressant-StemCells, Inc.-neuronal ceroid lipofuscinosis
- Development stage
- Discontinued
- Lead developer
- StemCells
- Modality
- Small Molecules
01
Overview
This refers to the specific immunosuppressive drug regimen used by StemCells, Inc. in clinical trials for the treatment of neuronal ceroid lipofuscinosis (NCL), also known as Batten disease. The regimen was designed to support the engraftment and survival of HuCNS-SC (human central nervous system stem cells), which are allogeneic fetal-derived neural stem cells. Typically consisting of a 12-month course of tacrolimus and mycophenolate mofetil, the protocol aimed to prevent immune rejection of the transplanted cells. While the Phase I study (NCT00337636) demonstrated that the regimen and the cell therapy were safe and well-tolerated, further development was halted when StemCells, Inc. discontinued the program in 2011.
- Other names
- HuCNS-SC immunosuppression regimenBatten disease immunosuppression protocolTacrolimus + Mycophenolate Mofetil regimen for NCL
02
Targets
Nucleotide synth (Nucleotide synthesis enzymes)CN (Calcineurin)
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