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Carboxylesterase-expressing allogeneic neural stem cells are a cell therapy product consisting of neural stem cells derived from a donor (allogeneic) and genetically engineered to express carboxylesterase, typically rabbit carboxylesterase. These modified neural stem cells are designed to migrate toward tumor sites in the brain and convert the prodrug irinotecan (CPT-11) into its active cytotoxic metabolite SN-38 locally within the tumor microenvironment. This approach aims to enhance anti-tumor efficacy while minimizing systemic toxicity. The primary indication under investigation is for brain metastases, particularly from lung cancer, but also potentially other solid tumors with central nervous system involvement[1][2][6][10]. Preclinical studies have demonstrated selective migration of these engineered NSCs to brain tumors and significant inhibition of tumor growth when combined with irinotecan[6]. Early-phase clinical trials have focused on evaluating safety, tolerability, and optimal dosing in patients with recurrent or progressive high-grade glioma or metastatic brain tumors[1][2].
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