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VT1021 is a first-in-class cyclic pentapeptide derived from prosaposin (Psap) that acts as an inducer of thrombospondin-1 (TSP-1), a potent anti-tumorigenic protein. By binding to myeloid-derived suppressor cells (MDSCs) in the tumor microenvironment, VT1021 stimulates the expression of TSP-1, which reprograms the tumor microenvironment through multiple mechanisms. These include inducing apoptosis in tumor and endothelial cells via CD36 and CD47 binding, modulating immune cell activity to enhance cytotoxic T cell responses, converting macrophages to anti-tumor phenotypes, and inhibiting angiogenesis necessary for tumor growth. The drug is being developed primarily for glioblastoma (GBM) and other advanced solid tumors. Clinical studies have shown that VT1021 is well tolerated with promising efficacy signals in recurrent GBM and other solid tumors[2][3][4][6].
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