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DTGM is a recombinant fusion toxin composed of a truncated diphtheria toxin (DT388) linked to human granulocyte-macrophage colony-stimulating factor (GM-CSF). This construct is designed to selectively target and induce cytotoxicity in cells expressing the granulocyte-macrophage colony-stimulating factor receptor (GM-CSFR). It has demonstrated toxicity against blasts from patients with juvenile myelomonocytic leukemia, chronic myelomonocytic leukemia, acute myelogenous leukemia, and gastrointestinal cancer cells. Preclinical and early clinical studies, including those in cynomolgus monkeys and patients with acute myelogenous leukemia, revealed significant liver injury and limited tolerance at certain doses.
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