Definition

A Hedgehog (Hh) pathway inhibitor, with potential immunomodulating and antineoplastic activities. Upon administration, Hh inhibitor NLM-001 targets the Hh pathway and disrupts the tumor microenvironment (TME) by decreasing cancer-associated fibroblasts and promoting immune cell infiltration into the TME. This may increase tumor penetration of chemotherapeutics and may enhance the activity of immunomodulating agents, such as immune checkpoint inhibitors. Hh signaling overactivation may increase tumor cell proliferation and may lead to tumor immunosuppression and drug resistance.