ACT001 + pembrolizumab

ACT001 + pembrolizumab is an investigational combination therapy being studied primarily for the treatment of recurrent glioblastoma multiforme and potentially other cancers. ACT001 is a novel small molecule that acts as a Plasminogen activator inhibitor 1 (PAI-1) inhibitor, while pembrolizumab is a humanized monoclonal antibody targeting Programmed cell death protein 1 (PD-1). Pembrolizumab works by blocking the PD-1 receptor on T cells, thereby enhancing immune system activity against cancer cells. The rationale for combining these agents is to improve anti-tumor immune responses by both modulating the tumor microenvironment (via ACT001) and releasing immune inhibition (via pembrolizumab). Pembrolizumab has been approved for multiple cancer indications, including melanoma, non-small cell lung cancer, head and neck cancers, Hodgkin lymphoma, urothelial carcinoma, colorectal cancer with specific genetic features, gastric cancer, esophageal cancer, cervical and endometrial cancers[2][4][5]. The combination with ACT001 remains investigational[3][6].

Brand names

Keytruda

Other names

none