Cancer

Patient sample, cell culture and mouse studies suggest inhibiting VEGF-C could help treat AML. In bone marrow samples from pediatric patients, levels of VEGF-C were higher than in samples from healthy volunteers. In two human AML cell lines, shRNA targeting VEGF-C decreased proliferation compared with scrambled shRNA. In six pediatric patient-derived AML cell cultures, VGX-100, a humanized mAb targeting VEGF-C, decreased colony formation compared with no treatment. In a xenograft mouse model of AML, the anti-VEGF-C mAb decreased white blood cell counts in the peripheral blood and numbers of AML blasts in the spleen compared with vehicle. Next steps could include testing the mAb in additional models of AML.

Opthea Ltd. has VGX-100 in Phase I testing for solid tumors...