GM-CSF
The first discovery by LICR investigators to make it to commerical application was GM-CSF, a molecule that stimulates the production of white blood cells.
The innate immune system provides the first-line of defense against infectious organisms. Central to both the innate immune and inflammatory responses are the white blood cells, or leukocytes, a group of specialized cells that includes neutrophils, eosinophils, monocytes and macrophages. Leukocytes are produced when hematopoietic stem cells in the bone marrow differentiate into the specialized cell types in response to regulators, such as the colony-stimulating factors (CSFs). Many chemotherapeutic regimens destroy leukocytes, leaving oncology patients susceptible to infection and unable to undergo further chemotherapy treatments.
Investigators from the LICR Melbourne Branch and the Walter and Eliza Hall Institute of Medical Research (Australia) cloned GM-CSF (granulocyte/macrophage-CSF) in 1984. The first-in-man clinical trial of GM-CSF was conducted by the Melbourne Branch, and assessed the ability of GM-CSF to stimulate leukocyte production in cancer patients. The GM-CSF discovery was licensed to industrial partners to move its clinical development forward, and GM-CSF is now part of a treatment regimen that supports bone marrow transplantation and some chemotherapies.
A simplified diagram of blood cell differentiation. Specific growth factors (only some of which are depicted in green, below) control the differentiation of blood cell types from hematopoietic stem cells.
