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Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF)

Dr. Donald Metcalf’s discovery of colony-stimulating factors (CSF) resulted in their widespread use to prevent infection in cancer chemotherapy patients, AIDS patients and persons with rare white-blood-cell diseases.

CSFs are hormone-like substances secreted by cells in tiny amounts. Their release influences white-blood-cell precursors and progenitor cells in bone marrow. When stimulated, progenitor cells divide and produce new white blood cells. The higher the level of CSF secretion, the greater the number of cell divisions and the higher the production of white blood cells. Healthy persons only need small amounts of CSFs, but patients with low white-blood-cell counts need larger amounts of CSFs to help generate white blood cells quickly.

The two major types of white blood cells affected by these factors are granulocytes and macrophages. Granulocyte-macrophage colony stimulating factor (GM-CSF) spurs production of both cell types. Because the body produces such low levels and the molecules are too large to be synthesized conventionally as a drug, GM-CSF is produced with recombinant DNA techniques.

RCT’s broad patent estate covering GM-CSF is licensed to Schering-Plough, Sanofi, and Amgen. Sanofi, through its acquisition of Genzyme, sells a recombinant form of GM-CSF called Leukine® to stimulate white blood cells in patients receiving chemotherapy and radiotherapy for cancer treatment. Amgen is developing a OncoVEXGM-CSF, an oncolytic vaccine, for the treatment of melanoma, head and neck cancer, breast cancer and pancreatic cancer.



  • Drs. Nicholas M. Gough
    Ludwig Institute for Cancer Research, Melbourne, Australia
  • Ashley R. Dunn
    Ludwig Institute for Cancer Research, Melbourne, Australia
  • Dr. Donald Metcalf
    Walter and Eliza Hall Institute of Medical Research, Melbourne, Australia

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