Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF)
This technology involves GM-CSF DNA sequences, GM-CSF protein, methods to make GM-CSF protein and uses of GM-CSF protein. Licenses are available for use of the technology in making commercial products, including therapeutics for human use.
Working at the Walter and Eliza Hall Institute of Medical Research, in Melbourne, Australia, in the 1980s, Dr. Donald Metcalf identified and purified the specific white cell regulatory hormones, the colony-stimulating factors, that are responsible for initiating the conversion of precursor cells into differentiating and differentiated blood cell types. Two of these factors are now used extensively to treat patients with cancer and diseases of blood cell formation.
Metcalf worked with Drs. Nicholas M. Gough and Ashley R. Dunn of the nearby Melbourne Branch of the Ludwig Institute for Cancer Research to isolate the DNA for mammalian GM-CSF. This technology enabled the production of recombinant mammalian GM-CSF protein in amounts sufficient to develop products based on the landmark discovery.
Although the four colony stimulating factors were known in the early 1980s, their study required painstaking manual isolation and purification from biological fluids for small amounts of material. Therapeutic use could not be supported by such production techniques. Isolation and use of the mammalian GM-CSF DNA to produce recombinant protein was among the first demonstrations that DNA cloning could lead to production of biologics in the amounts needed for clinical studies and sale as pharmaceuticals.
The first-in-man clinical trial of GM-CSF, conducted by the Melbourne Branch of the Ludwig Institute, demonstrated the ability of GM-CSF to stimulate leukocyte production in cancer patients. Rapid licensing to industrial partners quickly moved its clinical development forward, resulting in the marketing of Leukine® and Leucomax®. GM-CSF is now part of a treatment regimen that supports bone marrow and PBMC transplantation and adjuvant cancer therapy for chemotherapy-induced neutropenia.
New uses for GM-CSF continue to be discovered. The cytokine provides excellent support for patients in danger of contracting life-threatening infection after burns or trauma, if provided before the onset of infection. Recent clinical trials have demonstrated that co-administration of GM-CSF along with cellular antigens improves the ability of patients' immune systems to attack some cancers.
Inventors
Drs. Nicholas M. Gough and Ashley R. Dunn, Ludwig Institute for Cancer Research, Melbourne, Australia; Dr. Donald Metcalf, Walter and Eliza Hall Institute of Medical Research, Melbourne
In 2009, the Walter and Eliza Hill Institute of Medical Research celebrated Dr. Metcalf's 80th birthday with an extensive tribute to his groundbreaking work.
Patent Information
We encourage companies to contact us if they have further interest in or questions about the technology.
U.S. Patent
5,602,007, "Recombinant DNA molecules," issued Feb. 11, 1997
Canadian Patent
495,255, filed Nov. 13, 1985. This application recently emerged from Conflict proceedings in the Canadian Patent Office. RCT expects grant of the patent in the near future with a full patent term.
GM-CSF Overview
RCT Contact
Kurt R. Ghelsen, Ph.D., Vice President and Chief Scientific Officer