Discovering a growth factor and its incredible healing powers

1989

Challenge

For decades, nothing was available to prevent or reduce the severity of oral mucositis (ulcerative lesions of the mouth), a common side effect of high doses of chemotherapy and radiation, which increases the risk of infection in cancer patients. A therapy was needed to reduce the incidence of this painful and life-threatening side-effect of many cancer therapies.

Advance

In the late 1980s, IRP scientists Jeffrey Rubin, M.D., Ph.D., Paul Finch, Ph.D., and Stuart A. Aaronson, M.D., discovered and purified keratinocyte growth factor (KGF). Several studies later demonstrated that KGF occurs naturally and stimulates the growth of surface layer cells in the mouth, which speeds healing of ulcers, reducing infection risks. The NIH partnered with Amgen in 1992 to develop Kepivance, a therapeutic treatment based on KGF.

Impact

Clinical trial results showed that Kepivance decreased the incidence and duration of severe oral mucositis in cancer patients who were given intensive chemotherapy and radiation prior to bone marrow/blood cell transplants. FDA approved in 2004, Kepivance now benefits about 11,000 American adults who undergo bone marrow transplants each year FDA Approves Kepivance for Severe Oral Mucositis in Cancer Patients Undergoing Bone Marrow Transplant; Pivotal Phase 3 Study Published in This Week's New England Journal of Medicine.

Publications

Rubin JS, Osada H, Finch PW, Taylor WG, Rudikoff S, Aaronson SA. (1989). Purification and characterization of a newly identified growth factor specific for epithelial cells. Proc Natl Acad Sci U S A. 86(3), 802-6.

Kepivance FDA Approval Package: http://www.accessdata.fda.gov/drugsatfda_docs/nda/2004/125103s000_KepivanceTOC.cfm.