Keynote: Dr. Tom Curran

“Translating Science into Medicine: Lessons from Targeting the Hedgehog Pathway in Cancer”

In 1995, after meeting with children suffering from brain tumors and learning about the long-term side effects of current therapy, I refocused my research to develop better treatments for pediatric medulloblastoma (MB). At the time, there was a dearth of quality biospecimens and no clear molecular targets.

While I was establishing biobanking efforts, which later expanded through the Children’s Brain Tumor Tissue Consortium, initial reports started coming out implicating the Hedgehog (HH) pathway in a subtype of MB. We developed a high incidence, early onset MB mouse model and worked with Curis Inc., and later Genentech Inc., to demonstrate proof-of-concept of the use of Smoothened (Smo) inhibitors in cancer.

This led to successful Phase I/II studies of Smo inhibitors in MB that recapitulated all the major findings obtained in mice. Our work focused on mouse models because human tumor cell lines and xenografts failed to predict clinical responses in a range of cancers. The first Smo inhibitor (Vismodegib) was approved for treatment of basal cell carcinoma in 2012 and was tested successfully in Phase I/II trials of MB.


Dr. Tom Curran is the Donald J. Hall Eminent Scholar in Pediatric Research and Executive Director of the Children’s Research Institute at Children’s Mercy Kansas City. He is also Professor of Pediatrics, University of Missouri-Kansas City School of Medicine and Professor, Department of Cancer Biology, University of Kansas School of Medicine.

He obtained a Ph.D. from the Imperial Cancer Research Fund Laboratories and University College in London, UK in 1982. He served as President of the American Association of Cancer Research in 2001 and on Board of Scientific Advisors of the National Cancer Institute from 2000-2005. He is a member of the National Academy of Medicine, a fellow of the Royal Society, London, a fellow of the American Academy of Arts and Sciences and an inaugural Fellow of the American Association for Cancer Research.

He discovered the fos oncogene and described its role in gene regulation and signal transduction. He also identified the Reelin gene and its role in formation of laminar structures in the developing brain.

His current research is uncovering new approaches to pediatric brain tumors by integrating an understanding of normal brain development with the molecular genetics of brain tumors. He helped develop inhibitors of the Hedgehog pathway for the treatment of cancer including basal cell carcinoma and medulloblastoma.