LFHM: Targeted therapies for children's brain tumors: Cooperation among academia, biotech, big pharma and a flock of sheep

Tuesday, April 26 2011 11:30am - 1:30pm Eastern Time Presented by:

Hedgehog (Hh) is a secreted protein family that controls proliferation, differentiation, cell fate specification, left-right asymmetry and morphogenesis during development.  The Hh pathway is extremely complex and mutations in various components result in developmental abnormalities as well as increased tumor incidence, including medulloblastoma, in mice and humans. Loss of one copy of Patched 1 (Ptch1), a receptor for Hh, causes increased activity of Smoothened (Smo) and elevated expression of the transcription factor Gli1 in both familial and sporadic medulloblastoma. Fifteen years ago, we embarked on a long-term program to develop new therapeutic approaches for this devastating childhood brain tumor. Small molecule inhibitors of the Hh pathway, both naturally occurring and chemically synthesized, inhibit Smo activity and provide potential novel therapeutic agents. We generated a genetically engineered mouse meduloblastoma model, Ptch1+/-p53-/- mice, that exhibits a 100% incidence of medulloblastoma. Using these mice, we tested the effect of a Smo inhibitor (HhAntag), isolated by Curis Inc. on the growth of spontaneous medulloblastoma. We found that twice daily dosing, by oral gavage, effectively eliminated even large tumor masses in the cerebellum of mice 3 to 10 weeks of age. These findings led to Phase I clinical trials of a Smo inhibitor in pediatric and adult medulloblastoma.

Speaker
Tom Curran, PhD, FRS.
Deputy Scientific Director, CHOP Research Institute
Professor of Pathology and Laboratory Medicine

11:30am   Lunch & Networking
12 - 1pm  Presentation and Q&A

This event is free, but space is limited and you must register to attend.


February 28, 2011
3711Science
January 29, 2012
3711Science
University City Keystone Innovation Zone
Free


Tuesday, April 26 2011 11:30am - 1:30pm Eastern Time
Location: University City Science Center
3711 Market Street
Suite 800
Philadelphia, PA 19104
Contact: Sheila Greer
215-966-6000
Email Contact Form

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