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Targeting Aberrant Hh Signaling with BET Bromodomain Inhibition as a Novel Therapeutic Strategy Against MB and DIPG

Lucile Packard Children’s Hospital at Stanford University School of Medicine
Yujie Tang, PhD
Grant Type: 
Young Investigator Grants
Year Awarded: 
Type of Childhood Cancer: 
Medulloblastoma, Diffuse Intrinsic Pontine Glioma (DIPG), Brain Tumors
Project Description: 


The specific childhood cancers I am addressing in this proposal are medulloblastoma (MB) and diffuse intrinsic pontine glioma (DIPG), which are the two most common malignant pediatric brain tumors and the leading causes of pediatric brain tumor-related deaths. We propose to focus on an evolutionarily conserved signaling axis called the Hedgehog (Hh) pathway that is known to be aberrantly activated in these two cancers. Importantly, targeting this pathway has been shown to effectively inhibit the growth of these two types of tumors. However, acquired and a priori resistance to the currently available Hh-targeting drugs has been widely identified.

Project Goal

Here, we will explore an alternative therapeutic strategy by targeting the Hh pathway through inhibiting one of its far downstream components, to overcome the resistance. For this study, we will utilize patient autopsy tumor tissues, patient-derived tumor cells and therapeutic testing of these cells in mice. Successfully performed, this project will provide the necessary rationale to move forward to new pediatric clinical trials and will mark a significant advance in the way we approach Hh-driven tumors such as MB and DIPG.


"ALSF YIA grant is such a big gift and a great beginning for my future career as an independent investigator in the field of pediatric brain tumors. Thanks to ALSF for such an important opportunity and support!" ~Yujie Tang, PhD