Targeting the Ras-pathway for the treatment of embryonal rhabdomyosarcoma

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David Langenau, Ph.D.,  Massachusetts General Hospital
Recipient of the: $25,000 research award, PharmaMar Research Award

A transgenic zebrafish model of embryonal rhabdomyosarcoma was recently created that is molecularly similar to human disease and from which the cancer stem cell has been identified, a cell most similar to the normal muscle stem cell-the actived satellite cell. My hypothesis is that the RAS pathway is responsible for eliciting stem cell self-renewal programs in a subset of the RMS cell types, and that this in turn regulates tumor growth.  Targeting downstream RAS pathways and self-renewal may provide a new way to curb sarcoma growth in patients affected with this devastating disease.