Treatment and Screen for Synovial Sarcoma

Researchers in Prof. Gerald Crabtree's laboratory have identified the pathological mechanism for synovial sarcoma (SS) that could be used to develop targeted therapeutics. This approach aims to reverse the effects of the SS18-SSX fusion protein (the hallmark of human SS). The inventors discovered that SS18-SSX causes cell proliferation through a chain of events that induces Sox2 expression (see figure below in Media). Because this mechanism of transformation depends on only a small portion of the fusion protein, that region provides a viable foundation for therapeutic intervention. New compounds that treat synovial sarcoma could fulfill an unmet medical need because SS tumors that cannot be removed with surgery are nearly always lethal.

Stage of Research
The inventors have used their screen to identify several small molecules as potential therapeutic compounds and performed structure-activity relationship studies on those molecules.