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Some sarcomas in children are caused by two genes fused into one abnormal protein. That protein is the whole disease, but no drug binds it. Destroying it instead of blocking it could work.
EWS-FLI1 in Ewing sarcoma and PAX3-FOXO1 in alveolar rhabdomyosarcoma are single, tumour-specific, genetically validated drivers with no enzymatic activity. Degradation via recruited E3 ligases, or indirect destabilisation through their obligate cofactors (for example BRD9 in the ncBAF complex for synovial sarcoma, already showing clinical activity with BRD9 degraders), offers a route. Their absence from normal tissue means an unusually wide therapeutic index.
Shares C4 Therapeutics, Drugging the 'undruggable' cancer targets, The undruggable drivers, PROTACs & molecular glues (targeted protein degradation).
Shares C4 Therapeutics, Drugging the 'undruggable' cancer targets, The undruggable drivers, PROTACs & molecular glues (targeted protein degradation).
Shares Drugging the 'undruggable' cancer targets, The undruggable drivers, PROTACs & molecular glues (targeted protein degradation).
Shares Drugging the 'undruggable' cancer targets, CRISPR functional genomics, The undruggable drivers.
Shares Drugging the 'undruggable' cancer targets, CRISPR functional genomics, The undruggable drivers, PROTACs & molecular glues (targeted protein degradation).
Shares Drugging the 'undruggable' cancer targets, The undruggable drivers, PROTACs & molecular glues (targeted protein degradation).
Shares Drugging the 'undruggable' cancer targets, The undruggable drivers, PROTACs & molecular glues (targeted protein degradation).
Shares Epigenetic therapy roadmap: loosening silenced genes → mutation-specific enzymes → editing the epigenome, CRISPR functional genomics, Sarcomas (soft tissue, bone, GIST).