OnCo
ideasIdea

Can MYC be drugged directly, and will patients tolerate it?

MYC drives half of all cancers but has no pocket for a drug and is needed by normal cells too. The first direct MYC blockers are in trials; the question is whether there is a therapeutic window.

OMO-103 (Omomyc) showed safety and disease stabilisation in phase 1; MYC degraders and MAX stabilisers follow. Mouse Omomyc studies showed reversible, tolerable toxicity in proliferating tissues, but human tolerance at effective doses is unproven.

Hypothesis
Transient, incomplete MYC inhibition (intermittent dosing) achieves tumour regression in MYC-amplified cancers while normal tissues recover between doses, yielding a usable therapeutic index.
Rationale
Soucek's Omomyc mouse models tolerated systemic MYC inhibition; MYC-addicted tumours show non-oncogene addiction to MYC dosage; combination with PD-1 blockade may add via CD47/PD-L1 downregulation.
What would test it
Phase 2 randomised OMO-103 plus chemotherapy vs chemotherapy in MYC-amplified metastatic PDAC and TNBC with intermittent schedules; pharmacodynamic MYC target-gene signatures in paired biopsies.
Maturity
early clinical

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