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Intrinsic cell-penetrating activity propels Omomyc from proof of concept to viable anti-MYC therapy
Marie-Eve Beaulieu1,2, Toni Jauset1,2, Daniel Massó-Vallés1,2
1Peptomyc S.L., Edifici Cellex, Hospital Vall d'Hebron, Barcelona, 08035, Spain.
Abstract:
Inhibiting MYC has long been considered unfeasible, although its key role in human cancers makes it a desirable target for therapeutic intervention. One reason for its perceived undruggability was the fear of catastrophic side effects in normal tissues. However, we previously designed a dominant-negative form of MYC called Omomyc and used its conditional transgenic expression to inhibit MYC function both in vitro and in vivo. MYC inhibition by Omomyc exerted a potent therapeutic impact in various mouse models of cancer, causing only mild, well-tolerated, and reversible side effects. Nevertheless, Omomyc has been so far considered only a proof of principle. In contrast with that preconceived notion, here, we show that the purified Omomyc mini-protein itself spontaneously penetrates into cancer cells and effectively interferes with MYC transcriptional activity therein. Efficacy of the Omomyc mini-protein in various experimental models of non-small cell lung cancer harboring different oncogenic mutation profiles establishes its therapeutic potential after both direct tissue delivery and systemic administration, providing evidence that the Omomyc mini-protein is an effective MYC inhibitor worthy of clinical development.
Insights
Omomyc, a MYC inhibitor, can now be directly administered as a mini-protein. This approach shows therapeutic potential in lung cancer models with manageable side effects, paving the way for clinical development.
Area of Science:
- Oncology
- Molecular Biology
- Drug Discovery
Background:
- MYC oncoprotein is a crucial driver of human cancers.
- Targeting MYC has been challenging due to potential toxicity in normal tissues.
- Previous studies introduced Omomyc as a dominant-negative MYC inhibitor via transgenic expression.
Purpose of the Study:
- To evaluate the therapeutic potential of a purified Omomyc mini-protein as a direct-acting MYC inhibitor.
- To assess the efficacy and safety of Omomyc mini-protein in preclinical cancer models.
- To determine the feasibility of Omomyc mini-protein for clinical development.
Main Methods:
- The study utilized a purified Omomyc mini-protein.
- Investigated spontaneous cellular uptake and MYC transcriptional interference by Omomyc mini-protein.
- Tested Omomyc mini-protein efficacy in various mouse models of non-small cell lung cancer (NSCLC) with different oncogenic profiles.
- Assessed therapeutic potential via direct tissue delivery and systemic administration.
Main Results:
- The Omomyc mini-protein spontaneously penetrates cancer cells.
- Omomyc mini-protein effectively inhibits MYC transcriptional activity.
- Demonstrated potent therapeutic effects in diverse NSCLC experimental models.
- Observed mild, well-tolerated, and reversible side effects in previous studies.
- Showcased efficacy following both direct tissue and systemic delivery.
Conclusions:
- The Omomyc mini-protein is a viable and effective MYC inhibitor.
- Omomyc mini-protein exhibits therapeutic potential for NSCLC and warrants clinical investigation.
- Direct administration of Omomyc mini-protein overcomes previous limitations for MYC-targeted therapy.
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