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Published on: November 9, 2020
Stimuli-activatable PROTACs for precise protein degradation and cancer therapy
Jing Gao1, Lei Yang1, Shumin Lei1
1Center of Pharmaceutics, Shanghai Institute of Materia Medica, Chinese Academy of Sciences, Shanghai 201203, China.
Abstract:
The proteolysis targeting chimeras (PROTACs) approach has attracted extensive attention in the past decade, which represents an emerging therapeutic modality with the potential to tackle disease-causing proteins that are historically challengeable for conventional small molecular inhibitors. PROTAC harnesses the endogenic E3 ubiquitin ligase to degrade protein of interest (POI) via ubiquitin-proteasome system in a cycle-catalytic manner. The event-driven pharmacology of PROTAC is poised to pursue those targets that are conventionally undruggable, which enormously extends the space of drug development. Furthermore, PROTAC has the potential to address drug resistance of small molecular inhibitors by degrading the whole POI. Nevertheless, PROTACs display high-efficiency and always-on properties to degrade POI, they may cause severe side effects due to an "on-target but off-tissue" protein degradation profile at the undesirable tissues and cells. Given that, the stimuli-activatable PROTAC prodrugs have been recently exploited to confine precise protein degradation of the favorable targets, which may conquer the adverse effects of PROTAC due to uncontrollable protein degradation. Herein, we summarized the cutting-edge advances of the stimuli-activatable PROTAC prodrugs. We also overviewed the progress of PROTAC prodrug-based nanomedicine to improve PROTAC delivery to the tumors and precise POI degradation in the targeted cells.
Insights
Proteolysis targeting chimeras (PROTACs) offer a novel way to degrade disease-causing proteins. Stimuli-activatable PROTAC prodrugs and nanomedicine enhance targeted degradation and reduce side effects.
Area of Science:
- Biochemistry
- Molecular Biology
- Drug Discovery
Background:
- Proteolysis targeting chimeras (PROTACs) are an emerging therapeutic strategy.
- PROTACs utilize the ubiquitin-proteasome system to degrade target proteins.
- They offer potential against historically undruggable targets and drug resistance.
Purpose of the Study:
- To summarize advances in stimuli-activatable PROTAC prodrugs.
- To review PROTAC prodrug-based nanomedicine for targeted delivery and degradation.
- To address challenges of "on-target but off-tissue" protein degradation.
Main Methods:
- Review of recent literature on stimuli-activatable PROTAC prodrugs.
- Overview of nanomedicine applications in PROTAC delivery.
- Analysis of PROTAC mechanisms and potential side effects.
Main Results:
- Stimuli-activatable PROTAC prodrugs enable precise control over protein degradation.
- Nanomedicine approaches improve PROTAC delivery to tumors.
- These strategies aim to mitigate adverse effects from uncontrolled protein degradation.
Conclusions:
- Stimuli-activatable PROTAC prodrugs and nanomedicine represent significant progress in targeted protein degradation.
- These approaches hold promise for safer and more effective therapies.
- Further research is needed to fully realize their therapeutic potential.
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