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How Engineered Peptides Could Drug the 'Undruggable' — Targeting Protein Interactions Inside Cells

evidence
The takeaway

Peptide engineering techniques like stapling, macrocyclization, and scaffold grafting are opening the door to targeting intracellular protein-protein interactions — a vast 'undruggable' space that small molecules and antibodies can't reach.

~80% of targets are 'undruggable'

Most intracellular protein-protein interactions can't be reached by traditional small molecules or antibodies — engineered peptides are positioned to fill this gap

What the researchers found

The review highlights recent technological and chemical advancements in peptide design that enable targeting of intracellular protein-protein interactions, a class of targets traditionally considered undruggable by small molecules or biologics.

Why it matters

This work is important because it outlines how peptides can overcome limitations of existing drugs, potentially leading to new treatments for diseases involving intracellular protein interactions.

How the study worked

This is a review article summarizing current research and technological progress in peptide therapeutics, focusing on chemical strategies such as stapling and macrocyclization to stabilize peptides for intracellular targeting.

What this study cannot tell us

As a review, it does not present new experimental data and the evidence strength and clinical applicability of discussed approaches remain to be fully established.

How to read the evidence

This is a review article in a medicinal chemistry journal, synthesizing research on peptide engineering strategies. It describes technological approaches rather than clinical evidence. The individual techniques reviewed have varying levels of preclinical and early clinical validation.

When this study was published

Published in 2015, this review captured the field at an inflection point. Since then, several stapled peptide and macrocyclic peptide drug candidates have entered clinical trials, partially validating the approaches described here.

The bigger picture

The 'undruggable' proteome represents roughly 80% of disease-relevant targets. Protein-protein interactions control cancer pathways (p53-MDM2), apoptosis (Bcl-2 family), and transcription factors that drive disease. Since 2015, several of the approaches described in this review have advanced to clinical trials — stapled peptides targeting MDM2 and Bcl-2 have shown promise. The concept of 'beyond rule-of-five' drug design, where molecules exceed traditional small-molecule size limits, has become a major pharmaceutical research theme.

Questions still open

  • Which peptide stabilization technique is most likely to produce the first FDA-approved intracellular PPI inhibitor?
  • Can cell-penetrating peptide strategies be combined with stapling to achieve both membrane crossing and target binding?
  • How do the manufacturing costs of engineered peptides compare to small molecules and antibodies?

Common questions

What makes a target 'undruggable'?
A target is considered 'undruggable' when existing drug types can't effectively reach or block it. Many critical disease targets are protein-protein interactions inside cells — their binding surfaces are too large and flat for small molecules, while antibodies are too big to enter cells. Peptides are being engineered to fill this gap because they can cover large surfaces and potentially be designed to cross cell membranes.
What is peptide stapling?
Stapling is a chemical technique that locks a peptide into its active 3D shape (usually an alpha-helix) by adding a chemical 'staple' across two points of the peptide backbone. This makes the peptide more resistant to degradation, better at binding its target, and in some cases able to cross cell membranes. It's one of the most promising approaches for creating peptide drugs that work inside cells.

Read the original research

Peptide therapeutics: targeting the undruggable space.

European journal of medicinal chemistry, 94, 459-70

Citation

Tsomaia, Natia. (2015). Peptide therapeutics: targeting the undruggable space.. European journal of medicinal chemistry, 94, 459-70. https://doi.org/10.1016/j.ejmech.2015.01.014