Delivery to, and Reactivation of, the p53 Pathway in Cancer Cells Using a Grafted Cyclotide Conjugated with a Cell-Penetrating Peptide

J Med Chem. 2024 Jan 25;67(2):1197-1208. doi: 10.1021/acs.jmedchem.3c01682. Epub 2024 Jan 4.

Abstract

Peptides are promising drug modalities that can modulate protein-protein interactions, but their application is hampered by their limited ability to reach intracellular targets. Here, we improved the cytosolic delivery of a peptide blocking p53:MDM2/X interactions using a cyclotide as a stabilizing scaffold. We applied several design strategies to improve intracellular delivery and found that the conjugation of the lead cyclotide to the cyclic cell-penetrating peptide cR10 was the most effective. Conjugation allowed cell internalization at micromolar concentration and led to elevated intracellular p53 levels in A549, MCF7, and MCF10A cells, as well as inducing apoptosis in A549 cells without causing membrane disruption. The lead peptide had >35-fold improvement in inhibitory activity and increased cellular uptake compared to a previously reported cyclotide p53 activator. In summary, we demonstrated the delivery of a large polar cyclic peptide in the cytosol and confirmed its ability to modulate intracellular protein-protein interactions involved in cancer.

MeSH terms

  • Cell-Penetrating Peptides* / metabolism
  • Cell-Penetrating Peptides* / pharmacology
  • Cyclotides* / metabolism
  • Cyclotides* / pharmacology
  • Humans
  • Neoplasms*
  • Peptides, Cyclic / metabolism
  • Peptides, Cyclic / pharmacology
  • Tumor Suppressor Protein p53 / metabolism

Substances

  • Cyclotides
  • Cell-Penetrating Peptides
  • Tumor Suppressor Protein p53
  • Peptides, Cyclic