Computational design of peptides to target Na<sub>V</sub>1.7 channel with high potency and selectivity for the treatment of pain.

Nguyen, Phuong T; Nguyen, Hai M; Wagner, Karen M; Stewart, Robert G; Singh, Vikrant; Thapa, Parashar; Chen, Yi-Je; Lillya, Mark W et al. · Elife · 2022

basic_science · Level V

Where this comes from

Abstract

The voltage-gated sodium Na<sub>V</sub>1.7 channel plays a key role as a mediator of action potential propagation in C-fiber nociceptors and is an established molecular target for pain therapy. ProTx-II is a potent and moderately selective peptide toxin from tarantula venom that inhibits human Na<sub>V</sub>1.7 activation. Here we used available structural and experimental data to guide Rosetta design of potent and selective ProTx-II-based peptide inhibitors of human Na<sub>V</sub>1.7 channels. Functional testing of designed peptides using electrophysiology identified the PTx2-3127 and PTx2-3258 peptides with IC<sub>50</sub>s of 7 nM and 4 nM for hNa<sub>V</sub>1.7 and more than 1000-fold selectivity over human Na<sub>V</sub>1.1, Na<sub>V</sub>1.3, Na<sub>V</sub>1.4, Na<sub>V</sub>1.5, Na<sub>V</sub>1.8, and Na<sub>V</sub>1.9 channels. PTx2-3127 inhibits Na<sub>V</sub>1.7 currents in mouse and human sensory neurons and shows efficacy in rat models of chronic and thermal pain when administered intrathecally. Rationally designed peptide inhibitors of human Na<sub>V</sub>1.7 channels have transformative potential to define a new class of biologics to treat pain.

Medical subject headings