Pharmacological characterization of potent and selective NaV1.7 inhibitors engineered from Chilobrachys jingzhao tarantula venom peptide JzTx-V

PLoS One. 2018 May 3;13(5):e0196791. doi: 10.1371/journal.pone.0196791. eCollection 2018.

Abstract

Identification of voltage-gated sodium channel NaV1.7 inhibitors for chronic pain therapeutic development is an area of vigorous pursuit. In an effort to identify more potent leads compared to our previously reported GpTx-1 peptide series, electrophysiology screening of fractionated tarantula venom discovered the NaV1.7 inhibitory peptide JzTx-V from the Chinese earth tiger tarantula Chilobrachys jingzhao. The parent peptide displayed nominal selectivity over the skeletal muscle NaV1.4 channel. Attribute-based positional scan analoging identified a key Ile28Glu mutation that improved NaV1.4 selectivity over 100-fold, and further optimization yielded the potent and selective peptide leads AM-8145 and AM-0422. NMR analyses revealed that the Ile28Glu substitution changed peptide conformation, pointing to a structural rationale for the selectivity gains. AM-8145 and AM-0422 as well as GpTx-1 and HwTx-IV competed for ProTx-II binding in HEK293 cells expressing human NaV1.7, suggesting that these NaV1.7 inhibitory peptides interact with a similar binding site. AM-8145 potently blocked native tetrodotoxin-sensitive (TTX-S) channels in mouse dorsal root ganglia (DRG) neurons, exhibited 30- to 120-fold selectivity over other human TTX-S channels and exhibited over 1,000-fold selectivity over other human tetrodotoxin-resistant (TTX-R) channels. Leveraging NaV1.7-NaV1.5 chimeras containing various voltage-sensor and pore regions, AM-8145 mapped to the second voltage-sensor domain of NaV1.7. AM-0422, but not the inactive peptide analog AM-8374, dose-dependently blocked capsaicin-induced DRG neuron action potential firing using a multi-electrode array readout and mechanically-induced C-fiber spiking in a saphenous skin-nerve preparation. Collectively, AM-8145 and AM-0422 represent potent, new engineered NaV1.7 inhibitory peptides derived from the JzTx-V scaffold with improved NaV selectivity and biological activity in blocking action potential firing in both DRG neurons and C-fibers.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Action Potentials / drug effects
  • Amino Acid Substitution
  • Analgesics / isolation & purification*
  • Analgesics / pharmacology
  • Animals
  • Capsaicin / pharmacology
  • Cell Line
  • Drug Evaluation, Preclinical
  • Ganglia, Spinal / drug effects
  • Humans
  • Male
  • Mice, Inbred C57BL
  • Mutagenesis, Site-Directed
  • NAV1.7 Voltage-Gated Sodium Channel / drug effects*
  • Nerve Fibers, Unmyelinated / drug effects
  • Nuclear Magnetic Resonance, Biomolecular
  • Patch-Clamp Techniques
  • Peptides / chemistry*
  • Physical Stimulation
  • Protein Engineering
  • Recombinant Proteins / drug effects
  • Sodium Channel Blockers / isolation & purification*
  • Sodium Channel Blockers / pharmacology
  • Spider Venoms / chemistry*
  • Structure-Activity Relationship
  • Tetrodotoxin / pharmacology

Substances

  • Analgesics
  • NAV1.7 Voltage-Gated Sodium Channel
  • Peptides
  • Recombinant Proteins
  • SCN9A protein, human
  • Scn9a protein, mouse
  • Sodium Channel Blockers
  • Spider Venoms
  • jingzhaotoxin-V, Chilobrachys jingzhao
  • Tetrodotoxin
  • Capsaicin

Grants and funding

The authors were all Amgen, Inc. or Atheris Laboratories employees at the time the work was performed. No specific funding was received for this work. This work was fully supported by Amgen, Inc. Amgen, Inc. provided support in the form of salaries for authors, but did not have any additional role in the study design, data collection and analysis, or preparation of the manuscript. The specific roles of the authors are listed in the 'Author contributions' section.