A platform stratifying a sequestering agent and a pharmacological antagonist as a means to negate botulinum neurotoxicity

ACS Chem Neurosci. 2014 Aug 20;5(8):632-6. doi: 10.1021/cn500135h. Epub 2014 Jul 11.

Abstract

Botulinum neurotoxicity is characterized by peripheral neuromuscular blockade/flaccid paralysis that can lead to respiratory failure and ultimately death. Current therapeutic options provide relief in a pre-exposure scenario, but there are no clinically approved postexposure medical countermeasures. Here, we introduce a platform that utilizes a combination of a toxin sequestering agent and a pharmacological antagonist to ablate botulinum neurotoxicity in a well-defined mouse lethality assay. The platform was constructed to allow for ready exchange of sequestering agent and/or pharmacological antagonist for therapeutic optimization. As such, we attempted to improve upon the pharmacological antagonist, a potassium channel blocker, 3,4-diaminopyridine, through a prodrug approach; thus, a complete kinetic decomposition pathway is described. These experiments provide the first proof-of-principle that a synergistic combination strategy can be used to reduce toxin burden in the peripheral using a sequestering antibody, while restoring muscle action via a pharmacological small molecule antagonist.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • 4-Aminopyridine / analogs & derivatives*
  • 4-Aminopyridine / chemistry
  • 4-Aminopyridine / pharmacokinetics
  • 4-Aminopyridine / pharmacology
  • Amifampridine
  • Animals
  • Botulinum Antitoxin / pharmacology
  • Botulinum Toxins / antagonists & inhibitors*
  • Botulinum Toxins / toxicity*
  • Drug Therapy, Combination
  • Female
  • Kinetics
  • Mice
  • Neurotoxicity Syndromes / blood
  • Neurotoxicity Syndromes / drug therapy*
  • Potassium Channel Blockers / chemistry
  • Potassium Channel Blockers / pharmacokinetics
  • Potassium Channel Blockers / pharmacology*
  • Sequestering Agents / pharmacology*
  • Survival Analysis

Substances

  • Botulinum Antitoxin
  • Potassium Channel Blockers
  • Sequestering Agents
  • 4-Aminopyridine
  • Botulinum Toxins
  • Amifampridine