Idiosyncratic treatment of motor neuron disease: selective inhibition of voltage-gated sodium channels using spider venom peptide (ProTX-III) to treat motor neuron disease

Amyothropic lateral sclerosis (ALS) commonly known as Motor neuron disease is plaguing humanity for generations, impeding the basic vital functions. Both upper and lower, motor neurons facilitate voluntary and involuntary actions. Suppressing such action’s leads to muscle weakness, atrophy, paralysis and death. This however can be halted upon modulation of hyperexcitability by selectively inhibiting voltage-gated sodium channel subtypes 1.1, 1.2, 1.3, and 1.6 on motor neurons. ProTX-III is one such spider venom peptide which has shown to selectively modulate aforementioned subtypes and partially inhibit 1.4 (off target). This presentation will highlight the rationale behind choosing our drug targets, optimisation of ProTX-III and evaluating the efficacy of the drug. We hope this study will lead to novel drug candidates to treat ALS.