Human amyotrophic lateral sclerosis excitability phenotype screen: Target discovery and validation

Huang X, Roet KCD, Zhang L, Brault A, Berg AP, Jefferson AB, Klug-McLeod J, Leach KL, Vincent F, Yang H, Coyle AJ, Jones LH, Frost D, Wiskow O, Chen K, Maeda R, Grantham A, Dornon MK, Klim JR, Siekmann MT, Zhao D, Lee S, Eggan K, and Woolf CJ.

Cell Reports, 2021.

Scientists use Axion’s multielectrode array platform and other methods to identify ALS drug targets

Drug discovery has been challenging for amyotrophic lateral sclerosis (ALS), a neurodegenerative disorder characterized by motor neuron hyperexcitability. In this study, scientists develop a multi-step screening funnel using patient-derived motor neurons differentiated from induced pluripotent stem cell (iPSC) lines to identify targets for managing hyperexcitability associated with ALS.

To examine the effects of different compounds on hyperexcitability in the motor neurons, the researchers used Axion’s noninvasive Maestro multielectrode array (MEA) platform. Together with other testing methods, the researchers identified a number of ALS hyperexcitability modulators and demonstrated the suitability of human disease cell-based phenotypic screens for identifying targets for neurological disorders.