Fig. 2.

Combination of human iPSC approach, high-throughput drug screening, and biomarker application can potentiate clinical candidate selection. (A) iPSCs derived from patients with ALS can be used for high-content drug screening to identify pathways and targets of small molecules with therapeutic potential. After preclinical studies, these small molecules may enter clinical trials with suitable administration routes (B) and the use of biomarkers can be used to calculate and determine drug efficacy (C). P-NFH: phosphorylated heavy chain of NF.