Novel chemical inhibitor against SOD1 misfolding and aggregation protects neuron-loss and ameliorates disease symptoms in ALS mouse model

With ELISA-based screening, Woo et al. identify a new small molecule, PRG-A01, which can block the misfolding/aggregation of SOD1 or TDP-43 in a human neuroblastoma cell line. Its use in a mouse model of ALS results in improvement of muscle strength, motor neuron viability and mobility, highlighting its therapeutic potential in ALS.