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Background  Spinal Muscular Atrophy (SMA) is one of the most common inherited causes of infant death and is caused by the loss of functional survival motor neuron (SMN) protein due to mutations or deletion in the  SMN1  gene. One of the treatment strategies for SMA is to induce the expression of the protein from the homologous  SMN2  gene, a rescuing paralog for SMA.    Methods and results  Here we demonstrate the promise of pharmacological modulation of  SMN2  gene by BAY 55-9837, an agonist of the vasoactive intestinal peptide receptor 2 (VPAC2), a member of G protein coupled receptor family. Treatment with BAY 55-9837 lead to induction of SMN protein levels via activation of MAPK14 or p38 pathway  in vitro . Importantly, BAY 55-9837 also ameliorated disease phenotype in severe SMA mouse models.    Conclusion  Our findings suggest the VPAC2 pathway is a potential SMA therapeutic target.

VPAC2 receptor agonist BAY 55-9837 increases SMN protein levels and moderates disease phenotype in severe spinal muscular atrophy mouse models