A novel small molecule thought to be an agonist at the glial cell line-derived neurotrophic factor (GDNF) family receptor alpha-1 GFRalpha-1. It inhibits radiolabeled GDNF binding in Neuro-2A cells in a concentration dependent manner IC50=10.4 µM. Like GDNF, it induced Ret autophosphorylation and promoted neurite outgrowth in Neuro-2A cells1. Recent studies indicate that it enhances GFRalpha family receptor signaling in conjunction with ligand stimulation. Effective treatment for small-fiber neuropathy in mouse models of the disease.2,3 GDNF agonists may be a new class of Parkinsons disease therapeutics.4
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