144DG11 is a polyglucosan (PG)-reducing agent that targets the lysosomal membrane protein LAMP1 and increases autophagic flux in adult polyglucosan body disease (APBD) primary fibroblasts (50 μM for 24 hrs), resulting in an enhanced glycophagy, respiratory and glycolytic ATP production. 144DG11 prolongs survival and improves motor parameters of Gbe(ys/ys) mice in vivo (250 mg/150 μL/kg 2x per wk via i.v. for 1 month, then s.c.), a murine APBD model, by reducing PG and glycogen levels in brain, liver, heart, and peripheral nerve with good pharmacokinetic properties and no adverse effects to the animals.
Polyglucosan (PG)-reducing agent that targets the lysosomal membrane protein LAMP1 in cultures and in vivo.
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