AGT‑190 is an investigational re-engineered form of glial cell line-derived neurotrophic factor (GDNF) for the treatment of Parkinson’s disease (PD) and stroke.1 GDNF is a naturally occurring brain protein that has demonstrated neuroprotective effects in animal models. However, GDNF does not cross the BBB, and current forms must be delivered directly into the cerebrum to be effective. AGT-190, a neurotrophin fusion molecule, crosses the BBB via receptor-mediated transport.1

Preclinical validation studies demonstrated that AGT-m190 was neuroprotective in a mouse model of Parkinson’s disease.2


References:

1 Boado RJ, Pardridge WM (2009) Comparison of Blood-Brain Barrier Transport of GDNF and an IgG-GDNF Fusion Protein in the Rhesus Monkey. Drug Metab Disp. 37:2299-304

2 Fu A, Zhou QH, Hui EK, Lu JZ, Boado RJ, Pardridge WM (2010) Intravenous treatment of experimental Parkinson’s disease in the mouse with an IgG-GDNF fusion protein that penetrates the blood-brain barrier. Brain Res. 1352:208-13