Glial cell line-derived neurotrophic factor (GDNF) is important for the development and maintenance of dopamine neurons. In rodent and primate models of Parkinson disease GDNF can have restorative effects on dopaminergic neurons. But, as GDNF can not penetrate brain tissue from either blood or cerebro-spinal fluid, treating patients with this factor has proved difficult. In the March 31 Advanced Online Publication Nature Medicine, Steven S. Gill, and colleagues at the Institute of Neurosciences, Frenchay Hospital, Bristol, UK, show that direct intra-putamenal GDNF infusion in patients with Parkinson disease can be tolerated for one year and leads to significant increases in dopamine storage in the putamen and an improvement in symptoms (Nature Medicine, DOI:10.1038/nm850, March 31, 2003).

Gill et al. delivered GDNF directly into the putamen of five patients with Parkinson disease by chronic infusion using pumps. Infusion into the postero-dorsal putamen (its sensorimotor component)...