Clinicopathological findings following intraventricular glial-derived neurotrophic factor treatment in a patient with Parkinson's disease

As part of a safety and tolerability study, a 65-year-old man with Parkinso n's disease (PD) received monthly intracerebroventricular injections of gli al-derived neurotrophic factor (GDNF). His parkinsonism continued to worsen following intracerebroventricular GDNF treatment. Side effects included na usea, loss of appetite, tingling, L'hermitte's sign, intermittent hallucina tions, depression, and inappropriate sexual conduct. There was no evidence of significant regeneration of nigrostriatal neurons or intraparenchymal di ffusion of the intracerebroventricular GDNF to relevant brain regions. Alte rnative GDNF delivery systems should be explored.