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 Parkinson's disease (PD) received monthly intracerebroventricular injections of glial-derived neurotrophic factor (GDNF). His parkinsonism continued to worsen following intracerebroventricular GDNF treatment. Side effects included nausea, loss of appetite, tingling, L'hermitte's sign, intermittent hallucinations, depression, and inappropriate sexual conduct. There was no evidence of significant regeneration of nigrostriatal neurons or intraparenchymal diffusion of the intracerebroventricular GDNF to relevant brain regions. Alternative GDNF delivery systems should be explored.
Kordower
J H
JH
Department of Neurological Sciences, Rush-Presbyterian-St Luke's Medical Center, Chicago, IL 60612, USA.
Palfi
S
S
Chen
E Y
EY
Ma
S Y
SY
Sendera
T
T
Cochran
E J
EJ
Cochran
E J
EJ
Mufson
E J
EJ
Penn
R
R
Goetz
C G
CG
Comella
C D
CD
D000368:Aged; D001921:Brain / Q000473:pathology; D051100:Glial Cell Line-Derived Neurotrophic Factor; D006801:Humans; D007124:Immunoenzyme Techniques; D007276:Injections, Intraventricular; D008297:Male; D009414:Nerve Growth Factors; D009419:Nerve Tissue Proteins / Q000008:administration & dosage*; D018696:Neuroprotective Agents / Q000008:administration & dosage*; D010300:Parkinson Disease / Q000188:drug therapy* / Q000473:pathology
C495039:GDNF protein, human; D051100:Glial Cell Line-Derived Neurotrophic Factor; D009414:Nerve Growth Factors; D009419:Nerve Tissue Proteins; D018696:Neuroprotective Agents