The Pharmaceutical Journal
Vol 270 No 7244 p507
12 April 2003

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Molecule promising for alleviating symptoms in Parkinson's disease

A naturally occurring molecule injected directly into the brains of patients with Parkinson's disease may help alleviate symptoms, a new study shows.

The molecule, glial cell line-derived neurotrophic factor (GDNF), is involved in the development and maintenance of dopamine neurons in the substantia nigra and has restorative effects in rodent and primate models of Parkinson's disease. However, penetration into brain tissue from either the blood or the cerebrospinal fluid is limited.

In a phase I trial, researchers delivered GDNF directly into the brains of five patients with idiopathic, L-dopa-responsive Parkinson's disease who were poorly controlled on optimal medical therapy. GDNF was infused directly into a region of the brain most severely depleted of dopamine in Parkinson's disease.

They found that GDNF led to increases in dopamine storage and that symptoms improved in all five patients after three months of treatment. Reductions in many of the rating scores for Parkinson's disease were seen. GDNF can be tolerated for one year.

However, the researchers point out that precise functional effects of GDNF delivery have not yet been determined because of the open nature of the trial and the small number of patients. The study warrants careful examination of GDNF as a treatment for Parkinson's disease, they say. The study is to be published in the May issue of Nature Medicine.