Infusing a growth factor protein directly into the brain was safe and improved the symptoms of patients with Parkinson's disease.

Previous research has suggested that a protein called glial cell line-derived neurotrophic factor (GNDF) may help to protect or regenerate dopamine neurons. These are the cells that are lost in Parkinson's disease, leading to symptoms such as tremor and loss of co-ordination.

Now we have the first evidence of GNDF therapy in humans. Researchers at the University of Wisconsin-Madison say that the treatment is safe and it gives rise to marked improvements in the abilities of those with advanced Parkinson's disease. They studied a group of five patients at the Frenchay Hospital, Bristol, England. The GNDF was infused direct into the brain through a catheter. The patients had no severe side effects, and a marked improvement in their quality of life and ability to carry out daily activities.

Brain imaging revealed a marked increase in the brain's ability to store dopamine. It's not yet clear if the GNDF is helping make more dopamine neurons or preventing destruction of existing ones. This was a very small trial, whose main aim was to demonstrate the safety of the treatment. But the results are very promising and it's to be hoped they'll now be confirmed with larger trials.

Source

Nature Medicine 31st March 2003