GDNF (glial cell-derived neurotrophic factor)

What is GDNF?

GDNF is a special protein that is naturally produced inside the brain. It supports the survival of many types of brain cells, including the cells lost in Parkinson's.

Studies have suggested that when GDNF is given to brain cells it has the ability to encourage these cells to grow again. It may be able to stop the progression of Parkinson’s, something no current treatment can do.

But, because GDNF is a large protein, it cannot reach the brain if taken in a pill or injected into a vein. This means it needs to be delivered directly to the brain using a surgical approach.

Clinical trials

We were the major funder of a pioneering trial that tested whether boosting levels of this restorative protein could slow, stop or reverse the progression of Parkinson’s.

The results from the GDNF clinical trial are not clearcut, but offer promising signs that it may be possible to restore the cells damaged in Parkinson's.

The trial has advanced our understanding of the potential effects of GDNF on damaged brain cells. It has proved that delivering a therapy in this way is feasible and acceptable to patients. And it has shown that it's possible to deliver drugs with precision to the brain.

What's next?

We believe in GDNF’s potential.

Through our drug development arm, the Parkinson’s Virtual Biotech, in 2021 we announced that we would be investing up to £800,000 into Vivifi Biotech, a new company set up specifically to bring the focus and expertise needed to see if it was possible to plan and progress towards a new GDNF trial.

We have reviewed the scientific evidence from the first trial and listened to the views of people with Parkinson’s about whether it is right to move ahead. There’s still work that needs to be done on the device and drug manufacture to make sure they are the best that they can be. But the main focus now is to seek funding partners who can help make a future trial happen.