GDNF: where are we 1 year on?

While not clear cut, last year’s GDNF clinical trial results revealed signs that GDNF may make it possible to repair the cells damaged in people with Parkinson’s. We take a look at where we are a year later.

What is GDNF?

GDNF stands for glial cell-derived neurotrophic factor. It’s a naturally-occurring protein that is produced inside the brain. It supports the survival of many types of brain cells, including the cells lost in Parkinson's.

What happened last year?

We’re committed to finding a cure for Parkinson’s. And developing better treatments faster. This commitment led to us being the majority funder of the GDNF phase 2 clinical trial.

The pioneering trial aimed to investigate whether boosting levels of this restorative protein could slow, stop or reverse the progression of Parkinson’s. 

The results were released on 27 February 2019. Though inconclusive, the trial showed some signs that the treatment may have started to regenerate participants’ dopamine-producing brain cells.

And now?

A year later, many people who participated in this trial are not giving up hope that GDNF could one day provide a breakthrough in Parkinson’s treatment.

Steve, our Chief Executive met with some of the participants: 

“They tell me they’re still experiencing the benefits of taking part in the trial and that they are taking less medication now than before they started the trial 7 years ago.”

After a year, we remain as committed as these participants to accelerating breakthroughs in research. Taking the results from the recent trial, it may be possible to develop and fund a further, successful trial.

As Steve says, “I believe that this story isn’t over yet."

In his blog, you can read more about GDNF, and other global research developments that may hold the key to delivering treatments. Faster.

You may also be interested in the initial results from a study into a similar protein, called CDNF. Initial results from a phase 1/2 study of an experimental protective protein called CDNF have been published. This protein may hold potential to slow, stop or reverse Parkinson's.

Read more about GDNF

The trial has advanced our understanding of the potential effects of GDNF on damaged brain cells.

But there are still important questions to answer about GDNF's potential to be a life-changing treatment for people with Parkinson's.

Read more about GDNF, the results of the trial and what's next.