Making progress towards a potential new GDNF trial

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.

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. In February 2019, we got the results. They were inconclusive, but there were some signs that the treatment may have started to regenerate participants’ dopamine-producing brain cells. 

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.

Today, we are starting the last phase of its work: seeking out partners to move GDNF towards another clinical trial. 

A better understanding of the challenges

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 for this next phase will be seeking funding partners who can help make a future trial happen. 

Arthur Roach, Director of Research, said: 

"Through Vivifi Biotech’s work in the last 2 years, we now have a better picture of how GDNF might be studied further through a new clinical trial, and a specific plan that incorporates learnings from participants in the last trial, people with Parkinson’s who might be asked to consider participating a new trial, and scientific and regulatory experts. That includes a more detailed understanding of issues with drug supply and the device. And we still believe that a new GDNF trial is possible.  

"That’s why we’re continuing to invest in Vivifi to ensure that their work can keep making progress. We’ll be monitoring their findings every step of the way, so like with every project in the Parkinson’s Virtual Biotech, we can accelerate, pause or stop work as needed. And we’ll be looking for potential investors in the next stage of GDNF, alongside our other Virtual Biotech projects as part of a wider push for more investment in Parkinson’s research."

The evidence is showing that there could still be a way forward for GDNF and finding investors needs to be the next priority. And that’s why we’re not stopping.

Informed by the community and experts 

With what we now know from the work done to date, we’re continuing to make sure that the potential of GDNF is explored to its fullest. We believe that if another trial is possible, it could take around 4 years to see results. 

Reflecting on the decision, the GDNF Participant Group, shared the following: 

"The GDNF Participant Group have never wavered in their belief in GDNF and are delighted that their tenacity and determination to seek a further trial has been rewarded by Vivifi’s investigations. This has never been just about us, but about those we have lost, those with us now and those yet to come.
 
"The Bristol GDNF trial was a prime example of innovative thinking and bold endeavour, which deserves to be assessed with the same diligence and vision. The decision of the Board of Trustees is a shining light in the fight against Parkinson’s.
 
"We would dearly love to pass on the hope to the Parkinson’s community that we once had and call on all prospective investors to get behind this potential 'Best in Class' treatment to take the fear out of a diagnosis of Parkinson’s."

Working towards new partners to help make it happen

We’ve always known that we wouldn’t be able to take forward a new trial of GDNF alone. Now with a better understanding of the estimated costs, we know we’re looking for investors who can commit tens of millions of pounds. 

Gary Shaughnessy, Chair of the Board of Trustees, said: 

"In the last 2 years, with the insight and support of the GDNF Involvement Advisory Board, which includes members of the GDNF Participants Group, consultations with people living with Parkinson’s, as well as scientific and project advisors, we have made real progress. Together we believe that GDNF has real promise, but there is still a long way to go before we can test it again in a clinical trial. 

"Finding investors to fund the very significant costs of a new trial is an immediate priority. We will be able to fund some of the development ourselves but at this stage we need a partner who is willing to fund alongside us and to bring their expertise to help deliver a substantial and effective trial that is as safe as possible for all participants."

In the meantime, through the Parkinson’s Virtual Biotech, we’ll continue to invest in other projects too. Ones that can accelerate us towards other treatments, pursuing the most promising options available to us.