Finding partners — the future of GDNF

Recap

What’s GDNF?

GDNF stands for glial cell-derived neurotrophic factor. It is part of a group of proteins called growth factors and some refer to these as "the fertilisers of the brain". This is because these growth factors are naturally produced in the brain and support the survival of many types of brain cells, including the cells lost in Parkinson’s.

Why is it promising for people with Parkinson’s?

Previous studies in the lab have shown that GDNF could protect and even have the potential to regrow dopamine producing brain cells. However, this has never been successfully shown in people with Parkinson’s. That being said, trial results published back in 2019, although not clear cut, did show a glimmer of hope for GDNF as a potential treatment.

What did the previous trial show?

We were the major funder of a pioneering trial that tested whether boosting levels of this protein could slow, stop or reverse the progression of Parkinson’s. To get GDNF to the brain cells that need it, a specially designed delivery system was developed. The participants underwent robot-assisted surgery to have 4 tubes carefully placed into their brains. This allowed GDNF to be infused directly to the affected brain areas with pinpoint accuracy, via a port in the side of their head. The reason this is currently the best delivery method is because the protein is too large to cross the blood-brain barrier and therefore can’t be given simply via an oral drug or injection.

In total, 41 participants took part in the double-blind trial. The results were inconclusive, but there were some signs that the treatment may have started to regenerate participants’ dopamine-producing brain cells.

Read more about the results of the double-blind trial.

What’s happened since the trial?

The trial left many questions unanswered. We’ve been working with the companies, researchers and participants involved to find a way forward and in 2021 launched a new company, Vivifi Biotech, tasked with leading the planning and preparations needed to design a new trial. To build on the learnings from the previous trial, and put the wellbeing of participants at its centre.

Read more about our commitment to plan a new GDNF trial.

What’s new?

Seeking funding partners

Like all of our Virtual Biotech projects, we regularly check in on them to make sure we are agile and backing the research that is the most promising for the Parkinson’s community. We can accelerate, pause or stop work as needed. Over the last year and a half, Vivifi Biotech has made progress towards scoping a future GDNF trial that aims to overcome the challenges of the last trial.

This project is progressing well and will now start work on the final phase of planning: seeking out partners to help move even closer to a future GDNF clinical trial.

The continuation of this work comes after reviewing the scientific evidence from the first trial and listening to the views of people with Parkinson’s about whether it is right to move ahead. This includes input from past participants as well as a group of people who have no previous connection to the GDNF research. 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 at Parkinson’s UK, said:

"Through Vivifi Biotech’s work over the last one and half 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 work towards a possible new trial. We’ll be monitoring this project 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."

When could a possible trial start?

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.

We’ve always known that we wouldn’t be able to take forward a new trial of GDNF alone. So we’re looking for investors who can commit tens of millions of pounds.

We will continue to provide updates on this project. Please contact [email protected] if you have any questions.