Why we’re investing to drive forward a new trial of GDNF
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Quick summary:
- Parkinson’s UK has created a new company, Vivifi Biotech, to drive forward planning for a new trial of device-delivered GDNF.
- This experimental therapy was the subject of a recent clinical trial which produced inconclusive results.
- Despite this, we believe GDNF still holds the potential to be a life-changing treatment for Parkinson’s.
- Over the next 1–2 years we’ll invest up to £800,000 through our Virtual Biotech programme to try and make another clinical trial happen.
GDNF — or glial cell-line derived neurotrophic factor — is a special protein that is naturally produced inside the brain and supports the survival of many types of brain cells — including the cells lost in Parkinson’s.
GDNF was discovered in 1991 and experiments quickly highlighted its potential for protecting or even restoring dopamine-producing brain cells, giving hope for people with Parkinson’s.
Since these discoveries, the major challenge has been finding a way to deliver GDNF to the right part of the brain at the right concentration to understand whether it may have the same important effects in people with Parkinson’s.
The latest trial
In February 2019 we announced results from a clinical trial which used a new purpose-built device to deliver GDNF to the brains of people with Parkinson’s. Four tubes were carefully inserted into the brain to allow the drug (or a placebo) to be delivered to the right part of the brain with pinpoint precision once a month.
At the end of the trial results showed some tantalising signs of promise.
Brain scans showed that those who received GDNF had a 100% improvement in dopamine activity in the treated brain regions compared to those who received placebo. And many participants reported profound and long-lasting benefits in symptoms and quality of life.
However, despite these positive signs, the results fell some way short of demonstrating a clear-cut difference between GDNF treatment and placebo in crucial assessments of symptoms.
This means that ultimately the result was negative, a huge disappointment for the Parkinson’s community. The results also mean that many in the scientific community remain unconvinced of the potential of GDNF.
You can explore the full results in more detail in this previous blogpost:
We are stepping in
The results were not clear cut, but we believe that there are still important questions to answer and that GDNF holds the potential to be a life-changing treatment for people with Parkinson’s. The challenge is to create a new study that builds upon the learnings from the last one and that can provide a definitive answer.
Over the past two years, we as majority funders of the trial, alongside the international team that carried it out and the participants, have been seeking a path forward to create a new clinical trial.
Putting together a new study for a therapy as complex as this one, and where there have been previous failures, is a seriously challenging task that requires dedicated focus and investment.
So that’s what Parkinson’s UK is stepping up to do.
Through our Parkinson’s Virtual Biotech we have created a new company, called Vivifi Biotech, to focus solely on advancing this complex experimental treatment.
Thanks to a new agreement with MedGenesis — the Canadian company that was previously leading the development of GDNF — in the coming months, Vivifi Biotech will receive the full rights to use the existing patents, licenses, research materials, clinical data and know-how previously owned by MedGenesis, to advance the planning for further trials of GDNF.
Taking steps towards a new trial
Over the next 1–2 years, Parkinson’s UK will be investing up to £800,000 in Vivifi Biotech through the Virtual Biotech programme to drive forward planning and preparations for a further trial of device-delivered GDNF.
This will involve a wide range of important activities, including working with scientific experts and people affected by Parkinson’s to create a new clinical trial plan that gives the best possible chance of a definitive outcome, and crucially, is designed with participant wellbeing at its centre.
As part of this process, we will discuss our ideas and plans with regulatory agencies — such as the MHRA in the UK and the European Medicines Agency — to gain their input and advice. These authorities are ultimately responsible for approving new medicines for use, so understanding their expectations and requirements is critical if GDNF is ever to become an available therapy.
There is also significant work to be done on the manufacturing and supply of both GDNF itself and the delivery device.
Finally, we know that to make a new clinical trial happen we’ll need to secure significant investment from external investors as a new trial is likely to cost in excess of £10million. So, over the next 1–2 years, we will be seeking potential partners to help bring in the investment we need.
If all of this careful and complex work is successful, a new clinical trial will emerge and could begin as early as 2022.
However, we must also recognise that the ultimate result may be that we cannot find a way to overcome all the challenges and that a further trial of this particular form of GDNF therapy is not possible.
There are also other projects underway around the world that are exploring neurotrophic factors and alternative ways to get these important proteins to where they are needed in the brain. So if this particular approach isn’t able to proceed to a new trial, it won’t be the end of the story. As always we’ll be keeping abreast of developments with the wider research landscape so that we can learn from and support other promising research in this area.
Delivering better treatments and a cure
As well as focusing on creating a new plan for a new GDNF trial, we at Parkinson’s UK will continue to drive forward other promising new treatments and therapies for people with Parkinson’s.
No one treatment is likely to be the answer for everyone with Parkinson’s so we’re determined to push forward as many new therapies as possible to meet the unmet needs of the community.
We are already funding important trials to find new treatments for dyskinesia and to combat psychosis and hallucinations. We’re also investing in developing new drugs with the potential to slow, stop or reverse Parkinson’s and to explore promising non-drug therapies.
Because everything we do is guided and informed by people living with the condition, we won’t stop until everyone can live entirely free from Parkinson’s.
