What’s next for the GDNF trial?

GDNF is the only therapy which has been shown to restore the function of dying dopamine neurons in the brain of people living with Parkinson’s disease and with this there is reversal of symptoms.

With the release of the GDNF trial results (Results summary) it prompts the question…Whats Next?

The next and definitive step is for a further trial, before the approval of GDNF therapy.  It will include a dose escalation to see if they can increase the rate and magnitude of GDNF’s benefit. The team are now able to deliver a higher dose than was possible in this phase II trial. Due to experiments conducted in parallel with the trial, that have shown doses four fold higher are safe. The trial will  also validate a potentially better way of evaluating disease severity in people living with Parkinson’s, using a new scoring system. The system shows how much of the day their Parkinson’s Disease is well controlled and what they are able to do in addition to measuring their motor function. The current standard only looks at the motor function off medication and does not reflect what matters to patients.

Going Forward…
There are now major concerns that this neuro-restorative therapy. Which holds so much hope for those with Parkinson’s will be lost unless a small lead-in phase III study can be initiated, within the next 6 months  and demonstrate the enhanced efficacy of this therapy at a higher dose. Investors have already indicated their willingness to invest the large sums necessary to take this through to market authorisation.  Should such a study have a positive outcome. The cost of the lead-in study will be £4million.  Many of the trial participants are already fundraising for this. Funding Neuro have a dedicated fund for this vital lead-in study.  With your donations we can work toward reversing Parkinson’s disease!

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