Dr Richard Wyse (above) - Director of research and development, presenting at a recent research update meeting.

Getting going: trials of treatments to slow, stop, reverse Parkinson’s

A raft of new knowledge from many sources about the nature of Parkinson's (PD) has led to new attempts to match a range of innovative therapies to the emerging science with the hope of changing the way the disease progresses and so changing the lives of those who live with the condition.

Since its inception in 2012, our unique initiative called the Linked Clinical Trials programme (LCT) has seen some of the leading world experts in PD come together to study in great detail the merits of more than 70 potential new ‘disease-modifying’ treatments, each with different but compelling biochemical reasons for their potential to slow or stop long term decline in Parkinson’s. The LCT initiative, spearheaded by The Cure Parkinson's Trust and supported by The Van Andel Institute, Grand Rapids, Michigan brings the most promising of these treatments immediately into clinical trials (if funding is found). To date, this committee has prioritised around 50 of these new radical approaches to be tested in people with Parkinson's in specialist neurology hospitals around the world.

The first of these trials involving the repurposing of an anti-diabetic drug (Exendin-4) was completed in 2013 with extremely promising results in terms of intercepting long term PD decline. The Bydureon trial which followed on from this, resulted in further promising results.

Replication is the watchword in medicine and part of this global initiative are two new trials using similar diabetes drugs which are underway in California and in France (certain diabetes drugs influence how individual cells work in a way that we believe is highly beneficial to ailing neurons in PD). Another trial, in 25 hospitals around the UK, is testing Simvastatin (a cholesterol lowering treatment) for its disease-modifying potential in PD.

For us, one breakthrough would be incredible, but as everyone’s Parkinson’s is different, we need multiple breakthroughs if we are to defeat Parkinson’s once and for all.

Removing iron to slow Parkinson’s

Another clinical trial of promise is that of the use of an iron chelation drug to remove the build-up of excess iron from neurons in people with PD, a factor thought to contribute to disease progression. 

Mitochondria: restoring neuronal energy – restoring function

Mitochondrial dysfunction: In Parkinson’s neurons, energy production and use seems to be deficient and its biochemical restoration offers huge potential for slowing down Parkinson’s progression. As two ‘mitochondrial’ targeted drugs under the LCT global initiative (EPI-589 and MSDC-0160) move into their PD clinical trials, we now have new uniquely sensitive ways to monitor these energy processes, even to the point that for the first time ever we believe it will be possible to develop personalised medicine for people with Parkinson’s, with future therapies matched to their genetics and biochemistry as much as their symptoms.

Also within this area of research we are investigating the impact of oxidation and inflammation in the pathogenesis of Parkinson's with the trials of potentially anti-oxidant effects of certain drugs now underway. 

Understanding Parkinson’s, leading to finding and developing new treatments

Many feel that cell-to-cell transmission of abnormal forms of the alpha-synuclein protein help drive PD progression by a process which mirrors prion disease, possibly even starting in the gut neurons before migrating to the brain. This alpha-synuclein build-up in neurons in people with PD is currently being treated in the clinic in an immunisation trial in Austria. Other therapies, such as introducing a bacteriophage to disaggregate alpha-synuclein within neurons, and several other drug approaches we are involved in (such as Anle 138b) also offer great promise.

Reversing Parkinson’s

Regenerative medicine which also offers hope for a fundamental cure, continues to make substantial progress in PD. Large surgical trials of transplantation of neural tissue and of long-term infusions of a natural brain growth factor called GDNF are on-going in Cambridge and Bristol and offer potential promise. Gene silencing and RNA approaches are also emerging as potent future approaches to treat PD.

The Cure Parkinson’s Trust is constantly searching for new routes to slow, stop or even reverse Parkinson’s. Much of our drive is focused on developing new treatments that will affect people living with Parkinson’s within a five year period, but understanding how these treatments work on a cellular basis is also crucial. We are constantly screening for potential new treatments. For us, one breakthrough would be incredible, but as everyone’s Parkinson’s is different, we need multiple breakthroughs if we are to defeat Parkinson’s once and for all.

To help us drive our research forward, please consider donating today - your support is crucial for our work to continue - thank you.

Click here to download our helpful infographic 'Ten Routes to a Cure' which shows the key areas of research CPT is actively pursuing.