Q: What are the challenges in Parkinson's research that you feel Parkinson's UK can help address?
There are clearly massive scientific challenges we need to tackle - such as the lack of accurate animal models and biomarkers for Parkinson's, creating mechanisms to share patient data, and the translation of genetic discoveries into new treatments.
We are funding projects to address these, and there is lots of other excellent research happening all over the world - but it's often fractured.
It's clear that we need to be much more than just a funder, we also have a responsibility to lead the way to a more open and united Parkinson's research community that can tackle these challenges together.
As the world's largest patient-led Parkinson's research and support charity, we're ideally placed to act as a trusted and independent broker in developing partnerships between the key players in the research community. We're already using our influence to form a consortium of data custodians to discuss how we can develop a secure, online database for sharing patient data globally.
Another big challenge for Parkinson's research is recruiting patients to clinical trials. Many trials fail to reach their recruitment targets and this is hampering progress towards better treatments and a cure.
The UK provides us with the ideal environment to conduct patient-based research due to our unique National Health Service, and research support from the National Institutes of Health Research and DeNDRoN (Dementias and Neurodegenerative Diseases Research Network).
All this, coupled with our network of 37,000 members means that we're uniquely positioned to mobilise patients to take part in clinical trials.
Q: Tell us about your current research strategy and how that is pushing your search for a cure.
Our 2010-2014 research strategy identified four biggest barriers to progress towards a cure.
Why do nerve cells die? Without doubt, the most crucial and challenging question that we need to answer to find a cure.
Developing new animal models. Research is currently held back by the lack of realistic animal models that truly reflect what happens inside the Parkinson's brain.
Faster, better drug screening. Powerful techniques are needed to speedily and accurately identify promising compounds so that they can move into clinical trials as quickly as possible.
Finding biomarkers for earlier diagnosis. Without a reliable test we cannot diagnose Parkinson's accurately or measure how it progresses - which is a massive barrier to clinical trials. We've made exciting progress on all fronts:
- huge advances in our understanding of the genetic factors involved in Parkinson's which has pinpointed key pathways and processes involved in nerve cell death.
- developing a range of more useful animal models from simpler organisms like fruit flies, to a powerful new genetic mouse model developed by our research team in Oxford.
- identifying existing drugs that have great promise for Parkinson's, including diabetes drug 'exenatide' which is currently in clinical trials.
- funding the world's largest ever in-depth study of Parkinson's to collect the data and samples we need to find a biomarker.
As our current strategic period draws to a close, we're working on a new strategy to build on the huge progress we're making and take these discoveries from the lab into life.
Q: The Parkinson's UK Brain Bank is Europe's largest brain bank dedicated to Parkinson's. Why did the organization take on this ambitious initiative?
We've actually been funding a Parkinson's brain bank (in different guises) since the mid-1980s. Last year we collected 103 brains from people with and without the condition. Our tissue is high quality (most is collected within 24 hours of death) and we make it available to researchers all over the world who are studying Parkinson's free of charge.
The reason we continue to invest in this area of research is that Parkinson's is a uniquely human condition. It only occurs in people and current animal models fall some way short of truly representing the pathology of this complex condition.
Therefore, to really understand Parkinson's and uncover crucial clues to what happens to the nerve cells that are lost throughout the condition, we must study human brain tissue.
Research supported by our brain bank has massively improved understanding of the pathology of Parkinson's. Our brain bank tissue first revealed the problems with mitochondria inside dying dopamine-producing nerve cells, and the accumulation of iron inside the substantia nigra. Both discoveries have launched whole new avenues of research and led to new ideas for treatments that boost mitochondrial function and mop up excess iron.
And advances in technologies mean we can learn more from brain tissue than ever before. For example, new gene expression profiling techniques can measure the activity of thousands of genes across the whole Parkinson's brain. So tissue from our brain bank remains a crucial tool for researchers around the world.
Q: What do you consider the organization's greatest accomplishments over the last decade?
Over the past decade there's been exciting progress in Parkinson's research internationally and our pioneering research has made a massive contribution.
Our researchers have played a huge part in a genetic revolution, discovering key genes (LRRK2 and PINK1) that cause rare inherited forms of the condition, and several others which increase risk. These discoveries have transformed research, giving scientists vital new targets for developing future treatments, representing perhaps the most important breakthrough towards a cure.
In 2010, we established a new, world-leading centre for Parkinson's research. The Oxford Parkinson's Disease Centre brings together a multidisciplinary team of experts across basic and clinical research to tackle some of the biggest challenges facing Parkinson's research.
The team has already achieved significant breakthroughs, including creating a bank of induced pluripotent stem cells from people with Parkinson's, developing a unique transgenic mouse model, and growing a huge patient cohort.
In 2012, we launched the world's largest in-depth study of people with Parkinson's - Tracking Parkinson's - involving 70 clinics across the UK.
We've already recruited over 2,000 patients and their data and blood samples will be made available to researchers studying Parkinson's all over the world free of charge, creating a lasting legacy for future research.
Finally, in October 2013 we began an innovative new clinical trial to see whether a drug called GDNF can slow the development of Parkinson's. This trial is a huge collaborative effort, involving many partners, and is using the most sophisticated new technologies to deliver the drug to the brain.
Q: What are your top research goals over the next year, and what will it take to reach those goals?
As we come to the end of our current 5 year plan, this year our main goal is to develop a new strategy to take our progress in the search for better treatments and a cure to the next level.
Scientists across the world are making significant progress in developing our understanding of Parkinson's, and towards new ideas for treatments – but a more aggressive approach is needed to convert this progress into real benefits for people with Parkinson's.
We recognise that we cannot do this alone. We need to channel the energies of everyone involved in Parkinson's research – academic researchers, biotech and pharma, other funders and charities... and of course people with Parkinson's.
So, our new strategy will be quite different to our last one, with greater emphasis on how we can bring all these players together to tackle Parkinson's together in a more coordinated way by:
- Increasing leadership to focus Parkinson's research on the most urgent challenges and lead the way to a more open and united global research community.
- Increasing effectiveness by creating mechanisms for researchers to share data and tools, facilitating collaborations and campaigning to streamline research processes.
- Increasing investment by partnering with other charities, not-for-profits and industry to fund joint projects, and using our influence to access more support from the UK and European governments.
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