The Parkinson’s UK research strategy 2010-2014

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ContentsA cure for Parkinson’s 4

How do we get there? 6

Priority 1Understanding why nerve cells die in Parkinson’s 8

Priority 2Developing new animal models of Parkinson’s 12

Priority 3Faster, better drug screening 16

Priority 4Finding ‘biomarkers’ for earlier diagnosis 20

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ForewordWe’re the largest charity funder of Parkinson’s research in the UK. Major advances have come from research we’ve supported – helping to improve treatment and care for people living with Parkinson’s, both within the UK and worldwide.

More progress has been made in Parkinson’s research in the last 10 years than ever before. But we’ve still got a lot of work to do in our search for a cure.

Our new research strategy sets out our exciting plans for the next five years. Working with leading research experts, people living with Parkinson’s, and other major funders of Parkinson’s research, we’ve identified four central research priorities that will lead us closer to a cure – and they’ll be used to guide our future funding

Our strategy explains how we plan to tackle the key challenges facing Parkinson’s research through the voices of people who helped to shape our vision.

So far, we’ve invested more than £45million in groundbreaking research. By 2014, that figure will have reached £75million.

Our ground-breaking strategy ensures that every penny we spend works towards a cure and improves life for everyone affected by Parkinson’s.

Dr Kieran BreenDirector of Research and Development Parkinson’s UK

A cure for Parkinson’sWhen we talk about a cure, we mean that we want everyone with Parkinson’s to be able to live their lives completely free of symptoms. Everyone is different, so we’re going to need a range of treatments. This may mean a combination of drugs, nerve cell replacement and therapies like exercise, that together can slow, stop or reverse the symptoms of Parkinson’s.

“Current and future research remains active and assured, under the watchful eye and leadership of Parkinson’s UK.

I trekked 100 kilometres through the Sahara desert last November to raise money for Parkinson’s UK and it’s great to know that the money raised works towards our ultimate goal – a cure for Parkinson’s. It makes every mile worthwhile.

A cure would bring an end to our worries and my uncertain future with Parkinson’s. It would bring new hope to us and all those other people touched by Parkinson’s. It would be truly wonderful.”Roddy

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How do we get there?Over the next five years, we will:• encourage a flourishing community of

Parkinson’s researchers within the UK

• work with other funders and industry to boost investment in Parkinson’s research

• monitor Parkinson’s research worldwide to spot key breakthroughs and keep our research priorities at the forefront

• tell scientists about our research priorities to get the best out of our grants programme

• promote innovative projects to explore exciting new research avenues

• track the success of our research and our progress towards a cure

• show how our research helps people living with Parkinson’s

Priority areas for Parkinson’s researchOur four core priorities will guide our future research funding and lead us closer to a cure.

But it’s vital we maintain a flexible approach. So we’ll still be considering research applications from outside these areas for funding. While together with our Research Strategy Group, made up of scientific experts and Parkinson’s UK members, we’ll closely monitor the latest scientific developments to help us revise and update our priorities.

Coloured X-ray of human skull, brain superimposedMehau Kulyk/SPL

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Priority 1

Understanding why nerve cells die in Parkinson’sResearch has uncovered many changes inside nerve cells that may be involved in their death. But we still don’t know enough about how or why it happens.

Dr Mark Cookson is at the Laboratory of Neurogenetics in the USA. As a leading expert in genetics Mark is unravelling what causes Parkinson’s and explains why we must work out why nerve cells die:

“Parkinson’s research is like putting together a hugely challenging jigsaw puzzle. We know about changes to key proteins such as LRRK2 and alpha-synuclein. We know that both proteins form part of the sticky clumps called Lewy bodies that build up inside nerve cells. And we know about problems with the mitochondria – the power stations inside cells.

But what we need now is to understand how these pieces fit together. We don’t just want to treat the symptoms, we need to get at the underlying causes of Parkinson’s. That way we can develop new types of treatments that can slow, stop or reverse the condition.”

Embryonic stem cells, SEMSteve Gschmeissner/SPL

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“I’m delighted with our new research strategy. It’s realistic and straightforward and I think it ultimately gives people that feeling of hope, which is so crucial to people living with, or caring for a loved one with Parkinson’s on a daily basis.

Everyone knows a cure will not appear overnight, but science doesn’t stand still and one could be round the corner. It is this potential that is so exciting!

For me, having had Parkinson’s for 34 years, the more research that can be funded the better. Understanding why nerve cells die is rightly placed as number one priority and is fundamental to finding a cure. I would certainly like to understand why, my nerve cells began dying when I was only seven years old.

This innovative research strategy is very encouraging and a genuine boost to morale. Together we can find a cure.”Matt

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Priority 2

Developing new animal models of Parkinson’sParkinson’s is a progressive condition that develops over time. It’s vital that we study the whole brain and body as well as investigating what is happening inside individual nerve cells.

Professor J Timothy Greenamyre leads a team at the University of Pittsburgh, in the USA investigating Parkinson’s. He explains why current animal models aren’t good enough:

“Current animal models mimic some of the symptoms of Parkinson’s but they don’t recreate the changes that happen in the human brain. To do this, we need better models, where the nerve cells die slowly and develop characteristic features of Parkinson’s, such as Lewy bodies.

We need to be able to look at the gradual development of the condition in animals as they age. Better animal models will give us crucial information about what happens throughout the early, middle and later stages of nerve cell death. We can then test treatments that could protect the remaining nerve cells.”

Swarm of the fruit fly, Drosophila melanogasterClaude Nuridsany and Marie Perennou/SPL

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“This is probably the most exciting time to be a neuroscientist there’s ever been.

Decades of hard work on understanding the fundamentals of the human brain is beginning to be translated into treatments that will make a huge difference to people’s lives. New technologies and techniques are helping to pick up the pace of research in a wide range of neurological conditions.

In Parkinson’s research the great promise of the Human Genome Project is now being realised – with discoveries of important genes. And nerve cell replacement therapies may yet become a reliable and effective option thanks to the new ability to turn skin cells into stem cells.

Parkinson’s UK has played a pivotal role in supporting and encouraging the scientific community to produce excellent research and keep the goal in mind. We all want to see a cure for Parkinson’s and I believe we’re closer than ever.”Baroness Susan Greenfield CBEVice President of Parkinson’s UK

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Photography: Olly Scarf

Nerve cell, SEMSteve Gschmeissner/SPL

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Priority 3

Faster, better drug screening We urgently need the ability to move promising new drugs forward into clinical trials as quickly as possible. To do this, we need simpler animal models that reproduce rapidly.

Professor Heinz Reichmann of Dresden University of Technology in Germany is the Chair of our research advisory panel. He explains why better drug screening is essential to accelerate a cure:

“Rats and mice are the most commonly used models for testing Parkinson’s drugs at the moment. But simpler models will help us to test more new drugs over shorter periods of time. That way we can rule out the less effective ones quickly. Fruit flies, roundworms and zebrafish all have a lot of potential in this area.

Any promising drug could then be tested in an appropriate rodent model of Parkinson’s before moving on to the clinic. We may also be able to use cutting-edge technologies to grow nerve cells in the lab from people with Parkinson’s and use these as a test-bed for new drugs.”

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“My Dad first noticed something was wrong on holiday when I couldn’t swing my arm properly. The doctors initially thought I had RSI or MS. It took them over four years to diagnose me with Parkinson’s.

Every day wondering what was wrong with me made me feel stressed and anxious, and I became very depressed.

Being told I had young onset Parkinson’s was a relief in some ways – at least then I had a ‘name’ for the problems I was experiencing. And it helped me to deal with it and move on. It took a long time but eventually I began to look more positively at the future.

Early diagnosis is so important – understanding what is happening to you means you can begin to accept the fact you have a progressive illness and try and get on with your life.

One day I hope research will find a cure, or at least a better way to control the symptoms.”Karen

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Priority 4

Finding ‘biomarkers’ for earlier diagnosisDiagnosing Parkinson’s can be a long and difficult process. New tests to identify Parkinson’s as early as possible could give us the opportunity to protect or rescue nerve cells.

Professor Anders Bjorklund at Lund University in Sweden is pioneering new treatments for Parkinson’s. He explains why early diagnosis will be central to a cure for Parkinson’s:

“At present, there is no definitive test for Parkinson’s. We can make a diagnosis in the clinic based on a person’s symptoms, their medical history and perhaps a brain scan. However, these can still give uncertain results that lead to people being misdiagnosed at first.

That’s why we need to find biological markers or ‘biomarkers’ – molecules inside cells or body fluids that reflect the severity of Parkinson’s. We may then be able to predict whether a person might get Parkinson’s and monitor the condition’s progress accurately if they do. Biomarkers would also help us test whether drugs can slow its development rather than simply relieve the symptoms.”

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Tilo and Mike

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As President of Parkinson’s UK, I am especially proud of our intensive and well-targeted research funding. Our ultimate aim is to help create a world without Parkinson’s and this clear, straightforward and sensible five-year strategy will enable us to work towards that goal efficiently and effectively. It will help us to monitor the way we move forward, ensuring that the work we support is rigorously scientific and as productive as possible and that it makes the best use of the funds we raise and award each year.

Jane AsherPresident, Parkinson’s UK

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We’re here to find a cure and improve life for everyone affected by Parkinson’s.

Over the last 40 years we’ve made great strides – investing more than £45million in research, securing 280 specialist Parkinson’s nurses and providing information, support and friendship to hundreds of thousands of people.

But we’re still not reaching everyone who needs us. And we still don’t have a cure. So there is much more for us to do.

Over the next five years our ambitious strategy will push our search for a cure to a new level.

We’re also determined to reach out to everyone living with Parkinson’s, so that no one has to face Parkinson’s alone. And use our influence to demand better services.

But to achieve our ambitions we must grow strong. We need to increase our income by raising more than £110million by the end of 2014.

And we need your help. We need to harness the knowledge, skills and enthusiasm of everyone involved in the work of Parkinson’s UK, whoever they are. Only by working together can we make our vision a reality. Thank you for your support.

Steve FordChief Executive, Parkinson’s UK

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The Drake family

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© Parkinson’s UK, September 2010. Parkinson’s UK is the operating name of the Parkinson’s Disease Society of the United Kingdom. A company limited by guarantee. Registered in England and Wales (948776). Registered office: 215 Vauxhall Bridge Road, London, SW1V 1EJ. A charity registered in England and Wales (258197) and in Scotland (SC037554).

Parkinson’s UK215 Vauxhall Bridge Road London SW1V 1EJ

Free* confidential helpline 0808 800 0303Monday to Friday 9am–8pm Saturday 10am–2pm. Interpreting available. Text relay 18001 0808 800 0303(for text phone users) *calls are free from UK landlines and most mobile networks.

Visit us online at parkinsons.org.ukDrop us a line at hello@parkinsons.org.uk

Every hour, someone in the UK is told they have Parkinson’s. Because we’re here, no one has to face Parkinson’s alone.We bring people with Parkinson’s, their carers and families together via our network of local groups, our website and free confidential helpline. Specialist nurses, our supporters and staff provide information and training on every aspect of Parkinson’s.

As the UK’s Parkinson’s support and research charity we’re leading the work to find a cure, and we’re closer than ever. We also campaign to change attitudes and demand better services.

Our work is totally dependent on donations. Help us to find a cure and improve life for everyone affected by Parkinson’s.

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