Clinical trials are essential in assessing how effective or safe treatments are as well as advancing a greater understanding about a condition.

The process of medicine development from test-tube to patient can take a decade or more, and is extremely costly to the investing company. These costs can only be recovered and converted to profit in drugs that demonstrate efficacy in phase 3 clinical trials. Sadly, new treatments are comparatively infrequent and the development of each new medicine is a significant event for the Parkinson’s community.

Historically, clinical trials in Parkinson’s have a poor success rate, with many drugs failing to show they are effective at the phase 3 stage, when the evidence for licensing a drug is assessed by the FDA and other regulators. So, the failure of a clinical trial is a major setback to a drug company involved in drug development for Parkinson’s. Failure also has implications for the patient community, depriving them of potentially effective new treatments.

Why do some clinical trials fail?
Clinical trials can be unsuccessful for numerous reasons for example; failures of trial design, recruiting people to take part, communication and information. To try to understand more about the ‘barriers’ that hinder trials from succeeding, Parkinson’s Movement developed and carried out an online survey.
The results of this survey were the focus of the discussion at Rallying to the Challenge 2014, and led to the formation of the Parkinson’s Clinical Trials Charter.

24-25th September 2014 – Challenges at a Glance: Grand Challenges Meeting

• Mr David Van Andel, Van Andel Institute
• Jay Van Andel Award for Outstanding Achievement in Parkinson’s Research
• Professor Andrew Lees
• Tom Isaacs presents on The Urgent Unmet Needs of People with Parkinson’s
• Professor Jeffrey Conn, Taking compounds from the laboratory to the clinic: challenges in drug development for Parkinson’s disease 
• Professor Karl Kieburtz, New drugs for Parkinson’s – what are the Big Picture challenges? 
• Survey Feedback on barriers to effective clinical trials presented by the Parkinson’s Movement team and comments from audience
• Israel Robledo and Steve DeWitte Meeting Objectives and Format
• Dr Jon Stamford, The Role of Parkinson’s Movement in the Meeting
• Short Introduction to Other Organisations Present PDF, MJFF, Davis Phinney, Brian Grant, Parkinson’s UK and CPT
• Tom Isaacs, Summary of Survey Results – The Barriers to Progress with Clinical trials in Parkinson’s – Can We Really Make a Difference. The Linked Clinical Trials Model.
• Brian Grant, ex-NBA star, The benefits of teamwork
• Bernard Ravina, M.D., M.S.C.E, Voyager Therapeutics, USA, Challenges with trial design regarding disease-modifying therapies for neurodegenerative disease
• Kuldip Dave PhD, Michael J Fox Foundation, USA, The Parkinson’s Progression Markers Initiative (PPMI) Study update
• Tom Isaacs, Dr Jon Stamford (UK), Israel Robledo and Steve DeWitte (US) Rallying to the Challenge talk, How people with Parkinson’s can be a resource and contribute to more effective clinical trials
• Caroline Tanner, M.D., Ph.D. – The Parkinson’s Institute and Clinical Center, USA, Optimising outcome measures for disease modification trials in Parkinson’s disease
• Michael A Schwarzschild, M.D., Ph.D. – Massachusetts General Hospital, USA, Non-imaging biomarkers as support clinical trials in Parkinson’s disease
• David Brooks, Ph.D. – Imperial College of London, Brain imaging and disease-modification in Parkinson’s disease

Over a quarter of those involved also reported the importance of the subject matter of the trial, and problems associated with accurate measurement of results.
People with Parkinson’s were asked the reasons why they might not engage/participate in clinical trials and the top five reasons were given as:
• Potential of adverse consequences/side effects (58%)
• Disruption of medication regime (53%)
• Possibility of being on placebo (39%)
• Upheaval to life (38%)
• Not being informed of the progress and results of the trial (34%)

Over 25% of respondents also reported other important reasons such as:
• Possibility of not being able to reclaim all the costs incurred through participation
• Being excluded from future trials
• Lack of access to understandable information about what the trial involves

You can read about the survey in more detail in an article published in the Journal of Parkinson’s Disease.

This year’s theme for Rallying to the Challenge was the issue of data:  big data, small data, personal data, impersonal data, what’s useful and what’s not, and how data could and should be used.

Collecting information about our personal experiences can inspire greater accuracy and urgency in research and even shape scientific and clinical expertise.  This not only redefines the research landscape but even more importantly helps to personalise Parkinson’s. However, one of the pressing issues that came out of Rallying 2015 was a need to define how data is used, who has access to it, and what level of control does a person have over their own data?

We believe it is hugely important that we define how we want our data to be used, particularly as there is growing pressure for legislation to be introduced to tell us how it can be used. To address these issues we developed a survey which 389 people with Parkinson’s (PwP) completed. Overall the survey found that the majority of people are willing to share their data for research purpose but far fewer people are actually doing so. It also revealed that in many cases it is unclear whether data is fully anonymised, who owns the data that people share and who is accessing it. A published results of the survey results can be viewed here. These results formed the basis of the discussion and debate at the focus groups which took place at the Rallying to the Challenge 2016 meeting:

It was clear from this focus group that anonymity is vital and that the individual who is sharing their data should own the data. Exactly how the data is going to be used must always be transparent from the outset and any changes should be communicated in on-going dialogue with the person sharing their data.

It was concluded that a Parkinson’s specific Data Charter should be created recommending ‘best practice’ on the issues discussed, for both PwP sharing their data and groups collecting data. Specifically, a Data Charter would clarify what information PwP would like to share, identify how this information should be used, define how that data should be accessed, by whom and in what circumstances (e.g. frequency, viability).

MONDAY, SEPT. 26, 2016 – TUESDAY, SEPT. 27, 2016

• Tom Isaacs, Co-founder of Parkinson’s Movement, Welcome film,
• Dr. Jon Stamford, Co-founder of Parkinson’s Movement, Why data, data, data? What did we learn from Rallying to the Challenge: Outcome Measures (2015) that makes the theme for Rallying 2016 so pertinent?
• Peter Schmidt, National Parkinson Foundation, Presentation: Health Confidence,
• David Jones and Richard Windle, Parkinson’s advocates Survey findings
• Ray Dorsey navigates the measurement world in Parkinson’s
• Brainstorm chaired by Dr. Jon Stamford and other Parkinson’s Movement representatives, What do we mean by Big Data? What questions do we want answered by Big Data? What are the most useful data needed that would improve a) research and b) care?
• Focus groups: What data is needed vs what data is offered and is engaging for people to give? How we can collect relevant data from PWP with the quality and in the quantity required to allow experiences to drive the research agenda? How do we engage sufficient numbers of people with Parkinson’s in recording information about themselves? What controls do we need in place?
• John Dean, Davis Phinney Foundation, What data would improve health outcomes?
• What data supports research into cures? Panel discussion (chaired by Patrik Brundin, M.D., Ph.D.)
• Stephen Friend, Sage Bionetworks, Individual classifiers of an individual’s Parkinson’s
• Ethical considerations around data collection, sharing, ownership and evaluation, Motivation to contribute data, Data ownership
• Panel discussion: All your questions answered, Dr. Patrik Brundin and others
• Dr. Jon Stamford, David Jones, Richard Windle and others, Feedback presentation to Grand Challenges in Parkinson’s Disease: What data should we collect and how should that data be used? 

Rallying to the Challenge 2018: Non-motor symptoms 

The Stateside event, hosted by The Van Andel Research Institute (VARI) and organised by The Cure Parkinson’s Trust (who fund and support this website) took place this year on Wednesday 26 and Thursday 27 September. The theme of this year’s conferences was Non-Motor Symptoms – for many just as frustrating, if not more so, than the well-documented movement-related symptoms.

In addition to an overview of non-motor aspects of Parkinson’s, this year’s Rallying explored fatigue, exercise and sleep and this year’s survey was designed to understand the psychological impact of symptoms on people living with Parkinson’s and their families.

WEDNESDAY, SEPT. 26, 2018

10:20 a.m.
Welcome to Rallying to the Challenge 2018
Steve DeWitte
Dr Soania Mathur
David Jones

10:25 a.m.
For 185 years a number of non-motor aspects of Parkinson’s were ignored –  what are we missing now and can Fox Insight help us identify these aspects?
Dr Brian Fiske Fox Insight, MJFF for Parkinson’s Research

10:45 a.m.
Impact of non-motor symptoms from a clinical perspective. Personalised medicine, phenotypes and moving forward? 
Professor Ray Chaudhuri, Kings College London

11:15 a.m.
The language of fatigue
Led by Dr Jon Stamford, Leah Mursaleen

11:30 a.m.
CPP Survey findings
Claire Bale, Parkinson’s UK with Richard Windle

11:40 a.m.
More about fatigue
Dr Angie Kehagia and Dr Joy Duffen

12:30 p.m.
Your questions answered
Drs Patrik Brundin,  Nico Bohnen and Michele Hu

1:30 p.m.
Moving Evidence: The Impact of Exercise on Non-Motor Symptoms
Sara Garvey, Davis Phinney Foundation and Alison Sheltrown and Jillian Carson

2:30 p.m.
Parkinson’s Patient Phenomena Survey findings: impact on PwP and their families of aspects of Parkinson’s symptoms
Dan and Ginny DeWitt, Shaun Hindley, Bruce Mabee, Ken and Jill Peirce, Louis Bouma, Gloria Groner and Ron Rutowski

3:30 p.m.
Focus Group: Sleep, Cognitive, Personality and Behavioural aspects Tools needed for identification/measurement/evaluation of impact
Led by: Dan and Ginny DeWitt, Shaun Hindley, Bruce Mabee, Ken and Jill Peirce, Louis Bouma, Gloria Groner and Ron Rutowski

4:30 p.m.
Feedback from Focus Groups

5:00 p.m.
World Parkinson’s Congress

THURSDAY, SEPT. 27, 2018

9:30 a.m.
Monitoring and measuring non-motor symptoms: the challenge
Dr. Camille Carroll

10:00 a.m.
Issues in Parkinson’s: Sleep disorders 
Ben Stetcher

11:15 a.m.
Parkinson’s Movement: explaining research, celebrating advocacy and contributing to research
The Parkinson’s Movement Team: Eros Bresolin, Gaynor Edwards, Julia Jockelson and Helen Matthews

11:55 a.m.
Concluding comments
Dr Soania Mathur, Steve DeWitte, David Jones

12:15 p.m.
Your questions answered 
Panel of clinicians Drs Kathleen Shannon and Ron Postuma

12:15-45 p.m.
Facebook Live
Professors Patrik Brundin, Ray Chaudhuri, Viviane Lebrie and Dr Jon Stamford

1:00 p.m.
Presentation of the Tom Isaacs Award

1:15 p.m.
Presentation to Grand Challenges in Parkinson’s
Claire Bale, Parkinson’s UK with Richard Windle

Non-Motor Symptoms: Impact, correlation, symptom relief, symptom monitoring

With very many thanks to the Van Andel Research Institute and Team Spark, Grand Rapids’ Parkinson’s Community we once again hosted a terrific Rallying to the Challenge meeting in parallel with Grand Challenges in Parkinson’s.  This year around 100 delegates joined us to explore the impact of non-motor symptoms in Parkinson’s, both for the individual living with the disease but also their partners, spouses and loved ones.

Brian Fiske, Senior Vice President Research Programmes at the Michael J Fox Foundation opened the meeting highlighting the range and diversity of non motor aspects of Parkinson’s and invited the audience to contribute their experiences of their condition by joining Fox Insight, the open-source surveying too.

We were privileged that the 2018 Jay Van Andel Award winner, Professor Ray Chaudhuri from Kings College London, who has championed research into non-motor aspects of Parkinson’s also addressed the meeting.  He elaborated on the variety of symptoms that characterise the condition, with each person experiencing their own changeable combination of symptoms and explored the current research underway to tackle non-motor aspects of the disease.

The variety of symptoms that people experience has been an important theme for the Rallying meeting.  The 2015 Rallying survey identified that fatigue was significantly impacting the quality of life for a large number of people.  At this year’s meeting there was a fantastic focused discussion around fatigue.  Presentations by Dr Jon Stamford, Leah Mursaleen and Angie Kehagia explored the language used to differentiate fatigue from tiredness and how that can help us better define fatigue and therefore lead towards more accurate measurement of its impact on quality of life.

This session was followed by an action packed presentation about the benefits of exercise on non-motor symptoms of Parkinson’s by local advocate Alison Sheltrown with physiotherapist, and founder of Parkinson’s Wellness Project in British Columbia, Canada Jillian Carson and Parkinson’s wellness specialist Sara Garvey of theDavis Phinney Foundation.

The meeting also explored the impact of sleep in an inspirational presentation by Canadian advocate Ben Stecher, author of https://tmrwedition.com . Ben went on to call for more voices to join the advocacy effort to change the way Parkinson’s is treated and to add urgency to the research effort.

Dr Camille Carroll from Plymouth University highlighted some of the approaches that are currently being used to objectively measure and evaluate non-motor aspects of Parkinson’s, however this is a challenging field given that each individual has their own recipe of symptoms that impact their quality of life and therefore we need a range of evaluation approaches to suit each individual and their own circumstances.

Local Team Sparks Ralliers Louis Bouma, Dan and Ginny DeWitt, Gloria Groner, Shaun Hindley, Bruce Mabee, Ken and Jill Peirce, Ron Rutowski devised this year’s survey around the impact of Parkinson’s symptoms on quality of life for the individual with Parkinson’s and their partners, spouses and families.  The survey findings triggered a spirited discussion in the focus groups around the importance of communication when raising sensitive issues particularly around thinking and memory and the call for a communication tool to support conversations in a timely way.

In parallel a group explored the creation of a scale to prioritise potential therapies for clinical translation based on the needs of the patient community, which would help ensure scientists are in alignment with people with Parkinson’s. In a fitting tribute, Ralliers have suggested that it be called the “Isaacs Scale” in honour of Trust co-founder Tom Isaacs.

In summary, this year’s Rallying meeting not only highlighted the wide variety of non-motor symptoms people experience but also that these symptoms change, as does their impact on an individual’s quality of life. This variety and variability of symptoms has implications for communicating effectively about the disease not only to a PwP’s healthcare team but also to their spouses, partners and families. The focus groups led by Team Spark identified the need for a communication tool and working with the Parkinson’s Movement and CPT teams will be helping to design this in the coming months.