History

Previous Rallying to the Challenge Meetings
In 2014, co-founder and President of The Cure Parkinson’s Trust, and Parkinson’s Movement, Tom Isaacs contacted Professor Patrik Brundin to ask whether we could host a small think tank for people with Parkinson’s allied to Grand Challenges.  “Just a small group of 15 or 20 advocates with Parkinson’s,” Tom said persuasively.

That September, more than 100 people affected by Parkinson’s and Parkinson’s organisations gathered in Grand Rapids for the first Rallying to the Challenge meeting, with the sole objective of understanding how people living with Parkinson’s could best support clinical trials and research into new treatments for Parkinson’s.

2014

Clinical trials
Rallying to the challenge 2014 focused on how people with Parkinson’s (PwP) can strongly influence the effectiveness of clinical trials.

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
This survey was carried out to try to understand more about the ‘barriers’ that hinder trials from succeeding.
For those involved in conducting clinical trials the following top five reasons or ‘barriers’ were:
• Funding (66%)
• Administrative support (46%)
• Recruitment of people with Parkinson’s (PwPs) (44%)
• Practical support from other organisations involved in the trial (36%)
• Time taken for institutional contracting (32%)

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.

  

2016

Collecting data
Rallying to the Challenge 2016: Data, Data, Data 

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? 
 

2015

Symptoms
At Rallying to the Challenge 2015, clinical trial outcomes were put under the microscope.
Clinical trials put great emphasis on measurement scales such as UPDRS (United Parkinson’s Disease Rating Scale). These are questionnaire-based scales and provide a snapshot of a patient’s well-being at any one time. Because of the constantly changing nature of Parkinson’s, even within individuals, it is increasingly accepted that most clinically used measures of outcome in clinical trials give only a partial and non-specific picture of patient well-being. Moreover most existing outcome measures focus on movement components (e.g. stiffness, slowness, tremor), with less emphasis on non-movement symptoms (e.g. fatigue, sleep, pain).

For patients, outcomes that relate to quality of life are more important. Movement symptoms per se influence but do not solely determine quality of life. There is increasing evidence that non-movement symptoms have a particularly strong effect on quality of life for patients.

Variability between individual patients and fluctuations in symptoms over time means that measurements such as UPDRS fails to capture the variability of the condition with time, a critical component of the management of Parkinson’s.

Wednesday, September 30 – Thursday, October 1

  • Tom Isaacs (Co-founder and president, The Cure Parkinson’s Trust): Rallying to the Challenge II
  • Jon Stamford, Ph.D., D.Sc. (Scientific and advocate communication coordinator, The Cure Parkinson’s Trust): Clinical trials charter
  • Israel Robledo (Parkinson’s Movement Steering Committee member) and Richard Windle (Research Support Network Advocate, Parkinson’s UK): Experience to expertise
  • David Ashford-Jones (Owner, Sagio Ltd.; Parkinson’s advocate): Risk and benefit
  • Steve DeWitte (CEO, Connecticut Advocates for Parkinson’s) and Richard WindlePatient-centered research
  • Patrik Brundin, M.D., Ph.D.; Caroline Tanner, M.D., Ph.D.; and others: Panel discussion
  • Tom Isaacs: Outcomes and measures: How do Ralliers define “patient-centered outcome measures”? Is there the opportunity to introduce patient-centric change?
  • Dr. Jon Palfreman (Award-winning author, journalist and documentary film producer): Self monitoring in Parkinson’s: Who/what is it good for? Why is measurement important? Why are current measures flawed?
  • Dr. Jon StamfordCan we gain consensus on what should be measured? (includes survey result presentation and discussion)
  • Focus group: Parkinson’s symptoms – it’s personal: Investigating individual prioritized symptoms in terms of frequency, severity, individuality, impact on wellness, impact on other symptoms, “communicate-ability” about the symptom, measurability
  • Soania Mathur, M.D. – Introduction
  • Ken Kubota, Director of Data Science for Research Programs, Michael J. Fox Foundation for Parkinson’s Research: Fox Insight
  • Bruce Hellman, CEO, uMotifWhat questions can we ask of Big Data?
  • Walter Maetzler, M.D., Professor, Centre of Neurology and Hertie-Institute for Clinical Brain Research, University Hospital TuebingenPwPs+Clinicians+Technicians=How to measure
  • Anupam Pathak, Ph.D., Google
  • Bas Bloem, M.D., Ph.D., ParkinsonNet
  • Peter Schmidt, Ph.D., Chief information officer, vice president of research programs, National Parkinson Foundation—Parkinson’s Outcomes Project
  • Eli Pollard, Executive director, World Parkinson Coalition—World Parkinson Coalition’s exercise program in association with Brian Grant Foundation
  • Max Little, Ph.D.—Parkinson’s Voice Initiative
  • Caroline Tanner, M.D., Ph.D., Director of clinical research, The Parkinson’s Institute—Integrating patient-relevant measures into clinical trial design: The Apple app
  • Feedback presentation to Grand Challenges in Parkinson’s Disease: Applying the right measure to the right person in the right situation

The results of our 2015 Rallying survey formed the basis for the focused discussion at the meeting at Grand Rapids. This meeting looked closely at the variability of individual’s symptoms and how they can inform outcomes in clinical trials, where outcomes can be improved and how technology may be useful in developing new ways of measuring. The emphasis throughout was on improving the patient experience.

492 people with Parkinson’s (PwPs) completed our 2015 Rallying to the Challenge survey which explored which aspects of Parkinson’s are most important to individual PwPs with regards to quality of life and how these aspects can be measured.

407 respondents shared their top 5 symptoms which most impact on their quality of life. Lack of energy was the most reported individual symptom, with a massive 46% of respondents identifying it in the 5 symptoms that most impact their quality of life. Balance and falling (31%), slowness of movement (30%), tremor (28%), stiffness and rigidity (23%), speech issues (21%), pain (21%), urinary urgency (18%), memory issues (17%) and difficulty sleeping (16%) were also commonly symptoms that impact quality of life. No one person reported the same top 5 symptoms.

The results from the survey found that the majority of all respondents (83%) are interested in recording information about their Parkinson’s to monitor well-being, however currently only 36% of all respondents are currently doing so.

Further analysis of these results can be found in our article published in the Journal of Parkinson’s Disease.

2017

Drug repurposing
Rallying to the Challenge 2017: Drug repurposing 

People with Parkinson’s have a vital role in influencing research aimed at changing the course of the disease. That’s why Van Andel Institute and The Cure Parkinson’s Trust are thrilled to once again host Rallying to the Challenge, a patient-focused meeting designed to seek input and amplify the voices of people with Parkinson’s. This year’s meeting will tackle drug repurposing, access to novel treatments, clinical trials and more.

WEDNESDAY, Sept. 27, 2017

10:20 a.m.
Welcome
Steve DeWitte, Connecticut Advocates for Parkinson’s
Israel Robledo, Parkinson’s research advocate
Dr. Soania Mathur, Parkinson’s research advocate

10:25 a.m.
“Off-label” prescribing: Definitions, risks, the law and unintended consequences
David Jones, Parkinson’s research advocate, The Cure Parkinson’s Trust

10:40 a.m.
Distinguishing between hype and hope
Dr. Rachel Dolhun, The Michael J. Fox Foundation for Parkinson’s Research

11:10 a.m.
The challenges of repurposing old drugs: A clinician’s view
Prof. Tom Foltynie, University College London

11:40 a.m.
Patient access to novel treatments
Dr. Michele Tagliati, Cedars-Sinai
Dr. Joy Duffen, The Cure Parkinson’s Trust
Dr. Jon Stamford, The Cure Parkinson’s Trust

12:10 p.m.
Lunch

12:30 p.m.
Your questions answered
Over lunch question-and-answer session with Dr. Patrik Brundin and colleagues

1:15 p.m.
Presentation of the Tom Isaacs Award
NOTE: This session will take place in Tomatis Auditorium during the Grand Challenges in Parkinson’s Disease symposium

1:30 p.m.
Join Grand Challenges in Parkinson’s Disease for Exendin-4 treatment in Parkinson’s disease
Prof. Tom Foltynie, University College London
NOTE: This session will take place in Tomatis Auditorium during the Grand Challenges in Parkinson’s Disease symposium

2:00 p.m.
Is there a new way to run trials? If so, how can we run them effectively?
Dr. Peter Schmidt, Parkinson’s Foundation

2:30 p.m.
2017 Rallying survey findings
How to evaluate risk vs. benefit comparing findings from healthcare professionals to people with Parkinson’s
Leah Mursaleen, The Cure Parkinson’s Trust
Dr. Soania Mathur, Parkinson’s research advocate

3:00 p.m.
Break

3:20 p.m.
Focus groups

4:20 p.m.
Feedback from focus groups

5:00 p.m.
Day closes


THURSDAY, SEPT. 28, 2017

10:00 a.m.
From a researcher’s perspective, what can people living with Parkinson’s do to help research and clinical trials
Dr. Camille Carroll, University of Plymouth

10:30 a.m.
Disease-modifying trials in the PD pipeline: Highlighting funding, staffing and recruiting needs
Sue Buff, Parkinson’s research advocate
Gary Rafaloff, Parkinson’s research advocate

11:00 a.m.
Advocacy, engagement and making a difference: A discussion
Steve DeWitte, Connecticut Advocates for Parkinson’s
Israel Robledo, Parkinson’s research advocate
Dr. Soania Mathur, Parkinson’s research advocate
Leah Murasleen, The Cure Parkinson’s Trust
Helen Matthews, The Cure Parkinson’s Trust
David Jones, Parkinson’s research advocate, The Cure Parkinson’s Trust

12:00 p.m.
Lunch

12:15 p.m.
Panel discussion: All your questions answered (during lunch)
Dr. Patrik Brundin and colleagues

1:00 p.m.
Concluding comments

1:45 p.m.
Feedback presentation to Grand Challenges in Parkinson’s Disease
NOTE: This session will take place in Tomatis Auditorium during the Grand Challenges in Parkinson’s Disease symposium

Explore Rallying articles from The Cure Parkinson’s Trust below:

2018

Non-motor symptoms

2017

Drug repurposing

2016

Collecting data

2015

Symptoms

2014

Clinical trials
407 people with Parkinson’s shared the 5 symptoms which most impact on their quality of life in our 2015 Rallying to the Challenge survey. No one person reported the same 5 symptoms but grouped together, thinking and memory aspects were most commonly reported. That said, lack of energy (or fatigue) was the most reported individual symptom, with a massive 46% of respondents identifying it in their top 5 symptoms.

 

The majority of respondents were interested in recording information about their Parkinson’s to monitor well-being. Symptoms people wanted to monitor generally mirror those reported to be most bothersome, with a continuing emphasis on non movement aspects of Parkinson’s.

As a result of this survey, we have partnered with uMotif (a self-monitoring application) to collect information from individuals and create a shared symptom data resource for research purposes. This resource will help researcher further investigate the symptoms of Parkinson’s and how they differ between individuals.  If you are interested in recording information about your Parkinson’s, click here to read more about our study.

You can read the full survey results in our article published in the Journal of Parkinson’s Disease.

 

 

The free-text question in our Rallying 2015 survey which asked people with Parkinson’s to list five symptoms that most effect their quality of life highlighted the range and breadth of symptom description used by people with Parkinson’s. These symptom descriptors were further discussed at a focus group held at The Cure Parkinson’s Trust in August 2016 and together analysis of the survey results and focus group findings formed the basis of a recent article published in the Journal of Parkinsonism and Restless Legs Syndrome.

The paper provides insights into symptom complexity, patient interest and understanding, as well as possible communication barriers between the patient and the physician. It was found that patient descriptors were more extensive generally for nonmotor than motor symptoms, and that the terminology used to describe neuropsychiatric symptoms was particularly detailed with few clinical terms were used.

The results suggest that since many nonmotor symptoms are not visible and require interrogation by physicians and articulation by patients; these are areas of particular vulnerability in the patient–physician communication loop.

 

 

 

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