足S p e c i a l L i m i t e d E d i t i o n 2 0 1 3
To the hundreds of PPMI volunteers. Enclosed are letters from members of the Parkinsonâ€™s community thanking and honoring you for volunteering to participate in this critical research. It would not be possible without you. To view and share this book online, visit ppmi-info.org/participants/thankyou.
Special Limited Edition 2013
A Letter from Sohini Chowdhury
Dear Friend, Since the 2010 launch of The Parkinson’s Progression Markers Initiative (PPMI), hundreds of volunteers like you—people with and without Parkinson’s—have stepped up to play a personal role in the pursuit of Parkinson’s disease biomarkers. One clinic visit at a time, you are helping accelerate research that could transform millions of lives. The PPMI team and The Michael J. Fox Foundation (MJFF) have been overwhelmed by your willingness and enthusiasm to be part of this critical work. But we aren’t the only ones who have taken notice. Across the Parkinson’s community, patients, family members, caregivers and researchers have written letters to you, the PPMI volunteers, to thank you for your tremendous gift to Parkinson’s research. In this booklet we have compiled a small sampling of the letters we have received. We hope you will accept it as a token of gratitude from a worldwide community touched by your commitment and perseverance. Thank you for participating in PPMI. Sincerely,
Sohini Chowdhury Senior Vice President, Research Partnerships The Michael J. Fox Foundation
A Letter from the PPMI Steering Committee
Dear PPMI Participant, Thank you for all you are doing to make PPMI possible. Planning for this study began in 2007 and, while we successfully galvanized the expertise and funding to launch the study in 2010, this study would not be where it is today without you. As a result of your participation in PPMI, we are now amassing the largest data bank and biorepository in the history of Parkinson’s disease (PD) research. Scientists from around the world are accessing what we are building in PPMI, and it’s all thanks to you. In addition to the PD research community, others are taking notice of your efforts. Other disease states, media outlets, and the PD community are increasingly interested in PPMI’s structure and success and are eager to share in our lessons learned and scientific findings. When we launched PPMI in 2010, a PD research study of this size and scope had never even been attempted. All we could do next was open the clinic doors and wait to see if anyone would sign up to participate. Your response and willingness to contribute your time and energy have been and continue to be nothing short of amazing. Together, we are truly making PD research history. Thank you for being a part of PPMI.
PPMI Principal Investigator Ken Marek, MD and the PPMI Steering Committee 3
A Letter from Michael J. Fox
Dear Friend, I wanted to personally thank you for participating in PPMI. You have my deepest gratitude for being part of our mission to speed breakthrough treatments for Parkinson’s disease. I’m grateful you’re here because, if there’s one thing I know for sure, it’s that better treatments aren’t going to fall from the sky. Real challenges stand in the way of the results we need and it’s up to all of us to get involved and meet those challenges however we can. By participating in PPMI, you are doing just that. At the Foundation, we don’t like to pat ourselves on the back. But I hope you’ll give yourself one for your enormous contribution to this effort. The thoughtful engagement of people like you is the only thing that can help meet a major need for a Parkinson’s biomarker. Today, you are part of the solution. Thank you again for being part of something that could change everything for the five million Parkinson’s patients worldwide. All my best,
Michael J. Fox
Letters from the PD Community Dear PPMI Participant, As someone who loves and cares for a person who has Parkinson’s disease, I want to thank you for this incredible gift you’re giving and the sacrifice you’re making for the sake of PD research. Your generosity and courage to commit to a fiveyear study are amazing and inspiring. If it ever seems like too much, know that your continued participation will benefit the millions of people with Parkinson’s around the world—and the people who love them. Without people like you to step up and participate, new discoveries could never transform into real medicines and treatments. Thank you for joining us in the pursuit of better treatments and a cure. Collectively, we become a force of change—and together we really can make a difference! Best,
Robin Katsaros Caregiver and Loved One of a Person with Parkinson’s
Dear PPMI Participant, First, let me say, as a study coordinator for PPMI at Northwestern University, seeing each PPMI participant come in for their visits is one of the best parts of my job. It’s inspiring to see how willing you are to give of yourself for the benefit of others in the pursuit of a biomarker. Working toward identifying a biomarker is essential to the future of Parkinson’s research. It could have the potential to help doctors diagnose the disease sooner and eventually generate new treatments that may be used even before motor symptoms present themselves. Your involvement in this study is crucial to its success. As a coordinator, I consider it my job to make sure that you have everything you need to stick with the study. We are all in this together and the potential for what we could learn from so many years of data and specimens being generated in PPMI is staggering. What you’re doing as a participant in PPMI is nothing short of extraordinary—and the field of Parkinson’s research will be eternally grateful. Thank you.
Karen Williams Senior Clinical Research Coordinator, Northwestern University PPMI Site Coordinator
Dear PPMI Participant, Thank you so much for being a part of PPMI. I have benefited so much from the outpouring of support from my family, friends and co-workers, clients, etc., since being diagnosed with PD. I am inspired by the people who have been willing to become PPMI participants. I am so grateful to all of you.
Richard Person with Parkinson’s
“If it ever seems like too much, know that your continued participation will benefit the millions of people with Parkinson’s around the world— and the people who love them.” Robin Katsaros Caregiver and Loved One of a Person with Parkinson’s 8
Dear PPMI Participant, I have worked as a scientist studying Parkinson’s disease for almost 15 years. In that time I’ve seen great progress. I’ve also been privileged to meet many people affected by Parkinson’s. I see a common thread among those who live with this disease: a need to understand, a drive to see a cure, and always, a desire to help speed progress. There are many ways to help: donating money, becoming part of a support group, telling others about your journey or, of course, taking part in a research study. The Parkinson’s Progression Markers Initiative is one such study, and one that I believe will be critical to our success in treating this disease. PPMI aims to help identify markers that will allow us to track the progression of this illness in an unbiased way. This is important for many reasons, but perhaps most crucial is its potential impact on treatment trials. Today’s trials take a long time and cost enormous sums of money. If we can find a marker that indicates progression in a simple and rapid manner we can reduce the cost of such trials, reduce the time taken to complete them, and likely entice drug companies to invest more time and effort in Parkinson’s disease trials. The work of PPMI is only possible because we are able to follow the same subjects month after month, year after year. Without this incredible commitment on your part, it just wouldn’t work. I have seen the data coming from PPMI, and I know that you are donating a lot of time and effort to this study. So thank you. I am genuinely grateful for your help.
Andrew Siderowf, MD, MSCE PPMI Scientific Advisory Board
Dear PPMI Participant, When the three of us came together in 2010 as a team for PPMI, we had no idea how our recruitment of study volunteers would go. What we did know was that we faced some daunting hurdles. Would we be able to convince anyone to take part in a five-year study including multiple lumbar punctures? In January of 2011, we got our answer. We were contacted by a woman whose husband had Parkinson’s disease. He was unable to participate in the study, but she offered to do so. Just like that, we had our first study participant, and the rest is history. Many PPMI volunteers we work with have said that their participation is a way to take control of their disease. Some do it to help future generations and contribute whatever they can to make a difference in the search for a Parkinson’s cure. Many of those without PD have a personal connection to the disease in the form of a pastor, a grandmother, a husband, a wife. While PPMI volunteers have joined for a variety of reasons, you all have one thing in common—you truly astound us with your generosity. You give us your invaluable time and equally invaluable biosamples to make this study possible. We ask you to give, give, give and you always oblige. None of this remarkable endeavor would be possible without you. To our many volunteers, please know that we are amazed by your altruism and have benefited from getting to know each and every one of you. You are laying the groundwork for a cure to Parkinson’s to be found. Thank you! Sincerely,
Irene Richard, MD Professor, Department of Neurology, University of Rochester PPMI Site Principal Investigator | MJFF Scientific Advisory Board
Cheryl Deeley, MS, RNc Nurse Programmer, University of Rochester PPMI Site Coordinator
Courtney Bishop, MS, MPH Clinical Coordinator, University of Rochester PPMI Site Coordinator 10
Dear PPMI Participant, Thank you for your wonderful contribution to Parkinson’s research. As a person with Parkinson’s, I have hope for the future because perhaps this trial will lead to significant results that could benefit millions. Thanks to your generosity, we will learn from this research, whatever the outcome. Please know that your volunteerism is greatly appreciated!
Al Person with Parkinson’s
“I see a common thread among those who live with this disease: a need to understand, a drive to see a cure, and always, a desire to help speed progress.” Andrew Siderowf, MD, MSCE PPMI Scientific Advisory Board 11
Dear PPMI Participant, On behalf of my loved ones and friends who wish I did not have Parkinson’s disease, and on my own behalf, thank you! I cannot express my gratitude strongly enough for your empathy, compassion and dedication in the search for the answer to all the “hows” and “whys” of Parkinson’s disease. Life with PD is not always easy and if it weren’t for people like you, it would be even harder. You show the best of human nature—the common search for everyone’s wellness and happiness. Thank you again.
Claudia Revilla Person with Young-Onset Parkinson’s
“What you’re doing as a participant in PPMI is nothing short of extraordinary—and the field of Parkinson’s research will be eternally grateful. Thank you.” Karen Williams Senior Clinical Research Coordinator Northwestern University PPMI Site Coordinator 12
Dear PPMI Participant, I am a basic scientist in whose laboratory a molecule was discovered that is currently being studied in a PD clinical trial. In the long process of therapeutic development, my team and I did the easy part. The hard part is in the translational and clinical studies that define how to effectively and safely use the molecule we discovered, and to determine if it truly modifies the course of the disease and improves the quality of life of PD patients. Clinicians and scientists cannot accomplish these latter objectives without the active collaboration of PD patients and, in the case of PPMI, individuals who provide the critical basis of comparison. PPMI is a landmark study that will facilitate the difficult process of therapeutic development. Being a participant in a study such as PPMI is hard. I appreciate this from personal experience, as I have been a research participant in a similar study (developing biomarkers for prostate cancer). I have experienced the inconvenience, the hassle and the discomfort that being a participant in a longitudinal study entails. I have the privilege of serving as the Chair of the Biospecimen Review Committee of PPMI. Our committee reviews applications from scientists to gain access to the biospecimens (blood, CSF, etc.) that you generously provide at your PPMI visits. We take this responsibility very seriously and work hard to make sure, by many criteria, that these samples are used in a way that is highly likely to provide information that will advance the mission of PPMI. I want to personally thank every PPMI participant for what you have done and will do in this noble effort. Sincerely,
Gene Johnson, PhD Professor, Washington University School of Medicine Chief Scientific Advisor, MJFF Scientific Advisory Board Chair, PPMI Biospecimen Review Committee
Dear PPMI Participant, I am a 58-year-old male who has had PD for 8 years. When I first heard of the overwhelming willingness of volunteers to enroll in PPMI, I was blown away. I have worked in the pharmaceutical industry for over 30 years now. I am very familiar with the difficulties there are in recruitment and enrollment for a study that requires such a time commitment. Personally, my fight against PD has been energized by your willingness to help us all. As we learn more about this disease, we increase our chances of eradicating it. Just imagine a world without PD. Thank you for your contribution to this study and to PD research as a whole.
Tom Picone, PhD Person with Parkinsonâ€™s MJFF Patient Council
Dear PPMI Participant, Thank you so much for your commitment to PPMI. Participating in a clinical trial is a selfless action that few people take, but one that is crucial for developing new treatments (and a cure!) for all diseases, including Parkinson’s. While I didn’t have a personal connection to Parkinson’s disease before working at the Foundation, I have met and come to know many inspiring people in the PD community since being here. Though I probably have not met you, your participation in PPMI instantly proves that you are one of these inspiring people. By participating in PPMI, you are making an invaluable contribution to the lives of five million worldwide living with Parkinson’s and the roughly 30 million more who love and support them. Thank you for this enormous, incredible and unique gift and for being someone who is willing to take action in this effort to cure Parkinson’s.
Elizabeth Joyce Marketing and Communications Officer The Michael J. Fox Foundation
Dear PPMI Participant, As a physician, wife, and mother of three young daughters, I’ve now lived with the challenge of PD for over 14 years. This disease has affected every aspect of my life and that of my family, as well. Despite its progression, I’ve learned to live as well as possible and with optimism for the future. I believe that there will ultimately be a cure, but I fear that this goal cannot be reached expediently unless we all do whatever we can to increase participation in the clinical trials that will get us there. PPMI is one such trial. The results of PPMI will serve to increase our understanding of this disease and find that elusive biomarker, which I believe is key to developing new treatments. Not to mention the other research that shared PPMI data will help to move forward. It’s always shocking to me when I hear the statistics about clinical trials—how 85% are delayed in their completion due to recruitment issues and a shocking 30% of clinical trials fail to recruit a single participant. What a huge waste of resources and more importantly, time. Time with this life-altering disease, time without improved treatments and the cure we desperately need. More time struggling with daily disability, more time not living to our full potential, more time living with the burden of our illness. But there’s hope. Better treatments and even a cure are in our future. It’s not a matter of if but when. And we will reach that goal because of people like you. I know that participation in a landmark trial such as PPMI requires selfless commitment on your part. You are giving of your time, energy and literally yourselves! But through your dedication to participate in PPMI, the day will come when we will no longer have to face this challenge and when those that follow will not have to embark on this journey in the first place. You are playing a huge role in moving us closer to that day and are part of something that has the potential to make a life-changing difference for all PD patients and their loved ones. Please accept my heartfelt gratitude and admiration.
Soania Mathur, MD 16
Person with Young-Onset Parkinson’s MJFF Patient Council
Dear PPMI Participant, Please accept my whole-hearted thanks! This is likely to be one of the most important studies done in Parkinson’s disease research in this decade. We can’t do it without you. Many thanks for your ongoing contribution to this landmark study.
Holly Shill, MD Director, Sun Health Research Institute PPMI Site Principal Investigator
“When I think about the thousands of samples and all of the data pouring into the PPMI database already, I am excited about the possibilities for PD research.” Claire Meunier Director, Clinical Trial Strategies The Michael J. Fox Foundation 17
Dear PPMI Participant, As a member of The Michael J. Fox Foundationâ€™s PPMI team, I am integrally involved in the day-to-day management of the study. In my role, I also get a seat at the table with all of the researchers, logistics managers, coordinators and industry partners who are all collaborating on PPMI. I am constantly on the phone, in meetings and emailing back and forth about the many details and decisions that need to be made to continue to make progress toward finding a biomarker for PD. However, despite the many of us behind the scenes who are working tirelessly to make the study happen, our efforts would be for naught if it werenâ€™t for you. I remain in awe of each of you who give up time and energy (and blood and CSF!) to make all of this possible. When I think about the thousands of samples and all of the data pouring into the PPMI database already, I am excited about the possibilities for PD research. Thank you for everything you are contributing to PPMI. On behalf of all us working on this study, I hope you know how much we appreciate everything you are doing to make this work possible.
Claire Meunier Director, Clinical Trial Strategies The Michael J. Fox Foundation
“But there’s hope. Better treatments and even a cure are in our future. It’s not a matter of if but when. And we will reach that goal because of people like you.” Soania Mathur, MD
Person with Young-Onset Parkinson’s MJFF Patient Council
Dear PPMI Participant, Imagine being 47 years old, a physician and surgeon with three teenaged kids and being told that you have Parkinson’s disease. I am that person, and since that scary day over four years ago, I have hoped for a cure. And although you do not know me, your courageous and extremely generous enrollment in PPMI has me more than hopeful that a cure will be found. While I was not able to be in the study myself, my husband, brother-in-law, and several friends stepped up to the plate just like you. As a physician, I know that this study has an important task because finding a true biomarker will change the face of PD research and drug development. And yes, maybe even help find a cure. So, no matter how much money is raised or how many clinicians work together, no progress would be possible without the time, energy and enormous contributions (urine, blood, and spinal fluid!) made by you and the other brave individuals who make up this study subject population. I am very proud to tell you that I am a member of MJFF’s Patient Council and as such I get a firsthand account and assessment of where PPMI stands. I am one of many who are too young to have this disease. I know I speak for all of us when I say, “You are our hero.” Thanks so much. Sincerely,
Karen Jaffe, MD Person with Young-Onset Parkinson’s MJFF Patient Council
Dear PPMI Participants, Your dedication to this new frontier of PD research is invaluable. The time and effort you commit to the completion of this trial will pay off as the worldwide community of Parkinson’s disease researchers access this rock-solid, well-controlled data set. This unprecedented effort would not have been possible without the commitment of each individual. As a researcher and as a member of MJFF’s Scientific Advisory Board, I would like to thank you personally and assure you that the research community, starting with my research unit focusing on neurodegenerative diseases, will build upon your efforts to define the natural course of the disease and hopefully identify reliable diagnostic and prognostic biomarkers. With my most sincere thanks,
Erwan Bezard, PhD INSERM Research Director Member, MJFF Scientific Advisory Board
“I know I speak for all of us when I say, You are our hero.” Karen Jaffe, MD Person with Young-Onset Parkinson’s MJFF Patient Council 21
Dear PPMI Participant, What an astounding commitment you have undertaken. The five years of extensive testing and assessments for which you have volunteered are nothing short of heroic. Your extraordinary efforts will lead us toward one of the most critical needs in PD research today—identifying biomarkers—and are an enormous step in getting us closer to our ultimate goal of finding a cure. Thanks so very much.
Kathleen Vestuto Family Member of a Person with Parkinson’s Meeting Coordinator The Michael J. Fox Foundation
“To our many volunteers, ple amazed by your altruism an getting to know each and ev laying the groundwork for a found. Thank you!”
Irene Richard, MD
Cheryl Deeley, MS, RNc
Professor, Department of Neurology, University of Rochester PPMI Site Principal Investigator MJFF Scientific Advisory Board
Nurse Programmer, University of Rochester PPMI Site Coordinator
Dear PPMI Participant, I just wanted to write a quick note to say thank you for participating in PPMI. It means a lot to patients like me to have your help in advancing research on this terrible disease. I was diagnosed formally six years ago at age 36 and am hoping this study helps lead to better treatments or a cure within my lifetime. Thank you and God bless,
Bill Person with Young-Onset Parkinsonâ€™s
ease know that we are nd have benefited from very one of you. You are a cure to Parkinsonâ€™s to be Courtney Bishop, MS, MPH Clinical Coordinator, University of Rochester PPMI Site Coordinator
“This is likely to be one of the most important studies done in Parkinson’s disease research in this decade. We can’t do it without you.” Holly Shill, MD Director, Sun Health Research Institute PPMI Site Principal Investigator
Dear PPMI Participant, I just wanted to take a brief moment to personally thank you for your participation in the PPMI study. Iâ€™m sure you are already aware of this, but important research such as this could not move forward without the efforts of individuals like yourself. Unfortunately, it is not enough to just sign up for the study at the beginning. As a statistician, I have had numerous experiences where great ideas and studies have been derailed by a large amount of missing data. Efforts like PPMI are much more successful when a large number of individuals complete all of the study assessments. Think of it this way: at the end of the study when we want to ask important questions, it would be much more difficult to find the answers if there are holes in the data. For that reason, I want to stress the importance to you of remaining in the study and participating in as many scheduled visits as possible during the remaining years of PPMI. I fully understand that I cannot put myself in your shoes. Each one of you has a different set of unique circumstances that led to your participation in the study. The time that you are giving to participate in this study is a valuable gift. I can assure you that, on behalf of the PPMI investigators, we will work diligently to make sure that your gift is utilized to the fullest. Sincerely,
Christopher Coffey, PhD Director, Statistical and Data Management Center, University of Iowa Chair, PPMI Statistics Working Group
Dear PPMI Participant, I would like to thank you for participating in PPMI. I know this is no small effort on your part. As a person living with PD, I know how important it is to find biomarkers. Without your contribution, this would be very difficult. Because of information researchers are learning now through PPMI, I have hope for my future, and hope for all people with PD. Since my diagnosis 10 years ago I have done my best to talk about PD and advocate for more participation in clinical trials. I have participated in over 10 trials myself, and Iâ€™m looking for my next one. So, I have at least an idea of what your commitment to the PPMI study entails. I thank you from the bottom of my heart for your participation and all that you are doing for people with PD.
Jean Burns Person with Parkinsonâ€™s PPMI Patient Committee
Dear Friend, I know I can call you thatâ€”my friend. Because of what you have done for the whole PD community: patients, physicians, researchers, family members and friends. The choice you made to be a participant in PPMI, a courageous and selfless step, is going to prove invaluable in the quest to cure the disease I have had for almost 15 years. I have been lucky in that my version of PD is progressing slowly. I can walk, exercise, take care of most of the house chores and even went skiing recently, though I kept to the easier slopes. But I do know that it progresses and will be progressing for the duration of the disease. I also know that I have a gene responsible for my PD, that my two sons inherited it, and so might my grandchildren. And that increases their chances of developing PD themselves. Finding biomarkers for the disease will help diagnose it early, predict its progression and, eventually, find the cure. I do believe that. That is why I cannot thank you enough for what you have done for this study. And I wish you all great health.
Genia Brin Person with Parkinsonâ€™s MJFF Patient Council
Dear PPMI Participant, Thank you! PPMI is one of the most ambitious projects we have ever done and one of the most critical. Finding a biomarker that could track the progression of Parkinson’s disease would make testing new drugs cheaper, easier and more informative than is possible today. Which means getting treatments faster to the people who need them. As a staff scientist at MJFF for more than eight years, I’m always amazed at not only the dedication of the Foundation where I work, but the seemingly unlimited energy and hope that people with Parkinson’s bring to the cause every day. It’s a long battle, but as I sit in the trenches with all of you, I see a victory ahead that is inevitable. Thanks again.
Brian Fiske, PhD Vice President, Research Programs The Michael J. Fox Foundation
“Personally, my fight against PD has been energized by your willingness to help us all. As we learn more about this disease, we increase our chances of eradicating it. Just imagine a world without PD.” Tom Picone, PhD Person with Parkinson’s MJFF Patient Council
Dear PPMI Participant, As a researcher, and both the son of a father and the student of a mentor who both had PD, I am dedicated to finding a cure for PD in our lifetime. PPMI is so important for giving the research community access to the most valuable biologic reagents for better diagnosing and treating PD. My own work focuses on studying new biomarkers and therapeutic targets that circulate in cerebrospinal fluid and blood, and the PPMI samples will allow our work to move forward much more quickly and with the highest level of precision. Discovering a biomarker for the progression of PD will not only represent ways we can diagnose earlier and monitor disease course, but, even more exciting, will help discover new therapies and potentially a cure for PD. Thank you all so much for making this huge gift to this wonderful program that is already bearing fruit for new treatments and hopefully a near-future cure for Parkinsonâ€™s.
Dennis Steindler, PhD Professor of Medical Research University of Florida College of Medicine
Dear PPMI Participant, My husband of 34 years was diagnosed 16 years ago with Parkinson’s disease. While the first few years with PD were relatively easy, the last few have presented new challenges. It is hard to put into words how much we thank those who have volunteered for clinical trials. Identifying biomarkers is vital work to bring us all closer to a cure for Parkinson’s. Again, we thank you for volunteering your time so that our family has a chance for more time together. YOU ARE OUR HERO!
Betty Caregiver and Loved One of a Person with Parkinson’s
â€œYour response and willingness to contribute your time and energy have been and continue to be nothing short of amazing. Together, we are truly making PD research history. Thank you for being a part of PPMI.â€? The PPMI Steering Committee
About the Parkinson’s Progression Markers Initiative The Parkinson’s Progression Markers Initiative is a landmark observational clinical study to better understand the progression of Parkinson’s disease. The goal of PPMI is to identify and assess biomarkers, or indicators, of Parkinson’s disease. The discovery of a PD biomarker is critical to the development of new and better treatments for PD, particularly treatments that could slow or stop the progression of the disease, something no currently available treatment can do. PPMI is the first clinical study to assemble a population large enough in size to draw meaningful scientific conclusions over time to develop better ways to measure the progression of Parkinson’s disease. PPMI is a worldwide effort, with 32 study sites across the United States, Europe, Australia, and the Middle East. It is sponsored byThe Michael J. Fox Foundation for Parkinson’s Research.
This book is dedicated to the hundreds of volunteers participating in PPMI. This critical research would not be possible without you. To view and share this book online, visit ppmi-info.org/participants/thankyou.