Dr. Alberto Espay is professor and endowed chair of the University of Cincinnati James J. and Joan A. Gardner Family Center for Parkinson’s Disease and Movement Disorders. He trained in neurology at Indiana University as well as in clinical and electrophysiology of movement disorders at the University of Toronto, where he obtained a master’s degree in clinical epidemiology and healthcare research.

Dr. Espay served as chair of the Movement Disorders Section of the American Academy of Neurology; associate editor of Movement Disorders, the official journal of the International Parkinson and Movement Disorder Society (MDS); and in the executive committee of the Parkinson Study Group. He currently serves as chair of the MDS Task Force on Technology and as secretary-elect of the MDS Pan-American Section. Dr. Espay is also an honorary member of the Mexican Academy of Neurology and has received numerous awards, including the Cincinnati Business Courier’s Health Care Hero award, the Patients’ Choice award, the Compassionate Doctor award, and the Spanish Society of Neurology’s Cotzias award.

Dr. Espay has published more than 200 peer-reviewed research articles, 25 book chapters and five books. His research efforts have focused on the measurement of motor and behavioral phenomena in—and clinical trials for—Parkinson’s disease as well as in the understanding and management of functional movement disorders. With his colleagues at the Gardner Center, he recently launched a novel biomarker development program for neurodegenerative diseases with the aim of identifying small but biologically suitable subgroups most likely to respond to therapies already available for repurposing.

Megan Feeney, M.P.H. is Senior Manager, Community Engagement at the Parkinson’s Foundation. In this role, she manages several national patient leadership programs that engage the community in addressing critical unmet needs in Parkinson's. She manages the Women and PD Initiative, an innovative program that addresses long-standing gender disparities in Parkinson's. Within the Women and PD Initiative, her team received a $250,000 Patient-Centered Outcomes Research Institute (PCORI) Engagement Award titled, Women and PD Teams to Advance Learning and Knowledge (Women and PD TALK). Throughout her work, Megan collaborates closely with people with Parkinson’s, care partners, allied health professionals, researchers and industry representatives to promote patient engagement and involvement in Parkinson’s research and care. She received a Bachelor of Arts from the University of California, Los Angeles and a Master of Public Health from the New York University, School of Global Public Health.

Jessica Shurer is the Center Coordinator and Clinical Social Worker of the Movement Disorders Center at the University of North Carolina at Chapel Hill, a Parkinson’s Foundation Center of Excellence and a CurePSP Center of Care. She graduated from Penn State University with a bachelor’s in psychology and minors in Human Development & Family Studies and Gerontology. She received her Master of Social Work from UNC Chapel Hill in 2012, where she also obtained a Certificate in Aging and was a participant in the Hartford Partnership Program for Aging Education. In her current role, Ms. Shurer works as a team with the movement disorders specialists to address patient and care partner psychosocial needs, mood and coping, as well as offers short-term problem-solving counseling and connection to community resources. In addition, Ms. Shurer works to grow a referral network of Parkinson’s-specialized rehabilitation clinicians across the state, coordinates two specialty interdisciplinary clinics, one for Parkinson’s and one for atypical Parkinsonism disorders, facilitates both the Chapel Hill Parkinson’s support group and a PSP & CBD support group, and organizes educational programs for patients and care partners.

Dan Keller 
Welcome to the second episode of substantial matters, life and science of Parkinson's. I'm your host, Dan Keller. At the Parkinson's Foundation, we want all people with Parkinson's and their families to get the care and support they need. Better care starts with better research and leads to better lives. In this podcast series, we highlight the fruits of that research, the treatments and techniques that can help you live a better life now, as well as research that can bring a better tomorrow.

Dan Keller

Today, we're speaking with Peter Schmidt, PhD, Senior Vice President and Chief Mission Officer of the National Parkinson Foundation, a division of the Parkinson's Foundation. He was interviewed at the World Parkinson Congress last September by Lotta Levy, our Corporate Relations Manager. They discussed improving standards of care for people with Parkinson's and where to go for great interdisciplinary care. Key to those discussions are the Parkinson's Foundation Centers of Excellence and Parkinson's Outcomes Project. Currently, 42 Parkinson's Foundation Centers of Excellence deliver care to about 110,000 people with Parkinson's in the United States and around the world. These top tier Parkinson's centers comprise an international network where dedication to patient care is paired with groundbreaking research. Leading the way in research, the Parkinson's Outcomes Project is the largest ever clinical study of Parkinson's disease. Nearly 10,000 patients from four countries are enrolled, along with almost 6000 caregivers, leading to new discoveries and best practices in care that will change the course of Parkinson's. Lotta first asked Dr Schmidt to explain the vision and focus of the Parkinson's Foundation.

Dr. Peter Schmidt
So when we talk about, what is our vision, what are we trying to do with the work that we focus on? We have a center of excellence program where we evaluate the structure in which care is provided. We have our Parkinson's Outcomes Project, where we look at what patients are being provided in their care and what outcomes are being achieved through that process. And our goal with this is to every day, every month, every year, measure the things that are occurring in the context of Parkinson's care, look for the things that are delivering the best outcomes, and push our centers to shift their care to the things that achieve the greatest good for their population. And as, over time, we introduce new therapies, the things that work for patients are going to change, and we need to continuously pursue and continuously optimize the care patients receive so that they're getting the things that make a difference in their lives. With a process of continuously measuring and improving care, I know that we can make a difference, and I am reminded of the John F Kennedy line when he was talking about the Apollo program and launching the Apollo program, where he said, we choose to do these things, not because they are easy, but because they are hard. These are hard problems. By facing them and fighting to achieve the goals that are possible with our capabilities, we can make a difference for people. And it may not be easy, but it is worth doing. Two years ago, we hired a great neuroscientist who understood the treatment of neurological disorders but was not a Parkinson's specialist, and we said to him, talk to the neurologist in our community, and let us think about what is the science that's going to drive forward the best benefit to patients. And he came back to us and he said, Pete, there is a false dichotomy between care and cure. The things that we can do to deliver better outcomes to our patients are going to help them, and they're going to help scientists to develop new therapies and get them through the process of proving that they work and distributing them into the community. If we can treat patients in the ways that can most effectively manage their disease, then the first thing that we do is we give them a great quality of life, but the second thing that we do is we make clinical trials better. It is easier to find patients who are well managed and who can be included in a clinical trial and will have low variability with other similar patients, so that when we look at the effect of a new drug or a new therapy or a new way of organizing a clinic, that we can see that effect faster and with fewer patients, driving our goal of lower cost and faster access for everyone with the disease. So my team and I all believe that the things that we do to help people live a better life with Parkinson's today are going to pay dividends for those people now, and are going to pay dividends down the road in terms of faster insight into how this disease can be effectively treated.

Lotta Levy
So with your Parkinson's Outcomes Project, what is it that you hope to learn, and how are you achieving success in that?

Dr. Peter Schmidt
So what we're looking at now is, what are the aspects of care that make a difference in patients' lives? What are the things that drive the greatest capabilities to improve the status of people with the disease? One of our early findings, we've all looked at exercise and physical therapy and occupational therapy and speech therapy and all these different modalities of care, and we know that those things are making a difference in patients' lives, but the fundamental feature of treatment that enables all the rest of those things is great medication, getting patients to a neurologist who knows the way to manage the symptoms of the disease. The whole interdisciplinary team model of care for Parkinson's stemmed out of the introduction of levodopa, the introduction of a medication that can effectively replace the dopamine that patients lose when they have this disease. Prior to that, exercise was not enough. We really couldn't effectively manage the disease. With adequate dopamine replacement therapy, we can take the other modalities of care and use them to make a difference and to help patients to live a better life and to prevent falls. So our first focus in this is to figure out what is optimal medication, what are the things that change patients' life and can enable them to engage in the other activities that are going to ensure that they're more mobile and able to engage in the activities that they enjoy. But the fundamental feature of this is getting patients the medications that are going to help them replace the lost dopamine.

Lotta Levy
Based on what you've gathered so far, what kind of places can patients go that deliver that level of care?

Dr. Peter Schmidt
So we wish that patients could get that level of care in any setting, but in today's healthcare system, the best place to go is a center that's got a academic movement disorder neurologist who practices medicine in the context of a great interdisciplinary team. And that team may not be all co resident in the same clinic, but can be dispersed across the system. If for patients who have to drive an hour or two hours to see their neurologist, a great neurologist will know who to send them to in their own community, who can help them with their disease. But the fundamental feature is the first thing that you need to find is a neurologist who truly understands Parkinson's disease, and those neurologists are movement disorder specialists.

Lotta Levy
So what is the most interesting thing that you've seen in the data so far?

Dr. Peter Schmidt
So I think there are three really interesting things we've been able to discern from the data so far. The first one is that by looking at the patients in our cohort, we've been able to construct a delayed start trial of exercise. The delayed start trial is the way that people show whether or not a treatment will change the course of the disease, change the way a patient experiences the disease. Now we're not looking at the biology of the disease, we're looking at how patients experience their lives with it, but at the end of the day, if patients are happier and feeling better and experiencing their symptoms less, we've made a change that makes a difference in people's lives. So what we found is we were able to identify patients who, at baseline, did not exercise, and who by the time two years had gone by had started to exercise. And then we split those into two groups, the people who started to exercise in their first year in the study, and the people who started to exercise in their second year, and we were able to show that the earlier patients started exercising, the greater the benefit that they were able to achieve, and the longer it lasted. The insight from that is it's not about early in the disease course, it's about early from now. If you're not exercising, start exercising now. That's where the difference comes. Don't wait. So that's the first really interesting thing that we've been able to do in the database. A second thing we've been able to do, we've been able to basically construct a trial of a medication. So we've done this with a drug called amantadine very recently. Amantadine is to treat dyskinesia, and we are able to look at patients who did not take amantadine and did not experience dyskinesia, who then started to experience dyskinesia and were treated with amantadine, versus patients who did not. And we were able to show that the patients who experienced dyskinesia and were treated with amantadine had a significantly higher probability of a reduction in their dyskinesia, showing us that that drug appeared to be effective. Now this is the gold standard for a clinical trial, is a randomized clinical trial with blinded raters so nobody knows who's getting the drug and who's getting the placebo. But those things can take years and can take an enormous amount of money. We were able to create this analysis in an afternoon, and so the fact that we were able to draw this insight in such a short time and in such a compelling way can help us to jumpstart, it can help us to screen out those clinical trials that we might initiate and might wait 18 months or two years to get an answer from. If we could say I believe that this is going to work or I believe this thing is not going to work based on the data that we've got, then we can say to people don't spend your time or money or effort on this trial, let's look at something else that might really make a difference. So the data that we've collected on amantadine has said to us we should be looking at this. This does seem to be a powerful treatment for dyskinesia. And the third thing we've looked at is psychosis. So one of the things that we try to identify are what are the factors that have the greatest impact on quality of life in patients with Parkinson's, and we've identified a couple of things. If you suffer from depression, that has a very strong impact on quality of life, or if you've been hospitalized, that has very strong impact on quality of life. Both depression and hospitalization affect a large number of people. A much smaller number of people in any given year experience psychosis, but when they do, it has a much more dramatic impact than either depression or hospitalization. So although it is less common, it is much more dramatic in its impact. And so we've been spending some time looking at psychosis and looking at the treatment of psychosis and what works and what doesn't work, and we found that in many cases psychosis may be caused by medication, and by changing medication that's able to address the psychosis. But in some cases, patients experience psychosis because of changes in their physiology because of the disease, and in those cases we've been able to show that often the use of an antipsychotic drug can make a difference in their lives. These are all things that for the most part were not well understood. So if you look at trials of antipsychotic drugs, it always starts with the patient having psychosis. But because we've got this very large data set where we followed patients for many years, we're able to back up and look at those patients at the beginning of the problems. How were they doing before the neurologist even identified that they were experiencing psychosis? So we've been able to look at that and look at how that condition develops over time, and be able to get greater insight into what can be done early in the course, what can be done at the very first signs that somebody is experiencing a challenge of a hallucination or confusion, and what are the things that we can do to help address that. And I think that we're going to be able to provide recommendations to clinicians that will help them to address those things.

Lotta Levy
So it sounds like you have done a large volume of analysis already. Do you feel like you're going to have a point in which you want to make this data available to qualified researchers and let them do their own analysis?

Dr. Peter Schmidt
Probably the majority of the analyzes we've done have been ones that have been asked of us by researchers out in the community, so we have to date never told anyone no, you may not have access to the data, and it may not have an analysis from it. This is designed to be a resource for the community. In our grant making, we think of the funds that we're able to provide in issuing a grant as a resource that we use to help improve care by funding research that's going to change the way care is provided. Similarly, we can provide data to be a resource that researchers can use to better understand Parkinson's. We've created infrastructure around this database that can then help other people to very quickly identify issues that they can treat or to plan trials that they can do to improve care.

Lotta Levy
Thank you so much.

Dan Keller 
I hope you are excited about the research Dr Peter Schmidt described, and that you will start to put together your interdisciplinary care team. For help finding expert care near you or to get involved in a research study, call our helpline. I'll give you the number in a minute, and if you want to leave feedback or comment on this podcast or any other subject, you can do it at parkinson.org/feedback. We will respond to some questions in future episodes. At the Parkinson's Foundation, our mission is to help every person diagnosed with Parkinson's live the best possible life. To that end, we'll be bringing you a new episode in this podcast series twice a month. Coming up in our next episode will be a conversation with Dr Boss Bloom, explaining why exercise is so valuable for people with Parkinson's. Till then, for more information and resources, visit parkinson.org or call our toll free helpline at 1 804 4PD Info, that's 1-800-473-4636. Thank you for listening.

Peter Schmidt, PhD is Senior Vice President, Chief Research & Clinical Officer at the Parkinson’s Foundation and oversees research, education, and outreach initiatives. Dr. Schmidt leads the Parkinson’s Outcomes Project, the largest clinical study ever conducted in Parkinson’s disease, and is active in research on diverse clinical areas in Parkinson's.

Schmidt serves as an advisor to several government, industry, and foundation initiatives. His focus has been setting clinical standards, and he is involved in several national-scale quality initiatives, including the US National Quality Forum and the Fresco Network in Italy. Schmidt is bringing to Parkinson's ideas from across health care and is on advisory committees for projects in wearable sensors, Huntington’s disease, Glut1 deficiency syndrome, cystic fibrosis, inpatient rehabilitation, and telemedicine. His work has been covered widely in the press and he has contributed to AHRQ and Commonwealth Fund publications; he has been an invited speaker for the NIH, and internationally at patient and professional conferences.

Schmidt joined the Parkinson’s Foundation from Cronus Partners, an investment bank, where he focused on health care innovation. Previously, Schmidt created chronic disease management systems and served as Chief Operating Officer of a joint venture of Oxford, Stanford, and Yale delivering on-line education.

Schmidt earned his bachelor's degree at Harvard University and was awarded an MS and PhD from Cornell University, Sibley School of Mechanical Engineering, where he studied gait and balance and total joint replacement. He completed a fellowship at the Hospital for Special Surgery in New York.

Dan Keller 0:08
Welcome to this episode of Substantial Matters: Life and Science of Parkinson's. I'm your host, Dan Keller. At the Parkinson's Foundation, we want all people with Parkinson's and their families to get the care and support they need. Better care starts with better research and leads to better lives. In this podcast series, we highlight the fruits of that research, the treatments and techniques that can help you live a better life now, as well as research that can bring a better tomorrow. That research is a long and complicated process, moving from laboratory methods to animal testing and hopefully to clinical trials involving people with Parkinson's, and people with Parkinson's can play a valuable role in that research even before the clinical trials begin. Through in-person trainings and an online course, the Parkinson's Advocates in Research program at the Parkinson's Foundation, or PAIR for short, gives people impacted by Parkinson's the knowledge and skills needed to advise researchers on clinical studies. Their perspective helps prioritize research projects and improve the study designs in ways meaningful to people affected by Parkinson's. I spoke with Karlin Schroeder and Kevin Kwok about PAIR. Karlin is the Director of Community Engagement at the Parkinson's Foundation, and Kevin is a research advocate in the PAIR program who holds a Doctor of Pharmacy degree and has been living with Parkinson's for 10 years. I initially tossed the first question to Karlin.

Dan Keller 1:53
Let me ask you about patient engagement in research. This is something that in the old days just wasn't even considered. Why is it important to engage patients at all stages of research?

Karlin Schroeder 2:05
Well, what we say at the Foundation is that it's important to engage people early and often and throughout the research process. And the reason for that is that when you design a clinical trial, you want to reduce as many barriers as you can to participate. And if you have people with Parkinson's contribute to designing the clinical trial, they can point out barriers, like too many study visits, it will be hard for me to get to the site, and so you can design the trial with that in mind. Patient engagement in research for us is really that meaningful partnership between people with Parkinson's and their care partners and researchers to design and implement clinical trials. It's more than just recruiting for a trial.

Dan Keller 2:42
It had been a technique to get patients into trials. I guess that was the engagement that was seen from the research standpoint. But do the researchers accept or welcome patient input at all stages of trial design?

Kevin Kwok 2:59
I think it's an evolving field. But yes, I would say that more and more we're seeing more investigators and industry including patients earlier and earlier on in the design of the trials. It wasn't always that way, but I think you're starting to see more and more of it happening.

Speaker 1 3:14
Kevin, you've got a particular inside view. You had been in the biotech industry before, and now that you're a patient, I guess you have a unique perspective on how the two can work together.

Kevin Kwok 3:29
Oh, absolutely. The other area is, I'm also a clinician. I've trained as a clinical pharmacist. In some ways, I feel like I'm a triple threat. My ability to sort of transcend the industry, though, is really by putting on the eyes and the glasses of a patient, and that is uniquely different. And oftentimes, I'll be in the room with people that are far more experts in their field. And yet, when it comes down to it, they say that Parkinson's is a disease where no two patients are the same. And I think the complexity and the heterogeneity of the disease allows us to sort of speak from the patient voice. So while I do have an industry background, I think I mostly speak from the patient side when I get involved in advocacy.

Dan Keller 4:13
What is the Parkinson's Foundation doing to encourage this and bring more of it together?

Karlin Schroeder 4:19
We train people with Parkinson's and their care partners in the research process so that we have a cadre of people with Parkinson's and their care partners who are ready to engage, and then we also reach out to researchers and explain to them what patient engagement is. It can still be very new to people, although, as Kevin said, it's happening more and more often. So we can provide resources and materials and best practices in patient engagement to research teams. So a lot of it is educating the community and then demonstrating how patient engagement can make an impact, showing how we can make clinical trials more efficient and effective.

Dan Keller 4:53
Do you have anything to add, Kevin?

Kevin Kwok 4:55
No, I think that the training that we receive through the Parkinson's Foundation is tremendous. They get a group of people in a room who, for all intents and purposes, come from all walks of life. There are a few of us that have industry experience, but for the most part, they go through the whole process of, you know, what is an interventional trial versus an observational trial? You know, what are the various stages of endorsing clinical research? One of the biggest issues, I think, that we play is a bridge between academicians and patients and industry. We're sort of right in the middle of that triad, right? And so our role, in some ways, we become almost a reference to other patients who trust us, who say, well, what trial should I get involved in? Right? And so I think we play a very unique role in the way that we are able to sort of catalyze the role of engagement and research.

Karlin Schroeder 5:47
The Parkinson's Foundation also works to move the field of patient engagement in research forward across disease areas. So we work in national and international coalitions to define best practices in patient engagement and push the field forward with innovation, and we also work in coalitions to better define how we measure patient engagement so that we have metrics around our work.

Dan Keller 6:09
Are you educating patient advocates in all aspects of research? I mean things like how research is conducted, the steps in doing a trial, not only how to go out and lobby your congressman?

Karlin Schroeder 6:24
Exactly. So actually, the work in our program doesn't really include policy work, except for with FDA, the Food and Drug Administration, but what we do to educate people with Parkinson's and care partners is train them, as you said, in the different phases of clinical trials. We help them understand what's in the Parkinson's pipeline now. We help them understand how to analyze research and understand how to interpret a journal article and about ethics around research, such as the informed consent process. So we're really talking about all aspects of the research process, even as early as basic science, where we're thinking about therapies that we might want to study that could be on the market in the future in 15 years.

Kevin Kwok 7:02
You bring up an interesting point, though. I think patient advocacy has many dimensions. There's policy, there's research, there's personal, there's the caregiver issues. And so I think that in the journey of a patient advocate, I see the entry point being different places and the involvement being different, but at the end of the day, they somewhat all merge together. There's a research component, there's a policy component. You do develop dimensions as a patient advocate.

Dan Keller 7:31
As part of the Parkinson's Advocates in Research project, are there meetings, or how do you keep people involved and knowing each other and swapping ideas?

Karlin Schroeder 7:41
So there's two of us on staff for the program who have a very in-depth touchpoint with all of our advocates on an ongoing basis. So we talk to people regularly by phone or through teleconference. We travel around the community to see people face-to-face. And we really know the people with Parkinson's and care partners that we work with well. We know their skill sets. We know what stage of Parkinson's they're in, what type of therapies they might have tried. So it's a high-touch program. We do like to bring people together where we can. So we might bring in local advocates to a scientific meeting that's happening in a particular community, like the American Academy of Neurology meeting. So we do convene people locally as much as we can to bring the research advocates together, and we're thinking about right now how we do ongoing continuing education for advocates who've come through our program years ago. So perhaps we can do like WebEx courses on building your skill set to be a research advocate.

Dan Keller 8:36
When did it start? How long have you been doing it?

Karlin Schroeder 8:39
The Foundation's been doing patient engagement in research for over 15 years, and when we initially started, it was about educating the community about clinical trials and training people to be peer-to-peer advocates. But we quickly realized that challenges with getting people into clinical trials were related to how trials were created. The trials may have been created with a lot of burden on people with Parkinson's and their care partners to participate, so maybe a lot of study visits, and if you include people with Parkinson's and care partners early in the process, you can design a study that doesn't have so many burdens. So we shifted to really training people about clinical trials and how to design and implement them.

Speaker 1 9:17
Have you seen results already? Can you put your finger on some things that it has changed?

Karlin Schroeder 9:21
Sure. So one of the things that's most easy for people to understand is that we can help clinical trials recruit more quickly because we help design them so people are more likely to sign up for these trials and stay involved. So we know that we can make research more efficient and effective and get us to the goal that we all want—better therapies and hopefully a cure—but in a very concrete way, some of the things that we do with the trial, for example, we ensure that the trial has better endpoints. So is it important for people with Parkinson's to reduce the number of hours that they're off their medication to two hours or to six hours? What's really important so we can define that. And then when we define that in a clinical trial, we can cut back on the other tests that are involved that might not be so important to that endpoint, because all those other tests can make it very burdensome for somebody to take part in a clinical trial.

Dan Keller 10:14
The trials that come out of it this way, are they just as rigorous and informative or even more so than in the old system?

Kevin Kwok 10:23
I would say they're more rigorous, but they're more meaningful. You know, at the end of the day, we're looking at endpoints that make a difference. It may not necessarily be pure UPDRS rating, but it may be, you know, quality of life. Are you actually feeling better? You know, can you travel again? Can you continue work? These become the patient-reported outcomes that we feel maybe make more of a difference in our lives.

Karlin Schroeder 10:47
We're not trying to change the science. We're trying to change how the science is done. So we know that clinical trials have to be statistically sound, and there are certain things that have to happen, and scientists have their expertise in the science. We're adding the expertise of people with Parkinson's and their care partners to that process so that we can strengthen the process of how the science happens.

Kevin Kwok 11:09
One particular situation where I'm from the Bay Area in California, a local thought leader at one of the teaching institutions there approached me and said, would you review my research and would you write me a letter of recommendation? Because he said, I'm applying for an NIH grant. And so I actually wrote it on letterhead of the Parkinson's Foundation after reviewing it as to why I thought this was really solid research. This happened to me in the field of deep brain stimulation. The notice I got back, the email was, congratulations, we won the grant, and I had no idea the magnitude, but it was several million dollars over a several-year period of time. I now sit on the steering committee of this teaching institution to review updates on the protocol. Are we hitting milestones? And they really, truly do want our input on that. And I was told that the issuance of the grant in a very competitive market was based in part on the strong voice of the patients, you know, to winning that grant.

Dan Keller 12:09
I wonder if other institutions will sort of pick up on this idea also and put people with Parkinson's on their steering committees for those studies.

Karlin Schroeder 12:18
Actually, with NINDS-funded studies, so government-funded studies, it is fairly common for Parkinson's researchers to do this. We have people with Parkinson's not only on the steering committee but the data safety monitoring board, which is a board that looks at safety within the process of the clinical trial. So big Phase 3 clinical trials, such as Study PD and Sure PD, which look at delaying disease progression to see if these therapies can delay disease progression.

Speaker 1 12:45
So how can people get involved if they want to become advocates and join the program?

Kevin Kwok 12:51
You know, it's really funny you asked that question. When I was first diagnosed almost a decade ago with Parkinson's, as I said to my neurologist, you know, I've lived off the back of other patient volunteers in my entire career, and now I am a patient. I feel like I've come full circle. So I want to be involved in my disease. And her comment to me was, what do you mean by that? And I said, I have no real idea. The first place that she recommended then was, I believe that the PAIR program was looking for nominees. And she said, you know, I'd love to fill you out as an applicant for that program. And so that just started off really my “career” in patient engagement. So, you know, my view is that PAIR was a great, great stepping point, because I really had no idea how to formally get involved. Through the program, though, I've gotten other people involved. I've recruited other members into PAIR. You keep an eye out, you know, would this person be a good person? You know, are they inquisitive? Are they curious? Do they want to help others? You know, I think there are certain criteria that we look for in our advocate community.

Karlin Schroeder 13:57
And of course, people can contact me directly. Karlin Schroeder, my number is 646-388-7641, or my colleague, Casey Gallagher, who's an assistant in the program, at 646-388-7612. We run our training programs once a year, but there are ways that we can engage people in between those programs as well.

Speaker 1 14:18
Is there anything important we've missed or to add?

Karlin Schroeder 14:22
I think the main message is just again that people with Parkinson's and care partners are experts at living with their disease, and they have a unique voice to add that would otherwise be missing from the research process, and it makes a real impact in the process.

Kevin Kwok 14:34
We had the chance to testify at the FDA a number of years ago, and afterwards, the head of the neurology division approached us and said, you know, we know a lot about neurology, but hearing your voice really does make a difference, and we heard it.

Dan Keller 14:49
Very good. Thank you both. So far, more than 300 people with Parkinson's and caregivers have gone through training to become research advocates. They work closely with the Parkinson's Foundation and their peer network peers to identify research opportunities, take part in ongoing education, share resources, and to interact with the research community. If you would like to become a research advocate, you can find out more about the program, how it works, what the training entails, and how to get involved by visiting our website at parkinson.org/pair. Applications for the 2019 Learning Institute will open March 1, 2019. The next Parkinson's Foundation Learning Institute will be held June 23 to 26, 2019, in Minneapolis, Minnesota. You can also talk with our PD information specialist on our toll-free helpline. They can answer your questions and provide information about the PAIR program or anything else having to do with Parkinson's. You can reach them at 1-800-4PD-INFO. If you have any questions about the topics discussed today or if you want to leave feedback on this podcast or any other subject, you can do it at parkinson.org/feedback. At the Parkinson's Foundation, our mission is to help every person diagnosed with Parkinson's live the best possible life today. To that end, we'll be bringing you a new episode in this podcast series every other week. Till then, for more information and resources, visit parkinson.org or call our toll-free helpline at 1-800-4PD-INFO, that's 1-800-473-4636. Thank you for listening.

Karlin Schroeder is the Senior Director of Community Engagement at the Parkinson’s Foundation, where she leads the Parkinson’s Advocates in Research program (PAIR). Through this program, Karlin runs the Parkinson’s Foundation Learning Institute, an intensive multi-day course that has trained over 300 people with Parkinson’s and their care partners in the research process and provides tools and resources for successful collaboration with research teams. Karlin creates and directs projects to incorporate patient expertise into research with industry, academic centers and government. Her work includes improving diversity in patient advocacy in Parkinson’s disease and defining metrics in patient engagement in research. She is a member of Patient Focused Medicines Development and the Clinical Trials Transformation Initiative Steering Committee.

Kevin Kwok is the Head of Patient Engagement at Theravance Biopharma in the Bay Area. Previously, he was a partner at a leading executive recruiting firm, where he led the North American Life Science practice. Kevin earned his doctorate degree in clinical pharmacy from the University of Michigan. An avid lifelong wannabe athlete, Kevin was diagnosed with Parkinson’s in in his late 40’s. He has taken control of his Parkinson’s with bold irreverence, optimism and biting humor. As a self-proclaimed amateur “PD satirist”, he has been a speaker and patient advocate for several foundations and has been featured on national news programs and webcasts, speaking about living a high-impact life, thanks to traditional therapies and a “zen-aggressive lifestyle.” Kevin joined the Parkinson's Foundation Parkinson's Advocates in Research program in 2014.

Priscila es asesora genética certificada y licenciada en el Departamento de Genética Médica y Molecular (MMGE) de la Facultad de Medicina de la Indiana University. Obtuvo una Maestría en Ciencias de la Northwestern University en el 2015 y trabajó como asesora genética prenatal durante 5 años antes de unirse a MMGE en el 2020.

Priscila es miembro de un equipo de asesoramiento genético en la Universidad de Indiana que ha proporcionado asesoramiento genético telefónico a más de 11,000 participantes de estudios de investigación de la enfermedad de Parkinson. También proporciona asesoramiento genético bilingüe a los participantes del estudio de investigación PD GENEration. PD GENEration es una iniciativa internacional patrocinada por la Parkinson’s Foundation que ofrece pruebas genéticas gratuitas y asesoramiento genético para las personas con la enfermedad de Parkinson. Priscila también ha estado involucrada en los esfuerzos de vinculación y educación para la comunidad hispana y es colíder del Comité Latino de Asesoramiento sobre PD GENEration. Algunos de estos esfuerzos incluyen el desarrollo de materiales educativos culturalmente apropiados, así como también eventos educativos abiertos para la comunidad internacional de habla hispana.

Es miembro de la Sociedad Nacional de Consejeros Genéticos (NSGC por sus siglas en inglés) y es colíder del Grupo de Interés Especial de Desarrollo en Español. También es miembro de la Junta Consultiva de Recursos de Asesoramiento Genético de All of Us. Ha dado numerosas conferencias y presentaciones sobre competencia cultural, incluyendo temas sobre diversidad, equidad e inclusión para la comunidad de asesoramiento genético.

Los intereses de investigación de Priscila incluyen el uso de intérpretes y el papel que desempeñan en la sesión de asesoramiento genético, la competencia cultural en el asesoramiento genético y la diversidad en la investigación con un enfoque especial en la comunidad hispana.

Dan Keller 0:08

Welcome to this episode of Substantial Matters: Life and Science of Parkinson’s. I’m your host, Dan Keller. At the Parkinson’s Foundation, we want all people with Parkinson’s and their families to get the care and support they need. Better care starts with better research and leads to better lives. In this podcast series, we highlight the fruits of that research—the treatments and techniques that can help you live a better life now, as well as research that can bring a better tomorrow.

The journey to new therapies for Parkinson’s and other diseases depends on clinical trials involving people with the disease to prove the efficacy and safety of any new treatment. The road to clinical trials starts with preclinical studies—laboratory experiments involving animals, cells, or isolated biological compounds or systems as models.

Richard Smeyne is a professor of neuroscience at Thomas Jefferson University in Philadelphia focusing on Parkinson’s disease. His laboratory uses preclinical models to study mechanisms of neurodegeneration and neuroprotection, talking about preclinical models of Parkinson’s disease.

What does preclinical mean first of all, and how does this add to useful knowledge that may eventually help people with Parkinson’s?

Richard Smeyne 1:38

When I think of preclinical models, I generally think of studies that do not involve humans. Often they’re the early studies using animals, or we can use cell lines, or we can use single-cell organisms such as yeast to study a variety of biochemical mechanisms that we know or think underlie development of Parkinson’s disease.

And these are very useful because they give us the clues on the pathways that are affected, the biochemistry, sometimes even anatomical circuitry underlying the disease. And it’s a way that we can use them to intervene for potential therapies down the road that can then be used in human trials.

Dan Keller 2:20

Do they generally pan out? What’s your batting average there?

Richard Smeyne 2:24

It’s interesting when you ask that question. Many of the therapies that we know work in animal models or in cell lines in terms of rescuing do not work in humans, and that’s always a potential problem.

And one thing we have to recognize in our preclinical models, especially related to Parkinson’s disease, is that Parkinson’s disease is a uniquely human disorder. In any of the models that we’ve made to examine Parkinson’s disease, they do not recapitulate what we see in the disease spectrum seen in people.

So we have to choose. When we’re doing animal models, we can study the loss of cells, or we can study biochemistry or processes, but not the whole disease.

Dan Keller 3:07

Is Parkinson’s disease even one disease in people? You have various genetic causes, interactions of genes. So when you study it in a preclinical model, do you have to pick what kind of underlying deficit there is?

Richard Smeyne 3:23

We do. But your initial question is actually critical to our understanding of Parkinson’s disease, and we don’t quite have a handle on that now.

Many years ago, we thought of Parkinson’s disease as a single entity, and it was sort of all-encompassing. With the advent of modern genetics and the identification of many different genes that lead to Parkinson’s disease, we now have to ask the question: is Parkinson’s a better term than Parkinson’s disease? Because many different ways can eventually give you similar symptoms.

And this is a critical question when we’re thinking about therapy. Do we have a single treatment that would be good for all forms of Parkinson’s, or do we have to identify these subgroups of people because they’ll be treated differently?

I often think about Parkinson’s disease using a cancer analogy. 15–20 years ago, you would tell someone you had cancer. Nowadays, if you say “I have cancer,” it becomes virtually meaningless—it is what type of cancer, what mutation underlies it, because individualized therapies are coming now.

That’s likely the future in many neurological disorders, including Parkinson’s disease.

Dan Keller 4:36

I suppose like in cancer, the more defects you find, the more each case becomes a rare disease. You’ve got A, B, C, and Z, and different types of cancers or reasons leading to Parkinson’s.

Richard Smeyne 4:53

Absolutely. And the more we know about genetics, the less we know about the disease in a way. Once we get a better understanding of underlying genetics, we may have more personalized medicine available to everybody.

As you said, you may have a defect or mutation or epigenetic alteration controlling gene expression. We can see this in screening as it becomes cheaper and technology improves.

Then we can personalize treatment. This is where preclinical studies are at the forefront, because depending on the process interfered with, you may use one class of drugs, and if another process is involved, we may use a different approach, hopefully still achieving the same positive outcome.

Dan Keller 5:49

Can you give me one or two examples of preclinical studies that have progressed and led to phase 1, 2, or 3 studies or even an actual drug?

Richard Smeyne 6:00

The ones that are furthest along now are studies examining alpha-synuclein. Alpha-synuclein is the protein that is misaggregated—it forms clumps within cells—and is thought to underlie much of the disorder.

We know this process normally involves non-aggregated alpha-synuclein, but with mutations or other defects it clumps. So we have studies preclinically using different drugs that stop aggregation, and we are moving into therapies using either passive or active antibodies that interfere with transmission of this altered synuclein, aiming to reduce misfolded protein.

Those are now being transferred from the laboratory and pharmaceutical companies into human trials.

There are also other studies, for example from Hopkins, looking at a molecule called LAG3, which interferes with aggregation. Other studies from Georgetown are looking at drugs affecting the c-Abl gene, originally an oncogene but now known to be involved in the proteasome system.

A drug called nilotinib, a small molecule, interferes with c-Abl signaling and may help disaggregate synuclein or prevent aggregation, lowering synucleinopathy.

Dan Keller 7:42

What you’ve described is often termed bench to bedside. What about going the other way—bedside to bench? What you learn at the bedside?

Richard Smeyne 7:55

Absolutely. When I think of bedside to bench, I don’t think so much about drug therapy alone, but about alternative therapies—things people do that we know can interfere with disease progression.

Exercise is a perfect example. Epidemiology shows people who exercise tend to have slower progression or lower risk of Parkinson’s disease.

My lab and others have taken that observation and studied it in animal models where we can control exercise and examine biochemical changes in the brain. From that, we’ve identified pathways that change with exercise that can be targeted.

Another example is MPTP in heroin users who accidentally developed Parkinsonian symptoms. This led to discovery of MPTP, which helped identify mitochondrial dysfunction and bioenergetic failure in substantia nigra dopaminergic neurons.

From that, we’ve identified genes and biochemical processes that can now be targeted in therapies.

Dan Keller 10:10

As Yogi Berra said, “You can observe a lot just by looking.” It sounds like serendipity plays a role.

Richard Smeyne 10:27

Absolutely. This is a secret of science. Look for things that are out of the ordinary. The MPTP story required detective work, and many people contributed.

It happened in different places, including Baltimore with Barry Kidston and later work by Bill Langston, who is often credited for advancing the field.

Yes—keeping your eyes open for unexpected findings is critical. Many discoveries come from outside Parkinson’s research and are incorporated by scientists who read broadly across fields.

Dan Keller 11:37

Do you think there’s even more need for reading outside your field?

Richard Smeyne 11:48

Absolutely. Both clinicians and basic scientists need to read outside their fields. Parkinson’s disease is not just a motor disorder—it includes gut, immune, and cognitive systems.

It is difficult for one person to master everything. The field is now highly interdisciplinary, involving immunologists, molecular biologists, anatomists, pharmacologists, and physiologists working together.

We can no longer be siloed. We need collaboration across disciplines.

Dan Keller 13:24

What have we missed that’s interesting?

Richard Smeyne 13:24

One important point is how much we can learn from different organisms. Yeast, C. elegans, flies, rodents, and non-human primates all contribute important insights.

These models are not meant to cure animals but to improve understanding of disease processes in humans.

Dan Keller 14:15

Good. Thanks.

Dan Keller 14:24

To learn about research and drug development, visit parkinson.org and search “research” or “drug development.” You can also call our toll-free helpline at 1-800-4PD-INFO.

If you have questions or feedback, visit parkinson.org/feedback.

Our mission is to help every person diagnosed with Parkinson’s live the best possible life today. New episodes are released every other week.

For more information, visit parkinson.org or call 1-800-473-4636.

Thank you for listening.

Richard Smeyne earned his bachelor’s degree in Biology and Psychology from St. Joseph’s University (Philadelphia, PA) in 1977, attended the M.A. Program in Experimental Psychology at the University of Hartford through 1992 and earned his Ph.D in Anatomy from Thomas Jefferson University (Philadelphia, PA) in 1989. He did a postdoctoral fellowship from 1989-1991 at the Roche Institute of Molecular Biology in Nutley NJ where he studied with Jim Morgan and Tom Curran.

Following his postdoctoral work, he started his own lab at the Bristol Myers Squibb Pharmaceutical Research Center where he became recognized for his analysis of mice carrying mutations of the neurotrophin receptors TrkA, TrkB and TrkC. Dr. Smeyne was subsequently recruited in 1994 to become the Head of the Neurogenetics Program in the Division of CNS Research at Hoffmann-LaRoche. In 1996, following the closure of the Roche Institute of Molecular Biology, from 1996-2016, Dr. Smeyne was a faculty member in the Department of Developmental Neurobiology at St. Jude Children’s Research Hospital (SJCRH) in Memphis TN. IN 2016, he moved to his current position as a Full Professor in the Department of Neuroscience at the Vickie and Jack Farber Institute for Neuroscience at Thomas Jefferson University. He also serves as the Director of the Jefferson Comprehensive Parkinson’s Disease Center.

Dr. Smeyne has had a long-standing interest in the cell biology of Parkinson’s disease, with studies examining both environmental and genetic causation. His lab was one the first to show, in animal models of PD, that exercise could modify disease progression, and more recently has been examining the underlying molecular programs responsible for this neuroprotection. He is also recognized for his work examining interactions of the peripheral immune system with that of the brain’s innate immune system immune as well as the role of viruses in the etiology of Parkinson’s disease. Currently, Dr. Smeyne serves as a member of the Clinical and Scientific Advisory Board for the Parkinson’s Foundation. His lab is currently funded by the NIH, the Parkinson’s Foundation, and the Michael J Fox Foundation for Parkinson’s Research.

Dan Keller 0:08
Welcome to this episode of Substantial Matters: Life and Science of Parkinson’s. I’m your host, Dan Keller. At the Parkinson’s Foundation, we want all people with Parkinson’s and their families to get the care and support they need. Better care starts with better research and leads to better lives. In this podcast series, we highlight the fruits of that research—the treatments and techniques that can help you live a better life now, as well as research that can bring a better tomorrow.

The most efficient development of therapies depends to a large extent on identifying a specific problem in the body and then designing a way to correct or mitigate it. In the case of drugs for Parkinson’s disease, knowing the underlying molecular, physiological, or genetic defects that lead to the disease and its symptoms significantly helps to design or discover treatments.

I spoke with Michael Okun, National Medical Director of the Parkinson’s Foundation, at the World Parkinson Congress, where he described how knowledge of disease mechanisms is facilitating the hunt for better treatments. Let’s first talk about new mechanisms in Parkinson’s disease, and possibly as targets for drugs and drugs in development—what’s coming along?

Michael Okun 1:34
So there’s a whole host of interesting new approaches to how we might attack Parkinson’s. We, in general as scientists, look for targets, and the targets can be drug targets, they can be device targets, or they can be targets by modification using other techniques, such as behavioral techniques. So even exercise affects targets in the brain. And so it’s important to keep in perspective the wider picture.

Right now, there are a couple things that are hot that people are really keying in on. Now, the idea that could we take away the abnormal accumulations in the brain—some of these plaques—and use drugs or vaccines or other approaches to clear away these abnormal deposits. And if we clear them away, will that lead to an improvement?

And so I think that there are a group of companies that are working on different vaccination approaches. There are several safety studies that have been completed there. There are a few people that are working on it from the drug angle, and a few of our colleagues in Alzheimer’s have some interesting drugs who are trying to do the same thing.

And the lesson from Alzheimer’s in the past was that when they cleared the plaque away, it didn’t necessarily improve the symptoms, and there may have been some side effects. We have more optimism in Parkinson’s, and there are multiple different approaches of how we can do it.

There’s also a class of drugs called c-Abl inhibitors, and c-Abl is part of the degenerative cascade. And it turns out that there’s an approved chemotherapy drug called nilotinib. And this is a drug that is of interest because, again, it works on that pathway that may clear plaque or these abnormal deposits out of the brain.

And people are interested to see how this is going to pan out. There was a very small study that was published by our colleagues at the Parkinson’s Center of Excellence at Georgetown, but only 10 or 12 patients have been done. And there are a couple of trials now that are being planned, and there are other groups that are working on c-Abl drugs, and they think if they can go after this particular enzyme in the brain, that’s going to be a viable therapeutic approach.

And so there are a couple of really interesting scientific avenues that are being pursued by a lot of people all over the world.

Dan Keller 3:42
What about the time frame here? I mean, they’ve identified targets, some drugs exist for other uses which could be imported, but in a realistic sense, when do you think these things might actually help a patient—not in a trial?

Michael Okun 3:56
So your point about repurposing drugs—drugs that are already on the market that have a known mechanism of action, and then once we identify a target we think, “Oh, well, let’s take this drug because it’s already been through the FDA or has some sort of stamp on it”—that’s a great notion, and I’m glad that you brought that up. That speeds the drug development and getting it to market and to trial quite a bit, by a number of years.

The disappointing thing for Parkinson’s patients and for patients with other neurodegenerative disorders, and for that matter cancers, liver issues, kidney issues, heart issues, is this long latency that can be anywhere from five to 15 years to get a drug through development, several billion dollars in terms of trying to move it to market.

So there’s been a big push from the foundations, from the patient advocacy groups, to work with the Food and Drug Administration and to try to decrease that latency so we can get things to market quicker. People are anxious—they want to get on with different therapies.

There’s a famous picture by McGlynn of a man standing on top of the world looking at his watch, and I think that’s probably what every Parkinson’s patient is thinking about: “Can we get this done faster?”

So it’s a problem. I think it’s getting better. I think that the repurposed drugs, like the cancer chemotherapy drug, will get into a larger trial here within the next year. I think we are going faster. I think the FDA is responding faster, and we are starting to see things move through there. And so there are a number of symptomatic drugs that we’ve seen move through a little faster than we’re used to. So we’re liking the trend—we’re hoping it’s going to speed up.

Dan Keller 5:27
What about vaccines? There’s passive immunization, there’s active immunization. If you give a vaccine that’s active, it’ll keep working in your body. Passive would be antibodies you keep administering. In the Alzheimer’s area, the monoclonal antibodies you administer haven’t really done much—there have been a lot of trials. Do they think that in Parkinson’s there would be something different?

Michael Okun 5:49
Yeah, so it’s an interesting question. And just to back up and talk a little bit about how this may work—the idea is that you could utilize your own immune system, which is designed to fight disease, to try to fight off some of these abnormal depositions in the brain and perhaps attack inflammation and some of these other potential underlying causes of Parkinson’s disease.

And so you can do this a number of ways. You can inject the antibodies into a vein and then have them travel around, hopefully get across the blood-brain barrier, which is obviously one of the things we worry about the most.

So the brain is a privileged area—it’s protected, as it should be—but that makes it harder for us to get treatments there. And so the first idea is when you do treatments like monoclonal antibodies, they’ve got to get across the blood-brain barrier, they’ve got to get to the target, and they’ve got to do their job. And that’s a lot of “what ifs,” so we have to take them to trial to see.

And so when we see a failure, I like to tell people we shouldn’t look at this as yes or no—we should look at it as process refinement. How can we refine it to make it better?

The other type of therapy is injecting something that then raises your immunity, kind of like a flu shot would do. And so then it can fight off these abnormal issues going on in your brain by raising your own immunity.

So there are two different approaches, and actually there’s a whole host of different ways this can be done. Another thing people don’t realize about vaccines are off-target effects. Sometimes you’ll have something designed to do one thing that actually has a beneficial effect you didn’t anticipate—but it’s a good effect.

And we’ve seen this in other areas like small nucleotide RNA therapy and eye diseases and other areas of medicine. An off-target effect isn’t necessarily a bad thing. We see this in deep brain stimulation surgery too—sometimes you see something you didn’t expect, but it can be a good thing.

Dan Keller 7:44
I suppose if you do active immunization—giving someone a protein like alpha-synuclein and have them make their own antibodies—there’s evidence recently that lymphocytes do end up in the brain. So I guess that would overcome this idea of antibodies having to cross the blood-brain barrier to get in.

Michael Okun 8:01
Yeah, there’s some evidence that there are ways to permeate the blood-brain barrier, that some things get across that we don’t expect. And then there are clever things like using ultrasound to temporarily open the blood-brain barrier and break it down. So there are really clever ways scientists have come up with to try to combat that problem.

Dan Keller 8:22
Are you encouraged at this point that there are new mechanisms, new pathways, and new targets that have come up recently? Because the field has depended essentially on levodopa for so many years and its brethren.

Michael Okun 8:35
Sometimes when you’re out there fighting the battle day by day, it’s hard to interpret: has the needle moved? Have we done something that’s helped people and helped the field?

I think I can say without a doubt—between this World Parkinson Congress and the last two, which occur every three years, and this year we’re in Portland—the needle is moving on a lot of things, particularly symptomatic therapies and symptomatic drugs.

We think of these therapies in three buckets: symptomatic therapies, disease-modifying therapies (trying to slow progression), and then the cure bucket. We are certainly furthest away from the cure bucket, but some of these single-gene defects and DNA problems might one day be amenable to gene silencing or other new technologies.

Disease modification—we’re still fighting that battle. But in the symptomatic, meaningful symptomatic therapy bucket, we’re doing pretty well. And if you look across other neurodegenerative diseases, frankly, they’re fairly jealous of the Parkinson’s community because we are moving the needle on a lot of things.

Dan Keller 9:48
To learn more about what’s coming along in drug development and research, visit parkinson.org and search “drug development.” You can also call our toll-free helpline at 1-800-4PD-INFO to speak with PD information specialists.

If you have questions about the topics discussed today or want to leave feedback, you can do it at parkinson.org/feedback.

At the Parkinson’s Foundation, our mission is to help every person diagnosed with Parkinson’s live the best possible life today. We’ll be bringing you a new episode every other week.

For more information, visit parkinson.org or call 1-800-473-4636.

Thank you for listening.

Dr. Okun is Chair of the Department of Neurology at the University of Florida, as well as Co-Director of the UF Center for Movement Disorders and Neurorestoration. Dr. Okun has long been dedicated to the interdisciplinary care concept, and since his appointment as the National Medical Director for the National Parkinson Foundation in 2006, he has worked with the 43 international Parkinson’s Foundation Centers of Excellence to help foster the best possible environments for care, research, and outreach in Parkinson disease, dystonia, Tourette, and movement disorders.

In addition to his role as National Medical Director for the Parkinson’s Foundation, he is the Medical Advisor for Tyler’s Hope for a Dystonia Cure and Co-Medical Director for the Tourette Syndrome Association (TSA).

Dr. Okun has enjoyed a prolific research career exploring non-motor basal ganglia brain features, and he has participated in pioneering studies exploring the cognitive, behavioral, and mood effects of deep brain stimulation (DBS). Dr. Okun holds the Adelaide Lackner Associate Professorship in Neurology, has published over 200 peer-reviewed articles, is a published poet (Lessons From the Bedside, 1995), and has served as a reviewer for more than 25 major medical journals. He has been invited to speak about Parkinson’s and movement disorders all over the world.

Dr. Okun earned his BA in history from Florida State University and his MD from the University of Florida, with honors. He completed an internship and neurology residency at UF. Following residency, he was trained at Emory University.

Dan Keller (00:08)
Welcome to this episode of Substantial Matters: Life and Science of Parkinson's. I'm your host, Dan Keller at the Parkinson's Foundation. We want all people with Parkinson's and their families to get the care and support they need. Better care starts with better research and leads to better lives. In this podcast series, we highlight the fruits of that research, the treatments and techniques that can help you live a better life now, as well as research that can bring a better tomorrow. Clinical research studies are central to developing new treatments that will ultimately improve people's lives, and in the case of Parkinson's, that may even slow down or halt progression of the disease. At the heart of clinical trials is the willingness of people with Parkinson's to volunteer to participate. Christine Hunter, the coordinator of the Parkinson's Foundation Center of Excellence at Baylor College of Medicine in Houston, is in close contact with study volunteers. She talks about what is needed to improve and speed up clinical trials, and why it's important to get involved.

Dan Keller (01:21)
Who does it benefit—the patient, the scientists, the community?

Christine Hunter (01:25)
Everybody. The more patients that we can involve in clinical trials, we can bring therapies to them much quicker. Part of the problem with clinical trials is the slow rate of recruitment into studies. So if we don't have people that are enrolled, it takes that much longer. So we want to bring new therapies, and some of them are very promising. Some may not be. Some we have to try to see if they're going to work, and we have to have the data to do that. But it helps everyone—patients, the scientists, the centers, the community—because as more people are aware and more involved, it's better for your health.

Dan Keller (02:05)
What kind of feedback do you get from participants?

Christine Hunter (02:08)
They love it. At least at our center, I have repeat participants, so they finish one study and then they go into the next one, depending on whether they're allowed or not. The Parkinson's Foundation has a registry also that they can participate in. So that's an ongoing thing that doesn't require intervention, but they're helping with data and the future knowledge of how the disease progresses, how we serve our people with Parkinson's.

Dan Keller (02:34)
You're at Baylor—that would bring up an idea of what kind of diversity do you get in studies, or is that lacking?

Christine Hunter (02:40)
I think it's lacking. It's lacking everywhere. In San Francisco, they have trouble with diversity in studies. Even though our cities are very diverse, we have one of the most ethnically diverse communities in Houston, but it's typically the same Caucasian participants. So we really do need minorities to step up. Things may be different for them, but we don't know that. And so until we have the opportunity to study with them and have them participate with us in studies, we don't know if there are ethnic differences, reactions to medications, all of those kinds of things that still have yet to be studied.

Dan Keller (03:15)
Do you think there are barriers or just not enough outreach?

Christine Hunter (03:20)
I think there are barriers. I think partly an outsider coming into a community—I think it usually has to come from within the community. Some communities are doing a great job at that. The Phoenix group has done a lot of outreach in the Hispanic community. They have done enormous work. And so I think there are some success stories, and so what we need to do is learn how to replicate those successes in other centers, in other communities.

Dan Keller (03:50)
Do you, I would say, market in a broad sense, participation in studies by telling people what has come out of patients participating in the past?

Christine Hunter (04:01)
Yes, we let them know whether they were on the active treatment or the placebo. After a study is over, we give them copies of the papers. A lot of the studies we do with the Parkinson's Study Group have webinars with the patients and participants to give them the data from the study, to let them know before it's broadly publicized. So there are lots of efforts to keep people involved and aware of what's happening. We go to our support groups. We go to different groups and let them know this study was positive. This is what you can expect. This is a new therapy that's just been approved. We have some other studies that are looking at similar things. As much information as people can have, then they can make an informed decision about what they want to do going forward. And I know sometimes the requirements for clinical trials are so narrow—the inclusion exclusion—people don't understand why we have such narrow inclusion exclusion. But to get a drug on the market, we have to go through so many different phases, and that broadens as they get larger. But the smaller studies, especially early on, they want to be very exclusive about who's put in a trial and making sure they don't have other comorbidities and that kind of thing. We want the success of the drugs. We want to be able to bring new therapies.

Dan Keller (05:16)
Do people get discouraged by the exclusion criteria? They're all hyped up to do it, and then they can't?

Christine Hunter (05:21)
Sometimes, sometimes that's a tough thing. Things as simple as weight. I mean, I have a funny little story. I have a patient who was ready to participate in a trial, and he went through all the screening and was excluded on his body mass index, but because the early studies were only done within a very narrow body mass index, he was excluded. His comment to me was, "I didn't know fatties couldn't be in a study," which—that's not the reason for that—but we did find another study that didn't have the same exclusion that he could participate in. So, you know, sometimes where they close the door, you've got a window that opens, and that's why the Parkinson's Outcomes Project is good, because everyone can participate. And so it's open to everyone, so everyone feels like they're involved in doing something.

Dan Keller (06:07)
Naturalistic, real world.

Christine Hunter (06:09)
Yeah, and do you receive physical therapy, speech therapy? Have you changed your medicines? What's going on? Have you been hospitalized? Do you have falls? Just kind of a global snapshot once a year to see how things are progressing in different categories, but it's been used to look at hospitalizations and to make some changes so people with Parkinson's aren't at higher risk when they're hospitalized. So there is good that comes out of everything. So I think that we're all in this together, and the more that patients can work with their centers, their nurses, their doctors, the more impact we will have on this disease.

Dan Keller (06:45)
Great. I appreciate it. Thanks.

Dan Keller (06:56)
There are lots of factors to consider when you are thinking about participating in a clinical trial. To learn more about what's involved, or if you want to find out which trials are currently enrolling participants, call our toll-free helpline at 1-800-4PD-INFO. To recruit enough volunteers, many trials involve several medical centers and medical practices around the country, so there may be trials starting or going on near you. You can also visit clinicaltrials.gov to look for studies in your area. One ongoing study Christine mentions is the Parkinson's Outcomes Project, started in 2009. It is the largest ever clinical study of Parkinson's disease, with about 10,000 people enrolled. Tracking so many participants over time allows researchers to examine the most effective Parkinson's treatments, benefits of various exercise programs, best candidates for treatment, the disease's impact on caregivers, and more. To learn about this study and other Parkinson's Foundation initiatives, listen to our podcast episode, "The Parkinson's Foundation's Role in Improving Standards of Care." If you have any questions about the topics discussed today, or if you want to leave feedback on this podcast or any other subject, you can do it at parkinson.org/feedback. We'll respond to some questions in future episodes. At the Parkinson's Foundation, our mission is to help every person diagnosed with Parkinson's live the best possible life today. To that end, we'll be bringing you a new episode in this podcast series twice a month. We'll continue our focus on research and how people can volunteer in upcoming episodes. Till then, for more information and resources, visit parkinson.org or call our toll-free helpline at 1-800-4PD-INFO—that's 1-800-473-4636. Thank you for listening.

Ms. Hunter is the Director of Clinical Research for the Baylor College of Medicine, Parkinson’s Disease Center and Movement Disorders Clinic (PDCMDC) and serves as the coordinator of the Parkinson's Foundation Center of Excellence. As part of the PDCMDC’s leadership team, she serves as a resource to the Parkinson's community both locally and regionally. Her duties at Baylor College of Medicine include oversight of the research program, as well as serving as the primary coordinator for a number of PD-related trials.

Through educational efforts, Ms. Hunter is committed to increasing awareness of Parkinson's disease and improving care for individuals with PD and other movement disorders. She is an active member of the Movement Disorders Society and the Parkinson Study Group, for which she currently serves on the Executive Committee. She is a founding mentor of the Parkinson’s Foundation Mentoring and Networking Program for Centers of Excellence.

Ms. Hunter joined the Baylor Department of Neurology in 1995. She earned her AA degree in Nursing from Houston Community College and her Bachelor’s degree in Nursing from the University of Texas, Arlington.

Dan Keller 0:08
Welcome to this episode of Substantial Matters: Life and Science of Parkinson’s. I’m your host, Dan Keller. At the Parkinson’s Foundation, we want all people with Parkinson’s and their families to get the care and support they need. Better care starts with better research and leads to better lives. In this podcast series, we highlight the fruits of that research—the treatments and techniques that can help you live a better life now, as well as research that can bring a better tomorrow.

To best serve people with Parkinson’s and the health care providers who treat them, it’s essential to know how many people have the disease—for current treatment purposes and to allocate resources and funds for the future. But reliable figures for the prevalence of Parkinson’s in the U.S. population are lacking and have been based on outdated and possibly skewed estimates. In an effort to accurately capture these figures, the Parkinson’s Foundation has taken a leading role in determining the prevalence of PD and related conditions through the Parkinson’s Prevalence Project.

James Beck, the Foundation’s Chief Scientific Officer, says he believes the number of people living with Parkinson’s in the U.S. has been dramatically underestimated and does not represent the ethnic diversity of people affected by PD because of how we gathered older data. He explains how the Parkinson’s Prevalence Project is designed to give a clearer picture of PD in the country. First of all, what is prevalence, and what’s the goal of the project?

James Beck 1:53
Sure. Prevalence is just a fancy word for saying how many people have Parkinson’s today. What’s really surprising, I think, to many people with PD, is that they don’t appreciate that we really don’t know who has Parkinson’s today. It wasn’t until the efforts of the Foundation to better understand the prevalence of Parkinson’s that we had a firmer ground upon which to stand when we talk about who has PD today.

Dan Keller 2:15
The best evidence, or best numbers, were from quite a while ago until this project happened.

James Beck 2:22
That’s right. So probably the last study that really tried to capture the prevalence of who had Parkinson’s disease at one particular time was done in 1978. It was a really well-done study. It was a $15 million study—door-to-door epidemiology survey in a rural part of Mississippi called Copiah County, Mississippi, population right around 40,000 people.

And the goal of the study was to go door to door and identify people in a biracial community—equal Caucasians and African Americans—to see who had Parkinson’s disease. And as part of the process, they actually identified some people who hadn’t been diagnosed yet with PD.

You know, in 1978 Microsoft had 11 people in the company. It was a startup. In 1978, the black-and-white TV sat adjacent to your kitchen table. There was no social media. Our understanding of Parkinson’s disease has changed since 1978, and our ability to diagnose it—still clinically—but it has improved a lot.

So there really was an importance to be able to improve upon that and to take into account the fact that there were only 26 people identified as part of this study who had Parkinson’s disease in this rural county. Those numbers were never meant to be extrapolated to count the entire United States. Actually, the authors say so in the discussion of their paper—that it’s limited to this particular area.

Nevertheless, as people tend to do, they are given numbers and then extrapolate them to the entire United States. So for a rural county that identified 26 cases of Parkinson’s disease, it has been utilized to extrapolate to our entire country of roughly 330 million people that is ethnically and geographically diverse. And as a result, it has led many people to believe there are fewer people with Parkinson’s disease than there actually are.

If you hear numbers in the 500,000 to 600,000 range for people with PD in the U.S., that’s where those numbers are coming from. We as a community always knew in our gut that there were more people with Parkinson’s than that. One of the problems was the community was all over the place. Some people were more willing to accept these government numbers, others were more optimistic. We had settled on about a million people with Parkinson’s disease and convinced some other organizations that was about the right number.

Some of that was due to back-of-the-envelope calculations that I did a number of years ago. But I’m not an epidemiologist or statistician, and it was shaky ground. So when people would ask, we could give them numbers, but it was not substantial evidence in the way that would carry real weight.

So as part of an effort the Foundation is doing for the entire community, something we can all stand behind, we worked with a number of leading epidemiologists and movement disorder specialists who had access to rich but disparate datasets from different parts of the country. And we used these to create what’s called a meta-estimate. This meta-estimate was used to generate a revised estimate of who has Parkinson’s disease.

This is not the absolute last word, but it is a big step from where we were before. We worked with people who had administrative health care databases like Medicare, integrated health care systems like Kaiser Permanente in Northern California, and the Rochester Epidemiology Project at the Mayo Clinic in Olmsted County, Minnesota, where they effectively have every person diagnosed with PD in that county.

We pulled all this data together and used it to calculate prevalence across different age ranges—for example, ages 45–54 and 55–64. What was really interesting is that despite different methods of data collection, the numbers almost all lined up. They were slightly different, but essentially the same. So we had a lot of confidence in the estimate we generated.

As I mentioned, it’s not the final word. We still recognize variation in the United States. Some states have more people with PD than others. It doesn’t appear to be a geographic hot spot like you might see with cancer, but it is not evenly distributed either.

But the point is we now have a number upon which to speak as a community. That number is nearly 1 million people by 2020 who will have been diagnosed with Parkinson’s disease. That represents a floor—we can say with confidence at least this many people have PD, if not more. And part of that “if not more” comes from those who have not yet been diagnosed.

That remains a problem—getting accurate diagnosis quickly—and also reflects disparities in access to care, which is central to our mission: improving care and quality of life for everyone with PD.

Dan Keller 7:21
So I suppose knowing the more real number than you had before, and the distribution of the disease throughout the country—if there are geographic differences—would affect resource allocation and funding and things like that.

James Beck 7:38
Absolutely. These are important numbers upon which to build a case to talk to members of Congress, the federal government, and state governments to say Parkinson’s disease is bigger than we thought, and we have numbers upon which to stand to defend that case. We need to allocate more resources to it.

Even with imperfect studies estimating the economic burden of Parkinson’s, we are working with another organization to improve that number as well. But using these updated prevalence numbers with older cost estimates suggests almost $20 billion a year in societal costs.

The federal government, in turn, invests roughly $160–$180 million a year in Parkinson’s research—about 1% of the total cost. Even doubling or tripling that would make a tremendous difference for researchers and clinicians working on Parkinson’s disease.

It could help bring more scientists and clinicians into the field. It could also help insurance companies and other organizations prepare for increasing numbers of people with PD and ensure appropriate care services are available.

Dan Keller 9:04
Is that going to be the major benefit to people with Parkinson’s—that resources may be better allocated and more research will go on? Or are there other benefits you see right now?

James Beck 9:17
I think this is probably more of a policy benefit rather than a direct practical benefit, although there is strength in knowledge. For people with PD to know they are not alone and to have confidence in these numbers can be empowering. It allows them to be their own advocates for change.

Dan Keller 9:47
Now that you have good numbers in hand, do you have next steps as part of this project?

James Beck 9:52
Absolutely. Prevalence is one leg of a stool. Another important number is incidence—how many people are diagnosed each year. We have rough estimates of 40,000 to 60,000 per year, but we want a better estimate using the same datasets and researchers.

The other side is mortality—whether people with PD die sooner or live a full lifespan. This is difficult but important to understand so we can measure progress, including whether we are improving diagnosis rates or slowing disease progression with new therapies.

This study will be published in an open-access journal, NPJ Parkinson’s Disease, in partnership with Nature Research. Open access means anyone can download and read the article for free and review the data themselves.

Dan Keller 11:39
Good. Thank you.

Dan Keller 11:50
To find out more about the Parkinson’s Prevalence Project, including state-by-state estimates and other findings, go to parkinson.org and search “prevalence.” You can also call our toll-free helpline at 1-800-4PD-INFO to speak with PD information specialists.

You can also leave feedback at parkinson.org/feedback.

At the Parkinson’s Foundation, our mission is to help every person diagnosed with Parkinson’s live the best possible life today. We bring you a new episode every other week.

For more information, visit parkinson.org or call 1-800-473-4636.

Thank you for listening.

James Beck, PhD, is the Parkinson's Foundation Chief Scientific Officer and has led the scientific affairs of the foundation since 2008. Dr. Beck oversees the foundation’s research strategy and programs, including management of grants that support research centers, individual investigators, fellows and collaborative projects. In his role, he has changed the parameters of investigator-initiated grants programs to provide additional funding and multi-year support to Parkinson’s-focused scientists; expanded funding for early-career Parkinson’s researchers, established Conference Awards and instituted the inclusion of Parkinson's Foundation’s Research Advocates in the foundation's own programs. Dr. Beck is a member of the Forum on Regenerative Medicine of the National Academies of Sciences and the Udall Center Coordinating Committee.

Dr. Beck is currently an Adjunct Associate Professor in the Department of Neuroscience and Physiology at the New York University School of Medicine. Previously, he taught at Long Island University as Assistant Professor in the Biology Department. Additionally, Dr. Beck was a recipient of National Institutes of Health (NIH) funding and has been published in journals, such as the Journal of Neuroscience, Journal of Neurophysiology and Journal of Comparative Neurology. His research focused on the neural control of vertebrate motor systems and behavior.

Dr. Beck holds a B.S. from Duke University and a Ph.D. from the University of Washington, Seattle. He completed his postdoctoral training at the New York University School of Medicine. Dr. Beck frequently comments in the media about the implications of the latest Parkinson's science headlines.

Dan Keller (00:08)
Welcome to this episode of Substantial Matters: Life and Science of Parkinson's. I'm your host, Dan Keller at the Parkinson's Foundation. We want all people with Parkinson's and their families to get the care and support they need. Better care starts with better research and leads to better lives. In this podcast series, we highlight the fruits of that research, the treatments and techniques that can help you live a better life now, as well as research that can bring a better tomorrow. Over the past several decades, new treatments have been developed that can help control the symptoms of Parkinson's, but no therapies so far have been able to slow or stop its progression. Every episode of this podcast, we say the fruits of research are the treatments and techniques that will help you live a better life today and in the future. Dr. Tanya Simuni of the Parkinson's Disease and Movement Disorder Center at the Parkinson's Foundation Center of Excellence at Northwestern University Feinberg School of Medicine in Chicago describes some of the current research and why she thinks these potential new drugs are promising. However, she says the tree can only bear fruit if people with Parkinson's are willing to participate in clinical trials. Dr. Simuni discusses a couple treatments that are currently in phase three trials. This means they have already been tested for safety and are being tested in larger study groups to see how well they work. One she mentions is the drug isradipine, which is already approved as a blood pressure medication. The other is inosine. While there are many treatments to reduce the impact of Parkinson's symptoms, the hope is that these new therapies might slow disease progression.

Dr. Tanya Simuni (02:07)
Parkinson's, compared to other neurodegenerative diseases, compared to Alzheimer's disease, is really, on the one hand, privileged, because we have a larger armamentarium of medications that treat the symptoms. We can improve functionality of the patients. We can improve their functional capacity. However, what remains the holy grail of Parkinson's is finding the intervention that would slow and ultimately stop the progression of the disease, and that has been the hottest area of research for years. And specifically now

Dan Keller (02:47)
You carry out trials, you coordinate them, you design them. What's coming along?

Dr. Tanya Simuni (02:52)
So this is an exciting time in Parkinson's experimental therapeutics, and I will position the studies in the phases of the development. Phase three are the studies that were vetted out in the earlier phases of the development and the studies that are asking the ultimate question: is the intervention efficacious? So as of today, there are two disease modification studies being carried out. One of them, with a full disclaimer, I'm fortunate to spearhead, is the drug called isradipine. It is actually a drug that has been approved for management of elevated blood pressure for years and has been found in the lab and in the animal models of Parkinson's to be neuroprotective against development of Parkinsonian features. The mechanism of neuroprotection has nothing to do with reduction of the blood pressure, but has to do with the presence of unique channels present, largely on the cells that are susceptible to Parkinson's pathology, and reducing the activity of those channels translated in a neuroprotective effect in animal models without causing negative implications. That is the data that led to the launch of the early phase studies. We completed a phase two study that demonstrated that the drug had the parameters sufficient for safety to move on to the phase three study. And the phase three study now is in the third year of its development, and we will have the readout of the data in two years. It remains to be seen. The other phase three study is spearheaded by Dr. Schwarzschild from Mass General, and the drug that is being tested is inosine, based on the observation in large epidemiological studies that people with high levels of urate—and urate is one of the most potent antioxidant chemicals in the body—have both lower risk of development of Parkinson's, and if they develop Parkinson's, less of the rate of the progression of the disease. So the team led by Dr. Schwarzschild also conducted earlier phase studies, a phase two study demonstrating acceptable safety of moving on with the study into phase three, and that study just started recruitment of the participants a couple months ago. So as a clinical trialist and as a clinician treating a lot of patients with Parkinson's disease, I cannot resist putting a pitch into the fact that people with Parkinson's should participate in clinical trials. That's the only way for us to find better therapeutic options and ultimately, hopefully, the cure for the disease. So these are the phase two studies, moving on into the earlier phases of the development and jumping all the way into the phase one studies. Probably the most exciting target for the development are the molecules that are approaching the abnormal protein associated with Parkinson's disease. So some of the listeners probably have heard about synuclein-targeted therapies. Synuclein is that protein that is present in every person, but in the Parkinsonian state, it gets clumped, it gets misfolded, and it is believed that that misfolding of the protein and cells having trouble clearing that protein from the cells contributes to the mechanism of the development of Parkinson's and progression of the disease. So there are a couple companies around the world that are developing vaccines—immunization, simplistically saying—against that synuclein. These are early phase development studies, but definitely the studies that are targeting the underlying biology of the disease. All of us are certainly very hopeful that they will be ultimately successful.

Dan Keller (07:11)
In the later-stage trials that you just described with the urate pathway and isradipine, what are the primary endpoints? What's the outcome you're looking for—limiting progression, fixing symptoms?

Dr. Tanya Simuni (07:25)
So these are the studies to slow progression of the disease. So that's the primary readout. That's the primary objective of the study. How do you read out progression of Parkinson's disease? You use what we call validated, reproducible clinical scales in the field of Parkinson's. As of 2016, we do not have objective measures in the form of blood sugar level or cholesterol level to apply an objective variable for the progression, so we use clinical outcome measures, and those will be the readouts of both of the studies.

Dan Keller (08:04)
So you're really looking at the severity of the disease or the clinical symptoms.

Dr. Tanya Simuni (08:10)
Ideally, you want to have the happy marriage of both. Obviously, for the phase three studies, in order for any regulatory agency to approve the molecule, you need to have a readout that is clinically meaningful. So the clinical outcome—the role of biomarkers, and that is an extremely hot word in neuroscience in general, and Parkinson's is an example of that—is that they provide the biological readout whether the clinical improvement was triggered by and linked to underlying improvement of the biology, or whether we are improving compensatory mechanisms of the body, which is no less significant for the clinical outcomes but gives us the other part of the story. The other significance of biomarkers is that they're very important in early phase clinical development because they allow us to separate the winners from the losers much faster than tracking the clinical outcomes that take time.

Dan Keller (09:13)
How long does it take, say, in one of these phase three trials, to accumulate enough symptoms or on a rating scale to see a difference, to know whether the drug is working or not? It seems like a long study.

Dr. Tanya Simuni (09:26)
Again, disease modification studies are looking not at the immediate improvement of the symptoms, but rather at slowing progression of disability. And obviously, in a disease that spans a decade plus, an immediate readout three months from initiation is not going to give us the answer to the story. So the majority of disease modification studies in Parkinson's disease span at least a year, some of them two years. The study of isradipine actually is the longest to be conducted study of disease modification in newly diagnosed people with Parkinson's disease. The duration of that is three years of exposure for every participant. And the reason why we've done that is because it's important for us, if the molecule shows benefit, to have at least a readout over a three-year period of time. We understand that it's not 10, but that if there is a benefit, it is sustainable.

Dan Keller (10:32)
How encouraged are you at this point, from what you see in clinical trials, various pathways, various approaches, that you're going to see really meaningful improvements in therapy, say, within five, six, even 10 years?

Dr. Tanya Simuni (10:46)
You know, if you asked me that question five years ago, I would have said I am cautiously optimistic but will not promise you the timeline. The field has changed remarkably in the last couple of years. A number of pharma companies are coming into the field. There is really exciting research. There is common understanding that Parkinson's is not one disease, but an umbrella under which there are kinds of different ethnicity, different behaviors, so to say, and that we really need to start thinking of personalizing our interventions to different members of the constituency. And there are companies that are stepping into that arena. So really optimistic—cautiously, but really optimistic.

Dan Keller (11:42)
Anything we've missed—interesting to add, important?

Dr. Tanya Simuni (11:46)
Again, I would strongly encourage people affected by the disease to take an active role in management of their disease, meaning a positive outlook, meaning participation in clinical trials, meaning a healthy lifestyle. Everyone with Parkinson's has heard about the role of exercise, should hear about the role of exercise, and really should make that part of daily living. If we're talking about disease modification, animal studies are very convincing on the role of physical activity. In humans, it's more difficult to prove it, but common sense always prevails—you will be in better shape, you will be able to sustain the disability related to the disease better.

Dan Keller (12:35)
Great. Thank you.

Dr. Tanya Simuni (12:46)
If you would like to find out more about what's in development for Parkinson's, or if you want information about finding and participating in a clinical trial, call our toll-free helpline at 1-800-4PD-INFO. Many clinical trials involve medical centers around the country, so there may be opportunities near you. Dr. Simuni mentioned early phase studies that target the protein alpha-synuclein, which is known to be involved in Parkinson's. We talked about these so-called Parkinson's immunizations with Dr. Mark Gutman, so check out our podcast episode of vaccine for Parkinson's. If you have any questions about the topics discussed today, or if you want to leave feedback on this podcast or any other subject, you can do it at parkinson.org/feedback. We'll respond to some questions in future episodes. At the Parkinson's Foundation, our mission is to help every person diagnosed with Parkinson's live the best possible life today. To that end, we'll be bringing you a new episode in this podcast series twice a month like this one. Our next couple of episodes will focus on research, what's new in clinical trials, and why it's important for you to get involved. Till then, for more information and resources, visit parkinson.org or call our toll-free helpline at 1-800-473-4636. Thank you for listening.

Tanya Simuni, MD, is an Arthur C. Nielsen Jr. Professor of Neurology and Director of the Parkinson’s Disease and Movement Disorders Center at Northwestern University Feinberg School of Medicine in Chicago, Illinois. She also serves as Residency Program Director in the Department of Neurology.

Dr. Simuni graduated with her medical degree from Leningrad Medical School and completed an internship in medicine in Leningrad, Russia. She subsequently completed an internship in internal medicine at Albert Einstein Medical Center and a neurology residency and a clinical neurophysiology fellowship at Temple University in Philadelphia. She remained in Philadelphia to pursue a movement disorders fellowship at the University of Pennsylvania, where she served on the clinical faculty for three years and was Medical Co-Director of the Parkinson’s Disease and Movement Disorders surgical program. She is board certified in neurology and clinical neurophysiology.

Dr. Simuni joined the faculty of the Northwestern University in 2000 to build and lead a multidisciplinary movement disorders center that is now a Parkinson’s Foundation Center of Excellence and serves as a training model in the region. She is currently the investigator of more than a dozen PD clinical trials, one of which is a multicenter, phase 3 clinical trial to evaluate isradipine as a potential disease-modifying agent in early PD. Her works have been widely published in peer-reviewed scientific journals and she has lectured nationally on PD and other movement disorders.

Dr. Simuni is an active member of the American Academy of Neurology, American Neurological Association, Movement Disorders Society, Parkinson’s Study Group, Restless Legs Syndrome Study group, and REM Behavior Disorders Study Group.