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1 Michael J Fox: [00:00:30] [00:01:00] [00:01:30] [00:02:00] [00:02:30] [00:03:00] [00:03:30] This is Michael J. Fox. Thanks for listening to this podcast. Learn more about the Michael J. Fox Foundation's work and how you can help speed a cure at michaeljfox.org. This is Dave Iverson. When we think about medical research, certain images often come to mind. Laboratories, expensive equipment, and time-consuming experiments that only white-coated scientists can perform. But a new Michael J. Foundation research project called Fox Insight aims to change that picture. The goal of the online Fox Insight study is to harness the power of the patient voice by getting tens of thousands of people to record what it's like to live with Parkinson's. And in so doing, help inform and perhaps even reshape clinical research. Dr. Katie Kopil is Director of Research Partnerships at the Foundation and leads the newly-launched Fox Insight effort. We sat down recently to talk about what Fox Insight is and what the Foundation hopes it can accomplish. We always think about needing data to solve problems. And traditionally, we think of it being like scientists collect the data. The data comes by way of scientific observation or experiment. How does Fox Insight in some ways turn that on its head in terms of who's collecting the data and what that data is? That's one of the powers of having a platform like Fox Insight that can engage tens of thousands of people to contribute self-reported health data. Data about quality of life, about symptoms that may be variable from person to person. Those are considered data and they become very valuable when you have the statistical power of a cohort, a group of people, on the scale that Fox Insight captures. That data then informs people about the prevalence of Parkinson's disease, the prevalence of particular symptoms that might occur within Parkinson's. And for someone contributing data to Fox Insight, they might think, "Well, I didn't measure this with a ruler. I didn't measure this with a microscope. I'm just telling you about my day." But there are actual tools and instruments, surveys, questionnaires, that are scientifically validated as a way to capture patient-reported outcomes, patientreported symptoms to actually turn the patient voice into a science. And turn that into meaningful data for our researchers. That patient data is all collected online. Participants receive regular survey questionnaires that pose a diversity of questions about life with Parkinson's. There are a variety of questions that are asked routinely, over time, in study visits within Fox Insight. One example is a physical activity scale that's asking participants how often do you go for walks during the week. How often do you exercise? Are you able to maintain the physical activity that you had previously? That type of survey response would allow researchers to say, on individual levels, how active/inactive people are. And you could extrapolate from that an understanding of someone's mobility limitations, the physical impairments that someone might be experiencing. When you apply the scale of Fox Insight, you can look for differences

2 between people that have Parkinson's disease, people who don't that are of similar ages, and understand then what the impact and burden of Parkinson's disease really is around physical function and activity levels. [00:04:00] Other questionnaires get about the frequency of non-motor symptoms, asking about within the last month, have you had issues with sleep. Have you had issues with over-production of saliva? There's a scale and a factor of looking at multiple people across time, looking at people's other type of health information that's reported within Fox Insight that allows meaningful insights on a whole. [00:04:30] [00:05:00] [00:05:30] [00:06:00] [00:06:30] The science painting a picture of what people are experiencing, Fox Insight aims to assess which Parkinson's symptoms are most bothersome, and use that data to influence how the Foundation allocates its research dollars. I think one of the activities that our in-house research team at MJFF is interested in looking at Fox Insight data for is to understand whether the investments that we're making in certain disease areas, symptom areas, are reflective of how burdensome those symptoms actually are for patients. Being able to look at not just a small group of people on the order of hundreds to say really how burdensome is this issue of constipation or gait, but to look at the scale of tens of thousands of patients and to say, "Oh, this is where the real issue is and we should invest the most of our research dollars in that areas to solve a meaningful problem for patients," is one way that the data on symptoms could be utilized. Let's take that as an example, because you just mentioned two symptoms for which there's sometimes not great solutions. If you can fast forward this for a moment, Katie, and imagine that you've now collected all this data and you learned something about the prevalence of constipation or the prevalence of falls or whatever it happens to be. How does that in turn then have a potential impact on the development of new treatments or things that would assist people in contending with those particular problems? At a high level, I think understanding the frequency of those symptoms within the patient population will incentivize research dollars, either from the Foundation, or other groups, pharmaceutical companies, biotech, investors, to prioritize those areas that really have a high unmet need for patients because that is an opportunity to come into Parkinson's disease and solve a problem that no one has yet solved. And I think that's really important in terms of driving research investments. Is some ways part of the goal of this to shine a bright light on something that might not have been understood before? That through the collection of all this data, it will provide insight, literally, given the name of the project, to something that might not have been understood. And therefore, change perhaps the way in which that problem is looked at, change perhaps the way in which we could find solutions for it.

3 [00:07:00] [00:07:30] [00:08:00] [00:08:30] [00:09:00] [00:09:30] [00:10:00] That's beautifully put. And I think that's the goal... And it's not researchers working in isolation. It's not just one person contributing data. It's about an ecosystem of bringing tens of thousands of volunteers to contribute that data, attracting researchers to understand those insights, and then thinking about the applications for other stakeholders in Parkinson's disease, which include regulators like the FDA, like payers and insurance companies, and health care providers to understand truly the holistic experience and the lived experience of Parkinson's disease. Does that also mean that part of what you're doing is making it clear in kind of a data-driven way what it's like to live with Parkinson's? What the burden of that can be? That is, at its core, the value of Fox Insight for researchers is to understand what the lived experience is of Parkinson's disease. And that's true for people with Parkinson's, and it's true for their loved ones and their care partners. And it's important to note that while a lot of the Fox Insight material talks about symptoms of Parkinson's disease for the patients, there's an important component of Fox Insight that's also going to be probing the ways in which care partners are affected by Parkinson's disease. Whether that is emotional burden, financial burden, or otherwise, that there's a whole separate component that is relatively understudied about the burden of Parkinson's on the person's family at large. In terms of who you want to participate in Fox Insight then, it's not just people who have Parkinson's? It's also their partners, their spouses. So is there also a need for controls? Who else should participate, I guess, in this effort? Fox Insight is open to anyone over the age of 18 that's able to complete, have internet access, and is English-speaking. And we would love to see anyone that has an interest in helping speed a cure and speed better care delivery for people with Parkinson's participate in this. There's value in controls. Sometimes people call them healthy controls, but we're actually interested in people that don't have Parkinson's, that are of similar ages as people that have Parkinson's. So people that might be 60 and older because Parkinson's is not the only disease that people usually are living with. I think it's important to understand that in the holistic sense of someone with Parkinson's might also have diabetes, might have cardiovascular issues, might have asthma. And to understand how the quality of life of those people are changed by Parkinson's compared with people who might also have asthma or diabetes or cardiovascular disease, but don't have Parkinson's is a really important contribution of data from control volunteers, in addition to unique insights that people might be able to provide if they're also closely connected and providing care for someone with Parkinson's. How would you measure success? What would, to you, constitute Fox Insight being a successful project? Is it that it would help speed a cure? Is it that it would help provide better care for Parkinson's patients? Is it that it would help generate, as we've been discussing, a broader understanding of what it means to live with it? Is

4 it all of the above? [00:10:30] [00:11:00] [00:11:30] [00:12:00] [00:12:30] [00:13:00] It's all of the above. And I'll take any of them. One place that I'm extremely excited to see improvement of care, because that can be defined a number of ways, is making Parkinson's care more patient-centric. There is a study that's upcoming, one of these surveys that will be deployed through Fox Insight by an investigator at the University of Toronto named Dr. Connie Marras. Dr. Marras is interested in understanding the ways in which patients and their care partners talk about the experience of off, when medications wear off. Physicians call that many things. Off is one of them. Motor fluctuations is another. But rarely are those terms that are used by patients, or rarely are those terms that are used by care partners. People might say, "My medications aren't working. I'm having a bad day." And being able to use Fox Insight to capture data at scale about the ways in which people talk about the symptoms that they experience when their medication wears off would allow for a dictionary, a lexicon, that could bridge the gap between the way patients and care partners talk about Parkinson's and the way their care providers talk about Parkinson's. So it would be a way of matching up here's what patients are saying about the lived experience, here's perhaps how scientists or clinicians tend to define it, and trying to bridge the gap between those two means of communication? Exactly. And understanding how big or small that gap is today and identifying opportunities, tools for improving and narrowing that gap. There's been a kind of trial fun of Fox Insight going now for some time? I've participated in it, many other people with Parkinson's diagnosis have participated into it. What have you learned from that trial run so far? Have there been new insights that have developed from it? One of the most valuable lessons we have learned through the pilot phase, this very large pilot phase of Fox Insight, is the importance of mixing very wellcharacterized defined tools for assessing patient-reported outcomes with novel approaches. And there have been very important feedback from people that have been using Fox Insight that actually totally changed the types of questionnaires that are being included in the study to ensure that they are appropriately measuring what we think they should be measuring. And a good example was feedback we had gotten around a question that was assessing whether or not someone had nighttime urination or urgency around needing to urinate in the evening and whether people thought it was linked to their Parkinson's. And talking and getting feedback from people that have taken these surveys, and someone said, "Well, I do have that but I think it's related to a prostate issue and not my Parkinson's, so I'm going to say no, I don't have that

5 [00:13:30] symptom." Well, that doesn't actually help researchers if we're trying to have everyone respond to the question in the same way. And so we have learned that it's really important to capture data in a standardized rigorous fashion that has been tested so that people understand the meaning of the question. And so that has led to an evolution in the types of questions that are being captured in Fox Insight to make it more valuable for researchers going forward. [00:14:00] [00:14:30] So does that mean you rephrase the question? You don't ask a question like, "Do you have problems with nighttime urgency to go to the bathroom?" You change it somehow? And how so? Yes, in that particular instance, it was asking people if they had that symptom related to Parkinson's disease. And we've realized that it is important, and these are lessons learned from other fields as well, that it's important to not ask people to attribute it to their Parkinson's. And I think this comes up a lot where now in research, we know, for example, things like constipation, loss of smell, or maybe early signs of Parkinson's disease. And people have experienced them, but thought, "Oh, I didn't realize it was part of Parkinson's." And if people don't identify those symptoms because they think, "Well, it's because of a different reason," those insights get lost and get missed for a long time. So a lot of the questions that are being asked now in Fox Insight are being asked just within the last month, a time-bound period. Or have you ever experienced this? But not asking individuals to attribute it to Parkinson's or not. And that also makes the data valuable for control volunteers as well. [00:15:00] [00:15:30] [00:16:00] [00:16:30] One thing you mentioned a moment ago, Katie, was this idea that a researcher from elsewhere is interested in exploring this one question of what does it mean to people who are not clinicians, who are experiencing so-called off episodes. How do they characterize it? How do they describe that? Is part of the value of Fox Insight that you're going to have thousands of people signed up for this? And then from time to time, not only are they going to ask a regular series of questions about whatever symptoms they're experiencing in life with Parkinson's, but you will have these occasional specific surveys that are designed to capture information about one particular problem. It solves a lot of survey problems in a way because you have this ready-made group of people who you can say, "You want to find out about sleep problems in Parkinson's? We can send that question out to 8,000 people." That's exactly right. And one of the things we are excited about, the potential for Fox Insight is to speed research timelines. That many investigators are interested in asking these questions, and either don't have the resources or time available to recruit at a scale that's necessary to answer those questions. And by having a platform that is available for researchers to ask the Parkinson's community, ask care partners, about surveys they have designed for their own specific interests. We can shorten timelines for getting answers to those questions so that people can

6 move onto the next important finding. [00:17:00] [00:17:30] [00:18:00] [00:18:30] [00:19:00] [00:19:30] [00:20:00] One more thing I wanted to ask you about, which is, and I don't know if Fox Insight helps solve this problem or not, but I was intrigued yesterday when I heard Todd Sherer, the Foundation's CEO, make the point that increasingly we need people to participate in research who might fit a particular profile. That as medicine becomes more precision-oriented and personalized, we realize that we're going to have to solve a variety of Parkinson's conditions. So we'll need someone who has a particular genetic mutation. Or we'll need someone with a certain set of symptoms. Or maybe someone who's been exposed to a particular pesticide, whatever it happens to be. Does Fox Insight help us deal with that at all so that you have... You know because you have this big database, "Well, as a matter of fact, we have 300 people who have this particular mutation and so we can hone in on them." I think the precision medicine angle, this idea that medications can be tailored to someone's type of Parkinson's disease, or their holistic biology is important for Fox Insight and it's a way that we think Fox Insight could advance research. And there are two main aspects of Fox Insight that I think can help us get to that idea of personalized medicine. One is the genetics component that is now available through Fox Insight, which is the collaboration with 23andMe where individuals can remotely contribute their DNA for genetic testing and their genetic results can be included alongside their self-reported health information. That matched with health data, self-reported data, symptom data, not just from 10 people, but from 14,000 people, thinking even larger, that's where you can start to have those insights. Because in many historical studies, the scale of the genetics in terms of number of people against the health-reported information are just too narrow. I think there's another important factor with Fox Insight being remote, that people can participate in the study anytime, anywhere. And that opens up, relieves restrictions that often are part of research participation. I think that there are challenges with making broad interpretations of clinical trial data to the entire Parkinson's population because most people that participate in research are living close to an academic medical center, and they have the flexibility in either time or mobility to actually participate in research. And by having an online study and having patients and other volunteers contribute data easily, at their own convenience, it really enables a broader understanding of PD epidemiology that people living in the middle of Nebraska, who might not be able to participate in studies otherwise, their data are being captured. I think it's really going to be exciting to see a broader section of what Parkinson's looks like than what we have been able to capture historically through research participation at clinical sites. I think that's such an interesting point because I often think about, and just musing about this or speculating about this, that I wonder if sometimes the same people who come to Parkinson's conferences or who participate in Parkinson's activities,

7 [00:20:30] are the same people that participate in research. We keep testing out things on the same people essentially. And that we're missing often a broader swath of the Parkinson's community. You see that at conferences that are so often largely white upper middle class populations. We have a lot of work to do in that regard. And it seems to me, one exciting thing about this is that it essentially makes research more democratic in the best sense. It's a broader indicator of who has this condition. [00:21:00] That's perfectly stated. The Fox Insight study has the opportunity to capture the broader swath of people. And there's an interest in Fox Insight recruiting individuals who are under-represented in research. And as you articulated, those people are typically of lower education status, lower socioeconomic status, in a geographic white space, so to speak, in the middle of the country, not near major urban areas, people that are older. Traditionally, most people that participate in research skew younger. [00:21:30] That by removing some of the inherent barriers to research participation through an online study, we can capture a more representative picture of Parkinson's disease. [00:22:00] Michael J Fox: That's Dr. Katie Kopil, Direct of Research Partnerships at The Michael J. Fox Foundation. She'll join us, along with Parkinson's researcher, Dr. Roseanne Dobkin and Parkinson's patient, Kevin Kwok on our next third Thursday webinar, November 16, when we focus on the Foundation's Fox Insight initiative. To register, visit michaeljfox.org/webinars. I'm Dave Iverson. This is Michael J. Fox. Thanks for listening to this podcast. Learn more about the Michael J. Fox Foundation's work and how you can help speed a cure at michaeljfox.org.

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