A Conversation with the Chief Science Officer of the Michael J. Fox Foundation

CM:
I know that you lead a team of scientists and researchers with the goal of funding an aggressive research agenda and pushing forward the agenda, and you also have some scientists and PhDs on staff. Can you talk to me about why the foundation is structured that way and how that helps you fund your agenda?

MF:Sure. So we started hiring PhDs on staff around 2004, a couple years after we were founded. And one of the reasons was to have some peer-to-peer interaction from the foundation to our awardees, our grantees. We really see ourselves as not just a check writer, but a strategic partner and an intellectual partner in the research that we fund. And so having PhDs on staff really allows us to hold our awardees accountable to milestones and deliverables that are a part of each grant, but also it provides some support to our awardees in that we see a lot of different research all over the world. We see over a thousand different ideas of research projects, and we're funding a lot of research.

And so through our interactions with our grantees, we're able to problem-solve with them and connect certain dots that they may not be able to connect individually because we have this network of research that we fund all over the place. And so we problem-solve with our awardees. We help make connections and introduce them to other grantees that may be good collaborators for them. And so it's really more of a partnership, and having that peer-to-peer relationship, I think, fosters a really nice collaboration.
CM:That's great. And you joined the foundation in 2006, so you've really seen the growth leading up to 2022, 2023. Can you take me through some of the evolution in terms of the science and where you are today?
MF:Sure. In the last 20 years, the science has really exploded, and it's remarkable to think about the progress that has been made. There's been a number of genetic discoveries that have really uncovered what happens in Parkinson's disease, what's really causing Parkinson's disease. And that has unlocked a flood of research to not just understand the disease better, but it has identified specific targets that can be prosecuted and pursued for new treatments, new treatments that not just help the symptoms and reduce tremor or stiffness, but actually treatments that we think might slow or stop the progression of the disease, and ultimately, result in a cure. So the progress in the science is remarkable, but also that has led to more and more investment, not just from the foundation, but other partners like from pharma, biotech and government. So it's a really exciting time in Parkinson's research.
CM:That's amazing. And I know you work closely with biotech and pharma on a lot of projects. What types of research are you currently funding now?
MF:Well, I would say there are two general categories. One is what we call translational research, and this is a real gap in the field. And that is taking the discovery that happens in a laboratory, mostly in universities, and translating that into meaningful treatments. And there's a real gap in funding and intellectual strategic thinking to do that translational work. So that is one major area that we support. We fund both academic biotech and pharma partners to really do that, focus on that translational research.

The other area is tool development, and we see this as a real opportunity for us and a goal for us that will develop and fund tools that will help all different therapeutic approaches. And these are things like laboratory tools, antibodies, maybe animal models, but also developing tools like measurements and biomarkers that can help diagnose Parkinson's more precisely and track the progression of Parkinson's disease. So these are things like brain imaging techniques or blood markers or fluid markers. And so those tools really can lift all boats regardless of the actual therapeutic that's being developed. And so it's both tools and translational research.
CM:And I know that discovering the biomarker is a priority for the foundation. Can you give me a little bit more background of exactly what that means and why you think it will really change the research field?
MF:Sure. So currently the way that Parkinson's is diagnosed is really through a neurologist's evaluation of someone's symptoms. So they do certain tests to measure tremor, measure their movement, measure their symptoms, their stiffness. And because Parkinson's can change from person to person and from hour to hour, those symptoms, especially early in the disease, are really difficult to track and diagnose as Parkinson's. As we age, there are a lot of things that change, and we all slow down. And so the diagnosis of Parkinson's disease can be very difficult and the journey to a diagnosis can be very long and have multiple different opinions. So having a more objective marker like a brain scan or a blood test in the way that we use cholesterol in heart disease would be a more precise and better way to diagnose Parkinson's disease so people would be diagnosed earlier and potentially be treated earlier with medicines that work.

The other area that biomarkers are really important is tracking change over time, so progression of Parkinson's disease. And a misconception about Parkinson's disease is that everyone experiences it the same. But symptoms can really vary from person to person, and people may progress faster or slower depending on their Parkinson's journey. And so it's very difficult to really track how someone is progressing through the different stages of Parkinson's disease. And so having a more objective marker, like a blood test, to know how the disorder is changing over time would really allow doctors and researchers to understand whether treatments are actually stopping the progression and doing what we hope these new treatments will do, which is to slow the progression of the disease.
CM:Absolutely. And I know it's so important to the foundation to get donor dollars immediately deployed to research projects. How many research projects are you currently funding?
MF:We're funding about between four and 500 different research grants. We fund research all over the world. We're really agnostic to where the science and where the research happens. As I mentioned, we fund biotech companies, pharmaceutical companies, universities, government labs, and it's really about the idea and the science and regardless of where it happens or who's doing it. And so all of our projects are what we call hypothesis-based, and they have a beginning and an end, and they have specific milestones and deliverables that we expect to be met throughout the course of the grant. So it is a robust portfolio. Our team is very busy managing all of these projects and finding new research to fund.
CM:That's amazing. One of the things that I think is troubling for everyone to hear, of course, is the impact of the pandemic on supply chain issues, I imagine scientists being deployed to other areas, et cetera. So can you just talk me through the impact that it had directly on your research and maybe how you were able to overcome some of that?
MF:Yeah, it was a huge impact. It had impacted everything in our lives. From a research perspective, the major issue was the people resource, that researchers really couldn't go to the laboratory to run their experiments, and people living with Parkinson's couldn't go to the hospital to participate in clinical trials that were testing new treatments. And so this slowed down the progress of a lot of our grantees.

What we did was essentially to award them what we call no-cost extensions. So we kept the money, and our money is tied to meeting specific deliverables, and then we allowed them additional time, usually about six months, to finish the project, and then we allocated the money and the funds as deliverables were met. So allowing this time, these six to 12 months of an extension, really enabled the researchers to kind of find solutions to the challenge that the pandemic was introducing, figure out protocols for individual staff to work in laboratories, figure out virtual visits for people living with Parkinson's to have their visit not in the hospital but over Zoom or using the computer.

So this actually fostered a lot of innovative approaches and some that we still use today. For example, we have many more virtual meetings where we bring in scientists to present their work, whereas we used to invite them to come to New York and present for a day. And so this has actually fostered a lot more collaboration, sharing of information more rapidly and actually accelerating progress based on this sharing.
CM:That's great. So a little bit of good out of a difficult time.
MF:Absolutely, yeah.
CM:In terms of the research itself, what area do you think is currently most underfunded?
MF:I think it's the translational research. So again, this is taking an idea that typically a university researcher professor might have, an aha moment, if you will, and translating that into therapies and treatments that actually make an impact. There's a big gap, many, many steps that have to happen, many, many different expertise that are needed from chemistry to biology to clinical trial experience. And that translational gap is really underfunded. And this is where I think we are poised to use the patient capital, the donor dollars from people living with Parkinson's and people that care about Parkinson's, really to accelerate and reduce that translational research gap.
CM:So the foundation is then filling a void that perhaps the government or pharma companies would be uncomfortable necessarily jumping in right away. They want to see more proof of concept and the foundation's able to move it to that stage.
MF:Yes, and we use the term de-risking a lot within our team. And what we mean by that is to really lower the risk of investment for groups like pharmaceutical companies or even the government to invest in Parkinson's disease. So if we can fund that one experiment that will make it more attractive, make Parkinson's more attractive to drug developers, we'd like to do that. We often say we're in the silver platter business because we really want to tee up Parkinson's disease as an attractive opportunity for pharmaceutical companies to develop new treatments. Because really, ultimately, we can fund a lot of research, but to develop new treatments, it takes billions of dollars just for one new therapy, and we really need biotech and pharma as partners to really invest in Parkinson's research.
CM:Absolutely. What would you say is the biggest misconception about Parkinson's?
MF:Yeah. I mean, there are a lot of misconceptions, and I think if I had to choose one, it's that everyone experiences Parkinson's the same way. So typically Parkinson's is thought of as a movement disorder, which it is. So people experience tremor and slowness and stiffness. But what's unique about Parkinson's disease is that people might have many other symptoms, maybe sleep changes, smell loss. Some people experience depression, some people have some cognitive impairment. And it really is this snowflake disorder that if you've met one person with Parkinson's, you've met one person with Parkinson's. And so everyone experiences it differently. They progress differently. What their symptoms are are very different. And so this is a real challenge when developing new treatments because it may be different for every person.
CM:What would you say has been the biggest win for the foundation so far with respect to the research?
MF:Well, I can say that we've de-risked a lot of science, and we are now seeing a robust portfolio and pipeline of new treatments that are marching through clinical testing now. So I think that is one win. But I would also highlight a study that we funded called the Parkinson's Progression Marker Initiative. It's a mouthful, PPMI for short. This study is focused on developing better ways to measure Parkinson's disease, so developing better biomarkers. It's happening at 50 different sites around the world and collecting information on people with Parkinson's disease and people without. And it's a lot of information. We collect a lot of data in this study. It's brain scans, it's clinical evaluations by a neurologist. It's digital sensor work using smartphones and smartwatches. We collect fluids and DNA and blood from all of these individuals, about 1,500 individuals around the world. There's a lot of unique things about this study, a lot of unique features about this study.

The most unique, I think, is that we make the data available in real time. So we have a web portal where researchers can access the data. Any researchers around the world can request access to the data, and the data are uploaded every week as it's being collected. We've now had over 11 million downloads of the data. The study started in 2010. What's more exciting than that is that we are actually seeing clinical trials using this data from the PPMI study to guide their clinical trials and introduce new biomarkers into their studies to make their trials more informative and improve the outcome of the trials. So this was kind of a revolutionary study when we first started it in 2010, making the data available in real time, collecting all of this data from the same individuals. And to see the interest and the impact of the study in the 13 years that it's been going on, I would highlight as a real win for the foundation.
CM:That's amazing. Mark, you've been with the foundation since 2006. Can you give me a little bit about the history of the foundation?
MF:Sure. So the foundation was started in late 2000 by Michael Fox himself. And at the time, as it still is today, the science was ahead of the money, meaning that there was a lot more science to fund and the money wasn't there. And so he founded it with the goal of accelerating a cure and ultimately going out of business, but urgently moving the funds to the researchers very quickly. And so we still do that today. We have no endowment. We have raised over $1.5 billion, and that's been deployed to research around the world. Last year we supported over $300 million in 2022 alone. And our goal is, again, to go out of business and ultimately fund a cure for Parkinson's disease.
CM:Amazing. And I would be remiss if I didn't ask you, how close do you think we are to a cure?
MF:Well, we're closer than ever, to be honest. We have funded a lot of really exciting breakthroughs that have resulted in clinical trials. They're now in early stage clinical trials. And these medicines and these treatments are really targeting what we think is the underlying cause of Parkinson's disease. There's a couple of different targets that we're really excited about, and they're in mid-stage clinical testing. And what's exciting is that we really think they have the potential for slowing or even stopping Parkinson's disease in its tracks. So we think we're pretty close.
CM:That's incredible. I want to thank you so much for joining me today. It's been an absolute pleasure.
MF:Thank you.
CM:And I look forward to speaking soon.
MF:Thanks so much.

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