Inside the National Ataxia Foundation’s Mission to Empower Families and Accelerate Treatment for a Rare Neurological Disease
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Show Notes:
In this Mission Matters episode, Adam Torres interviews Andrew Rosen, CEO of the National Ataxia Foundation, as part of the Milken Global Conference Series. Rosen shares the challenges of diagnosing and treating ataxia a rare, inherited neurodegenerative disorder and how his foundation is funding critical research, supporting patients and families, and pushing for greater awareness and federal support.
About Andrew Rosen
Andrew has an MBA in Marketing and a Master’s in International Studies and has more than 15 years of nonprofit leadership experience after a 20-year business career. He leads NAF’s strategy, oversees staff, and works closely with the Board of Directors. Having joined NAF in 2019, Andrew is continually motivated by the resiliency and caring of the ataxia community. He is honored to work with such a talented team as we get ever closer to treatments and an eventual cure for Ataxia.
About National Ataxia Foundation
The National Ataxia Foundation (NAF) was established in 1957 to help persons with Ataxia and their families. Our mission is to accelerate the development of treatments and a cure while working to improve the lives of those living with Ataxia. NAF’s vision of a world without Ataxia will be accomplished through our primary programs of funding Ataxia research, providing vital programs and services for Ataxia families, and partnering with pharmaceutical companies in the search for treatments and a cure. We work closely with the world’s leading Ataxia researchers and clinicians, promoting exchanges of ideas and innovation in Ataxia discovery.
Full Unedited Transcript
Hey, I’d like to welcome you to another episode of Mission Matters. My name is Adam Torres, and if you’d like to apply to be a guest on the show, just head on over to mission matters.com and click on Be Our Guest to Apply. All right, so today’s guest is Andrew Rosen and he’s CEO over at the National Ataxia Foundation, and today’s interview is part of our Milken Global Conference.
Coverage special where we interview attendees participants and really just top guests that we meet at, at the Milken Conference and all of the above. So first thing for Andrew, welcome to the show. Thanks, Adam. Thrilled to be with you today. All right, Andrew. So as I mentioned, this is part of our Milken Conference series, and one of my goals is to, you know, get out and get the word out there about what a wonderful conference, what the event’s like.
I, I know you were there this year. Have you been in the past years or is this a first year? Like, give us a little bit of your history with the conference. this is my second global conference. I’m actually Oh, you’re a vet. You’re a veteran now. Yeah, yeah, yeah. I’m part of a Milken program that part of their Faster Cures program.
I’m part of Leaders Link, which takes nonprofit CEOs. That whose foundations are focused on medical research and puts us together and, and lets us network. And we do kind of a capstone project. And part of that is an invitation to the global conference, which is an absolute treat, I can tell you that much.
Hmm. What’s one for those that you know, haven’t attended or have thought of attending? Like what, what are some of your standouts? Like, what are some of the reasons what brings you coming back year after year? I’m guessing you’ll be there next year. And by the way, this is my, yeah. Third or fourth year now, and it’s every year it’s automatically blocked off for the next year, like immediately.
So I already know a year in advance and we don’t really have any other conferences like that. But you know, for you, what keeps you coming back? I mean, it’s just, it’s the level and breadth of speakers, I think, mainly. So, I literally in, within about three hours of each other, I think I got to hear like my, childhood idol, magic Johnson.
Describe how he threw a no look pass to Michael Jordan in the 19. 84 Olympics, right? I mean, like live on stage. He like acted it out. Mm-hmm. And then I, stepped into a smaller session and heard amongst other speakers a professor from Berkeley, Fider OV talk about this sort of first patient ever treated with a personalized CRISPR therapy and they saved this little boy’s life.
And, and literally that broke about a week after the conference. And so I. Wow, we gotta hear directly from one of the participants. And it’s that kind of depth of, content that’s just sort of so amazing. So for me, and, and then I guess the only other thing I would say is it’s just a, it’s like a wonderful, I, you know, a lot of us we nonprofit CEOs don’t spend a lot of time on professional development.
But this was just a chance to exercise the mind. And boy, there’s no better place to do it than Milken Global, I don’t think. Yeah, it’s amazing. So speaking of let’s get into the foundation. So maybe start with a little bit of the history and background, the National Ataxia Foundation. Yeah. So so first off, ataxia just I’ll do kind of a quick description of what the, condition is.
So, ataxia is actually a Greek word that really just talks about a lack of coordination. Mm-hmm. And ataxia is, is somewhat complex ’cause it can both be a symptom of other diseases or conditions. Plus it’s its own hereditary disease, which is really the part that. NAF the National Taxi Foundation focuses on, but for instance, if you had too many drinks.
One night you would show symptoms of ataxia, as in, you might stumble, you’re right, you’re, you’re, you’re walking, or your gait might be off, you might slur your speech. Mm-hmm. Those are two classic symptoms of ataxia. Now obviously if you’re, if you’ve had too much to drink, it tends to go away and you, you go back to being, having normal gait and speech with hereditary ataxia.
It’s really a progressive neuro neurodegenerative disease. Passed from parent to child. There are many different forms of hereditary ataxias they can affect. They can hit kids very young, but also can come on much later. In life. We call that later onset ataxia. It is certainly. Significantly life altering, right?
People will end up very, most people with at taxi will end up in a wheelchair, and many forms of ataxia are life shortening. So it affects the ability to swallow and all of those sort of fine motor skills that the cerebellum, that’s the part of the brain that the genetic mutation occurs in.
The cerebellum really impacts your ability to do all those fine motor skills. And so NF has been around for a long time. Our mission is to accelerate the development of treatments in a cure while working to improve the lives of those living with ataxia. And so, Adam, we really do two main things.
We give money to research, right? Mm-hmm. You’d be hard pressed to find an ataxia researcher in the world that hasn’t received a grant from NAF over the last 60 some years. And we do a number of other things in the research space, including a no cost genetic counseling and testing program. We sponsor the largest natural history study.
In ataxia et cetera. And then the other part of our mission is to really help support patients and their families that live with the disease. And we do that again through, we’ve got about 60 support groups spread around the country, and, and many are. International now, as well as some purely online, but these groups get together as often as every couple of weeks to support each other.
we have the largest annual patient conference in the world where we bring together almost a thousand at attacks in their caregivers and families. I. And then we obviously do a whole bunch of things online dozens of webinars a year and, other ways to support those families.
So that’s just a little bit about at taxi it’s probably, it’s, considered a movement disorder, right? So yeah, the movement disorders, you know, that’s Parkinson’s and then a LS and Huntington’s. You know, for a long time I think ataxia was sort of the ugly stepchild of those. But we’re working to change that narrative and the foundation has grown substantially in the last several years.
And we’re excited about what our future holds. Yeah. what originally Andrew drew you to this mission and to this organization? Like a lot of things we can do with our time, a lot of missions we can support. What drew you to this? Yeah, you know, I had about a 20 year corporate career largely in healthcare and, had sort of come across the term at various times, but for a number of reasons, kind of a classic, maybe, sort of midlife crisis.
I had felt so fortunate in my life and, and really wanted to take some of the business skills that I had developed and put ’em toward something that I felt like mattered more. So I made a move to non-profit. Management about almost 20 years ago now. And this organization, I was in between roles.
It’s, I love the size. We’re about 20 people now. And I’ll be honest, I love to, focus on the strategy and, and I get to do that as CEO, but I also mm-hmm. Like to do some of my own work and when you’re as small as we are, I can, only delegate so far before I actually have to do it.
And I must admit, I kind of like both sides wearing both those hats. So it’s been a great fit for me. Now, you know a lot’s in the news. And just for context for people that’ll be listening to this far into the future. And we’re recording this in June of 2025 a lot of the space about funding, federal funding, who’s gonna get it, who’s not gonna get it?
Why is federal research support so important to the ataxia space? Yeah, so it, it’s critically important. So as I mentioned, you know, we, do our grant program every year, but we can only give away. I. You know, we can give away maybe a million dollars a year in our grant programs. And, and really what we’re trying to do, these are often early career grants to PhDs that are just starting out in their career.
And what of course we’re hoping is by almost giving them, say seed funding for their work. They can then turn that funding into. A much larger grant from, for instance, a federal source and then perhaps set up their own lab and continue the work that they’re doing. And so we just aren’t able, we, we aren’t able to raise enough philanthropic money to provide the level of support that you need for frankly, a really complex.
Neurogenerative set of diseases like ataxia. Mm-hmm. And so the federal sources, and we have, there are literally countless examples of young researchers receiving a grant from NAF and then turning that around and being able to leverage that into a much larger federal grant. it is simply critical to our space.
Mm-hmm. And so, you know, we, like many other patient advocacy groups, especially in the rare disease space, ataxia is considered rare. Are concerned about what’s been happening in Washington DC and we are doing our best to raise our voices and make sure our, legislators understand how important it is to continue to fund those sources. You mentioned the research and also possibly like even the difficulty and like what it takes, like what makes ataxia a difficult condition to study. Yeah, that’s a great question. So I think there’s a, couple things that make a taxi and, other things like it really difficult.
So first off, it’s, it’s a rare disease and, by definition in the United States, any condition that affects fewer than 200,000 people is considered rare. We think it’s difficult to know in the ataxias, but certainly it affects maybe tens of thousands of people in the United States. But because it’s rare, you can imagine that doing clinical trials and studies can be more difficult because you’ve gotta locate the patients to recruit for studies.
So, you take something like happened with the pandemic and trying to get a COVID vaccine approved, and boy, guess what? The whole world, you can recruit the whole world, right? Everyone’s a potential mm-hmm. Candidate for a trial. Not true in a rare disease like ataxia. The other thing I would point out is that many of the are very slowly progressing.
And so what that means is, you know, it will take. You can’t do a trial just in a few weeks or a month and see the kind of change that you’d like to think you might see with an effective drug. it can take years for someone with ataxias the progression of their disease to show enough change.
So that you can see if a drug is having an impact or not. It makes it very difficult to do clinical trials, Adam, because patients don’t want to stay on trial, especially a placebo controlled trial for years, not knowing if they’re actually even receiving the drug or not. And so it, just adds a level of complexity to the ataxia.
The last thing I will mention is we are really still searching for really good, what are called clinical endpoints. So what are we trying to measure in a clinical trial to show that a certain treatment is effective and we continue to look for biomarkers, right? So not necessarily just a physical exam.
Performed by a doctor, but perhaps a, bodily fluid or blood that can show a specific marker where you take the subjectivity of a patient doctor interaction and a physical exam sort of out of it. we continue to look for better clinical endpoints in the ataxia and that that’s a challenge for our space.
Yeah. Well, I’ll tell you Andrew, it’s been really good getting to, have you on the show, of course, and also learn more about what’s going on in the ataxia space. I was not completely aware of it and what was taking place. So this is one of my favorite things about doing this show is getting the opportunity to bring on and learn, you know, on a daily basis and to introduce new content and new things that I think my audience should know as well.
So thank you for that. And if somebody wants to. Follow up and they wanna learn more and maybe even continue the dialogue. And you mentioned you’re raising funding, other things like that. how do people do that? I will point people to our website. I think that’s the best way to get started.
It’s www.ataxia.org. One last thing I’ll mention and, and if you add so one last thing I’ll mention is that bill I, the science guy, I. Who I’m guessing you, know of and obviously most of America loves Bill as a, as a science educator. Many of us grew with him. I’ve seen him in the airport a couple times.
It’s so interesting seeing him in the airport. Like, oh, there goes Bill NI don’t know. There goes Bill. The only other person that I got that like that feeling on was I saw Mike Tyson walking through the airport. I’m like, oh, there goes Mike Tyson. Oh, there’s Bill Knight. They’re the same. You, you with Stockton?
Both. I loved it. Both, both instances. Bill is, bill is a terrific guy. He actually has ataxia in his family. Oh wow. And it was a form, it was a specific type of a taxi that was just identified a few years ago. And since then, bill is really engaged with us at, the National at Taxi Foundation to help raise awareness.
he’s got a voice out there that people listen to. And so if you just go to a taxi.org/bill Nye, you’ll see a, a series of really short kind of TikTok like videos that. Bill recorded for us just explaining the disease a little bit, and they’re really fun. They’re in, they’re in bill’s inimitable style and you’ll be seeing more from us in Bill in the future here.
But that would be the best way to learn about [email protected]. Oh, that’s wonderful. And for everybody listening, just so you know, we’ll definitely put some links in the show notes, so you can just click on the links and head right on over. And speaking to the audience. If this is your first time with Mission Matters and you haven’t done it yet, hit that subscribe or follow button.
This is a daily show each and every day, bringing you new content, new ideas, and hopefully new inspiration help you along the way in your journey as well. So again, hit that subscribe or follow button. And Andrew, thanks again for coming on the show, Adam. Thanks. It was my pleasure.
