Building The Noise with Dean Suhr: EPISODE 024

SPEAKER_02 0:00

You know, I made a whole shift of who I was and what I did. When my youngest passed away, which was that same year, we were at her memorial service. And um I I remember distinctly being up on the podium or whatever, talking for a few minutes and thanking people and all that. But I said two things. I said, I've up to this point been an engineer in a cubicle, and I'm not gonna be that anymore. I mean, looking out at all of you, and we had the second largest memorial service that church has ever had. The first largest was a sports um giant. Um and uh I said, I'm telling you that I'm no longer gonna constrain myself to that cubicle that I'm going to engage and I'm going to be a hugger.

SPEAKER_04 0:42

Welcome to the Building the Noise Podcast.

SPEAKER_00 0:45

Ladies and gentlemen, welcome to the Building the Noise Podcast. I'm Matt Teresco, and today we're joined by Dean Sir, a rare disease and biotech executive whose work connects patient advocacy, policy, market access, newborn screening, genomics, and ethics. Dean brings decades of leadership across rare disease, gene therapy, data systems, and cross-sector collaboration with work spanning patients, clinics, researchers, industry, regulators, payers, and the government. Through his connection to the MLD Foundation, one you'll hear quite a bit about and why such a strong found uh connection, and his focus on newborn screening and access, Dean brings a systems-level view of what it takes to turn patient and family needs into real policy, infrastructure, and therapeutic progress. What makes this conversation important is Dean's ability to bridge worlds that often move separately. Lived experience, science, policy, and commercialization. Dean, welcome to the show.

SPEAKER_02 1:44

Well, thank you, Matt. I'm tired already. It's early in my day, and I'm tired already just listening to your introduction.

SPEAKER_00 1:50

Thank you. It's uh some amazing work that you've done. And as president and co-founder of the uh Metacromatic Leucodystrophy Foundation, what do you do and why do you do it?

SPEAKER_02 2:02

What do I do and why do I do it? Uh I uh I run this uh organization and co-founded it with my wife uh 25 years ago now. Uh great partner? Yeah awesome. Yeah, it it has its challenges, right? Working with your spouse. But we're well matched. I am a business development operations uh engineering type guy, and uh she is a former pediatric intensive care uh registered nurse. And I say former only because her license has expired. Uh but between the two of us, um we founded the foundation, sounds redundant, um, and have been doing uh all those things you talked about uh as uh requirements and stepping stones to serving the MLD community and hopefully serving the rare disease community, not just the patients, but the whole ecosystem. And and why do I do that? Um it's become a passion, it is an unpaid passion, so it truly qualifies that way. Um we do this, uh we got launched into this because two of our three kids were diagnosed with MLD back in 1995 after a six or seven-year diagnostic odyssey. Our oldest was 14 at the time. Um, and uh MLD is a neurometabolic condition where there's uh cognitive decline in this form of MLD, cognitive declines. And that was, you know, we were misdiagnosed from medical medical um um uh sorts of diagnoses to behavioral diagnoses to she's a teenager to we're young parents, to da da da. And that went on and on and on. Um and ultimately when we found out what the disease was uh through a CT scan, I'm I'm a turn of the before the turn of the century guy, and then MRI, and then a blood test. Uh, we figured all that out uh with the help of some really smart people. Um my youngest daughter was 10 at the time. She went into an experimental, truly experimental bone marrow transplant, predating stem most of the stem cell work. And uh she actually engrafted uh she shows a success in the publications, uh de-identified, of course. Uh but uh she passed away shortly thereafter from some reactions to the graph versus host, the anti-rejection drugs. We've learned a lot about that since then, but that we we knew what we were getting into thanks to my wife's uh uh perspective and experience on that. And my oldest at the time, again, she was 14, she's supposed to live to be uh early 20s according to the publications, not the internet. We didn't have the internet like we do today. We actually had to go pick up, you know, magazines and and uh visit the universities and hospitals. Uh said she lived to her early 20s. And uh, of course, that's life-changing just a few years down the road back then. And I'm pleased to say that uh in a week and a half we're heading to Hawaii. I use uh we use our rare disease airline and hotel points, and take our daughter to Hawaii each year to uh that's fantastic celebrate, take her out in the in the ocean, hopefully it's calm, the hurricane's gone. Uh and uh we'll just we'll just enjoy that time. So why do we do this? Uh it's certainly there's a family component to it. Uh but also on behalf of uh all those other families, again, in the MLD space, but in in the rest of the rare disease community, where um uh they're alone. They you know they are struggling to get the diagnostics, the diagnosis, um and then there, you know, there may be a therapy, there may not be a therapy. In the case of MLD, a gene therapy. We started working with some researchers in 2005 and uh about two years ago the therapy was approved here in the U.S. a gene therapy. I'll call it curative if identified pre-symptomatic, and hence as you've already taken a sneak peek at uh newborn screening and our work there.

SPEAKER_04 5:50

Yeah.

SPEAKER_02 5:51

But um not all families can access that. There may be issues because most families still identify and are diagnosed with MLD because their kids are symptomatic. It's a rare disease. There's no history in their in their family that they know of. So those kids aren't eligible for the therapy. And so we walk that journey with them with compassion uh of clinical care and quality of life. And that's just become so important to us. Um I think it's important to us as humans, you know. We look at each other and all day long we're kind of doing this sort of thing, looking down at our phones or our computers or whatever. And um uh, you know, I drive home, I actually work out of my house, but when I drive in, I'm not that guy that puts the car in the garage and closes the garage door before I get out of my car. I go talk to the neighbors. And and I so this has been life-changing for me as well. So it's uh it's one of those terrible situations which I don't wish on uh you know, on anybody, but uh those of us that are that are in it, we need to support each other. And um, you know, we're not we we may or may not get the other, you know, it's one in ten with a rare disease. We may not get the other 90% of everybody to be empathetic to us, but with 10% of us with rare disease, we can certainly um uh carry our weight in terms of supporting each other. And as you mentioned, then uh dabble and make accomplishments in policy and research and uh so many other areas.

SPEAKER_00 7:17

Unbelievable what you were able to do as you as you reference, uh if I'm not mistaken, you were in semiconductors at the time, correct? I was, yeah. High tech.

unknown 7:26

Yeah.

SPEAKER_00 7:27

And you you made the move, I think, if I'm not mistaken, 1999, the foundation was established.

SPEAKER_02 7:33

Well, legally in 2001, but we started the work in 1989. Um I wrote an email list server. I I'm an engineer, I'm a nerd. Uh I wrote an email list server. This is before Facebook and Google and any of that. And email was was just barely there. And so it connected us all so that you sent the message once, it went to everybody, anybody could reply, it would come back. You know, it seems pretty simple these days, but way back. Listserves and and dial-up modems, and and but you know what? We could all communicate with each other. We could get there, we we we develop these bonds in in a way that you know that I well, turns out I do have some neighbors, some people within uh three or four miles of us here that do have MLD. Um but uh you know, generally back then everybody was scattered. And we reach and continue to reach families around the world uh because of uh you know the advancements of the internet, but basically building off of those early, early roots.

SPEAKER_00 8:32

Yeah. I mean, I was gonna ask, how did your tech background help? And obviously, uh being able to create a listserv that early, uh obviously it was a benefit. But as you look forward, where do you where do you think AI will be able to take us both positively or negatively?

SPEAKER_02 8:48

So from dial-up modems to AI, that's a big jump. Right? So I have I have this partner in my office uh named AI that I've been really working closely with probably the last six or months, six months or so ago. And it's a great tool. Uh it's partially, you know, 40 years old and and can do the work of you know thousands of people, and it's partially uh uh a four-year-old and a six-year-old that you need to keep reminding and you need to keep focusing. And you can't quite say. Um so I guess my parental experience is is helping me there. Um AI certainly has its place in a in a, you know, I'm doing coding now with it. I'm actually doing a um uh an application that we can talk about another time. It's not quite ready yet. Uh but you know, we hear about drug repurposing and uh analyzing clinical trial data, um, uh all those sorts of things, and of course the efficiency of business operations. AI will help us in all of those things. And uh I I believe it's gonna uh displace some jobs. It's certainly not going to eliminate lots and lots of jobs. I think we will rise up to uh AI as our partner. And and honestly, you know, the audience that I'm talking to, I don't I don't know the whole breadth of it. But there are people out there, me and you included, that have been using AI for a decade. We just don't know it. You know, the fundamentals of Google and Facebook and advertising that comes back, mind you, they are advertising companies. But how they know what to present back to you and all that, that's really the roots of AI. The the AI that they're branding AI today is just more in your face.

SPEAKER_00 10:32

Um that's important, I think, for people to recognize that we used to call it what computerized learning or or um uh if I'm not mistaken, it was just watching and and seeing your patterns and feeding it information to you. I mean, it wasn't uh six, seven years ago that people were talking about is it listening to me? You know, no, we just figured out your patterns, right?

SPEAKER_02 10:58

Right. Well, that's a way of listening, right? It observes.

SPEAKER_03 11:00

Yeah.

SPEAKER_02 11:01

And uh, you know, I can learn a little bit about you uh through the screen here. And if I knew what you were typing and and what you were getting back in in response to that, I'd certainly learn more about you. Um so I think AI is is a tool. It's uh you know, that whole six-year-old thing, uh, I I hate to say it, but it's got to go through puberty too. That's another whole process.

SPEAKER_00 11:21

Um and I'm in the middle of that right now. I'm not looking forward to AI puberty, but my daughters are 12 and 13. And man, is this fun?

SPEAKER_02 11:30

Yeah, we'll we'll pray for you. Um uh and hopefully your intelligence is not all artificial, it's real. Um but uh but I do believe, you know, we've heard, and I don't want to spend all of our time talking about AI, but but we've heard of AI quote escaping, where uh of recent, or at least those of us on the tech side of this. And that has happened, I mean that's happened uh uh a lot of times, but but it's happened in a big way um uh here of recent. And we'll figure that out. Uh I'm I'm sure that they will. And uh frankly, um, you know, as we talk about even rare disease policy and we talk about privacy, for example, but also I think that relates into AI, and and they're quote, blaming AI for for privacy issues, but AI is is enabling fundamental activities. Um so we're gonna need as a community to engage in uh some policy that overlaps in you could think of AI as computers and data centers and all that stuff. No, it's it's it's part of um it's like you and I getting out on the road and knowing the laws and the rules. Um I drove in, I drove in a Tesla, I drove, I was a passenger in a Tesla about two weeks ago, and my friend was really proud of it, and and he took his hands off the wheel, and it literally got us from one parking lot out on the street, around a roundabout, into another grocery store parking lot, and it backed in. It backed into the space because it knew he would like to back into the space. But I tell you all of that because when we came to a stop sign or a stoplight, it actually stopped. When was you don't have to say this on the air, but I can't remember the last time I really stopped at a stop sign, right? Yeah. True stop, yep. Yeah. So, you know, there's there's things that um we'll we'll we'll adjust to and we'll adapt to, but we we need to we we we are learning to coexist with that.

SPEAKER_00 13:21

Yeah, I think my my only concern with AI is is how well we as humans, especially in in the patient advocacy community and and especially the rare disease community, understand it and prepare um our organizations for utilizing it. I think it was just a few years ago, everyone was still focused on SEO, and now you have new acronyms coming at you. And how do you ensure that uh, you know, I go back to when when you had your child, your children were diagnosed and you were trying to figure out as much as you could about what this diagnosis was. Now with AI and people going to AI for those answers, are they getting the right information? Are they getting to the advocacy organizations that we need them to get to? Have the sites been curated in a way that the information is readable by AI? All new problems, right?

SPEAKER_02 14:14

Right, right. And and you know, I I remember um as you were saying that, just kind of reflecting back, I've uh got a 50 plus year career. So when when I started, um we we barely had desktop computers. Most of them were connected with big cables back to you know central servers and stuff. Ironic, we're kind of back there again today, Ira. Um but uh you know, laptops and those sorts of things, they they were they were desktops and they were big. But I remember we were just you know, we're a bunch of electrical and software engineers, and uh, we were talking to each other, and and somebody said, you know, these these computers are awesome. They allow us to make the same mistakes we could make yesterday, just a hundred or a thousand times faster. And AI today, you know, maybe it's a million times faster. So um we we, you know, it I I don't particularly like the word artificial, and I don't particularly like the word intelligence, to be honest with you. Uh, but the idea, you know, if if you look at that, it it it is how do you manage that? How do you use that? Um, you know, we none of us had had phones, you know, the mobile phones we know today, the smartphones, you know, well, not all of us have been around for 20 plus years. Sure. Um we have. Uh, but but you know, the um or 25 years or whenever it came out, uh, but we've learned to adapt to that. We've learned uh we've learned good things and bad things, right? When we need to get somewhere, we plug in the address. The phone takes us there, usually. Yeah. Um, sometimes it's a little easy how it does that. Um we we have we have voicemail, we have this ability to to talk to each other with pictures and record and converse and then share that with others to inspire and help them. Those are things we've learned to uh not only manage, but how to how to use them to impact the world. And I think part of this is, you know, to take take a take a look, you know. I I've got a fake background behind me, but there's some flowers up there. You know, turn around, sniff those paws, enjoy those, you know, uh smell the roses, so to speak, and and recognize what we're able to do. You know, I'm able to to contact a researcher in another part of the world, and then someone in a different part of the world, and put us all together on a conference call and we can learn together. We can share, we can talk about a patient. We can um I was involved with some um uh legislative activity uh just earlier this week, and we were speaking to people in Washington, D.C. without having to pay thousands of dollars and spend tens of hours, in my case, I'm on the West Coast, tens of hours to fly there and back and stay in a hotel and all that stuff for a 20-minute meeting. You know, that doesn't make much sense, but doing it this way is awesome. So so these tools can be tamed and and used, uh, but we do need to respect them too, right? Uh because still still pre-teenagers.

SPEAKER_00 17:08

Yeah. And and you know, with that in mind, it's it's also how do we help others use them? And and I want to actually use this as an opportunity to go back to when your children were first diagnosed, because I can I can't imagine what the Odyssey was like, and and having been told so many different things at that time period, right? Uh, you know, it's your fault as a young parent, or the child is developing in this way, it's just normal development. Then to actually get a diagnosis, what was that process like?

SPEAKER_02 17:38

Well, looking back, uh uh, you know, it's a mixture of emotions. So my wife and I, in all the decisions that we've made uh and all of the things that we've done, we've, you know, part of that decision is we're making the decision. We think we've done as much research and have insight to it. We know the the the positives and the negatives, the risk versus the benefits. And we're not going to look backwards. Because coulda, woulda, shoulda doesn't change a decision. And it it it it will blow up your head. Uh it will uh it's an emotional burden um like guilt that that uh uh just will it will overwhelm you. And we've lost an smart and we're losing another daughter, and we know that and we've accepted that. Yet Lindy, my oldest, was in the hospital in February for 28 days, in the in the ICU for 18 of those days. First time ever in the hospital for for an extended period for for that kind of serious event. It was it was an aspiration. Um it's it's ironic that that happened, you know, twenty-five years after we started the foundation. We've worked and served, you know, hundreds of f you know, nearly a thousand families actually in that time frame. And most most of those, most of them are kids, you know, ninety percent or so of them are kids, and those those those kids who we know because we visited many of them. We've had them at conferences, we visited them when I when I when when my wife and I travel for policy or for researchers or conferences, which they tend to move around. They don't move DC, but they move everything else around. And uh we try and visit families in the local area. We've been international from from Australia to Germany and and uh northern Europe and even Canada.

SPEAKER_00 19:23

That's amazing.

SPEAKER_02 19:25

Uh but meeting families. Um and yet at the same time we thought we were prepared for our daughter and the worst. And when it when you're face to face with it, it's always different. And so we had those conversations again and again, and held hands and cried a little bit, prayed a little bit, and said, Okay, we think this is this is the the the best or optimal decision. And here we are, we're traveling to Hawaii, but you know, next time it may not be um uh that that outcome. I I love that premise with that.

SPEAKER_00 19:55

Yeah, I love that premise of we made the most educated decision that we could, and we're not gonna go back to it. I I think so many of us, you know, outside of our health, uh kick ourselves time and time again too many times uh about decisions we made in the past when there's nothing we can do about it. So keep keep living the way that it's been going uh and make the best decision for the future. Um so with that in mind, you know, as you think about parents today uh who may be going through similar diagnostic process, trying to understand what their child is facing. Obviously, they now have the MLD foundation to come to, but what guidance do you give to parents today?

SPEAKER_02 20:38

That's a great, great question. And the world has changed in these past 20, 25 years. With that email list, we were the virtually the only source of information for families if they could find us. Uh and so we we we had our different search SEO problem. There was no Google. Um the doctors didn't know about the disease, you know, the the the smart head doctors at the hospitals didn't, but they had access to some resources, and so we connected with the NIH and we got in their directories and those sorts of things. And so uh families actually were more naturally connected to us. Then along comes um a whole there were a number of stepping stones, but what what what ultimately Facebook kind of took over the bulk of the social media, and of course we've got things that I don't understand because that path keeps going with TikTok and Instagram and all that. Yeah um uh but uh uh as as other ways came around to connect for families to connect, that's awesome. That's awesome for day-to-day support. But the challenge with that is the families are desperate for information and recommendations and solutions to a to a problem or a concern they have. And the person on the other end of that conversation or the people on the other end may have a different form of the disease, or they may be further progressed. Or honestly, they may be using kind of an older style uh medication regimen for quality of life. So they don't have the most up-to-date information because they're working with their local care team. And that doesn't make the local care team bad. It just means they didn't learn about uh, you know, what the the new drug or the new whatever, the new approach. Um so today, families don't always come to us first. They come to you know, they Google it, right? Or whatever search engine you use. And then they search on Facebook. And I again I don't know how they do that on the other platforms, but um they come to when they do come to us, uh, often because they're saying, hey, we want to get connected with uh this gene therapy, and how do we do that? And yeah, and and maybe I can't afford it because it's a multimillion dollar therapy and we're concerned about the insurance or my medic medic uh paid access for my kid and so on. Um to so you ask, kind of asked how do we connect with them, how do we support. First thing we do is we remind them, just as I did your listeners at the beginning of our conversation, I'm an MLD dad, first and foremost. Uh, and I start to put this on my slides at professional conferences now. My title, MLD dad. I have some other skills and some other stuff. Uh, but but that's the most important uh title. And so we develop that rapport. We love to uh I love uh you know setting up a Zoom or a video call with them because we can see them and they can see us and they can see that compassion in their eyes, in our eyes, in their eyes. We might be able to see their kid in the background. We can get an idea of, you know, are the are the parents on the same page? Are there other kids in the house? Sure. They get a sneak peek at uh you know the kid wandering around if he still has motor skills and so on. There's so much insight to that. So we build that personal connection. And along that way, we're having a casual conversation because they've all been diagnosed with MLD. But they're not. Uh more often than not, the they've gotten an MRI back that says indicative of a leuchodystrophy, the umbrella MLD is a leuchodystrophy. Um, or uh it says uh uh might be uh you know MLD. And what the doctor's really saying is we need to do a confirmation that it's MLD. But the family took that and ran with it. And I get it because their kids, their kids sick and they they want something and they want it now, and they're grabbing onto what they perceive as a lifeline. But we always go back to this diagnostic confirmation because we don't want to assume that when they say it's MLD, that that they actually haven't been confirmed. And that could be newborn screening, which is a is more straightforward, but newborn screening is screening, it's not diagnostic confirmation. Um we talk about that in a bit. Um but uh so we we kind of rebuild that and we get a sense of um of where they're at and what is what is fact versus what is uh extrapolation or presumption, both on their parts, but also on their doctor's parts, because right, the doctors in you know the local hospital local hospital here would not know MLD. We go downtown to Portland for our for our medical services. And even they, we have a partnership with them, we have a care team there that we've developed where we bring things to the table, they bring things to the table, and more often than not, we're saying, well, we've learned this because of either this researcher or uh uh seen this impact on other families because we're talking about treating our daughter. Um and you know, we throw it on the table and say, well, does that make sense? And given what we're also doing with either these drugs or these therapies, um, would this conflict? Would this be you know uh a contraindication for one of the other drugs because we want to try something different or whatever? Um and so we we do this as a team member, and we don't expect our doctors to be experts in MLD. We know we're teaching them. That's okay. Yeah. We just want they are experts in neurology, they are they are experts in biochemistry. Um that's things that we don't know. And so as a team, we can pull all of that together. Um, and so one of the first things we do with parents is after this conversation, get to know them and see if they can be referred to therapy and/or what are the emotional states and all those sorts of things down that road is we start to gently remind them they need to take care of themselves because they're no good for their kids or their family, if they're not sleeping, if they're not panicked. And that's the other thing you can pick up on a on a phone call is whether they just look totally exhausted, which they often do because we're into the first two or three days of their journey. But um, you can get a sense for if they're taking care of themselves. And uh also, uh, and I particularly focus on on the moms, because nine times out of ten it's it's the moms, and I say, you're gonna learn to be a tiger mom. You're gonna learn to be the one that's the MLD expert, because they're always frustrated they can't find the experts. I said you've got to bring that to the table. And don't worry, you'll learn it. Just call it MLD, don't call it metachromatic leuko dystrophy and talk about all the enzyme numbers and all that stuff. You you can bring that step by step to the table and work together with a care team. And if and if your doctor is, um uh we call them God doctors, they think they know everything. There's only one way to do it. It's the way I've done it for 40 years. Well, 40 years ago, we didn't have MRIs, we didn't have therapies, we didn't have all these drugs or whatever it is, um, all this fancy equipment, uh, expensive equipment. Um so we we say if if you know just because he's the smartest, he or she is the smartest person at that hospital or the most, you know, the longevity there, that's okay. Maybe, maybe you want to be with the the med the you know the new doctor that's out of school for five years or ten years and is trying to build a career and has learned a lot of new things and is open to ideas, um, they may be more receptive to working with you, and frankly, they may have maybe not all the doc all the information the other doctor has, but they're willing to learn and absorb. And um, whether they do it to your face or not, they'll acknowledge they don't know everything. And uh they they they will go look into something and get back to you. And guess what? They're wizards at all these little handheld devices and and uh my chart and all those things. They don't they don't come to the terminal kicking a scream and they'll they'll they'll they'll type a response back to you, maybe only a line or two long, because that's kind of the style we communicate and and all the time they have, but you'll be able to have that conversation and get information. So the world's a different place in how you get your medical care. And I'll circle back to AI, which drives all of the Google results and so on. Just because the the most common favorite answer on Google comes up and says choose red, you may want to still consider blue or green, or if somebody else is saying blue or green, you know, spend a little time talking about that. And AI, for what it's worth, if you take an AI answer and put it back into AI, or maybe even a separate AI tool, and say, you know, double check this, give me the references, or what do you think about this? Um for better or worse, it sometimes comes back and it corrects itself or says, Oh, I hadn't really thought about that. Or now that you mentioned one little thing, you know, MLD Foundation says. Sure. So they now check the MLD Foundation site and they come, it comes back and it says, Oh, well, we've got we've I found something else out. And that would change. And they're very polite because they never really want to say they're absolutely AI never really wants to say it's wrong. But it you can you can, by the way, say, don't be so polite to me. I want you to be short and concise in your answers, and don't tell me I'm always doing a good job, because I'm just here struggling to keep my nose above the water and save my kids' life. And and to tell AI that's AI how you want it to treat you, like an expert. Um uh and and uh and and when you say like an expert, say if I don't understand, I will ask you. But otherwise, assume that I'm I'm an expert and you'll start to find that level area. Then you have a team partner. Um so sorry.

SPEAKER_00 29:49

That is but that that that is amazing insight, though, on building your own care team. Uh I think so many folks that I've spoken with, and even in my own care journey, uh, after breaking my neck and dealing with chronic pain, you know, you reach the end of a provider's knowledge base. And as you've said with with with MLD, there are going to be those that know and those that don't. It's it's okay to go find another doctor, but it's also okay to understand that this might be a rare condition that they've never had experience with, or I'm the first, and you can choose to work alongside them, work with them, bring resources, as you've said, uh to help them understand it or have another point of view. It's it's not just to show up and listen, it's a true partnership.

SPEAKER_02 30:37

Yeah. Yeah. And and and everybody wins when you do that, right? The doctor's more comfortable. The doctor gains their knowledge. And while you're you're generally most concerned about your loved one or yourself if you're the patient, they also develop a new skill as how they connect with the next patient. And they they they are thinking, oh, well, maybe I don't know that. Maybe I should ask and find you know how I can work better with this patient to learn from the patient, not just diagnose and treat the patient.

SPEAKER_00 31:05

So that would be golden if we could you know get the entire system to come to that realization that there's a lot to be learned from each of us as patients and and our care care caregivers and care partners in this journey, they have a lot to provide as well.

SPEAKER_04 31:21

Yeah.

SPEAKER_00 31:22

And and I I know that you guys serve the entire family, not just the the diagnosed patient uh and have tons of services available there. And I know historically you were one of the first organizations to rally support for caregivers and care care partners. Tell me more about how you saw that need and and the work being done there.

SPEAKER_02 31:44

Yeah, it was kind of an interesting journey as uh you know, as as as you introduced me. I'm an engineer, um, so I I could spell MLD. I couldn't spell any of the rest of that. And I I took, you know, truth be told, I took past fail chemistry in college because uh chemistry was not my thing. Had a good time, it's all the dental hygienists there and so on, but it was past fail, and that is a good thing. Um so we started attending some of the medical conferences uh pretty early on, and my I I just my eyes would glaze over, and I was taking pictures back then, it was 35 millimeter cameras, but you know, we're taking pictures and stuff uh so that I could try and figure this out later, and I just couldn't. I mean, I don't I can I can say the word carbon bond, but I don't know what that means. Uh carbons with the black stuff, that's all I know. When it burns, it's you know, I I don't know. Um but um uh it it we we started with that and we engaged with that, but then we realized that uh while we were and we were just we were quote just really important. We were loving on families, we were connecting families because that had never been done before. But I quickly realized with my business development and operational sense, so I while I'm an engineer, I'm really uh more of a an executive style guy, that uh we had we had to move policy because there were issues in rare disease, not MLD, but rare disease that needed to be addressed, that the idea of uh you know how the NIH did some of its research, how the FDA did some of their reviews and processes, that there were things that we could do better for rare diseases. Don't want to lower the bars, don't want to change the fundamental scientific study process, but if but but we don't have a million patients. We we have you know hundreds of patients, maybe you know, hands you know, under 10,000 around the world, kind of that sort of thing. You you can't do a clinical trial, say you know, we need to have um 2,000 people in the trial. It's just not gonna happen. And if and if you had to do that, it's gonna be over so many years that that the pharma company or the academia just couldn't couldn't afford it. So we got involved in policy. Um one of the first places uh was with Genetic Alliance, uh went to the Capitol Hill first time in 2007. Uh Genetic Alliance was building off of that uh that newly discovered human genome, or I should say it was discovered a long time ago, but kind of newly documented and and became part of the scientific tools. And from that we got involved uh in privacy, uh ironically, um, and little did we know how important that was going to be. Um and that led us also, and be again because of my my new nerdiness, when the newborn screening um and the secretary's advisory committee, the precursor to that was ACMG, the American College of Uh Medical Genetics, um or genomics, what I'm not sure what their last word is. Um they they were chartered by I think it was by either HERSA, I I think it was by HRSA, by HHS, to set up uh some structure with newborn screening. Newborn screening started in the mid-60s with PKU and and had grown, but it had only grown to a few dozen diseases uh that were being screened. And there was there was obviously this um sorry, I've got a phone ringing here. Um there obviously was a um uh uh uh a sense that more diseases, more th more therapies were coming, and hence more diseases need to be screening. So they proactively asked ACMG to develop some some foundations and some structure around newborn screening at the federal level, because newborn screening is state public health, and so it's managed in 50 states and six territories and literally hundreds of sovereign Indian Native American uh nations, um so uh and the military, all on top of that. Uh, but they're they're they wanted to create some more federal structure with that. And I got involved in it because I thought, oh, structure, organization, a process, um, you know, rules. I I can deal with that. I'm an engineer, right? I stay in my here, I'm in a round box, I'm not in our oval screen here. Um and uh of course I don't stay in the box anymore. But uh so I got involved in newborn screening, and Taryn now has joined me because there's the whole family medical component. But that led to um uh a really important transition where we engaged with the FDA. Uh we were we were blessed as a community to have a number of academic sites and a number of both small and large uh pharma and biopharma companies interested in MLD. We never had a direct project with the NIH. We built on the shoulders of a lot of work that had been done at the NIH. Sure. Um we never, as an advocacy group, had to raise millions of dollars to fund a research project. There were partners, just like we talked about with the doctors, we partnered with lots of pharma companies who said, look, I'm I've raised, I've raised millions of dollars in my high-tech world. I'll help you raise money, right? I'll talk to your investors or potential investors, and I do that regularly still. Um and we'll help with the awareness, and we can connect you with, you know, the experts and all those kind of things. Um so we didn't have to kind of do all of those struggles. We still had to get patients to clinical trials because, again, patients were being identified symptomatic. We needed early or pre-symptomatic patients. But the whole engagement with the FDA, um uh, I became very active with something called PADUFA, the predictive prescription drug user fee agreement. And many people don't know that with the FDA, um, a lot of their certainly their drug reviews, but a lot of their other uh revenues that are supporting a lot of the work they do come from fees that are paid when drug companies submit their reviews. So the FDA's budget has a has a federal component, but it has a very big uh commercial 75%. Yep, yep. Right. So those of you that think drug companies are bad, and nobody in your community probably thinks they're bad. Um uh but you know the general populace is oh so much costs so much and so on and so forth. And we can talk about that separately too. But um uh we established uh we, it wasn't just me, but I was very active in uh Padu, I think it was Padufa 5, where uh we set up the PFDDs, the patient-focused drug development meetings and some other patient engagement opportunities that allowed uh that required the FDA to set up an infrastructure that allows patients and disease communities to come together and either in at the umbrella, like us with leuko dystrophies, uh, or uh specifics, as we've also done with uh MLD, and share what we know with the FDA. A lot of that is one way still. We we're trying to work on it to make it more interactive, uh, but it's shared outside of the context of a drug application, of a review application. So the idea is that the FDA has a much better perspective on the patients and on what are our goals and priorities. Um so when they review the drug, it's like, well, that's awesome that that you know, your chemical number went this way, that way, your marker, but I want my kid to be able to say I love you. My kid hasn't talked for years, you know, right? So, and and you know, I want my kid to be healthy and comfortable and happy and so on. But when you really ask me what do I want, I want her to be able to tell me, I'd love her to say that she loves me. I know she does, she still smiles.

SPEAKER_03 39:19

Yeah.

SPEAKER_02 39:19

But I'd love her to be able to tell me what hurts or where does it hurt, or did you want, you know, this or that? And that's much more complicated. So I I digress a little bit, but that's what got us into that uh into that policy space and into dealing with the agencies uh in the government and how important uh that is. Um and of course, doing that proactively as we were really was paving, and I didn't know this was paving the ground for me as we got and me and my wife and and others as we got into um uh the assessments and the and the regulatory reviews for the gene therapy as we're launching uh MLD newborn screening, and as I touched on earlier, we have the uh the blessing of having a therapy. It also is the world's most expensive therapy. So we were sure and this cut this comes partially out of my experience with um selling semiconductors, you have to show the value of something if you're bringing something new to the new to the market, right? You know, if you're if you're deciding on a car, you're trading off one feature for another and so on and so forth, and you want the big one or the small one or the two-door or the four-door, whatever it is, yeah. Uh electric or gas, you know, all those kinds of things. Same thing when you're doing therapies. And so price is an issue, but it's really about value. And so the my my underlying message for your audience is I'm glad we have a therapy. I'm glad we have other pharma companies that are looking at being competitive with that therapy. The good news is a high price here means there's more opportunity here, and that will both of these will serve the world. I'm glad that our biopharma partner is able to have a success with MLD because it in our case MLD was their first product, but I can see how this is playing out in their subsequent products, that some of the MLD profits are going towards future development. And that's okay. That's good for their company, it's good for their shareholders, it's good for rare disease families. Yep. But it also has to have value. And so when we talk about the price, I call it the above the fold. For those of you who don't know what that is, that's the newspaper when you unfold it, that's what you see in the newspaper machine. You've probably never seen one of those. That's where all the big headlines show up. And you know, four and a quarter million dollars is the list price for uh for argene therapy in the U.S. That's above the fold. Very, very few people pay that. Those that come in truly with a millionaires or or have some rich pocket behind them uh actually come into the U.S. because they can't get the therapy in their country and they will pay list price for the therapy. U.S. U.S. citizens don't pay that. It's uh Medicaid has a mandatory 19 to 23 percent discount to start with before they even talk about pricing, and then they do negotiations. And there are all there's pricing agreements and so on, but we've shown the value of this therapy, even at its full list price, it pays for itself in a matter of a handful of years. Because your kids don't spend uh 30 days in the hospital, mine only once, but that bill was over a million dollars. I know families that are in the hospital two, three, four, five times a year. Um, and and all of that adds up. And we as a society, and I get it, you know, a doctor would much rather put you on a pill and you pay, you know, I don't pay, the insurance company is paying, you know, $500 a month for that pill. Add that up. It's $5,000 a year. Over ten years is fifty thousand dollars times, you know, a million people or whatever. Yeah. But we'd rather pay by the month than pay for it all up front. And so that dynamic of how you pay for it and when you pay for it is challenging. And of course, in the case of rare disease therapies like ours, there's only uh there's a half dozen or so, I think maybe seven now, centers of excellence where you can get the therapy in the U.S. And paying across state lines with state A's, your home states funds, into going into somebody else's medical system creates its own challenges. But those are those are business issues we can work with.

unknown 43:17

Yeah.

SPEAKER_00 43:18

I mean, just to be but to get there in the first place, uh to get a therapy to the patient population, to ensure that they were included in the development of that product and the trials, I'm sure, as well. Um, and even to hear that you helped to set the ground rules for patient-focused medicine development as a as a a lifeline, so to speak, for the patient community to provide input is unbelievable. The access now that didn't exist before. I don't think people recognize that that these may it be externally led or internally led PFDD meetings just didn't happen 10 years ago. Um, and and now it's it's as you said, what's the value? For the patient, where do what do they need uh to see to make it make it valuable and to be able to reduce those barriers uh uh to a clinical trial as well? And you you had touched on um you know how many patients for a trial and uh how do we find those patients? Well, you can start with newborn screening if it makes sense for the patient population. And I know that you guys were recently added uh helped to add uh MLD to Rust, correct? Uh as of December of this past year? Um what was that process like?

SPEAKER_02 44:34

Um Yeah, so a year ago, the um almost a year and a half ago now, the uh Secretary's Advisory Committee was disbanded. It was disbanded because it was a provisional committee. Uh it was thrown out with a dozen plus other provisional committees. That means that their federal authorizations had expired. Yeah. Um the Newborn Screening Saves Lives Act has not been reauthorized for 14 years. It expired 10 years ago. Um, that's that's on us, by the way. That's on us, then the community, because we let that expire. Um but the reality is it expired, the committee went away, you know. Doge came through and trimmed a lot of things. Yeah. Um the RUSP never went away. The work that everybody had done and all the signed, because RUSP is very recommended uniform screening panel, that's the federal advisory list. Um, all of the evidence that had gone into those reviews and those decisions and those recommendations, that still stands and still stood and stands today. But as a community, we had just completed all of that evidence with literally about a week and a half to go before we were expected to be voted on by the advisory committee, which was the second to last step in getting on the RUSP. The advisory committee reviews all the evidence, the final evidence review reports, and then they would make a vote and recommend it to the Secretary of Health and Human Services. So that's the final step. And we had no advisory committee. So, you know, some people panicked, and uh, of course, a lot of panic, um, but some people panicked and and said, Oh, there's no way you can get newborn screening for MLD. I said, Nah, come on. We're still people. We still we still can figure this out. It's only the government. You know, have we ever reformed the government? Yeah, we can do it. Does it happen overnight? No. Um so we pivoted, and we piv, my wife and I in particular, we pivoted before everybody else pivoted and started saying, well, what can we do? And who do we know? And what is the process and what was the goal of the advisory committee? And for all of the things we loved about the advisory committee, and hopefully their yes vote, and we're still confident they would have voted yes. There were a lot of things we didn't like about how that committee operated, and what were their priorities, and what were, you know, how did they approach certain things and and what were their what's their bandwidth? Um, how long certain things took place. So it was far from perfect. Um but we moved on and we we worked the process. Uh Every Life Foundation came alongside us and the Duchenne community. Those were the two to be voted on at that May meeting. And uh uh we held the equivalent of that advisory committee meeting with similar and some overlapping experts from the advisory committee. It had no authority, but we had that in a much more public way, in a much more interactive way, with other scientists and researchers and clinicians and with the public there. So we held that meeting. HERSA put a federal register notice, which is like the official document of the U.S. government. They put a question up in in the FRA, a federal registry notice. Um FRA, I don't know what that stands for. Um but they had a uh, I don't know, a 30 or 45-day period where anybody could comment. And to my knowledge, there were no negative comments about MLD. There may have been, but I wasn't made aware of it. Sure. Um and overwhelming, not just of course, you know, all the families said yes, but also the the clinicians and and the scientists were saying yes, and this is why, and so on. That then went up to uh Secretary Kennedy's office, and I mentioned that newborn screening started 50 plus years ago, 60 years ago, maybe. Um it was his great uncle, I think it is, President Kennedy, that started newborn screening. And there's another whole story for another person to uh uh but there's a connection there. Um and that, by the way, wasn't why Secretary Kennedy signed MLD and DMD on, uh, but uh it was an interesting connection. And so we were approved by the Secretary last December, and we're working through that process of state-by-state implementation. So a couple of things just through that story and from something else you said, I want to encourage advocacy groups out there. If you're frustrated or you feel overwhelmed or you don't know how to get something done, reach out to other advocates. We do a lot of sharing in this community, we do it through forums like this, which obviously they're not going to talk to us, you know, today, but they may talk to you or they may talk to me. You know, you mentioned you broke your back. Well, that that may ring a few bells with some people. Um but the other thing that I would I would encourage of advocacy groups is that you have certain passions and skills and emphasize those in what you do. Bring people alongside you, partner with people that can that can fill those gaps of things you're not an expert in. You may become an expert over time in them, but don't feel like you have to do everything. It's really important. You know, a lot of times when we talk about, and I hear other other groups talking about, you know, what does a brand new uh advocacy group need to do? Well, you need to have a natural history study, you need to have a PFTD meeting, you need to find a researcher, you gotta find somebody that's got money to be able to support this. Stop that. Yes, those are important things. But if you just look at that laundry list, you'll never get there. Well, some people I've seen them do it, it's amazing. But but generally you've got to you've got to find people that can come alongside and talk about the people not just in your community that are affected, you do need to connect with your community. But who do they know? You know, you may have somebody who is a legislator, you may have somebody that's that's CEO of Armor Company, you may have somebody that's in in public health or at a at a university or something. Use those connections um and you will get there. Do what you can, do it well. Don't try and do everything because somebody says you have to. Um our first registry, we call it now our patient, our MLD patient profile database, because it got confused with registry. We have a thousand families in there. Wow. And it's a it's a glorified contact database. Um it says what form of disease you have, when was a patient born, when were they deceased? Again, most of our patients are only living a handful of years up to the therapy. Um, and where do you live? What's your email? There's some other stuff in there, and there's some notes. Sure. But it's a glorified contact registry, and we use that almost every day. Either we're supporting somebody or we're trying to understand how many patients are nearby this, or nowadays we're doing some policy work. It's like, I know you lost your kid 10 years ago, um, and we're reconnecting with the family, which is always a a good moment, even though it may stir up some of those emotions. But but we say, you know, you we you can help other families because, you know, I I love to come to Florida, uh, but they don't care about me because I live in Oregon, right? So we need somebody in Florida that uh has a connection to MLD that can testify because you're your constituent, your words matter. And andor we've got this this other rare disease that looks like MLD, and uh we want to connect them with your doctors, or we'd like you to help connect this family with your doctors because at least they understand the clinical care aspects of this disease, because the symptoms are similar. So sure, there's so many things that we can do and be and be proud and accomplished with. As we said, you know, we talked about bad decisions, or you know, you look back and you say, I wish I made a different decision. The um uh the same thing is true here. Don't don't uh don't fret about what you couldn't do or didn't do well, just keep at it.

SPEAKER_00 52:06

I think the one of the other keys to to just pivot uh real quick as well was you know don't be afraid to work with an administration that you may not like. Oh yeah because you might be able to get something done. Um it's unfortunate, and and I uh we're not gonna play play politics in this, but I hear too many organizations say, well, we can't do anything with this administration, we're gonna have to wait. Um when if that's not the case and we're not serving the patient or constituents either by waiting. So, you know, sometimes you have to work with people you don't like uh or don't want to like, and you may end up liking them at the end of the day, though, too. Um but you don't know unless you try. And obviously you guys uh made it happen, uh, which is unbelievable.

SPEAKER_02 52:48

Well, and yeah, and I would say that's true with the pharma company you work with, or the advocacy organizations you work with, or the person who's interviewing you on the other side of the screen, whatever it is, um, it's a small world. And uh particularly with in the governmental side, you know, I don't know what the number is, but but the appointees, that's a small percentage of the government. And they're the people get all the press and all that sort of stuff. But this machine that we call the federal government, and in fact, with with our Rust journey, getting on the RUSP, the people that did all that heavy lifting, those are long-term employees. Those are people that were at HERSA through that administration and this administration, they'll be there for the next one. Um and so the people that are doing the work, build those relationships. Don't don't worry so much about what you saw in the evening news yesterday. Um honestly, when we put the press release together, or we were we were inspiring the press release uh in the coordination for how they would announce MLD newborn screening, we wrapped it around gene therapy because gene therapy was the exciting thing that the you know the secretary was trying to wrap around is you know the moon launch and all those kind of priority, you know, whatever they call their program of the day. But so there's some opportunities in that, and you need to seize those, and sometimes you need to show up and smile and don't tell them you didn't vote for them because guess what? Rare disease doesn't care. Um and and I've had this uh and and besides my medical work, I do some other policy work here in the state, and I I've taken on some transportation issues, and people are really, really, really, you know, you know, polar, you know, by bipolar is not what I mean to say, polarized on the transportation and transportation funding. And so I made some quote enemies, uh, people that I was thinking, oh, you know, I don't I know where you stand, and I'm not gonna talk to you. And the reality is when I started talking with them about health issues, we found lots of common ground. And the reality is some of the other assumptions I made, even the transportation space, were because they were being influenced by other people. They were being either their their leadership in the party, and by the way, well, it seems like some people are elected forever, but they're not. Um and so um I and I say this by the way when I go into the same thing you said. Don't don't get wrapped around the axle about red versus blue. Yeah, neither, neither is perfect. I can tell you, neither is perfect, and neither is really in the center. Sorry. Yeah. Um because we're because they're forcing themselves to the sides. And again, we won't go any further into that. But uh, I think uh Every Life said this at one of their legislative conferences. We're not red or blue, we're purple. And um uh we need we we need to respect and carry on with everybody we work with. Um it's just important.

SPEAKER_00 55:25

And healthcare is just like rare disease is purple. It affects all of us at the end of the day. So um, you know, uh doing some of the health policy work that I've been involved in, I've never found anyone that disagreed with our premise, which is that patients and doctors should make the decision that's best for them. And it's it's amazing how you can pull people from or think that they would feel one way about it, don't actually, uh, or they were influenced, as you referenced, from someone else. But the minute you get in conversation, things do change. Um, you know, but before we close, uh I do want to ask about your work with industry because I think that that's um an important thing for organizations to get a sense of of how you approach that work, um, but also what you find important about working with industry.

SPEAKER_02 56:13

So I'll respond with it with a couple of flavors. One is that uh I think this has changed over time, and our approach to industry uh may not, you know, your mileage may vary, right? It depends on who else is in in the you know, who other who the other industry partners are, how many, what's the history, and so on. So I'll just preface with that. My background in semiconductors and high-tech meant that we were doing a lot of collaborative work, we were doing a lot of fundraising, we were uh working with you know we were startup companies, I worked in a handful of them, and we were also working and selling our products to startup companies. And and these were products that's that um that were new and novel. That that really has kind of been my basis. Um playing a little bit of long game. You have to think about strategic and tactical, what's driving this potential pharma partner or this academic partner, by the way, because it's the same thing over there. They have a different way that they fund their labs and their careers and their publications and and their credibility. Um so with industry, as as I mentioned earlier, we've been blessed to have uh industry partners at the doorstep uh throughout our journey. In fact, the first time an industry partner uh came to us and wanted to meet us and buy us lunch here in Oregon, we thought, oh, well, we'll do that. Free lunch is a good thing. We had a great conversation. And they said they said, Well, what can we do for you? And we had talked a little bit about the research they're doing and so on. I said, Well, just keep working on that, get that, get that approved. And they said, No, no, no, what can we do for you? He said, What do you mean? He says, Well, do you need any money for anything? And I'm thinking, we got a little bit of internet, we got a family conference, you know, we're we're doing okay. We were meeting in in hospitals, the costs weren't too much. And they said, Well, can we write you a check? And and I had to pause and say, Oh, this is interesting. But I come to learn, and certainly as the world has changed with with families and and patient engagement, that industry needs us as much as we need them. They need to survive, they have different bosses, they're regulatory bosses. If they're a public company or they want to go public, they have a different set of uh uh regulatory bosses for that. Um they have leaders of a whole variety of different personalities, they've got other departments or divisions, or sometimes they're uh subsidiary of a big company, and they may or may not be getting financial support, or they may be squeezed by the big company. Yeah. Um and and so you need, in my opinion, my ex my experience has been, I shouldn't say you need, we've been successful because I always try and understand, I always talk about what is the shape of their sandbox. Their sandboxes are never square, they have zigzags in them. And you you don't just walk in their shoes, but you need to know what it is they can do, what it is they can't do, and why, and uh try and understand that because it makes it easier to ask for things that you know you know they can do. Um and those things they're struggling with, if you understand why that is, you either don't push them as hard, or sometimes you help them work around that problem because a sandbox has the sand on the inside and it has the grass on the outside, and we can come at it from two different directions, right? Maybe we remodel the sandbox, uh, or maybe somebody just didn't understand, or they didn't think, you know, they they think of you as, you know, you're just thinking about MLD. And I say, no, no, no. I'm thinking about how can you posture for for all of your therapies to go through regulatory review and um get on newborn screening, whatever it is, or get reimbursement, right? Because that makes you a better company, and when you're a better company, my patients get served better. The rising tides file. That's right. Uh but we've we've we've seen projects, really exciting projects, come and go because the companies ran out of money or because the overall market changed. We had one company, a very large company we worked with, who I loved working with, that uh was doing really good benchwork. They hadn't, they were designing clinical trials, but hadn't gone to clinical trial. And all of a sudden, they said, Oh, any any disease, any any project that has less than this amount of total revenue potential. We're a rare disease. We don't have that many patients. You can't charge $100 million per patient. That's a no-go.

SPEAKER_04 1:00:30

Yeah.

SPEAKER_02 1:00:30

Um, they said, we're canceling all those programs because we just we can't be divided into so many small parts. We have to work on on um on bigger swings, I guess. You know, they're looking for home runs and they're looking for a series of base hits. Uh and you can make a lot of money on base hits. You can win the game that way. Uh home months get you on the TV news, right? Um That's right. We've had that happen. We've had a clinical, we've had clinical trials that uh in one case where we said, nope, we won't recruit any patients for that trial. We actually said no to a clinical trial because we didn't think it was safe. We talked with our medical and scientific advisory board, they didn't think it was safe. The risk profile was was wrong or was too risky. We also um had a clinical trial where the families were seeing results, and everybody was excited about it. Of course, we're excited about as well. We know most of the families. And the the clinical trial was a success, meaning that it finished, they were able to gather all the data, and the answer to the clinical trial was no. So it was a clinical trial success, but it wasn't a therapy success. And it turns out there was a disconnect between what the endpoints were, what was being measured and was to be observed in the clinical trial, because they defined that all up front, versus what the families were seeing. The endpoints were messed up. I never paid much attention to endpoints. Actually, once we went into a meeting and said that will never work. But uh, and that's where having a database, by the way, allows you to reach out to a lot of families and get a non-scientific profile that says, you know what, I met a lot of families, I think you're wrong. And they said, Oh no, we checked the publications and the this and the that. We surveyed hundreds of families overnight, brought that into the next morning and said, Do what you want. I'm just telling you what the real world is. Warning. But uh we we had um we've had those all those different situations. And so you you need you need to be partners with your pharma companies. You need to meet with them regularly, not just when they want you or you want them, build those conversations up, sign the CVAs. Um, you know, there's a lot of talk these days about advocacy groups owning IP, and I think that's great if you can figure that out and how that makes sense. But isn't a therapy better than owning IP? Don't think about how you're gonna pay your salary or the expenses for your your Wi-Fi or whatever it is. Um, be thinking, be flexible and so on, and and uh always be talking to more people. The therapy we have today is uh too expensive and too too expensive for a lot of countries, you know, sec second, third order countries. It's um uh too expensive to get regulatory approval in smaller countries, so it's not just the cost of therapy, but the approval. And then it requires frozen blood product with some specialized equipment when you do the infusion, so it doesn't transport well. Uh it does in first world countries, you know, in the first world. Yes, but it doesn't in others. And so there's an opportunity for other approaches. And we see this with what are they, jeep GPL ones or whatever the weight loss. Yeah, GLP ones, yes. GLP ones. I would know, I'm a skinny guy. Um but uh you know, they're they're they're they're shots, right? And now their companies coming out with pills that are doing that. Well, pills generally, if they're just as efficacious, is a better therapy. It's it's easier to comply with that therapy. It's it's easier, you know. If if we had a pill therapy for MLD, even if it was a gene therapy, it was a pill, theoretically you could get that in any state. You wouldn't have to go to a specialized center. You know, somebody may monitor the monitor guidelines and so on. But so um work with everybody and then talk with advocacy groups, hang out at the big research conferences. We we um we actually recognized after 20 years attending a research conference uh with I don't know, six or seven thousand people that attend, um, as Patient Advocacy Leadership Award, which was really uh it was really a pat on the back and an acknowledgement, not something we sought out. But that was that same conference I went to where they said carbon strand, you know, carbon chain, whatever it is, you know. I can know what it is. And and I we struggle and struggle, but we go there and we talk to the researchers and we talk to the industry people and we ask them questions, and more often than not, in those environments, you'll learn, and it might not be from the people working on your product because they're all concerned about compliance and and IP, but but their peers, they'll tell you lots of stuff about processing questions. I learned more about um um uh infusion um infusions, um uh that kind of stuff from somebody who wasn't our partner back then, it was from somebody else. And they had that other somebody else actually invited me because I'd asked so many questions and learned so much that um um and the name is escaping me of what those infusions are called. I apologize. It's been a long hour here, but it's been really exciting. Um but uh they actually invited me because they had some manufacturing problems, and they said, Dee, can you come in and learn a little bit more and you know kind of be our our our voice out into the wilderness that things are back on track because I have a scientific background, even though I'm not a chemist. And um uh I did that. And it's like those those are wonderful opportunities. And then guess what? Those people, some of them have moved on, they're in companies who we're working with, or companies we want to work with, and so on.

SPEAKER_00 1:05:50

It's just it's just it's a small world, you know. It really it really becomes a small world. Yeah. Well, D, this has been uh an absolutely phenomenal and and fan uh Fantastic conversation and truly eye-opening. A all of the work that you've done, but more so how amazingly positive you stayed through all of it. Um, always looking for the brighter side and always looking for the opportunity that's just around the next corner. I I I love your your take on both how to approach being a patient advocate, but also how to work with industry and the government. Um it it's it's all so vital. My last question is the same question I have for all my guests. I just tweak it a little bit uh based off of age range. But if you could go back, if you could go back to 1995, um when your your daughters were first, your oldest was was first diagnosed, right? Um what would you tell Dean, that Dean, at that point?

SPEAKER_02 1:06:49

Oh my goodness. Well, I told him a lot of things, uh, but uh probably most of those uh weren't right. You know, I made a whole shift of who I was and what I did when my youngest passed away, which was that same year, we were at her memorial service, and um I I I remember distinctly being up on the on the podium or whatever, talking for a few minutes and thanking people and all that. But I said two things. I said, I've up to this point been an engineer in a cubicle, and I'm not gonna be that anymore. I mean, looking out at all of you, and we had the second largest memorial service that church has ever had. The first largest was a sports um giant. Um and uh I I said, I'm I'm telling you that I'm I'm no longer gonna constrain myself to that cubicle that I'm going to engage and I'm going to be a hugger. And I guess that was fortunate or maybe fortuitous because in the rare disease community we all greet each other with hugs. I greet my industry partners with hugs. I uh greet some of the regulatory people, not so much the politicians. Uh but uh, you know, and and we just do that. And I think that's the connection that we have on a personal level. Um I I don't know, uh honestly, you know, um the world is such a different place. Uh and maybe this is the reverse of my don't look back, don't look too far forward. I I don't know where I'm gonna be in 10 years. In fact, my wife and her are talking about this now. It's like we've 25 years of MLD Foundation and we're getting older. And uh you know, what does transition look like? And and it's like I don't know. You know, we we'll just do the best that we can. So um I'll just I'll just readapt my answer and say, you know, just like you don't shouldn't feel guilty about decisions that you have made, do your best. Yeah, I think you should always, and and this is built into me, but always be strategic and tactical at the same time. And um, you know, as a person, I would just say, you know, look look for look for the blessings in every day. There are those little joys. If your if your child or your loved one um is sick, look for the smile, look for the look for the encouragement, uh, look for the hug from your spouse or partner. If your child has passed away or you're you know you are the patient or or you know your spouse has passed away, there there may be something in your day. The logo in our community is a butterfly. And a butterfly, of course, has that whole life cycle of caterpillar and the rebirth and so on. But the logo for, and I doubt that this is gonna work. Uh the logo Oh yeah, we see it. You can barely see that. Do you see the two faces in the profiles right now? I guess one. But there's two faces each other. Look for look, yeah, look for the those aren't my kids. That's somebody that that didn't even really know all of our story that design was a graphic designer, put the butterfly together and said, this is community. And um and so look for those things that remind you every day. Um, and those people that are special to you, tell them you love them every day. Because you do. Don't just assume that you know, flowers on Valentine's Day or a hug once a week is good enough.

SPEAKER_00 1:10:01

Dean, absolute privilege. You're one hell of an advocate, a great MLD dad, and just a wonderful human being. Thank you for your time.

SPEAKER_02 1:10:10

Oh, thank you, Matt. I've really enjoyed this. You know, I we started off and I said you made me tired to talk about my introduction. And uh now I'm all energized up. So I appreciate the opportunity.

SPEAKER_00 1:10:21

Thank you. You're very welcome. Thank you.