Building The Noise with Monica Dudley - Weldon - EPISODE 013

SPEAKER_03 0:00

It's helpless. I remember sitting um in Texas Children's Hospital and I remember sitting in a little kindergartner chair, right? While my son was over by the window, you know, uh I think it was we were on the sixteenth floor of the Wallace Tower, looking out the window, and it was storming raining. It was gray. And I talk about this in my book that day. Um when we got the diagnosis, we were told he has intellectual disability, and they sat there and told me he'll never drive, he'll never he'll never get married, he'll never uh he'll never have a date, he'll never be able to do what his twin sister does. I remember seeing I'm sorry, I'm gonna get emotional. That's when I had to activate and and I had to find somebody. I knew he was not the only one because they already found five others. This was just this was just the beginning and um and you know, and he's beat all the odds. He walked. They said he wouldn't walk, they said he wouldn't talk, and now he tells me he can say a few words, and I and I still have it on on video with when they when he finally told me at night, just out of the blue, I love you.

SPEAKER_02 1:28

Welcome to the Building the Noise Podcast.

SPEAKER_00 1:31

Ladies and gentlemen, welcome to Building the Noise. I'm Matt Teresco, and today I'm joined by Monica Dudley Weldon, a strategist, rare disease advocate, and policy leader, working at the intersection of health policy, biotech, national defense, and patient advocacy, and fierce mama bear, to beckin, and simgap 1 patients all over the world. Monica was appointed by Governor Glenn Youncan to Virginia's health research board, where she helped shape research priorities aimed at improving health outcomes across communities. She's also the founder of the SIMGAP One Foundation, the first organization dedicated to advancing research awareness and support for families affected by SYNGAP 1 related disorders. Her work has helped turn patient and caregiver experience into concrete action, from building a patient registry and a natural history study to contributing to the establishment of an ICD 10 code for SYNGAP 1 related intellectual disability. Monica brings a rare combination of lived advocacy, legal and regulatory insight, biotech strategy, and public policy experience. Monica, it's a pleasure and a privilege to have you here on the show. And as we always start out with, why do you do what you do? And actually, let's go before that, let's go into what are you doing now? We heard obviously Syngap One Foundation, a lot of policy, and you find yourself in DC these days. What is it that you're doing now? And how did you get there?

SPEAKER_03 3:05

First of all thank you for having me. I uh it is a pleasure uh uh to be speaking with you today, Matt. And that you've invited me on to share all this craziness that I have been living this last decade and well more than a decade, almost almost 15 years in in rare diseases that has that has landed me in uh Washington DC.

SPEAKER_00 3:33

Um Texan in DC. It's the movie. That's a movie, I think.

SPEAKER_03 3:38

And I got a lot of hell for moving here too.

SPEAKER_00 3:42

I bet.

SPEAKER_03 3:43

You move in there. Well, you know what? I I got marble fever, um, but I got the bug, and I I really felt that that uh well this was a God calling. I I know that sounds kind of cliche, but um you know when when you see opportunity, you've got to go for it. It's similar to when I started, you know, uh the nonprofit uh bay back in 2014, and since, you know, in December of 2024, we sunset that organization. Um, but the foundation is still moving. We have you know, we still have an organization in the United States that's pushing that research. Um, and then other organizations all over the world, and and and I still have the charter uh in the UK. Um, so yeah, from a sixth grade science teacher to running an international nonprofit. And then you ask me what I'm doing now, it's it has molded and shaped my adventure into national security and national defense and taking the lessons uh I've learned through rare disease advocacy, believe it or not, to move into creating solutions for our federal government in the bio and chemical defense side, which interesting it has just been an amazing uh journey and um a lot of applications of things that we learned, you know, that I learned in rare diseases and how how I you know started the nonprofit to accelerate incentivizing biotechs to work on rare diseases. Well, the government has the same problem in national security. It's just we're defending our nation and we're trying to prepare, you know, medical countermeasures for civilians. And and a lot of those same business models and business policy ended up helping me. And so the federal government came across me uh in uh I actually in um September of 23. Wow um recruited me in to work as the lead on the biotechnology manufacturing portfolio for chemical and biologic defense. And um, I'm currently pending a um a contract and an opportunity to go back in um to working in chemical and biologic deterrence in the programs and policy office. Um, but you know, that of course, you know, in the situation right now when we were talking appropriations, that contract is kind of on hold. But in the meantime, I've been working uh with a law firm here in DC as a consultant on national security issues around critical minerals because we need those for medicines. Yeah, but also, you know, for other national security capabilities that these companies are trying to navigate. And so, yeah, so this is what I've been doing and learning national defense strategy and our you know, yeah. It's just been a wild ride, and I never thought I'd be recruited into working in the Department of Defense, Department of War now.

SPEAKER_00 7:01

Um unbelievable. I mean, Monica, I I met you now 10 years ago, and I can still remember in Philadelphia at a patient advocacy conference, me being that consultant trying to understand what's going on between industry and nonprofits, and most of the nonprofits in the room are quiet, and you know, when there's there's talk of policy, people people get really quiet, and then there's you who is the exact opposite of all of that. And I remember I remember thinking to myself, man, got a really small patient population, number one, right? Sendgap one, how many total patients in the U.S.

SPEAKER_03 7:46

currently in the US? I would say there's probably right now, and now I'm guessing uh about 300. I know there the numbers worldwide have gone up from uh to about 17, 1800 now worldwide.

SPEAKER_00 8:02

Yeah.

SPEAKER_03 8:03

Um, and Beckett was number six.

SPEAKER_00 8:05

And uh and that's what got my attention is most of the disease state nonprofits that I've I've worked with are looking for guidance or looking for different things to do and and don't know how. You never let that stop you or be a challenge. I mean, let's go back to getting Beckett's diagnosis, and at that point, there was no support option for you. So take us back to that and and how you started even thinking of pulling this group together.

SPEAKER_03 8:39

Well, I will say that he was four years old when he was diagnosed. I'd gone through 19 specialists. Um, at the time in 2012, the human genome was just finalizing that mapping of the human genome. And and uh the whole exome DNA test was still on an experimental basis. I they I don't even think they had gone to the next generation uh test was whole genome. And um I remember my geneticist uh when I had gone in for a consultation said, you know, well, insurance won't cover this. Um, and it's gonna be close to about you know $13,000 to $15,000 test. And I said, I'm sorry, I've got to know what's wrong with my son. And he said, Well, that's uh it's like playing the you know, it's like playing the lottery, right? But I I had nothing to lose but money, right? I didn't have the money, so I went and took out the loan. I think insurance covered maybe thirty six hundred dollars of it or something like that. We have to go back to actually know the numbers, but I knew that I had to take out a thirteen thousand dollar loan, which my my Becca's dad was not happy at the time, but I said, I've got to know there was only a 20% chance. You know, we were both teachers, we didn't have the money, and of course, you know, that's when I started learning about access, access into the needed diagnoses, proper diagnoses and access to these tools, right?

SPEAKER_00 10:13

And yeah, the tests, the products, all of it. Yeah.

SPEAKER_03 10:17

This was my first experience on like, well, why? Why is it so expensive? But I had to know, and so I took the risk, and they gave me a 20% chance they'd find out. Sure enough, it was a 14-week excruciating wait for the results.

SPEAKER_00 10:33

Um at that time, yeah. Not not anymore. Now it's what three days max.

SPEAKER_03 10:38

Yeah, yeah. Now it's like the boom, it's like here. They've got, you know, they've got new digital tools that run through, you know, this this DNA uh spectrometer type tools and all that. It's just been incredible half-bassed innovation has moved. And then you know, I they gave me a 20% chance of knowing uh his genetic mutation. And sure enough, I get a call on in fact, this is so funny because I had him on a list, on a waiting list for uh the NIH undiagnosed disease network. Uh the uh they had called me the day after I found out his diagnosis.

SPEAKER_02 11:25

Really?

SPEAKER_03 11:26

Uh-huh. And I said, you know what? I don't need your spot anymore. Give it to the next person. I found out yesterday. And and I wrote it down in my little notebook. I still have the notebook today that said Syngap 1, intellectual disability. It was a fluke, it was a codon stop, which I had no idea. You know, yes, I took genetics in college because my undergrad sixth grade science teacher, right? And so I started digging, and um, and then and then um I there was one research paper identified with the first three uh ever identified in the world was Syngap One, Dr. Jacques Michaud out of the University of Montreal. I pulled that paper, I called, I emailed him, and then he pointed me in the direction of Scripps, Florida to uh Dr. Gavin Rumbaugh, which Dr. Gavin Rumbaugh served on our board uh of the organization for almost seven years. We're published together along with Beckett's doctor on one of the most world-rocking discoveries I uh that opened up uh autism research was a sensory processing um disorder mechanism we discovered in 2018. And in all that, two years after Beckett was diagnosed, I started the research organization. People said I was crazy because I think there was one other one, I think it was NGLI One that had started the same time I started. And we were the only two organizations in the United States with uh with an organization that represented a gene mutation. It was unheard of. And then with the database and the natural history study pilot, that was the first grant I ever won from the National Organization of Rare Disorders. Uh, and the FDA set up this pilot program. And and I have to say, we were probably one of the most successful um platforms, uh, natural history platforms, because we were publishing left and right out of that database. And then that's where we ended up publishing from in Nature Neuroscience, the sensory processing mechanism, that just burst open the doors for autism research.

SPEAKER_02 13:41

Yeah.

SPEAKER_03 13:42

And then then other groups started calling me. And I and I I think I can't even count how many organizations that I helped get started just on a single gene mutation with the same framework that we started with when with our organization. And then, of course, you know, as we started running through um uh the journey of, you know, pioneers get all the arrows, right?

SPEAKER_00 14:10

Oh yeah.

SPEAKER_03 14:11

So being a pioneer, we watched that rare disease landscape, you know, uh, it started a tidal wave of organizations, right? And then because everybody's trying to figure it out, trying to keep up with the technology, of course, that's when the competition comes in, right? And um, but I guess that said, looking back, it was the most excruciating, most painful thing, you know, that I that I had to um go through uh when when I ended up having to sunset the organization. But I didn't realize, you know, and I am a person of faith and a Christian, and I believe that God puts you on a path sometimes. And you know me well because you were on our board.

SPEAKER_00 15:01

That's right. And I I had to learn from you. I had to learn from you.

SPEAKER_03 15:05

I'm glad I I had to learn from you.

SPEAKER_00 15:10

Mutually beneficial is always great.

SPEAKER_03 15:12

And I I will tell you, it I went kicking and streaming, I went kicking and screaming, didn't I? I was like, I did. But I but you know, I realized I remember, you know, after I went into the defense department, I think I called you and I said, you know, sometimes, you know, God just tells you you better let it go. Yeah, because I've got something so much bigger for you.

SPEAKER_00 15:35

You got what you needed from that time period, that learning, the starting not only Syngap One Foundation, but so many other organizations, the natural history studies. I mean, you did more in 10, 12 years than most organizations do in 30 or 40 years.

SPEAKER_03 15:55

You know, and you know it's crazy because I don't even know that.

SPEAKER_00 15:58

It's like I know it's you just put your head down and win.

SPEAKER_03 16:03

Yeah. And I was like, what do you mean it takes 30 years to try to find a treatment? And here's, I guess, the reality of it is that that foundation, and when we had our first international meeting in 2016, the company that has the preclinical data being reviewed by FDA for the first ever potential clinical trial for my son's disease has been eight years. Wow. From the first international meeting, I I believe a couple of the representatives attended. In fact, 30. Eight eight eight years, eight years and less than a hundred million dollars put in one gene.

SPEAKER_02 16:42

Wow.

SPEAKER_03 16:43

And I've kept up with the money because I'm not done yet. I I have plans and I have been working on a project, and of course, you know, I'm not ready to disclose all that yet, but going to law school and learning the business, learning the legal regulatory landscape from beginning to end on every aspect, not just in patient advocacy, not just uh in the science, but you have I mean, it was critical that I learned the legislative policies, uh, regulatory rules, the framework of the government, how it works, and how to leverage it. Because I realized in the process, I'm a lateral thinker. Okay. I see this is and also through this whole process of of grieving the organization. I it this quiet time has really set me uh in a place to sit still and listen and learn and look back at my own mistakes, look at my own uh trials and struggle. And I realize that I'm a lateral thinker, and sometimes, you know, I was like, why don't people get what I'm trying to say? Yeah, yeah, you know what I'm saying? I you know, I see it, but why can't I articulate what I'm envisioning?

SPEAKER_00 18:18

Because most people think of their lane, right? And that's I mean, to just think about all the things you did with the Syngap One Foundation. Most organizations focus on disease awareness and education. We're gonna tell everyone about the disease, we're gonna talk about screening for it, or we're gonna try to get folks tested, and that's it. And you said, no, no, no. We could go this way, we can go this way. There's so many things that we can do. And that's where I think, as you said, the innovators get the arrows. That's where people felt threatened. Like, no, no, no, we want to do just this. And you said, no, there's so much more that we can do for these kids.

SPEAKER_03 18:56

Yeah, and I was really and still am, that this is why I'm working in national defense. I can identify these gaps. And I I didn't realize it. I'll be honest with you, you know, working in Washington, right? And also going to law school and learning these things, laws are created with loopholes.

SPEAKER_02 19:19

Sure.

SPEAKER_03 19:20

And very smart legislators, uh, policy makers are good at leaving open some of those gaps for a purpose. Uh later down the road or or however. Some of those gaps don't need to be there because they cause problems. It's not that it was malicious or intent, you know, or the intent was bad. It's just that's how it was because when you create policy, you don't really understand where it's gonna lead.

SPEAKER_00 19:48

Where it's going. Yeah.

SPEAKER_03 19:49

It's like testing a drug. We don't know the side effects.

SPEAKER_00 19:54

So that's so true.

SPEAKER_03 19:55

That's kind of how I think of policy. And and when I look at a situation, I'm not gonna just look at the problem that's being presented. Well, the problem was there because of a root issue. So let's move backwards and how can we move forward by providing remedies for what's happening now? And because if you don't know the route, you're not gonna find a remedy that is gonna be successful, and that's why you have to understand the whole landscape of business, business, the right structure. Um, you know, I I guess that's just how my mind thinks.

SPEAKER_00 20:36

I I I it's great advice though for for patient advocates today that are running their own organization is to stop thinking so one-dimensionally, especially as we see a lot of organizations not getting it back into the policy work. We you know, we know that budgets were hit during COVID and they stopped doing the policy work, so they're just focused on helping patients. And when I say helping patients, I mean just disease education and awareness, um, and even helping from a financial perspective, but there's so much more. So, Monica, um, as you were saying, we're we're talking about how most organizations are very one-dimensional thinking, but you're a lateral thinker, and I would like to say that that resulted in a lot of strong relationships with industry that many groups don't have because something may not go their way, or they may not get the funding, or they may, you know, think, well, why aren't they doing what we want them to do? And it's more so that you don't understand the business side of it or the regulatory side of it, and you're not seeing eye to eye because you don't understand each other's lanes. How did you really just dive in and and not only develop those great relationships that got you to where you got to in a product or at least uh you know eight years now, right? Evaluating that gene and and beginnings of a product. Product coming. But also those deep relationships that allowed you to do as much as you did.

SPEAKER_03 22:07

Well, I have to say law school helped for sure. I would have to say of course everybody ha has funding, you know, funding issues. A lot of profits, um, you know, I would have to say nonprofits, you know, with my experience, everybody's fighting for those funds, right? Um, part of the reason, you know, I think a lot of nonprofits struggle with funding, especially from pharmaceutical companies, is because of the regulatory issues. And and I didn't understand at first until I started understanding what anti-kickbacks were, you know, what all these different statutes, uh, because there is a chokehold on pharma being able to engage with nonprofits because of the conflict of interest.

unknown 23:01

Yeah.

SPEAKER_03 23:02

And and patient advocacy groups do not understand conflict of interest. They get upset with these pharmaceutical companies when they just can't hand over millions of dollars. I mean, you know, I said the same thing. I was I'd walk into a conference saying, Oh my god, look at this boot. You know what I could do with the money that they built that ice sculpture for?

SPEAKER_02 23:27

That's right.

SPEAKER_03 23:28

But of course, that's where regulation comes in. And this is what where I kind of started when I took business formations in law school. Uh, I realized that nonprofits, they are good. And however, when you're gonna ask for funding from a pharmaceutical company, uh, they they can only fund, say, like educational initiatives, and they only have a certain amount of money in that bucket, right? Pharmaceutical companies have many, many buckets that they can pull through commercial, you've got the marketing, you you've got all of these things.

SPEAKER_00 24:10

Sponsorships, right? They're they they have their own nonprofits now.

SPEAKER_03 24:14

Yes. Well, and then, you know, they're yeah, also owned by insurance companies now. Well, you know, the whole all, I mean, they're you've got insurance, yeah, you've got the whole lot of vertical integration, conflict of interest and and regulatory gaps that need to be uh filled, you know, are are are some of these companies and these mergers and acquisitions. And and I took that in law school too, and I understand and I can see those. Well, as a nonprofit, you can't dip into commercialization money because you they can't use a nonprofit to commercialize because that's a conflict of interest, that would be coercion, right?

SPEAKER_02 24:54

Yeah.

SPEAKER_03 24:55

Um, and I looked at some other uh business models. Uh and the, you know, of course, I studied what I have structured now for myself uh that I've been working on is a new business model where many of those things can be tapped into, but not necessarily under a nonprofit and get the same result.

SPEAKER_02 25:18

Yeah.

SPEAKER_03 25:19

And um a nonprofit is part of that piece, but it has to be a legal entity outside of say a benefit corporation or a B Corp and um or an LLC. Um there are just so many different business formations formations you can uh attach to to do some of the things that nonprofits or patient advocacy groups want to do.

SPEAKER_02 25:44

Yeah.

SPEAKER_03 25:45

And I've been consulting a little bit on the side with some of these newer genes that are coming up. The some families here that, oh, you started a nonprofit. And I actually honestly I'm advising against a nonprofit, a pure nonprofit. Uh and this is where my national defense experience came into the rare disease space to be able to create a potential bit business model that would serve better the rare disease space, like the ones that we're using in national defense. Just some of the same policy in rare diseases and presented and proposed them for national defense, and two of them are being are implemented right now. That are in a pilot that are in pilot programs, and I did that at the Defense Department when I was there before. Um, and one of those is a national priority voucher, and that I proposed what initially was the priority production voucher, but then I pitched it to Congressman McCall, who did was the author of the priority review voucher, and um said, Why don't we do this for medical countermeasures? And there he was like, Oh, so I sent him my draft and my proposal that I sent to the Defense Department, my leadership there. And and it turns out they they launched last July the national priority voucher, where those countermeasures can be created. Well, the gap I believe that rare disease has, and I'm gonna flip back to rare diseases now and these institutions, is the only way that you're gonna sustain research without depending on the federal government create something that you can commercialize. Yeah, and it doesn't have to be attached to your rare disease, it could be, but it's hard, right? A lot of these moms and dads that have these nonprofits, they're still at the kitchen table, they're taking care of sick kids. So I was willing to step out on a limb and I sacrificed a lot uh doing it, right? To pursue what I am now. You know, I went, you know, on a personal, I went through a really bad divorce, you know, and and moved states, uh lost a lot in between. Um, but that's okay because it gave me the quiet time to think about my next step. What really what God wanted me to do, what my next steps were.

SPEAKER_01 28:14

Yeah.

SPEAKER_03 28:15

Um, and I think rare disease uh nonprofits are are missing, missing part of a business model that they could grasp onto even as a um a collective effort on a business model that would fund their rare disease. And data, of course, is one of those. And and of course, you know that the model that I had for our nonprofit before was to take the data, license it out, continue to license it out uh to companies that needed it. And however, you know, you know, the nastiness that happened in the space and the competition scared people away, ran researchers off, ran companies off. We had 12, I believe we had 12 companies working on Syngap1 at the time and when I sunset the organization, I believe there's only really two or three still active. And that was devastating. So, how do you get that back? Because you're not gonna have enough money. Um, there's already a fracture in the community. How do you build that back? And you know what? I'm one of those who who, if you build it, they will come, but I need to build it right the neck of the second, I have another opportunity to build it right and learn from the lessons before. And so um I really do believe creating something that you can commercialize will bring a sustainable profit in to help take some of those profits to fund in your own research, to harness the data that is needed, because the rare disease data is diamonds. I mean, the gold. Oh, yeah, platinum. I mean, we're we're talking, and you have to leverage that. And everyone poo-pooed on the fact that you're selling my data, you're selling my data, but it's not for nefarious purposes, it is to create your own bio-ego economic bubble sustain the work that needs to be done for the patient community, and so um, you know, and I and might be given a little bit away what I'm doing, but the effort that I've been working on is going to take years, it's gonna take five years, and I had to come to the realization, just like when I started the nonprofit, that it might not ever help my son. And so that a lot of families believe they want to do whatever they can for their child, and I don't blame them, but it can't just be for that. They have to come to the acceptance and the reality that it's gonna take years and years, and it may not help them, but the attitude is that you're building a legacy, and once you've built a legacy, you can live and know that somebody that came behind you has hope that you didn't have, and that's why I built the organization to begin with. Also, I wanted my tribe. I needed to know that somebody was out there experiencing the same thing I was. That was that that kept me from going over the edge.

SPEAKER_00 31:38

Now that is what we hear from a lot of advocates, right? Is that is it just me? And maybe you can talk a little bit about you know that feeling in the first place of getting that diagnosis and knowing there were no other parents out there that you could turn to.

SPEAKER_03 31:59

Yeah, I it was it's helpless. I remember sitting um in Texas Children's Hospital, and I remember sitting in little kindergartner chairs, right? While my son was over by the window, you know, uh I think it was we were on the 16th floor of the Wallace Tower, uh, I think that's where it was, looking out the window, and it was storming raining. It was gray, and I talk about this in my book uh that day. Um when we got the diagnosis, when we were told he has intellectual disability, and they sat there and told me he'll never drive, he'll never get married, he'll never uh he'll never have a date, he'll never be able to do what his twin sister does. I remember seeing I'm sorry, I'm gonna get emotional.

SPEAKER_01 32:54

That's okay.

SPEAKER_03 33:11

Yeah. Because they put limits on him. They, you know, and I sat I don't know. Yeah, and I sat and watched his dad. This was his only son, you know, and I know we're you know, we're no longer married, but I remember that pain uh and seeing that devastation, you know. And part that's when I had to activate and I had to find somebody. I knew he was not the only one because he already found five others. This was just the beginning. And um and you know, and he's beat all the odds. He walked. They said he wouldn't walk, they said he wouldn't talk, and now he tells me he can say a few words, and I and I still have it on on video with when they when he finally told me at night, just out of the blue, I love you.

SPEAKER_00 34:19

You know, that's amazing.

SPEAKER_03 34:21

And those little things are kept me going to know that there was a glimmer hope, but you know, you know, Beckett has no idea what he did for the world and his twin sister.

SPEAKER_00 34:37

Yeah. I mean the tw you hear people hear all the time about twin studies, right? But when you're dealing with a de novo mutation that has not really been seen all that much before, to be able to have a pair of twins to even take a look at is immensely um helpful, not to mention just rare in the first place, on top of rare.

SPEAKER_03 35:02

Yeah. And you know, Piper, you know, she's had to go through a lot, you know, she couldn't have friends over. Uh um so it was important for that. Was another thing, it's important to get in touch with families who had siblings that were going through the same thing um that they knew they weren't alone because she was isolated. I mean, we didn't get to go to birthday parties like you know, like like a lot of people. We couldn't go out like a lot of people, um, as a family, right? It was each one of us, you know. And I have to say that put a strain on our marriage too. That was uh, you know, but Piper changed the world too. I mean, I still, you know, I have the pictures of her sitting giving her blood uh for the stem cell uh project that we that we funded at Texas Children's that helped find the the the array of drug molecules that they ran on I think it was 17, maybe 17, 14 to 17 kids that we that they had seen. And Piper was the control. You know, and Piper and Beckett was in their gate study, and we found a potential biomarker with the gate study that she attended. Right now, Piper, she's eight years old twins are 18. Can you believe it? 18 crazy and and Kennedy Krieger right now is doing a study, and Piper at 18 is gonna give her blood again so they can grow organoids uh to compare. And Beckett and Piper are gonna be in that study that they're doing at Kennedy Krieger. So it's still going on, even though I you know stepped aside.

SPEAKER_00 36:43

She's so giving of herself and her time.

SPEAKER_03 36:46

Yeah, yeah. And and the thing is now I can't tell her she's gonna go give blood.

SPEAKER_00 36:52

Yes.

SPEAKER_03 36:53

I have to say, I can't give consent anymore. So and what I'm building is for them too, the new venture that I have been working on the last few years, and I'm building building out a um an incredible team and the opportunity uh to create something that has never been done before again, and and to show show this community that you don't have to dissolve after 10 years if we can fill that gap, but it's gonna make work, and it's gonna take the know-how, and it's gonna take the people uh to you bringing people on with certain skill sets. And I believe a lot of these moms and dads who really want to do this, they don't have that expertise. And I guess I gave one up for a team, and I remember there was a friend of mine that said, Monica, when you do this, when you start this nonprofit and you start doing and running a business, she goes, I've seen it before. She goes, But Monica, you you're gonna sacrifice everything in the long run. And she goes, Are you prepared to do that? And I said, Yeah, I have to. And I, you know, of course I was hoping it would end differently, but it didn't. But but you know, God has a way of restoring your life after a sacrifice and making it so much better and seeing that unfold.

SPEAKER_00 38:26

You can recreate time so quickly.

SPEAKER_03 38:30

Yeah. And you know, we can't control what other people do, but you teach it and use it as a lesson moving forward. And I do believe that everything that's happened in the n you know, me moving into national defense and using that also as a vehicle to preparing what I have been working on the last three years. Because I started this venture about three years ago, and it's not public, uh, but but but it because it's not it's not hatched yet. I'm not it's not it's not ready to hatch yet.

SPEAKER_02 39:08

Sure.

unknown 39:09

Okay.

SPEAKER_02 39:09

Yeah.

SPEAKER_03 39:10

You know, and and I think and I'm hoping that my advice, my example to other rare disease nonprofits, that when this does hatch, that people will have the patience and the fortitude and the the persistence, the tenacity that goes with building something, that will last.

SPEAKER_02 39:36

Yeah.

SPEAKER_03 39:37

And I have to say, and okay, so I might ruffle some feathers out there when I say this, and that's okay because guess what? When you're uncomfortable with something and you don't like hearing something, then maybe it's time to think a little bit differently about things.

SPEAKER_02 39:52

Yep.

SPEAKER_03 39:53

But on a personal note, I get really frustrated when people allow policy, politics to get in the way of innovation. I don't care if you don't like the policy. I don't care if you don't like the person. If you cannot put your emotion and your personal opinions aside and focus on the mission, you will never get anything done because you've got to work through it and around it. And sometimes the best thing is to ride the wave. Where's the opportunity? You might not like what's happening, and you might not like what's happening is because you don't understand everything that's going on. I'm talking about the president's agenda. I think he's I think he's a brilliant businessman. I don't care what anybody says uh uh you know politically about him at all. Sure, I believe he's brilliant. The business model that he's creating and that I've learned from to be able to create what I'm working on and ride the wave and understanding geopolitics. If you don't understand geopolitics, then then honestly, you you're at a disadvantage.

SPEAKER_02 41:08

Sure.

SPEAKER_03 41:08

Because we're talking pharmaceuticals are everywhere, it's not just here in the United States. Research is everywhere, not just in the United States. And and if you don't understand how different regulatory avenues and and mechanisms are being used around the world, you're gonna be at a huge disadvantage because it limits your options. You've got to think big. It's like expanded access. You know, sure. You've got to think and understand models, and that takes a lot of hard work, takes a lot of reading and a lot of time to understand it. You know, I before we started, I showed you a binder I put together last week to help me understand better, because that's how I learned processes. If you don't understand the processes, but a lot of people just aren't willing to go that extra mile, and then they well, I'm gonna just gonna say it, they bitch and gripe about them staying stagnant in the same place they are, because they're not willing to get uncomfortable and do something they don't like or learn something they don't like to get.

SPEAKER_00 42:14

Sure, or work with someone they don't like.

SPEAKER_03 42:16

Exactly. Exactly, and and this is and of course that's why you have all the splinter groups in rare diseases. Yes, because everybody thinks they can do it better.

SPEAKER_00 42:28

No, I I don't think people recognize how big of a problem that is in rare disease. Um you know, uh we see the amount of impact that uh happens to the advocacy space when industry changes people in seats.

SPEAKER_03 42:47

Yeah.

SPEAKER_00 42:48

But it's I would say exponentially worse when there's fragmentation for patient populations because folks don't get along and someone thinks they can do it better, and they branch off and they create a different group. Now you leave the patient population wondering why is this? Why is it that this is happening? And as always happens, in you know, the behind the scenes talking and complaining always seeps in, and we lose track of what actually matters for patients.

SPEAKER_03 43:22

Yep. Yeah, and also another uh you're exactly right. And another problem is you've got a lot of bad actors out there, and there are a lot of charlatans, and trust you me, I can pick them out and I can spot them from not just a mile away, but I can sniff them out 10 miles away. And all it takes is asking a few questions because it comes down to motive, it comes down to what exactly are you trying to do? And a lot of these groups uh you know have their own agenda, and I'm looking at it going, oh my God, why is this? Parent putting $200,000 in this effort and the change. That is like playing blackjack in Las Vegas. That's real, and that's a bad investment. And they don't, and it's all because of emotional desperation. They're willing to now, granted, I took out a loan. Granted, I I took a shot at genetic testing. But also, you're not risking your child's life doing a genetic test. When you're putting all this money into a company that claims they can come up with your own precision medicine, whatever, you don't have a natural history study yet. You don't even have a defined biomarker yet. What are you doing? Those are charlatans. And then to see these same companies. Now I've watched this over the last five years, who's grabbed millions of dollars off of patients. And these communities they get called out or they get in trouble, they go bankrupt, they don't deliver on what they've promised, they go under, and then guess what? The same group of people crawl up somewhere else and pop up somewhere else under a different company name. And I watch all that. And patient communities do not track that stuff. I do because you do. Because a lot of that happened in our own patient community. And uh and I know who they are. And the thing is, everyone's scared to hold people accountable. I am not. I have nothing to lose.

SPEAKER_00 45:37

And I'll call you out in a heartbeat because they're they're worried that their kid is gonna catch some sort of you know pushback or that their kid's gonna get injured in the process when in reality, if it doesn't feel good, it's not good. If your gut's not if it if your gut doesn't feel right, it's not right.

SPEAKER_03 45:58

Nope. Nope. And yeah, and that's when my spidey sense comes up. Every time I hear, you know, some uh stem cell treatment for whatever, and I'm like, so can you provide to me the peer-reviewed studies for this? You know, show me, show me, show me what where where has this worked before? And the and what's frustrating for me is I do know the science. A lot of these other when you're talking about just the general population, they don't understand the cellular uh mechanisms. Now I don't know everything, but I know a lot because I have the background, but I mean, it's snake oil is what they're saying.

SPEAKER_00 46:42

Well, I think Monica, what you what we skip over that I think that everyone needs to understand when you say, you know, I studied the science, I didn't know it, but you did uh a population study, if I'm not mistaken, that was a sixth-grade science experiment.

SPEAKER_03 46:57

Yes. Yes, we designed the first autism gut study uh in partnership. I helped design uh um uh the autism gut study using a seventh grade lab that I did in digestion, where we took blue food dye because blue blue dye doesn't digest. And um, well, in okay, in school it had to be fun, so we put it in icing. Well, it's so funny. We could do that in school, but when we ended up approaching the FDA, they said no sugar. It has to it has to be free, sugar-free. Basically, it tastes like cardboard. No, I'm just kidding. So during COVID, um, we we uh partnered with um uh University of Miami, Miami University in Florida, uh and uh Atrium Healthcare in North Carolina, and um uh they created during COVID, we did all this, all this stuff, you know, we were still working even in COVID, you know, right? Uh all virtual. Well, the researcher at Atrium and uh Florida got together on Zoom and they made what they call the the uh Smurf cakes. So they took the blue dye, they created their own recipe, and then they would ship the cookies. Uh we did a pilot study with I picked a few, you know, I put it out there, and a few of the parents volunteered uh to um to have their child eat the cookie. We determined the the time, which is the morning when they're hungry, and then our kids will pretty much eat everything. But we had to get them to at least eat a uh three-quarters of the cookie, I believe, or two-thirds of the cookie. And um, and then we would time it, and then it turns the poop blue. So when you saw blue poop, you could track the time from the time they ate it to the time that they excreted it out.

SPEAKER_02 49:00

Yeah.

SPEAKER_03 49:01

So you could track motility. Because uh our you know, most kids with intellectual disability also have gut issues and and they can be toxicated and things like that. And and because we're thinking, well, we can't give them the radioactive beads, y'all do the zebra fish, because that they number one, we can't throw our kids in front of an x-ray to track all the things. So um, so we did the the the blue food dye from my the seventh grade lab that we did. And that's fantastic.

SPEAKER_00 49:33

Um, but we helped design a bunch of different different things using real-world application to move the and this is why the this is why your patient population and and the the parents trusted you is that you took what was very difficult and frontier science and simplified it for them to understand in a way that allowed them to also have a voice. I tell this story often. I remember when we had the scientific meeting in DC and all of the you know innovative companies were there talking about the C-word, right? Curing, and um, we're gonna fix this. And the parent that stood up and said, you know, what about the epilepsy? Yeah, yeah, a cure is great, but my kid has seizures seven to twelve times a day. I don't know when it's gonna be the last one. Can you can we do something there?

SPEAKER_03 50:32

Yeah, right?

SPEAKER_00 50:33

And I remember all of the researchers sitting there looking at each other like, man, we're dumb. You know, like how did we miss that?

SPEAKER_03 50:40

Yeah, yeah. It well, yeah, and it and and that's why the patient voice is so important because it helps you target what's the most important. That's and you know, the patient-focused drug development meeting. I can't wait to believe we actually pulled it off during COVID.

SPEAKER_02 50:54

I know, yeah.

SPEAKER_03 50:55

But that report, which it took me, oh my god, how many years did it take me to do that report? Three.

SPEAKER_02 51:00

Three.

SPEAKER_03 51:01

I was in the middle of law school too, but I finally finished it.

SPEAKER_02 51:04

Yeah.

SPEAKER_03 51:05

But that helped uh navigate, you know, creating new new clinical studies for bio potential biomarkers.

SPEAKER_02 51:16

Yep.

SPEAKER_03 51:17

Um, new air, new places to look for what was important to the patient, right? Behaviors, behaviors, you got epilepsy, uh, intellectual disability. Like I can live with intellectual disability. It's the meltdowns, it's the behaviors, it's the seizures, right?

SPEAKER_02 51:37

Yeah.

SPEAKER_03 51:37

And so that was an amazing meeting that also drove more clinical research. And Europe's taken off, too. I have to give kudos to the German uh parents because um they probably now have, you know, I've suns since sunset the database, it's still available on your platform to be able for for scientists to retrieve that data that's left in there. Um, but the Germans have taken off with uh with a really big database, and they just published um a study a few weeks ago, I guess came out. And uh they're doing and so I look at that, even though I sunset the organization, but I look at that and said, you know what?

SPEAKER_02 52:21

I started it.

SPEAKER_03 52:23

And I'm so proud of them taking the baton and moving forward because it because honestly, it was killing me before. I mean, it was killing me. It was and and I think I had my own little nervous breakdown in the meantime, but but you know, that's okay because you know what? God had something else for me. And uh, but I can still dabble in it. I have a bigger, much bigger uh envision plan, as I said, it's just not ready to hatch. But uh we're gonna build off what's what's one thing at a time, right?

SPEAKER_00 52:58

One thing at a time.

SPEAKER_03 53:00

And well hey, but yeah.

SPEAKER_00 53:02

This has been this has been an absolute privilege. My last question is the same question I have for all my guests.

SPEAKER_02 53:08

Uh-huh.

SPEAKER_00 53:08

I just tweak it every little bit. If you could go back to 2012 and speak to the you that was there in the children's hospital, what would you tell her?

SPEAKER_03 53:24

I would probably tell her that um get rid of the imposter syndrome, be confident in what you know, and always be willing to learn even more from other people. Don't act like the smartest person in the room because you're not, um and listen more than you speak. And also, I would have to say use your energy to stay focused on the mission because there were so many times I kicked myself by not focusing on the mission. And I have to say I've grown up, I think, a lot uh from my experience then and into the person I am now where I pause before I respond. But then also I've learned that when you listen, you do not listen to respond, but you listen to understand and that's what that is probably one of uh the things that I've learned in this quiet time of mine is to open your mind to different perspectives and you don't get emotional about it. Sure, it's not personal. Um, you know, I think through pain you grow.

SPEAKER_02 55:07

Right.

SPEAKER_03 55:08

Uh you know, and and I'm sure the pain is not over. You know, we're living in a world of pain.

SPEAKER_00 55:15

Yes.

SPEAKER_03 55:16

And and but without pain, you don't grow. It's how you handle your pain.

SPEAKER_00 55:22

And Monica, you have taken the pain of so much from Beckett's diagnosis to your divorce to sunsetting the organization and so much more in between it all. And learned not to respond or lash back or any of that, but to learn and do the next right thing. And I can't wait to see what God has in store for you in your next chapter.

SPEAKER_03 55:50

Well, thank you. I can't wait either. I told God I'm not going down in a tragedy. We're gonna finish in a triumph. And you know, I I do have to say that uh, you know, I hope people see uh that if I died tomorrow that I hope that God that people would see that uh God and my faith in Christ is number one and that um to remind people never let fear keep you from doing anything. Face every challenge head on, and whether you get pushed, you know, two steps back, keep taking those steps forward because eventually you're gonna get there. And I say I hope I'm living proof of that.

SPEAKER_00 56:44

That's that's for sure. You know, as they say, you know, you don't have to not be afraid to do it, just do it scared. Yep, as long as you do it.

SPEAKER_03 56:52

And even if you'll know what you think anyway.

SPEAKER_00 56:55

That's right. Okay, someone's gotta do it. Might as well be you. Right.

SPEAKER_03 57:00

So exactly.

SPEAKER_00 57:01

Well, this has been an absolute privilege, Monica. I really appreciate your time. Thank you, thank you, thank you.

SPEAKER_03 57:07

Well, I'm so excited. Thank you so much, Matt. Uh I really appreciate it, and and there's more to come.