The Path with Becky Quick: Unlocking the Rare Genetic Code 2/10/26

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0:57We set up a state-funded newborn screening program to offer children whole genome sequencing at birth, free of charge. So it's initially... Any parent who wants to put their child into this? Yes, it's an opt-in program, so it involves their consent. When you think about Drew, is he forever four years old in your mind? Oh, that's a great question.

1:26This is The Path. I'm Becky Quick. Thank you for coming back. In my 25 years at CNBC, reporting on the stock markets in American business,

1:40this is the most important work I've done, bringing light to the sometimes dark and murky world of rare disease.

1:53Our nine-year-old daughter, Kaylee, our youngest child, has a rare genetic disease. Her journey and her battles have changed me in so many ways, all for the better. But walking that path, each slow step at a time, is not an easy one. That's Kaylee and Julie. Kaylee and Julie. And Julie. He is so beautiful. A rare disease diagnosis can be overwhelming, confusing, devastating. And for families navigating a new normal, it can be lonely. You know, the breath comes out of your lungs. Hi. Who's a happy baby? Who's my boy? Andrew Jones! Adam Anderson's story is like the story of many parents who face the unimaginable.

2:43Trying to get help for a child with a medical condition that doesn't make any sense, that they probably have never heard of. There was a list of, I don't know, maybe about two dozen or so conditions. And Tay-Sachs was the one that was the absolute worst. None of them were good, but it was, please, not that one. Rare diseases impact tiny populations. Just getting a diagnosis can be an odyssey. There were many, and they were all wrong. But maybe the path that we as rare disease families are walking doesn't have to be that lonely if we do it together.

3:24Drew passed away on just before Christmas of 2019, December 22nd. So he was only four years old? Four years old. Four years old. So I felt like if we canceled Christmas, we'd be given in. We couldn't do that. Adam lost a child and did the hard thing. We fought. He fought. He turned that loss into action almost immediately. What if we can get to them before they have symptoms? What if we can get to them before the neurological damage is done? He changed his life. He changed his career. We're celebrating the signing and the funding of HB907, the Sunshine Genetics Act.

4:09He changed laws, opening new paths to genetic testing and helping other families. This is the path to change, the path to answers. God, is this really where you want me to be in this room right now? I don't want to be in this room, right? The path to community. If that's where I'm led, that's where I'm supposed to be. The path to a cure.

4:38That was true. Happy little guy. So smiley. Yeah. That's probably what, like three or four months? Yeah, about that. Yeah. Yep. I'm Adam Anderson. I have the privilege of representing the northern part of Pinellas County in the Florida house. I am a loving husband, married my high school sweetheart, and a proud father of three beautiful children, Kaylee, Julie, and Andrew. it's been the greatest blessing of my life to be able to be a father and be able to have such an important role in the lives of your children. I don't know if there's anything more satisfying than that. What happened with Drew that was different than your other children?

5:21Well, Drew was born, he was our third child. And you don't think a whole lot when you have The third child's pretty routine. It was for me anyway. It's easy for a dad to say that, I guess. Hi. Who's a happy baby? Are you ready to do that? Are you ready to do that? He was a happy baby. He was perceived to be very healthy. There were no warning signals, no red flags. And I remember thinking after he was born that this is it. Our family's complete. What's today? January 2016. Drew's first sleigh ride. You know, this is now we have the classic American family, right? Two loving parents, two older sisters.

6:14Now I got my boy. We had the dog, right? So we had the whole package. But around six months or so of age, Brianne started noticing some things that weren't quite right. I didn't believe her. Our pediatrician didn't believe her. But moms know these things, right? And I always say that there's really nothing more powerful than the mama bear advocate. Dads are pretty good too, but there's nothing stronger than a mama bear who thinks her cubs threatened. And that's what it was. So we spent the better part of a year, a little longer. What's so funny? I love you, buddy. but Drew would not hit his marks.

7:00He wouldn't, he was missing some of his developmental milestones. And I remember the pediatrician saying, well, you know, you have to understand, you have two daughters that are also pretty far advanced. Our daughter Kaylee was literally running on her first birthday, and that's not common. So they tell us, well, your boys are just a little bit slower. You just, mom, you have to just accept it. It's what it is. And that was my opinion too. And then not making the milestones change to some regression. And that's where I said, okay, well, that's not, something's not right there. Regression in terms of what?

7:36He was sitting up and then was having a hard time sitting up. He was able to hold his head up perfectly fine and then, you know, wasn't able to hold it up all the time. Smaller things. And then he would get the regular colds that kids go through as they're building their immune system. Every time that would happen, he'd suffer from another regression of something. So then doctors started listening and we started receiving, unfortunately, the wrong diagnoses for several months. What kind of things were doctors telling you at that point? There was a whole list. there was you know just torticollis on your neck so you know things that were more physically developmental some more cognitive cerebral palsy was one there were many and they were all wrong and then it was probably around 15 months old that where we finally pushed very hard to get genetic testing completed on Andrew.

8:41And that gave us the ultimate diagnosis that he had Tay-Sachs disease. And Tay-Sachs, had you ever heard of it at that point? Never heard of it. We heard of it when we were kind of going through these missed diagnoses, but we really started learning about it when we finally got the genetic panel done. There was a list of, I don't know, maybe about two dozen or so conditions that could be detected on this particular genetic test. And I remember researching thoroughly every single one of them. And Tay-Sachs was the one that was if the absolute worst that it could have been outcome. So there's almost everything else on that panel.

9:23None of them were good, but it was please not that one. And unfortunately, that's what it was. What is Tay-Sachs for people who aren't familiar with it? Tay-Sachs is an ultra-rare genetic disorder. So in the rare disease sphere, they're all rare or they're considered rare. This is ultra-rare. So at the time, there were, I think about 16 children that were affected by Tay-Sachs disease in the United States. In the United States. In the United States. Not a whole lot more than that around the world. Over time, the community was so small that either Brianne or I had a personal relationship with every known family that had Tay-Sachs disease around the globe, as far as India, Brazil, South America, and throughout the United States.

10:11So it was a very small community. But it's a condition that is much more common in the Ashkenazi Jewish community, which we are not. So when a mom goes to the doctors the first time that they're pregnant, they're usually screened for this. They'll ask those questions if you have this ancestry. And the answer was no for us. And then you move on. But it's a very, very debilitating disease. So it affects your cognitive function, causes paralysis, loss of fine motor skills, gross motor skills. Probably the hardest really were the seizures. The seizures were pretty horrible. There were days when we were trying to get them under control where Drew would have 50, 60, even 70 seizures in a single day, which was very, very difficult as a parent to see that and not be able to do anything about it.

11:06Right. The seizure started before or after your diagnosis? Probably around that same time and then definitely got worse after. So the day you got the diagnosis, where were you? Do you remember how you felt that day? Yes, I definitely do. We were at CHOP in Philly. The Children's Hospital of Philadelphia. The Children's Hospital of Philadelphia. Can't say enough great things about that hospital. They do a phenomenal job. And we probably spent an hour and a half or so with the geneticist walking us through the results. And, you know, we knew that was the last thing that we wanted to hear come out of our mouth.

11:53And it was almost like just, you know, the breath comes out of your lungs. It was a debilitating message to hear. and he drew was with us um and i had to be the strong one there and that that was hard that was really hard um i remember leaving that visit and uh brian not even being able to walk out of the elevator it was it was hard to get back to the car was it just the three of you it was Who held Drew? Well, it was hard for her not to give him up, actually. But, you know, we've always both hold him all the time. Yeah. So you hear it's Tay-Sachs. At that point, they don't have any real therapy or anything they can really tell you to do.

12:49That might have been the hardest part of it is that, you know, we knew that there were no treatments. We knew that there was really nothing that can be done. But then when you hear a medical professional also tell you that, it really hits home. So the message was more or less spend as much time as you can with them. So what did you all do? We fought. We fought. So we regrouped. We had that gut punch that lasted a couple days. but and this goes back to the mama bear right mama bear went out and and found every every support group every non-profit that was somewhat even related to tay-sach's disease networked with with every family that that she could uh found all the best caretakers neurologists um not just from from this area but beyond we traveled all over the place Hello, my name is Brianne.

13:48I wanted to introduce you to my family, the Andersons. But first I wanted to meet the guest of the hour, Andrew. Andrew was diagnosed with Tay-Sachs disease. and we found it we found a research doctor up at UMass Dr. Miguel Estevez is his name still working on curing Tay-Sachs disease today and he was working on a clinical trial and that gave us a lot of hope a lot of hope so we almost immediately went and scheduled some time with him and drove up to Boston he got to know Drew he got to know our family and we realized they needed money to do this. So we set our sights to doing everything we could to raise money through the different nonprofits.

14:37The Cure Tay-Sachs Foundation was a phenomenal one. Brianne still serves on the board of the Cure Tay-Sachs Foundation today. And that organization and a collective group of parents helped get this gene therapy off the ground and it's working. Unfortunately, it's working. Unfortunately, it was too late for Drew to be able to receive it, But yes, it's working. It's halting the progression of Tay-Sachs. Wow. They're on the second round of it now. So there's some kinks that need to be worked out. But there's definitely promise for sure. The second round of it? Of the gene therapy. The gene therapy trial.

15:15How many kids were enrolled in the trial? The first one in the... I want to say just a handful. Definitely not a dozen, just a handful. Okay. Because there's not a lot of these kids. And it's very difficult for these research doctors to be able to find them. And that's one of the things that we really wanted to solve is how do we figure out a way where we can diagnose these children early on and be able to offer their parents an opportunity to receive a gene therapy, even if it's a clinical trial, to receive a treatment before they're symptomatic. So if we know that there's a condition that has a treatment that's halting progression of the disease, what if we can get to them before they have symptoms?

15:56What if we can get to them before the neurological damage is done? Can we stop it from ever presenting itself? Right. That's the promise. Hi. Come on, jump.

16:15Let's go back and just talk a little bit more about Drew. He was born in August of 2015? Yes. So that means you got your diagnosis around November of 2016? Yeah, it was around Christmas time. And the progression of his disease, did that happen pretty rapidly? It did. You know, sometimes when you're really in the thick of it, as a caretaker, it's hard to see that. You know, as they say, the days are long and the years are short, right? And it's very true when you're in an acute, intense situation of being a caretaker. But it did. Yeah, it was every time he had any kind of infection, a respiratory infection, a common cold, he would suffer from regression.

17:00His body just couldn't recover from the stress of having some other illness to deal with. So we were always very adamant about keeping as much of a germ-free environment as possible, which we had a toddler and and a seven-year-old right so they were in public school it's almost impossible to do right um but we did you know we made it through it um the girls were phenomenal caretakers for their little brother they were they were involved all the time yeah his sisters one of the pictures that you showed me his sisters are reading to him in bed too yeah A special time for everybody. It always was, yeah.

17:47We'd encourage that because they were learning how to read at the time. So they're practicing reading and getting to spend time with their brother. He loved it too. We'll be right back. From college send-offs to retirement dreams, life is filled with many important milestones. and making sure you have a plan in place to protect the people you love can give you confidence for whatever comes next. State Farm Life Insurance can help protect your family's financial well-being through life's milestones. Your State Farm agent can help you choose flexible coverage you can adjust as your family's needs change.

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19:29And that was right before Christmas, I think. I think he passed away maybe a week or two after that picture was taken. He's a Jets fan. Well, you know, they say you're born Jets fan, so that's how I became one. So yes, he was a Jets fan too. He was born Jets fan. It's the only way you become a Jets fan, I guess. I don't think Jets fans are made. I think they're born. And this was Christmas Day. Three days after.

19:58Drew passed away on just before Christmas, of 2019, December 22nd. So he was only four years old? Four years old. Four years old. What did your family do after that? Well, Christmas is a really big deal in the Anderson household. Many call me Father Christmas in my family. And I knew that we host Christmas every year. It's a tradition for many years, 20, 30 people in our house. And we planned to host that year, just like we did all the other years. And a lot of our extended family wanted to relocate Christmas dinner that night. They wanted to bring it to one of their houses. But I was adamant about not doing that for a few reasons.

20:43One, I own Christmas and I wasn't giving that up, but I wanted to make sure we kept the consistency of, for our family, for our daughters, and for the rest of our family too. This was a hard situation, not just for us. You know, sometimes we had to be strong for our parents and our siblings and our cousins. And that kept us going, though. It kept us going. So I felt like if we canceled Christmas, we'd be given in. We couldn't do that. So we hosted Christmas. But then you did move back to Florida. This was right around the pandemic? Yes, very, very beginning of the pandemic. And you moved back down.

21:27and I guess over the course of the next year, that's when you were deciding or you were kind of being pushed to run for state legislature. I was being motivated by my cousin from Lee at times, um, rejecting his motivation. And Brianne had asked me, she said, well, are you praying about this? And I said, well, I don't know. You know, I asked, I asked God for guidance and strength on a regular basis daily. She said, no, no, are you, are you asking God to lead you where he wants you to be. And I thought, huh, I don't think so. I don't think I've ever done that. Um, so I started doing that every day.

22:04I still do it every day today. And that was the catalyst because these doors started opening up opportunities were preventing, presenting themselves. And there were many times I didn't think like, God, is this really where you want me to be in this room right now? I don't want, I don't want to be in this room. Right. Um, But, you know, I've always had the belief that if that's where I'm led, that's where I'm supposed to be, right? And if the door opens and I don't walk into it, I'm just doing a disservice. And at the same respect, some doors closed. I mean, that wasn't the direction to go, but it definitely turned out to be the right thing to do as much as I rejected it myself.

22:42Yeah. Part of your experience as a parent and in particular as a parent to Drew has helped mold the type of state representative you are. How does your experience play into the way you are helping govern the state? That's a great question. I would say it's molded just the person in general as well, right? I never, when I first agreed to run for office, I never intended on working on rare disease policy. A lot of people think that's what I assume. Yeah, a lot of people assume that. And that's fair because it's made up such a giant portion of what I've worked on. But that wasn't the intent initially.

23:29I was just a free market pro-business guy that wanted to small business owner my whole life. But once I got elected and I, and I found myself serving on some healthcare committees, I realized the impact that we can have. And I also realized pretty quickly the lack of support for the rare disease community that existed in government across the board. Right. And I also realized that's something we can change. And that's something that I actually have the ability to change. And, you know, coming back to this regular prayer of leave me where you want me to be. That's where I kept going. That's where I continue to be led.

24:08And I'll fast forward. I can't give up. Senate Bill 1582. Importantly, the bill creates the Andrew John Anderson Pediatric Rare Disease Grant Program to provide grants to advance research and cures for rare pediatric diseases. This initiative was led by Representative Anderson from Pinellas County, who lost his son to an ultra-rare disease when his son was just four years old. Your biggest success to date has been the Sunshine Genetics Law. What is it? How did it come about? What does it do? We're celebrating the signing and the funding of HB 907, the Sunshine Genetics Act. It's truly a historic milestone that's giving Florida a once-in-a-generation opportunity to lead the nation.

24:59The Sunshine Genetics Act is a very exciting, transformative, truly landmark piece of legislation, which is wild to think that because it originated from my scribble notes on it on a cocktail napkin how did what were you doing with a cocktail napkin in your hand where what was the conversation you were having at the time it was just happened to be what i had in front of me that i could write on and i started having some ideas about and realizing some problems and um you know where how can we get these i was trying to think about how can we get these children to these preclinical and clinical trials before they're symptomatic?

25:40How do we do that? Because they had networked a lot with research doctors from around the country, and especially throughout the state. And there's a number of these even going on today where these therapies are providing tremendous promise. They're stopping progression of these diseases, but they're not able to reverse it. They can stop it or slow it, but they can't reverse it, right? And so many of these rare diseases, They have just so significant detrimental impacts on your overall health. So once you have deterioration of brain matter, it's very difficult or impossible to get that back, right?

26:17But what if you could stop it from happening in the first place? So I'm scribbling notes on, you know, how do we do this? What if we can add it to newborn screening? Another problem was, you know, how do we get our universities to work together? Instead of siloing them. Instead of siloing them, right? Why are universities competing, right? And if you think about it from a state policy standpoint, we're funding these universities, right? So it's a good business decision. Why are we funding Florida State for a viral vector manufacturing facility and University of South Farm is making up for something similar?