071 – John Crowley Saved His Kids’ Lives By Finding a Cure to Pompe Disease

Dad to Dad 71 – John Crowley Saved His Kids’ Lives By Finding a Cure to Pompe Disease

John Crowley: And it’s kind of the great irony of what we deal with that with many special needs children. Certainly what we’ve worked with in the field of rare diseases every day. What we’re trying to give families is time, time with each other, those special moments. Just to create more time and quality of life.

Tom Couch: That’s John Crowley, a special father of not one, not two but three kids with special needs. Two of those children have a rare genetic disorder called Pompei disease. John and his wife, Eileen were told that Megan and Patrick would not live very long. So they took matters into their own hands and help develop a drug that successfully treats the disorder.

They went on to from a drug company that helps treat other rare diseases. Their story was told in a book, a movie starring Harrison Ford and by the president of the United States in front of the joint session of Congress. And joining us in the gallery is a rare disease survivor. Megan Crowley. And today John Crowley tells his family story to our host David Hirsch on this dad to dad podcast.

Here’s David now.

David Hirsch: Hi and thanks for listening to the dad to dad podcast, fathers, mentoring fathers have filled them with special needs presented by the Special Fathers Network.

Tom Couch: This Special Fathers Network is a dad to dad mentoring program for fathers raising children with special needs. Through our personalized matching process, new fathers with special needs children connect with mentor fathers in a similar situation. It’s a great way for dads to support dads, to find out more, go to 21stcenturydads.org.

David Hirsch: And if your dad looking for help or would like to offer help, we’d be honored to have you join our closed Facebook group. Please go to facebook.com groups and search dad to dad.

Tom Couch: And now let’s listen to this fascinating conversation between very special father John Crowley and David Hirsch.

David Hirsch: I’m thrilled to be talking today with my friend, John Crowley of Princeton, New Jersey biotech industry veteran, and father of three children, including a daughter and son with Pompei disease. John, thank you for taking the time to do a podcast interview for the Special Fathers Network to speak with you,

John Crowley: David. Thank you.

David Hirsch: You and your wife Wayne have been married for 29 and yours and the proud parents of three children, John Jr. 24, Meghan 22 and Patrick 21. John jr. Was diagnosed with dyslexia, ADHD, and Asperger’s. Which is high functioning autism, both Megan and Patrick have Pompei disease, a rare genetic disorder and a rare form of muscular dystrophy.

Let’s start with some background. Where did you grow up? Tell me something about your

John Crowley: family. I grew up in New Jersey and they, uh, they ended up in the North part of New Jersey area known as Bergen County just opened the George Washington bridge, grew up in an Irish and Italian family, mom on the Italian side, dad on the Irish side.

Okay.

David Hirsch: And did you have any siblings growing up?

John Crowley: I did. I had my brother, Joe is three years younger than me.

David Hirsch: And what did your mom do?

John Crowley: My mom, uh, balance raising us. And she had begun her career as a secretary administrative executive assistant and worked for a time after, uh, you know, we were about five, six years of age after my dad actually passed away.

Worked as a waitress if apprentice, port Malden. Yeah. Okay.

David Hirsch: So some pretty humble beginnings.

John Crowley: We were. In fact, I remember my grandfather who had worked in a shipyard during world war two, after the war built a small little home in Bergenfield, New Jersey up North, after my dad had died as a police officer had an accident on duty.

We moved in with grandpa, was it 900 square foot home that he built with his own hands. And in one bedroom, it was grandma, grandpa, and my aunt Michelle, who was probably in her early twenties at the time. Three beds lined up in one bedroom. And then in the bedroom next door, it was mom, Joseph and I one bathroom, a little kitchen, a little living room that we thought it was the greatest thing in the world because we moved from an apartment.

So we had a little backyard and we had a softball field across the way. And Thomas Jefferson elementary school across the street where my mother had actually gone decades earlier.

David Hirsch: Wow. So that is amazing. Thank you for sharing. So, um, I remember in a prior conversation that you mentioned, your dad was also a Marine.

What were the circumstances around his short life?

John Crowley: Yeah, dad was a Marine, not just a Marine. He was a, a second special forces recon Marine, second force recon. And dad left the Marines right before actually Vietnam and started. So I think at the late fifties, early sixties, he was in the Marines and then came back, went through the police Academy to be a police officer in the town.

He grew up in, in Inglewood. New Jersey actually went through with his younger brother. His brother, Jim, my uncle Jimmy went through the police Academy and dad graduated, became a patrolman in Anglewood, then went on to become a Sergeant and you’ll learn at an early age. Uh, sometimes, you know, things happen in life that you don’t ever think about or predict, and remember waking up, being woken up early on a Sunday morning, January 12th, 1975 long, long time ago.

It was actually super bowl Sunday. And, uh, it was the knock on the door was from a couple of police officers. One of whom was my uncle Jim and my dad had been involved in a car accident earlier that morning, while on duty and, uh, and had died. And so in an instant life changes. Yeah. Well,

David Hirsch: I was really sorry to hear that.

Um, how could that not have a big impact on your mom, on you, on your brother, the rest of your family for that matter?

John Crowley: Yeah, sure. If that’s it. You know, help shape you. I think you’d find with children who go through these death of a parent, extraordinary, difficult times, it shapes them in different ways.

And in some ways it can break you as a kid. I think in some ways it’s, as I’ve learned from others who have struggled, David, it can, can help strengthen you for sure. It helped shape. How old were you at the time your dad lost too much.

 I was seven and a half years old. Joe was four. Okay.

David Hirsch: So he might’ve been too young to actually have firsthand memories of your dad.

Do you remember your dad? I know you were pretty young.

John Crowley: And do you know, you remember certain instances, things that we don’t have photographs of or videos that you know would have jarred my memory. So, you know, you remembered the one at the Bronx zoo. You remember swimming at the park at Darlington park.

Up in Bergen County. I remember my dad at one point was a motorcycle cop and they had a big parade in angle whatever year. And I remember him riding the motorcycle. And so, you know, you tend to remember all the good things,

David Hirsch: although you barely, you got to know your dad because of your young age, Maria, what were the most important takeaways from your relationship with them or advice that you sort of lean on when you think back on your dad?

John Crowley: Yeah, you need to look back that’s a long time ago. And you know, I remember one of the, one of the neat things was, you know, dad being a police officer, when I started kindergarten in ankle with what was then the kitty result elementary school, did you get dropped off in a police car? And that was pretty cool.

And, uh, every now and again, I flipped on the lights and siren and that would grab a little attention. Uh, but I remember we little routine when dad would drop me off here. Say do right. And at the end that have to finish it and say you’re nomad. So pretty, pretty good lessons from a cop and a brain for sure.

Yeah. Yeah. Well, so you should have told me, but do you write in fear? No, man. I think he was very specific and say fear. No, man. He didn’t say anything about a woman. So I think, just be sure if you’re your mother, so totally implicit.

David Hirsch: Yeah. Thanks again for sharing it to their teachers. Um, how about your grandpa’s uh, what role did they play on your dad’s side?

And then any

John Crowley: mom said, yeah, my grandpa, John, my dad’s dad. He died before my dad. I was quite young. I do remember him. Know, very kind man, very humble, man, you know, had only had a couple of years of education worked in a rubber battery, his whole life and died in his late fifties, you know, very, very kind man.

And that was very, very close to my grandfather, grandpa Frank, who is my mom’s father, you know, and after dad died, we lived for a couple of years with grandma and grandpa, you know, grandpa became a, a father figure as well. Yeah, Italian full of passion at one time, wonderfully carry a good sense of humor.

Always thinking about, you know, us and, you know, again, like my grandpa, John grandpa, Frank, my mom’s dad finished the seventh grade and at 13 years old, went to work in a pencil factory and then became a painter in his teens painting homes. And then it was a real skilled carpenter know, even I remember. As a young child, when my mom would go to work, I was probably five years old and mom and dad were at work, spend the day with grandpa.

And by that point we said superintendent at some very large apartment buildings. In fact, if you’re familiar with the area, when you come out of New York city today, you’ve over the George Washington bridge. And in Fort Lee, there are all those big apartments buildings and used to be an amusement park when I was a kid.

And then by the early seventies, they knocked down. Palisades amusement park and built the first of what would be many of those apartment buildings. And grandpa was the first superintendent, the first building. And I used to go with him every day and he had built me a little toolbox cause he had a toolbox.

I ended up in toolbox and I packed my lunch in there and I had little fake tools and would kind of mimic his work and follow him around. And you know, one thing he had actually done and was, and I didn’t realize it until many years later, later, David, he had actually. Put a book or two in there, and then over maybe the nine or 12 months, I was with him doing this almost every day during the week, he would take out a tool or two over time and put in another book.

So maybe

David Hirsch: planting some seeds about the importance of education. Yeah.

John Crowley: Yeah, no, I think very much, very much now we’ll thank you

David Hirsch: for sharing. Okay. I think the grandparents play a really important role for a lot of different reasons and are God forbid something. Happens like with your family, with your dad passing away at such an early age and as a result of divorce, right.

You know, on the almond down aren’t

John Crowley: mothers,

David Hirsch: you know, who’s going to fill that void when dad’s

John Crowley: not there. That is important. You know, that notion of a father figure in the absence of a father who can’t be there, you know, either cause God forbid, they passed away where they’re incapacitated, whatever it may be.

It’s just very important for somebody to have that father figure. Boys and girls. Absolutely. Yeah.

David Hirsch: So speaking of father figures, I’m wondering, who comes to mind, that’s sort of stepped into that role in addition to your grandpa’s.

John Crowley: You know, I think when I was younger, we, uh, you know, we had grandpa’s particularly grandpa Frank living with him when we had moved.

When my mom was remarried and moved up to a town called Norwood New Jersey, we went to immaculate conception parish, and it was a, a priest there. Father Ken Moore, A Carmelite priest, wonderful man, kind of a real model of what a priest should be. And as I became an older boy in that church, he became, you know, a bit of a father figure as well.

I think

David Hirsch: you mentioned in our previous conversation, he also had a brother.

John Crowley: He did father Tim. In fact, father Tim’s claimed event. You know, God bless them. They both passed away many years ago, but father Tim’s claim to fame in the 19. Forties. He was the pastor at Saint Cecilia’s church in Inglewood, and he hired a high school football coach for the high school team guy named Vince Lombardi, his first coaching job, Vince Lombardi, and the, uh, the more brothers I became very close with Vince Lombardi through that the early days of the NFL.

In fact, when I knew father Ken in Norwood, he was the team chaplain for the New York giant. You’d had that job with Vince Lombardi at the green Bay Packers years earlier. And, uh, he was the team chaplain for the giant. So when our parish would do a fundraiser, he would have about half the New York giants show up.

He was just, yeah. Beloved by the team. And so you get to meet the players and autographs and they always raffle off. The marrow family is always quite generous and they donate super bowl tickets to raffle off back in the late seventies, early eighties. So in fact, I can remember being a, an alter boy and going with father Ken on occasion to the old giant stadium, he’d say a mass for the team on Saturdays.

Many of them weren’t Catholic, but they would want to be there just to, to hear mass and hear his inspiration. So when I was young, I got to hang out with the New York giants.

David Hirsch: That’s pretty amazing.

John Crowley: Yeah. And this was the day of Phil Sims and build my company and Gary Jeter and Lawrence Taylor. So pretty, pretty amazing guy.

David Hirsch: I think you’d also mentioned in a previous conversation, um, your dad’s brother, your uncle Jim.

John Crowley: Yeah, sure. Uncle Jim was great. He was, you know, I think a real cops cop. Literally the funniest guy you’d ever meet. Great sense of humor. Great Patriot. He served in the Navy and again, went through the police Academy with my dad and uncle Jim ended up spending his whole career, went on to be the deputy chief of police in Inglewood.

And one of the great honors I had back in February of 2019 earlier this year was to give the eulogy at uncle Jim’s funeral.

David Hirsch: Oh, sorry to hear that he passed, but it’s quite an honor to be there.

John Crowley: Yeah. At that point. Yeah. He left a great legacy. He’d be proud of what he left behind.

David Hirsch: So, uh, there are a couple of other people that you had mentioned in a previous conversation and one of them being Eileen’s dad, Marty holler.

John Crowley: Yeah. We, uh, I met my now wife. I lean in, in high school when we were, I was 17. She was 16. And we were going out on that first date. And I remember meeting, uh, mr. Holleran back then. And I always thought he was, you know, Old wise fell. And I realized looking back on it, he was probably about 42 years old when his daughter out for a date and the first time.

And I remember picking her up and being very polite, polite, and I think I brought her flowers and going out to see a movie and dinner. And I picked her up in my old Camaro and he was very kind, but I remember as we were walking out the door of giving me this look like. We both know what you have, what you have in mind, young man.

And I’m looking at him, I’m like, this is an old man. This guy’s gotta be in his forties. How could he have any idea? So in any event, he became a great, great friend, a great mentor over the years.

David Hirsch: And I think he played an instrumental role in other aspects of your life as well.

John Crowley: He did. I’m sure we’ll get into, you know, as Megan and Patrick were diagnosed and with, you know, my father had a lot more and he was able to do to help us for sure.

Excellent.

David Hirsch: Yeah. So, uh, as far as your schooling is concerned, uh, you had mentioned you went to the Naval Academy and then from there you went to Georgetown. Um, what were you thinking about when you graduated drugs?

John Crowley: I was thinking I had no idea what I wanted to do next and thought about law school, but that’d be a great education.

Didn’t know if I wanted to practice law forever and thought about where to go and decided I would go to Notre Dame. Just talked about my dad. My dad was a huge Notre Dame fan. Never went to Notre Dame. I had never been to Notre Dame. And by the time it came to choose a law school, I have a couple, I had been accepted to the three or four and thought about it and said, you know, I think this is probably it’s just someplace I need to be.

And so I sent scrounged together that $200 deposit to send in and so said I would go to Notre Dame, a site on scene. They were, they were kind enough to accept me. Well, it

David Hirsch: sounds like you had a great experience there and you’ve become a loyal donor.

John Crowley: We did. Yes, we did. Eileen and I were married after my first year of law school and lived together in South bend for a couple of years.

And, uh,

David Hirsch: how did you start your career before you went to business school?

John Crowley: So I chose, uh, you know, I decided I want it to be a litigator. I like trial practice in law school and. I had some opportunities with some firms in Chicago, in New York, but, but found one in Indianapolis that we thought would be a great work life balance, Greek culture.

Sure. Indianapolis was a wonderful city to live in for a couple of years. We were able to buy our first home. I think we paid about $179,000 for a small start, her home in 1992. Yeah. And it was bigger than any home I had ever lived in. That was a great, great start to life. And for three years, Whereas I was blessed to work with some great people there in India.

David Hirsch: And then from there, I remember you went to San Francisco

John Crowley: to business first, and that was the connection. I think, you know, for me as a lawyer, I, you know, it was a great place to start a career, but I looked out, you know, like I tend to plan probably too much and looked out maybe 10, 20 years and thought, you know, I don’t think this is something I want to be doing.

For the rest of my life and like practicing law. I think, you know, I liked, I liked business. I like the diversity of what you could do in business and decided that the best way to do that would be to go to business school. I only applied to one business school. They were silly enough to accept me. So we sold our home in Indianapolis, packed up to go to a tiny little apartment in Boston, on the campus at the Harvard business school.

What was your experience

David Hirsch: like that?

John Crowley: Great, you know, with the education was very different than law school. What you have in business school is you stay together as a section and there’s maybe 10 sections per class. So those 70 or 80 students stay together in the same room. And the professors come to you.

Other generally are no textbooks. You have a case study. It’s a real life business study that would have been written by generally a Harvard business professor. And you study your real life. Incidents or business case. And the professor comes in really, as the facilitator class participation is a key part of your grade, which leads to a lot of entertainment.

Some of it actually intentional and, um, and some great learning, but you also build some great bonds, some friendship, and because many of us live right there, the vast majority lived on the campus at the business school. Some of whom were in the same apartment building at that, that was called soldiers field park.

Some of us were married, a small fraction of us had children. So it was a great bonding experience. Yeah.

David Hirsch: Well, I think that connection Jamaican business school are different than those that you have from your undergraduate experience.

John Crowley: Very different, right? You’re a little bit more mature where I was in my late twenties.

I was 30 years old when I graduated business school. Excellent.

David Hirsch: So where did your career take you from that?

John Crowley: So from there, went into consulting and, um, actually took a position with a firm based in Connecticut. Although I was assigned to live out in California, working on financial strategy for the bank of America.

Um, and then of course we found out and I’m sure we’ll get into a. Megan and Patrick now having their disease, they were diagnosed when we were living out there.

David Hirsch: I’m sort of curious to know before Megan and Patrick’s diagnosis, did you have any prior connection to the

John Crowley: special needs community? We really finished. We were blessed to grow up in very healthy families, even our extended families. On both sides. So we really didn’t have any exposure or experience. What

David Hirsch: was it like when you learned about Meghan’s and then subsequently Patrick’s diagnosis?

John Crowley: Yeah. You know, Megan was diagnosed on a Friday, the 13th David on March 13th, 1998. She was 15 months old and up until about a year of age and she was. Pretty normal kid and doing most of the things a kid would do, but by a year she wasn’t taking those first steps and something wasn’t quite right with their muscle tone.

And so we went from, you know, pediatricians and neurologists, blood test, deep muscle biopsy. Now, meanwhile, I mean, he was pregnant with our third with Patrick. Patrick was born March 6th and seven days later, we were called into the neurologist. The day after I leave out of the hospital from her C-section.

And the doctor told us that Megan had this rare form of muscular dystrophy called Pompei disease. And that she wouldn’t live to be two, maybe three years old. And that there was a 25% chance. Patrick had the disease as well, you know, Eileen and I are silent carriers for this disease, like any genetic diseases.

So there’s no history in our family. And I can remember it was kind of a cold, dark, rainy day living out in the San Francisco Bay area, town hall, Bowman Creek. Kind of a long car ride back to the home that day. My mother in law was there watching Vegas. We had Patrick with us and life kind of again, changes in an instant.

And I think our original, you know, we went through the, the, the shock, the grief, the denial, anger, um, but literally by that night, settled them determination. I think it was first determination to learn everything we could about this disease that we never heard of. Defined every researcher we could, and this was in a day, Google didn’t even exist.

So there were some pretty basic search engines online. And then there was something called the life or went to the library on Saturday and started reading everything I could. And it wasn’t a lot about that. If anything, about pay disease, but started to learn about neuromuscular diseases and clinical studies.

And I remember looking at a picture of a kid in a wheelchair in one of those books. And thinking, Oh God, please. A week earlier. I thought, God, please, I don’t want a child in a wheelchair. And it went from that in the course of a week to thinking, Oh God, please just let her live to see her second year.

It’s interesting

David Hirsch: how things can shift your perspective

John Crowley: or what’s your next area very quickly. I think it was also a bit of, in that shock part of, yeah, we’ve heard about this. We’ve seen it. We’re sympathetic to families with special needs. But that was kind of a classic case of this happens to other people.

It doesn’t, it doesn’t happen to us. And it certainly did happen to us as

David Hirsch: well. You described it. Um, it didn’t take very long to get to the determination factor as far as the creative fund denial and the anger.

John Crowley: Yeah. Some people languish

David Hirsch: and each of those stages for not just hours or days, but weeks, months, and years, what do you think it was that helped you and Arlene.

How are through that only look back on it,

John Crowley: the quarterly, it was the suddenness of it all. We really weren’t expecting that diagnosis. And then time realizing very quickly when the doctor said you have got a 15 month old and the doctor said she might not live to be two. You realize that time is the enemy as much as a mistake of nature.

And I think you intuitively begin to appreciate every small moment. Every next day is a victory. And it’s kind of the great irony of what we deal with that with many special needs children. Certainly what we’ve worked with in the field of rare diseases every day. And that is what we’re trying to give families is time, you know, again, time with each other, those special moments, um, just to create more time and quality of life and time for entrepreneurs like us to go back and figure out newer, better medicines maybe.

But that whole essence of what you’re fighting for is time. And the other side of it is the irony is the greatest impediment is time itself, the race against the clock, the race to beat nature. So I think, yeah, all of that, that gave us that termination, that, that frankly, that high sense of urgency and awareness of what needed to be done.

We never thought early on that we could change the course of the disease. We wanted to appreciate every moment, but I didn’t want to learn. We didn’t want to fight. And we knew the kids would want to fight. And it was as much for us though. I think as, as mom and dad, what we’d wanted to make sure without ever having said this to each other is that we didn’t want years later, if the disease would take our kids, we didn’t want to think, look back into wish we had done something different wished we had tried harder wished we had taken another risk.

Didn’t want to have any regrets. Yeah, that

David Hirsch: sort of coulda, woulda shoulda back. Okay. Well, thanks for sharing that. It’s very powerful. So what is Pompei? I know that it was named after Johann Pompei, but beyond that, I don’t remember.

John Crowley: Yeah. Named after a Dutch researcher. Um, remarkable man who, uh, characterize the disease in the 1930s.

So with pump disease, Megan and Patrick and people like them. And again, like many genetic diseases that can strike from infancy. All the way to adult forms of the disease, depending on the particular mutation that you have. And what happens is people living with pump pay or missing, where they make a defective protein or enzyme.

And it’s a housekeeping enzyme for you and I who live without Pompei. And most of our listeners here, it’s a housekeeping enzyme that breaks down a stored form of sugar, known as glycogen in a particular part of the cell, the lysosomes. And the kids can’t do that because of that. Yeah. The sugar, the stored glycogen builds up overwhelms the lysosomes.

It destroys the architecture of the muscle cell destroys the integrity of the muscle and it affects all muscles. It affects the, all of the skeletal muscles. It affects the diaphragm for breathing. It affects cardiac muscle. In fact, it was the severe enlargement of the hearts and the weakness of the kids’ hearts that would’ve killed them.

David Hirsch: That’s a, it seems overwhelming. When you think about the

John Crowley: consequences of Pompei disease

David Hirsch: house and a short life expectancy, I’m wondering what meaningful advice that you might’ve received early on that helped you put this situation in

John Crowley: perspective, lots of different advice. You know, literally that first neurologist to diagnose Megan.

Very good doctor, very caring, man. No, he apologized for having to give the diagnosis. It must have said three or four times. I’m so sorry. I’m so very sympathetic. And I appreciated that. And he did tell us, you know, when I asked the questions, can we get a second opinion? Is there research going on? He, uh, it was pretty noncommittal, I guess he just, there really wasn’t much and didn’t want to get our hopes up.

And the first piece of advice was enjoy the time you have. And there is some wisdom in that for sure. But we want it to do more than that. We heard. Yeah. Other pieces of advice with, we got to know other families Ali’s with pumping disease. I remember one dad we’ve been fighting the good fight for many years and his daughter was, was near the end and he told me, um, you know, we finally are at peace with it and what will be will be.

And I was because our kids were just diagnosed. So that’s so much fight. I was taken aback by that. Well then now I appreciate that perspective. The, you know, The realization at some point, there is only so much you can do and you want peace for your children. Um, and then lots of medical advice and technical advice and scientific advice.

Most of which really on I did not understand. Okay.

David Hirsch: Well, I remember reading in the book,

John Crowley: the cure about

David Hirsch: dr. Al slogan, this Australian Orthodox Jew.

John Crowley: Yeah. When did

David Hirsch: you meet him? Or

John Crowley: what was the kind of right after the kids were diagnosed at children’s hospital, we want him to meet some pump experts. So I reached out to a handful of them around the world at Duke university, at researchers in the Netherlands.

And then I reached out to Ellis Sloan, who is a practicing physician on long Island. So Megan out to beat him to confirm the diagnosis and to come up with a plan. One thing he recommended was that we put a feeding tube in Meghan. And she was still eating, able to swallow with the muscles in her esophagus, but he said that would she lose that?

And she would get sick soon. And when she got sick, she needed to be as strong as she could. And that was ended up being life saving advice. So while Megan was still relatively healthy at about 16 months of age, we put that feeding tube in been

David Hirsch: a. Hard

John Crowley: decision. It wasn’t the time it was Meghan’s first surgery.

It was, it seems so invasive. And next thing you knew, we had an IV pole with a feeding bag to supplement her meals and then eventually for quickly to replace them. And it was just the first of many pieces of equipment we have in our home.

David Hirsch: What were some of the more important decisions. In addition to that, that you made, as parents are raising a child with special

John Crowley: needs. We left California, went back to New Jersey. I left that job in consulting was able to get the job at Fritz DeMeyers. I didn’t go because I wanted to learn about the pharmaceutical industry or making medicines.

I went because it was a good company with good culture, had a good mission and really good health insurance. And it wouldn’t have to travel as much. So, those are the reasons we ended up doing that. You know, we made decisions. We relocate back in New Jersey to be close to doctors at Duke, dr. Sloan, him family for support.

We were seeing that, you know, Megan was starting to get weaker through that summer of 98. So we made in a couple of months, we made career decisions, relocation decisions, family decisions. We agreed that. You know, I would focus on job career, earning a living and on the side nights and weekends building kind of a coalition in Pompe disease while I lead would focus on raising the children with their needs.

And again, remembering as you indicated early on, although we didn’t appreciate it at the time John had our older son without pump disease, had his own challenges. And John was only three and a half years old at that point. And John, you know, with his dyslexia and ADHD. And as we learned later, Asperger’s, um, John, John was a handful as well,

David Hirsch: not to focus on the negative, but I’m wondering what some of the bigger challenges that you’ve

John Crowley: faced are, you know, I learned when I was in law school that a close friend of mine in law school, his dad told us that, or dinner one night said, you know, guys, he said, life is a series of challenges.

And your happiness in life is going to be directly proportionate to how you manage each of these challenges. And so while we had the big challenge of Megan and Patrick and that consumed our lives, we also realized, again, we want to be kids for as long a life they may have. And it may have been very short that they have as happy and fulfilled a life as possible, and that we make great memories.

So as we dealt with challenges, there were many, it was, you know, Megan by September of 98, when in the intensive care unit, she almost died three times in six weeks was put on a ventilator six months later in March of 99, when he was a year old Patrick crash and was in the ICU and went on a ventilator.

So in this course of a year, we went from perfectly seemingly healthy, normal family to a very different family, with very different challenges. She had those kind of backward challenges. And then you had, we had to hire nurses and bring nurses on board. And that was a whole other episode of the good healthcare we had finding and training nurses, our learning to be caregivers, how to change tricks in emergency situations, you have the challenges that every family faces and balancing life and career, and, you know, typical family challenges.

And then you have the challenges of realizing you needed to maintain a marriage. And that was very, very hard to, um, this was not how life was supposed to be. No Eileen and I grew up together and this tested us a great deal. So other than that, everything was pretty straightforward and we didn’t have any money.

We were just starting out. I had a better, $150,000 in student loans. Yeah. Well,

David Hirsch: it’s remarkable that you’ve traveled to Brown that you each have, and, uh, there are better than anticipated outcomes. Then what the original predictions or expectations,

John Crowley: or, you know, we learned early on to be grateful early on in life before we ever met each other.

And then I think certainly through what’s now been a 21 year journey with her, with her children. So

David Hirsch: I know that, uh, there’s a lot of information that is available through the book, the movie you’re your own memoir. And I remember you reflecting.

John Crowley: About your marriage

David Hirsch: and that there were points in time where you were at a real low and it must have been confusing for you.

And there was this one story that I remember you reflecting on about, uh, leaving the house with a backpack and just starting to drive around.

John Crowley: What was that about? I think you reach a point where, you know, you as a dad, a lot on your shoulders. And again, this isn’t how life was supposed to turn out. And you’re trying to balance work and raising the kids and marriage and probably a hundred other things.

And you get to the point, you can be tough and, you know, steel doesn’t bend it breaks, and you get to the point where you think, you know what? You could be better as a person. Maybe your wife would be better and happier if you weren’t together, you think maybe the kids would be better. They didn’t live with that stress of watching parents trying to balance a marriage.

So you rationalized, you know, Eileen and I had grown apart in that year, close at times and at times apart, and then, yeah, there was that one night, it was no one particular incident that, that, that kind of spurred it. But I just, that all kind of came crashing together and I thought, you know, there’s maybe a different path in life and I can still be a great dad and take care of the kids and provide for a family that maybe we’d be better apart in.

Spent a long time driving that night, any hours and conversations with my friend, ed and realize that, you know what, I think it would be better for the kids. Yes. But it would be better for me and Eileen to be together. And that was a great decision. I’m glad, glad we, I came to that and I’m glad I leaned was patient enough to suffer, suffered through my, uh, my, my challenges.

David Hirsch: Yeah. Well, you had mentioned there’s a lot of way to under shelters, right? You know, guys are sort of guys in that. We want to figure it out on our own. Yeah.

John Crowley: There’s something funny about this Y chromosome that’s for sure.

David Hirsch: And, um, what I appreciate most about you reflecting on that situation and the challenge is that it’s real right.

It’s authentic. And you know, one of the things that we each struggle with as men, as fathers, as husbands, is what do you do when you know your back’s up against the wall and. You don’t know what to do. Right. And I think it’s important to be in the company of others. Like you said, your friend ad was a confidant at the time and help you think through things and I’m relieved and pleased to hear that, uh, you chose the path that you did.

And, you know, I think each of us just has to be authentic and like knowledge that we need.

John Crowley: We do. And you realize it is a long journey in life. Hopefully. Um, it’s a long journey with your children and a long journey in marriage. It wasn’t that that night I came back home and everything is fine. It’s a continual work in project and there are times, you know, where you still feel.

Gosh, you know, wouldn’t it be nice if, and there are times that you grow apart when you’re together for your entire lives. Um, but if you come from back to that foundation, um, incredibly important together, you know, to rely on friends for advice and counsel. Um, to reach deep into our own soul, to rely on faith.

And we all, we all have a faith. I believe that we all have a religion. We may not all have a God, but we all have that. Something that reminds us that there is that element of life that is bigger than us. And that’s really important to, you know, for, for me, it was deep faith in God and in the church. Um, but for others, they find that faith and in whatever church they may be a part of or whatever is important to them in life that grounded so important.

David Hirsch: And I’m well-spoken, um, I just want to talk about one other challenge because it really talks to me. I was reading, um, the cure and it had to do with something you made reference to a few minutes ago, which had to do with the nursing staff and relying on others because you know, there’s only so much you could do as the mom and dad and these kids need 24 seven.

Care and attention

John Crowley: they do.

David Hirsch: And, uh, you know, it’s really hard to find, um, the right solution. And, uh, usually they’re not permanent solutions, right? There’s people that are coming and going people that treat this as a job and people that are really super compassionate and it’s not just about the paycheck or work, it’s something they take very personally.

And I know that you had some challenges along the way, you know, firing.

John Crowley: Nurses.

David Hirsch: I’m wondering if you can walk down

John Crowley: that we, you know, the nurses do help us take care of our kids are, are part of the family, took us awhile to figure out how we find that right person. Who’s a good fit, has the right chemistry who can provide the care and compassion, but also extraordinary medical and nursing skills.

And you know, we’re now in the point of having done this for many years, that we’re blessed that both Meghan and Patrick. You know, we were able to provide, you know, something we give thanks for every day that we’re able to provide for the care that they need. That it’s not only extraordinary medical care, it’s life care.

And that’s really important that takes a while to, to develop. Um, I, you know, when my, I know we’ll get into all the kids’ journeys, but my Megan’s journey, she wrote a paper in college. It was an autobiography for freshman year. The supposed to be 10 pages typed. Uh, single-spaced Megan’s was 37. She had a lot to talk about in her very short life.

And one of the things she wrote in that, she said, yes, she wishes she was stronger and wishes. She could do other things, but she’s not at all. Doesn’t have any regrets that she reported with Pompei disease. So this is, this is the way I am. This is the way nature and God made me. And one of the things she said was if I didn’t live my life with this disease, you know, I think of all the people.

In my life. I wouldn’t note today. Yeah. And for her, it was the vast majority of people outside of family, really. And some friends who she never would have been that in the core of that really are the nurses who take care of her. So finding those special people and think about it, how it’s shaped Meghan over her years, you know, she’s incredibly tough, precocious, quite a fighter.

I think that’s just her character by nature. But I think she was shaped because since she was. A year and a half old. She’s never been out of eyesight or earshot of an adult. She’s 22 years old now. Yeah.

David Hirsch: Well, it’s an amazing reflection that you’re just sharing that you have in this 37 page autobiography,

John Crowley: and

David Hirsch: even most of them have gone past the

John Crowley: Malloy that former president.

Yeah. Uh, at Notre Dame where she went to college was the teacher for that seminar. He said it was one of the best and certainly one of the lengthiest autobiographies he had ever read.

David Hirsch: Yeah. Well, thank you for sharing. Let’s focus for a minute on that, John jr. Who was also diagnosed with ADHD, severe dyslexia.

And Asperger’s, when did that transpire on the world of you and Irene juggle three kids with special needs?

John Crowley: John said his first word the day before his third birthday, and we were sitting up in my bed and I was reading to him. And I looked down at him and I said, John, you’re my little buddy. And he took his bottle out of his mouth and he looked at me, he goes, buddy.

And I kind of froze. And I said, say that again. He goes, buddy. And I kind of screamed yellow for Eileen. And she came in and we also have like, he can talk, he said a word.

David Hirsch: He would have been talking a year, a couple of years before

John Crowley: and talking at a year, you know, like a typical kid. And he wasn’t. Yeah. We didn’t know why we didn’t know what it was.

And that was a really special gift at that time, because it was right before John’s birthday is December 20th first. So it was just a couple of days before Christmas. We were out in California and it was about a week after Megan’s deep muscle biopsy, three months before her diagnosis and three months before Patrick was born.

So even in the midst of all that uncertainty and anxiety, you know that one small moment that okay. Small baby step forward, but he’s there. Um, and now he, uh, you know, John’s 24 years old now and adult living with Asperger’s and his ADHD and his challenge. And John never stopped talking since that day.

John talks talks an awful lot. And, uh, you know, we realized going through with John, you know, we walked, he was 19 months old before he walked three years before he spoke, but we figured it just delayed back then, frankly, nobody knew as much as we know today about. Uh, Asperger’s autism developmental delay, whatever it may be.

We got them early intervention. And then we went off to, you know, we, we moved a lot and he went into school and he was at least just a little bit behind, but made his way through. And you know, people ask us, David, you know, how help God? How do you deal with Megan and Patrick and the nursing and the challenges and all the life threatening incidents.

And shortly, those are certainly challenging. Um, but we, we kind of get around that John is more or work that Meghan and Patrick combined, he is a, a special soul and you know, God bless him. Now, he’s getting ready to finish college. It’s too taking them longer. And he’s 24 years old and he’s living pretty independently out at the Holy cross college in South bend, Indiana across from Notre Dame.

And so with John, it’s been constant learning, it’s been a lot of patients. For all of us, for me as a dad, there are many times I look back as a young dad and how I absolutely completely lost my stuff with, with John, not having the patients. In fact, I remember our Meghan four or five years old sitting on the couch, hooked up to a ventilator, watching a TV show, and John’s running around like crazy with his ADHD.

And Meghan just, just broke and just yelled at him and said, John, Said, Oh my God, stop. She said, I know exactly what’s wrong with me. Daddy needs to make one enzyme and I’ll be perfectly fine. I have absolutely no idea how many enzymes he has to make to make you fine. And John kind of froze and looked at her and he just done at the time and then have the sharpest wit and just look at her with kind of a sad face and said, that’s mean, and I said, Megan, with her little rapier wit, I said, Megan, don’t.

Don’t say that that’s not kind. And I rolled my eyes like, right. That’s actually pretty funny. And, uh, but you know what? John has been such a great gift to all of us, and we’ve learned as much from John as we have from Meghan.

David Hirsch: Yeah. Well, um, you have a lot to reflect on you and how they have a lot to reflect on.

And again, thanks for sharing. So I’m wondering about supporting organizations primarily for Megan and Patrick. I’m wondering which organizations that. They most important in their lives and your family’s life for that matter,

John Crowley: you know, organizations, uh, with, you know, involved with research and rare diseases and pup Pape disease, the muscular dystrophy association, those groups, all planning, incredibly important role.

And, you know, Meghan and Patrick and people living with puppet, like them may not be here today, but for those organizations, probably the one note that’s been most special in our lives has been the Make-A-Wish foundation. Um, Megan and Patrick, that we learned about the Make-A-Wish foundation. Yeah, right around probably the year, 2000 and friends with my mother and father in law.

We’re involved in volunteering for the foundation. I heard about it. Uh, you know, like many people, I thought it was for kids with, uh, only for kids with cancer, it was for kids with a terminal illness. And while that’s where the make a wish had its roots, its mission is much more broad now it’s to provide.

The heartfelt wish of every child with a life threatening illness. But when I heard Make-A-Wish, I always associated it with the last stage of a child’s life. And so while our friends had said the kids would be eligible, let’s introduce you to people that make a wish. Um, I didn’t want to deal with that.

Cause for me, it was almost an acknowledgement that we were at the end. And then finally we realized what make it really was and make it a Patrick were wish kids, both Disney wishes. And the Walt Disney organization was wonderful with the children. Yeah. And in 2001, and then 2004, respectively, Megan and Patrick went and had their wishes at Disney.

And, you know, it was so great about Make-A-Wish is what they were able to do for families. In 2001, we couldn’t afford to take kids to Disney with Megan and special needs on a ventilator. And with Patrick men on a ventilator, we had no idea logistically how to make it work. And the Make-A-Wish foundation made that happen.

And what it did was to give us just five days, just five days where we didn’t have to worry about nurses and hospitals and diagnosis and clinical trials. And it’s a wish that’s as much for the whole family as it is for the child. So, um, incredibly powerful effect on families, great memories and something that’s been part of our life now in trying to give back as well.

You know, went on to serve on the board of the national, make a wish foundation. I became national chairman of Make-A-Wish in, you know, our Meghan helped found and become precedent to the Make-A-Wish club, but Notre Dame and college. So it’s been an important part of our lives. And the great secret about Make-A-Wish is that it actually makes our lives better.

As volunteers, as donors. You know, I can guarantee when somebody volunteers for, or gives a contribution to make a wish or helps make a wish come true that you are. I can guarantee you 100%, you are going to have a profoundly positive impact on a child and a family. You can’t do that with any other not-for-profit.

You can say this may provide hope and they lead to a result. Make a wish and guarantee it.

David Hirsch: wondering beyond your own experience. If we can not focus on sort of the advocacy that you’ve had for finding the cure for Pompei and the work that you do in an Amicus therapeutics. If we can talk about what your focus has been.

John Crowley: So we were able to develop that medicine. We, uh, we went from desperate parents to being involved in the, not for profit world, to finding a researcher at the university of Oklahoma, brilliant clinical biologists who had a technology that could make a medicine to save the kids’ lives.

And that led us to find that first company called bill design and. From Novozyme we, uh, and that was wisdom. The focus of the movie and the book, the cure building a small biotech company that we started with some funds from individuals. What do you learn from Eileen and I, from what the liquid is, we had some credit card advances to build it, and it became a successful small company that we sold to a larger company that eventually develop a final medicine.

And now distributes that medicine around the world to people living with Pompei. Uh, that was the first journey. Then after the kids were treated and we saw very quickly that significant effects of the medicine and reducing the enlargement of the kids’ hearts within 12 weeks, their hearts went back to normal size for a while.

It made them particularly Meghan and stronger. But then after a year, we realized that improvements in muscle strength had plateaued and slowly but steadily, the kids were getting a little weaker. So we realized that making a medicine, we didn’t have a cure. We had a treatment. And we needed better treatments and ultimately a cure.

And I saw the good effect that medicine can have on people. And I wanted to build an organization of company that can focus not just on Pompei disease, but focus on dozens, maybe someday hundreds of the many thousands of rare diseases and could find the best scientists, best doctors that could look for the very best technologies.

And so when we went to, you know, help helps found a small company called Anacortes. We picked the Latin name Abacus for friend. We wanted it to be the most patient focused company in all of biotechnology. And a handful of us started the company. We raised money from venture capitalists then eventually became a public company.

And now nearly 15 years later, we’re a 600 person company. Um, And we have offices in 27 countries around the world, and we make medicines for people living with rare diseases, many of them, children. And we’re now in the world of gene therapies, which are incredibly exciting, potential new cures for these diseases.

But I think I’m most proud of the culture of what we built at Amicus that notion of investing in just the very best science. And what I ask everybody in our company every day is to think in every business decision they make. Think about it. You have this disease where you with a mom or dad of a child with this disease, what would you do?

Where would you invest? Who would you hire? Where would you build a facility? When would you start a program? When would you stop a program? Think about it from that perspective. And we’ll end up building one of the best companies in all the world in biotechnology. So we’re still in the early days of this journey and building this company, but it’s something we’re very excited about.

So in the 15 years

David Hirsch: that Amicus has been around, um, if you had to identify one or two highlights, breakthroughs, or discoveries that have been made, what would they be?

John Crowley: You know, we we’ve developed small molecule, precision medicine for a rare disease, known as Fabry disease, where we really had to understand the human genetics of that disease.

And we’ve developed what is one of the world’s most extraordinary precision medicines. And. With that, you know, it’s allowed us to fund research in many, many other diseases. We’ve had some successes. We’ve had many more failures though, than our successes. And that’s something I’m actually really proud of is, you know, we are a very persistent bunch, sometimes annoyingly resilience summit.

We argue and we never, never quit. And that’s an incredibly important trait. And I think when I look back now over these 15 years, look out over the next 15 years, I want to make sure that we’ve got. Four of them patient centered culture. And now I want to make sure that we’re always pushing things as far and as fast as yeah.

And that we, we tried hard enough that we fail more than we succeed, but that we take those learnings. We repackage them and we think about what’s next. When we started Amicus, we did have a pop pay program and it was an entirely different technology. It was a pill that could be taken. We spent four years, we raised about $50 million in research funds for it as a company investment funds and did failed.

We didn’t know why it took us six months to figure out technically why it had failed. We realized that technology did not have a future in Pompei disease. And so what we did was we went back to the drawing board with my chief science officer. How long ago was it a dear friend and one of my first collaborators on this nap, 20 year journey scientists.

And Hong came up with an entirely different idea, different how you treat this disease, a different technology. And we’re now to the point where that’s in very advanced stage studies and people have seen, you know, extraordinary, positive results on this new medicine that we’ve developed. So proud of that, taking those failures and turning it into success.

It’s the nature of what we do in biotechnology. You know, we could, in our business, we can. Hire the smartest people raise hundreds of millions of dollars, build the most elaborate research facilities in the world and still almost everything we do, it doesn’t work, but when it does work, I do think it’s one of the best jobs you can have incredibly rewarding.

And I think with that comes an obligation, and this is something that we tried to do different. The dynamic is realizing that, you know, ours is a sacred business then making medicines and we have what I believe is a moral obligation. To not only make the best medicines, but to make sure that everybody in VI has access and that then affects how you price your medicines, how you distribute them, how you ensure compassionate access to your medicines.

In our view, nobody in the world should ever be denied our medicine. So we have to figure out creative ways to get it to everybody.

David Hirsch: Yeah, well, very powerful. Thank you for sharing. And I’m, I’m hoping from your lips to God’s ears, like my grandmother used to say that you have more successes than failures, but I think, uh, in the world of biotechnology, there’s a lot of stops and starts,

John Crowley: right?

There’s a long road. It is never a straight, straight journey even for the most successful companies. Right.

David Hirsch: So.

John Crowley: I’m

David Hirsch: wondering under the category of advice out there, some important takeaways that perhaps you haven’t made reference to already about raising a child or talking about special needs.

John Crowley: I’ll give you an example.

When you know our Meghan blasts, you know, Pompei never affects the mind, incredibly smart young lady. And she went to Notre

David Hirsch: Dame. She graduated recently.

John Crowley: She did she’s in graduate school, in graduate school at university of North Carolina chapel Hill. When she went to college at Notre Dame, and that was a tough decision for her, for us 600 miles away from our home in New Jersey.

And Megan, you know, we knew would be the most disabled student ever to go through Notre Dame. And we’ve had students with lots of disabilities, but Megan being in a wheelchair, living on a ventilator and having 24 seven nursing and wanting, needing to be part of the university community. Every night of her four years at Notre Dame, living in her dorm in Ryan Hall at Notre to him, when she went out there, it was filled with a lot of hope and a lot of anxiety for all of us.

She got settled in and I went out to see her. It was father daughter weekend. In early October, we had had a real challenging 24 hours at, at Amicus. And I was out there and tried to compartmentalize, but it was hard. Megan got sick. She was six weeks into classes. She had failed her first statistics exam.

Damn her first psychology exam. She was struggling in math. And I asked her, you know, Megan, candidly, how are you doing? And she said, I feel overwhelmed. And so I really struggled until she wasn’t wasn’t happy. So we had a long conversation. And I came up with some advice. Then I remembered long, late Sunday night that weekend in 2015 in her dorm room, I said, Megan bowls we ever wanted for you is to be happy, safe, and fulfilled those three things.

And if you feel that you’re not any one of those three things, then come home. It’s okay to take a break. Maybe you come back. Maybe you don’t. And she listened to that and said, no, I fall break is only two weeks away. Let me get through the next two weeks and I’ll come home. We’ll talk again. And she did.

And she came home. You had a longer talk. And she said, no, I want to give it a try. I want to keep trying, but she is a persistent soul as well. And so she went back and. Came home and got her grade spelled a little bit better. And Megan ended up getting, I think about a 2.7 GPA at Notre Dame that fall. And she passed that one psychology class.

She got the email and she got to be negative. I’ve never gotten anything but an a in her life. And Megan was so darn happy to get that deep. And she said to me, dad, I think D is her diploma Irish. And she went back and finished and she finished, I think she had a 3.5 when she graduated and a 4.0 her last semester and found her way.

And you know, the wall street journal had written a great piece back in June of this year, 2019 about vegans journey. And it’s a great lesson for, for, um, the big people. You know, uh, adults with children. It’s a great lesson for moms, for dads in that, you know, we want a lot of things for our kids, but I do think for me again, for Eileen as a mom, and we’ve tried to convey as we want them to be happy, safe, and fulfilled.

And now as Megan moves on and her journey in life is a student now, a graduate student that UNC at chapel Hill in their school of social work, pursuing a master’s in social work. She wants to be a social worker and work with families of children with rare diseases. She’s going to find a way to give back.

David Hirsch: It’s just an amazing story. Thank you again for

John Crowley: sharing.

David Hirsch: your story has been covered really well initially by an investigative reporter at the wall street journal

John Crowley: Akita

David Hirsch: Anon. And then that sort of motivated her after following your story to write a book, the cure. Yeah. And then that was made into a movie and there were some forward and some others option right.

To the book and ended up making the movie.

John Crowley: Yeah. So ordinary measures, the movie came out almost a decade ago.

David Hirsch: When you think back at that point in your lives, what was going on? How do you put that all in perspective?

John Crowley: Yeah. No, we agreed to that. First story for Gita was just about a dad starting a biotech company to save his kids.

And then the second story was a front page story. After the kids had received their medicines after they were benefiting and then lives were saved for the medicine. And that got the attention of a lot of people, including Harrison Ford, who would read that story and the producers who had read it as well, when EDA said she would like to write a book when she was approached by a lot of publishers about a book.

You know, he is a wonderful lady and we trusted her completely to tell them the true, honest story, good. And the bad of everything that we had gone through in that, but had been at that 0.6 or seven year journey in our family’s lives. And we thought it would be important that people learn from everything that we had learned on this journey.

So we trusted Gita. And she said about for two years to write your book, um, took a year’s leave of absence for the wall street journal to do that. And then right about the same time. We got a call from the producers and from Harrison Ford, which was a surreal, I can tell you that. Yeah. The one piece of advice, when a guy calls your house says his name is Harrison Ford.

Read an article in the paper about your family and would like to come meet you. Um, don’t hang up on him. So I did and he was persistent and call back. So it wasn’t a practical joke. And I, that took a while. I have to tell you, um, because when they make a movie, you don’t get to control the script. You get input.

But you don’t get final say, so it took us a while to get to know Harrison and the producers. And they were wonderful people. Michael Schomburg, Stacy. Sure. Carlos Schanberg. We knew them. We trusted them. Michael and Carla have a daughter who was deaf, so they could relate to special children and special needs.

And ultimately we had to trust them to tell the families story or journey in the right way. And we’re very pleased, you know, the movie that they put together by. Being able to tell this at a Hollywood fashion, they made sure two things, they made sure. Number one, they got, yeah. The family dynamic family is spirit a hundred percent correct.

And number two, this time I answered the medicine. You know, whether they can composite characters and changed locations and condensed timelines, it took a lot of liberties with the business aspects of it. But, um, The key messages and themes are the right ones. And it was told in a beautiful mind.

David Hirsch: All right.

If I call you Bob, you got a check for a half, a million dollars.

John Crowley: Hell you can call me Peggy Sue.

David Hirsch: This is the first installment thinking. It’s the promised half week.

Tell you the truth. That’s more than I expected,

John Crowley: which is why you and me are going into business together, Jersey.

David Hirsch: Excuse me. Come tired of

John Crowley: begging bread comes from the

David Hirsch: university and giving them

John Crowley: the patents to my ideas. They don’t value my work. You do, which is why I’m setting up my own shop. Figure. Any dude in a business suit can help me raise venture capital and run the company.

But who’s going to be half as motivated as a dad is trying to save his own kids.

David Hirsch: So I can promise you longer hours, less money,

John Crowley: lousy working conditions. Plus, when we raised the money you’re going to have to relocate to Nebraska.

David Hirsch: Oh with the right business plan.

John Crowley: I can also promise you

David Hirsch: working enzyme for Bombay disease.

I can’t carry your kids, you know, that they’re always going to be in wheelchairs, but

John Crowley: I think I can save

David Hirsch: their lives. Yeah, well, it’s um, a well told story, a ketose story and the cure on that. I think the movie extraordinary measures and let’s just be clear. Harrison Ford. Didn’t bite. You.

John Crowley: No. He played a composite of a number of the researchers that I worked with, you know, including factor bill Canfield, who is the brilliant clinical biologist.

We worked with making that first medicine, but other, other doctors and researchers too were composited in that character. Uh, Brendan Frazier, the actor from the mummy movies, a playmate where’s my daughter, Megan will say that’s one of the great. Patients and the history of Hollywood casting director chose an actor.

Who’s six foot five to play her five foot six, that path.

David Hirsch: Um, and, uh, Carrie Russell played highly

John Crowley: beautiful, wonderful. Carrie Russell played at night.

David Hirsch: Yeah. So maybe more accurate if you will.

John Crowley: Yeah. Yeah.

David Hirsch: So, uh, I just had a couple of questions at twice the movie, um, did, uh, dr. Canfield. Who’s the real doctor, bill Canfield.

Did he really call you a Jersey?

John Crowley: Um, at times? Yeah. Yeah. I grew up very much in New Jersey and, and, uh, yes, he played from time to time. There was a

David Hirsch: scene in the movie that, uh, there was a backup generator that needed to kick in because the electricity had gone

John Crowley: out. Right. Was that an accurate, that was accurate.

We lost a couple of months of science experiments, uh, after a very rough series of thunderstorms in Oklahoma city. Okay.

David Hirsch: And, uh, I thought I saw you in that in the movie. Is it possible that you were in the crowd? And one of those,

John Crowley: I had a speaking role, David. I played a venture capitalist in the scene where they’re pitching the business to venture capitalists.

The producers asked me, we were on set a couple of times. They filmed the movie in Oregon and we flew out about four or five times in the three and a half months. They were filming. And, uh, one time we were there on a Friday night and the kids were out with us and they asked if I’d like to have a speaking role, play a venture capitalist.

I said, yeah, sure. So I had to get a screen actors Guild card and do all that, that night. And in the next week, the next two days, Saturday, Sunday, we filmed that one scene where they’re pitching the business. I had three lines. I think there were about 80 takes throughout. I learned about how grueling it is to make a movie.

Every angle they’ve got to shoot continuity. All of that. And, uh, I had three lines, they cut two of them, so, but one of them made it in. I get it. I get somewhere between two and $10 a month. Residual. Where am I speaking mine? So don’t

David Hirsch: quit your day job.

John Crowley: I think I have to keep the day work. Yeah.

David Hirsch: So one other question about the movie and it was really a touching part of it.

Um, and I don’t know if it was accurate. That’s what I wanted to ask is if there was a scene when the medicine was being administered to Patrick and Meghan. Sitting side by side. And when the medicine was being put through their IVs and the scene was like this sugar high, that experienced was that accurate?

John Crowley: It was. So what ended up happening was that the medicine that the kids have received, you know, had been receiving was given every other week as a four hour infusion of the enzyme that was missing in their body. And now over the course of those years, all that glycogen that’s stored sugar had been building up in their muscles.

And I asked the doctors and said, what would happen when, if the medicine gets in there and breaks down that sugar, is it toxic? Or could there be harmful consequences? I remember the doc said, no, they should just metabolize it, break it down. And you know, uh, it’ll be gone out of their systems. Okay. That night, I was there for the infusions and I went home that night with John junior, Eileen stayed at the hospital with the kids I called to check in.

It was after the fusions that ended asked how the kids were doing said, they’re great. She said, but they won’t go to bed. She said that like hyperactively into their beds. They were in the hospital rooms across the hall from each other, that kind of yelling to each other and just laughing. And you think about, you know, Joe kid, kids had a busy day, they were in the hospital for a couple of days.

And then it dawned on me. I’d ask that question. What would happen when they break down the sugar months earlier? And again, when the IB is flowing through your, you know, you don’t know, is it working or not? We knew it would be months to, we saw an effect on their hearts. And that was the first telltale sign that, that medicine was working.

And I just remember back to them, like the breakdown of the sugar, you know, maybe it is working and turns out that actually was one of the telltale signs. That level of hyperactivity was very short burst, but. Was very true.

David Hirsch: Yeah. Well, thank you for sharing. Um, totally unrelated to the movie. And I remember saying something, I think it was a YouTube video from a year, a couple of years ago now, and that, uh, Trump was addressed in Congress on rare

John Crowley: disease day

David Hirsch: back in February of 2017,

John Crowley: it was the end of February, 2017.

It was his first address to a joint session of Congress. I found out that that was going to fall on the 10th anniversary of world rare disease day. And so I, I sent a note in, through some folks I had met kind of on the periphery asking if they president would put in his speech a couple of sentences about people living with rare diseases.

And, uh, didn’t hear anything for a number of weeks and got a call on a Friday night that there were a couple of sentences in the draft of the speech mentioning rare diseases, which I thought was great. And then got a call on Saturday night that the oval office and the white house staff liked it so much that they’d like to make it a feature of the speech.

Three minutes out of an hour speech and asked if we would come to the white house and meet the president. And the vice president never met the president before. And we had a short visit in the oval office. We spent the afternoon in the white house and we’re guests that night. And I have to tell you, it was, there were those moments as a parent, as a dad that you’re incredibly proud.

Of your, your son or daughter, and that night, you know, again, in the category of surreal experiences, getting the presidential motorcade up to Capitol Hill, being whisked up to the president’s box. And they, in the house of representatives, I’d only been there a couple of times as a tourist and to be there and to be seated.

And during that speech to, you know, have the president, regardless of what your politics are, if there was ever a bipolar partisan moment, In Washington. And the last couple of years, maybe was it talking about the plant, you know, people living with rare diseases, the need for reform, the need to advance regulatory science at the FDA to enable these therapies and for him at that, you know, at that podium to talk about Megan, And to see, you know, all the leaders of our country, Supreme court justices, military leaders, every member of the house and the Senate, the vice president to stand twice.

And to give your daughter a standing ovation, an incredible young woman is with us this evening, who should serve as an inspiration to us all today is rare disease day. And joining us in the gallery is a rare disease survivor. Megan Crowley

Megan was diagnosed with Pompei disease, a rare and serious illness when she was 15 months old. She was not expected to live past five on receiving this news. Megan’s dad, John fought with everything he had to say, save the life of his precious child. He founded a company to look for a cure and help develop the drug that saved Megan’s life.

Today she is 20 years old and a sophomore at Notre Dame.

Megan’s story is about the unbounded power of a father’s love for a daughter. If we slash the restraints, not just at the FDA, but across our government. Then we will be blessed with far more miracles, just like Megan.

look out and to see Bernie Sanders and Nancy Pelosi standing and applauding, and to see all Ryan and, you know, John McCain, wherever you, wherever it was there, stand on both sides of the aisle. I think that was one small, good moment. Maybe for America. And it led to some really good reforms and good leaders continuing to do their work at FDA and help highlight what are rare diseases and the number of people who have them.

So, um, again, while I’m confused at times from many things that come out of many people in Washington, that was one very good experience for our fans. Yeah. Well, thanks

David Hirsch: for sharing. It was very powerful. So I’m sort of curious to know why you’ve agreed to be a mentor father as part of the special father’s

John Crowley: network.

We realized early on David, you know, this is a journey for our family. It’s a journey as an individual, a journey as a dad, a husband, a journey as a couple of family, and there is much that we’ve learned. And I think we’ve got an obligation to share that, you know, if we can touch people’s lives even just one at a time and, you know, help people realize the joy in life, how to deal with the sorrow in life and how to deal with the setbacks.

We don’t do it perfectly still by any means. And still struggle with it every day, but I think we’re, there’s some measure of peace. I think, you know what? I pray. I, I pray for grace. Great. Thanks. Prayed for peace. You know, it may be trite, but to literally look out to that next day, we were with a couple of weekends ago, Megan came back to Notre Dame for football weekend, where we’re union with her friends.

Great to have her back in South bend. And she started to talk to me, you know, Megan likes to plan and. So she did that in may of 2021. When I graduated, I think, yeah, I’d like to stay in North Carolina. Now I’m looking at places to live. And I said, Meghan, please. I said, can we get through this weekend? And then I’ll worry about may of 2021.

Um, but the fact that Megan still living with a rare disease life threatening, rare disease, still despite some good medicine that has enhanced and extended her life. The fact that she can have that perspective is, is a gift in itself. You know, and to think about my son, Patrick, you know, Patrick is living with us in New Jersey and Patrick after completing high school and a three year post high school program and told us earlier this year, he doesn’t want to go to school anymore, said, all right, what do you want to do?

And Patrick’s even more profoundly effective, weaker than Meghan I’m in a wheelchair on a ventilator. He said, I want to work. Alright. What do you want to do? You went out and found a job. Patrick works in a flower shop in Princeton and yesterday Patrick got his second job. He’s going to work at AMC movie theaters.

And over the weekend, he said that I’d also like to volunteer. We talked about opportunities and he decided he liked to volunteer at a homeless shelter and try on what a great way to give back and put strength and character there. These are the things that I think we can share with people along the way of their journeys and realizing, you know, all these great blessings.

Maybe it’s not life as we envisioned it years back, but it’s still a wonderful night.

David Hirsch: Yeah. Well, thank you for sharing. Thank you for being part of the network. Let’s give a special shout out to our mutual friend, Nicole Boyce, founder of global genes and LSO behavioral Califor, helping put us together,

John Crowley: Nicole and also a mom and wife and a fierce advocate for people living with rare diseases.

Uh, who’s also the mom of a student at Notre Dame. So we have that connection too, but black wonderful friend.

David Hirsch:  Is there anything else you’d like to say before we wrap up?

John Crowley:  Now David, I think you’ve captured these these questions very well. Thanks for all the great research you put into this. Thank you.

Thanks for coming out. Well, you’re welcome.

David Hirsch: Um, if somebody wants to get information on, make a wish or Amicus therapeutics or to contact you, what’s the best way to go about doing that, then.

John Crowley: Reach out through LinkedIn is a great way. Um, feel free to send a note, uh, to us here at, uh, at Amicus. Uh, the website is Amicusrx.com.

We’re just Google Amicus therapeutics and make a wish. You know, we have 62 local chapters get involved with your local chapter. If you have a child, you think may qualify for a wish reach out and start that process. Um, it’s a, it’s a wonderful experience in itself.

David Hirsch: Thank you, John. Thank you for taking the time and many insights.

As a reminder, John was just one of the dads. Who’s agreed to be a mentor father as part of the special fathers networks, a mentoring program for fathers raising a child with special needs. If you’d like to be a mentor father or are seeking advice from a mentor, a father with a similar situation, your own, please go to 21stcenturydads.org.

Thank you for listening to this episode with the Special Fathers Network dad to dad podcast. I hope you enjoy the conversation as much as I did, as you probably know. The 21st Century Dads foundation is a 501 c3, not for profit organization, which means we need your help to keep our content free to all concerned, please consider making a tax deductible donation. I would really appreciate your support. Please also post a review on iTunes, share the podcast with family and friends and subscribe. So you’ll get a reminder on each new episode. Thanks again, John.

John Crowley: Thanks again.

Tom Couch: And thank you for listening to the dad to dad podcast presented by the Special Fathers Network.

The special fathers network is a dad to dad mentoring program for fathers raising children with special needs through our personalized matching process. New fathers with special needs children connect with mentor fathers in a similar situation. It’s a great way for fathers to support fathers, go to 21stcenturydads.org. That’s 21stcenturydads.org.

David Hirsch: And if your dad looking for help or would like to offer help, we’d be honored to have you join our closed Facebook group. Please go to facebook.com roofs and search today.

John Crowley: If you enjoyed this podcast, please be sure to like us on Facebook and subscribe on iTunes or wherever you listen.

The dad to dad podcast is produced by couch audio for the special fathers network. Thanks for listening.