Don’t ever be embarrassed to ask questions. When you’re jumping into something you know nothing about, you have to figure it out in your own way. When I was learning about my son Ryan’s diagnosis with MPS I, I used to copy pages out of a medical dictionary at the library (breaking all the rules!) to make sense of everything. And, when I had the chance, I asked the researchers questions. I’m so grateful for their patience.
As part of my series about “individuals and organizations making an important social impact”, I had the pleasure of interviewing Mark Dant.
Mark is a retired Assistant Chief of Police who is the current Chairman of the Board of the Washington D.C.-based EveryLife Foundation for Rare Diseases, a science-based advocacy organization dedicated to accelerating biotech innovations for rare disease treatments through science-driven public policy. Mark is also the founder and Volunteer Executive Director of the Ryan Foundation and former President and CEO of the National MPS Society. Mark and his wife Jeanne founded the Ryan Foundation after their only child, three-year-old Ryan, was diagnosed with MPS I. At that time, life expectancy for children with MPS I was less than 15 years. Mark began an immediate global search for scientists and philanthropists who might aid in finding a treatment in time to help children and families living with MPS realize the promise of tomorrow. Today Mark spends his time now volunteering and helping to empower the patient advocate through the understanding that all of us have the power to turn action to hope and hope to reality.
Thank you so much for doing this with us Mark! Can you tell us a story about what brought you to this specific career path?
I was a newly married police officer, living with my wife, Jeanne and our new son, Ryan, just outside of Dallas. We’d moved from Kentucky, where Jeanne grew up, for my job. Our plans included a bunch of kids, and successful career for Jeanne in the airline industry and for me on the police force.
We were so happy. Then, when he was 3, Ryan’s pediatrician noticed his liver was enlarged. I remained convinced my son was fine, but Jeanne took him for follow-up appointments. After tests, and more appointments, a doctor delivered the news that Ryan had a rare, genetic condition called Mucopolysaccharidosis type I (MPS I). It was progressive and would be debilitating for Ryan. The life expectancy at that time (1991) was 10 years. We were told to take our child home and love him, there was nothing we could do.
From the night Ryan was diagnosed, and for the entire next year, my wife Jeanne or I would wake up in the middle of the night to find the other one missing, having walked to Ryan’s room to lay down on the floor next to his bed, just to listen to him breathe. We felt totally helpless. In the morning, we’d get up and go to work, then come home, and do it all again.
I started to spend my time off from work at the public library, reading up on MPS I and rare diseases, looking for anything I could find, any research at all. This was before the internet. I had to photocopy medical dictionaries to help me understand the words in the medical journals. At home, time went on, and everything the doctors had told us would happen — and that I’d read would happen — did.
On our next visit to the children’s hospital, I saw a flyer from the National MPS Society advertising an upcoming conference they were holding in Denver, CO for families living with MPS and ML, a related condition. I decided I would fly out. I needed to meet others who also understood the cause. When I left, Ryan was three, and he wasn’t that affected by MPS. When I got to Denver, I saw other MPS kids — 5-, 7-, 10-, 12-year-olds — but I didn’t see any children over the age of 12. The kids there were dying; I was scared for my son.
I learned about a coming international research conference for lysosomal storage disorders. Jeanne worked for the airlines, so we were able to fly standby for free and off I went to Germany. While there, I heard a presentation from a doctor who was working on a disease similar to Ryan’s, and he was showing pictures of a patient who looked just like Ryan. I expected him to end his talk by saying the patient had passed away. Instead, the patient walked up onto stage, to a standing ovation. I caught the presenter after his talk as he was heading back to his room in the hotel. I asked him if he thought what he was studying might work for Ryan. He said it might, but I’d need two things I didn’t have — money, and time. I flew home and as soon as I landed in the US, I found a payphone and called Jeanne. “He didn’t say no.”
One day I was at the library and by complete chance, I stumbled across a book about how to start a non-profit. Even after I left the library, I couldn’t forget that book, and I couldn’t forget about what I’d learned in Denver — the National MPS Society was seeking funds to help bring families to Orlando, for a bigger conference, where there would be scientists and researchers present who were working on MPS.
Lying next to Ryan’s bed that night I couldn’t sleep, but this time it was because I kept thinking about that book, and about those kids in Denver, and that doctor at the conference in Germany. While it seemed daunting, I knew that nothing would change for Ryan unless we changed it. If we didn’t get up off the floor, nothing would change. We also thought about the other parents out there like us, who needed the strength to stand up.
And so, Jeanne and I, and a handful of our friends and neighbors started knocking on doors, trying to raise money. Our first event was a bake sale raising 342 dollars. The Ryan Foundation was born and eventually, it was time to head to Orlando.
A researcher named Dr. Elizabeth Neufeld from UCLA came to the conference. I knew who she was because she’d written a lot of the materials about MPS. After a long day of learning in Orlando, I saw her in the hotel lobby and followed her into the hotel restaurant. I introduced myself, I told her our story, and I told her about Ryan. I also told her what the doctor had said to me, back in Germany. She told me about a brilliant former student of hers who was working on really promising research, similar to the type I’d seen presented. Then she told me he was about to put it on the shelf because he was running out of money. I called him the next day.
She was right, he was brilliant and understood the patient perspective fully. No one I’ve ever met since cares more about patients than Dr. Emil Kakkis. Dr. Kakkis didn’t say it was impossible. He gave us hope for the first time. We took all of the money we’d raised from bake sales, and golf tournaments, and donor appeals, gave it to Dr. Kakkis and asked him to keep his research going, and to find a way to help us save our son. It was a long road, and not without a lot of struggle, but he did.
A few years after the founding of Ryan’s Foundation, I sat down to put my thoughts to paper about being the parent of a child with a rare disease. The article, “Ryan’s Box of Dreams,” was about how as a parent you put all your aspirations for your children in a crystal box: sending them to college, seeing them get married, becoming a grandparent, and from the moment your child is born you can see all of the dreams adding up inside the box. For people with a rare disease and those who love them, the box exists, it just hurts too much to think about the dreams you’ve placed there. You can see them, but you aren’t sure if they’ll come true. Reflecting on that essay today, I am so proud to say Ryan’s box of dreams has grown. We have been able to add college, a job, and very soon, Ryan’s wedding, to the box. This TORCH Award belongs to him; without the opportunity to be his dad, I might not have been able to add to Ryan’s Box of Dreams. Other rare disease moms and dads out there might not have put dreams into a box for their children. And that’s what we want for all rare disease families — to inspire them to stand up, to dream for their children, and to find a way to share their voice and make a difference.
Can you share the most interesting story that happened to you since you began leading your company or organization?
In 1993, I met Ginger, a 17-year-old girl from east Texas, at a small golf tournament fundraiser we held for the Ryan Foundation. She and her parents had traveled to the event after hearing about it in the local paper — her Dad was a baseball coach and her Mom was a math professor. My son Ryan was just four at the time, so meeting a teenager with the disease was like fast-forwarding time and seeing into the future. I could tell the first time I met Ginger that she was really ill, but the entire event she was smiling. The next year we hosted the same tournament and Ginger came back with her family, only this time she was in a wheelchair. Yet, the same smile was on her face for the entire event. Ginger didn’t make it to the event the third year. She had passed away.
This might not seem interesting in the traditional sense — the story is actually just really sad. I tell it because knowing Ginger changed me dramatically, just with a smile. Even thinking on it now, I can call Ginger’s smile to mind. Her positive attitude in the face of incredible adversity reminds me of the hope we can find in the gift of enjoying today and hoping for tomorrow. No matter how ill she was, she felt each day was a gift. Talk about creating hope. Meeting Ginger made me realize I could find hope where I thought there wasn’t any. She taught me that in rare diseases, hope is often hard to find because the gift of time is taken away. Starting a non-profit, doing scientific research and developing therapies takes time, but each day spent doing the work makes hope possible. To gain time for patients tomorrow, we’ve got to put in the time and effort today.
Can you share a story about the funniest mistake you made when you were first starting? Can you tell us what lesson you learned from that?
I have made so many mistakes, and I still do. I’m sure my wife could tell you which ones of them are funny! Most of the mistakes I have made stem from being so passionate. I let my fire take over and I just keep on moving. The one thing I wish I had done more of would be really thanking those who helped me and my family along the way more often. Some of the people who volunteered alongside me never heard from me how deeply I appreciate them, and I wish I would be more vocal about my gratitude. Sometimes you can be so focused on the work, or getting from one day to the next, that you forget to thank the people standing beside you, helping, just because they care.
The list of people who helped our family and supported Ryan through the early years … that list isn’t long. There were eight families who would work alongside me until 2 a.m., their children were all healthy, but they, too, were parents and had their own full-time jobs, and still, they’d be with us all night, and they’d be back on the phone by 7 a.m. with new ideas. They changed the world and I don’t think they realize the immeasurable impact they had on our family. They don’t get the payoff of meeting all the families around the world they’ve helped and I fear they didn’t get the payoff of my gratitude for their hard work as often as they still should. The TORCH Award I’m receiving is in my name, but it is their faces we see when we look at it because what has happened wouldn’t have been possible without them. If they hadn’t believed in us, we wouldn’t be here.
Moving forward, it’s my ambition to thank others more. I’m still working on it.
Can you describe how you or your organization is making a significant social impact?
I’m fortunate to be a part of two outstanding organizations — the Ryan Foundation, and the EveryLife Foundation for Rare Diseases, which was founded by Dr. Emil Kakkis on the premise of uniting individuals with rare diseases. In the rare disease community, loneliness is the worst emotion, but it is experienced by so many. While family and friends often support those living with a rare disease or their caregiver, many still feel isolated because they don’t always know someone else living a shared experience.
At the EveryLife Foundation, we say that every voice matters. Earlier this year more than 900 rare disease advocates went to Washington D.C. — a number that started as a handful just a few years ago — to tell their personal stories and show that no one living with, or caring for, someone with a rare disease is ever lost. In the stories, others found themselves and found their own voices. These connections start to alleviate feelings of loneliness and are replaced with feelings of hope.
Just as important as the social impact of those connections is the advocacy itself. EveryLife Foundation empowers individuals to use their voice to speak up and out, advocating for policies that represent their needs to ensure they have access to appropriate, affordable medical care. It’s easy to ignore individual rare diseases — the communities are so small — but when we advocate together, as a rare community, we make a movement and there’s impact, and hopefully positive collaboration and change.
Can you tell us a story about a particular individual who was impacted or helped by your cause?
In 2017, I was contacted by a woman in California, Isabel Bueso. She had read Ryan’s story in a Reader’s Digest article when she was living in Guatemala. She, too, was living with MPS (type VI) and had moved with her family to the United States when she was 7 years old after they had been invited to participate in a clinical trial. Isabel told me that without any warning the United States Citizenship and Immigration Services had eliminated the “deferred action” program, which allowed immigrants like herself to stay in the country legally while undergoing life-saving treatment. Isabel and her family had been living here legally for 16 years, paying taxes, so that Isabel could receive life-saving treatment every Friday at a hospital in California. Isabel had 33 days to leave, or she’d be deported. If Isabel had to leave, she would lose access to her life-sustaining therapy, which was not available in Guatemala.
I chair the Board of Directors for the EveryLife Foundation for Rare Diseases, an organization focused on science-driven public policy, and I knew they would want to help advocate for these patients. We got right to work. This was about more than Isabel; it was about all patients who had come to the United States, following the rules, hoping for a chance at life-saving medical care. Rare diseases don’t know borders.
More than 100 patient organizations signed a letter to the Trump Administration requesting that people enrolled in clinical studies be allowed to stay in the United States. I asked Isabel and her family to meet me in Washington D.C. to share their story. We toured the Capitol, met with those on both sides of the aisle and spoke at a congressional hearing. At one point, she was offered a private bill that would provide her with a solution to stay in the United States. as a carve-out to the change in policy. She replied, “thank you, but what about everyone else?” Her courage absolutely blew me away. She was and is completely selfless.
In December 2019, the official documentation for the deferred action status was renewed for two years. I am certain that Isabel gave a face to the issue and made it impossible to ignore the human impact to this policy change. She made sure Congress knew who the action would impact and how the change would help.
I recently had the honor of receiving a TORCH Award from Sanofi Genzyme, which is an award that recognizes individual contributions in the rare disease community and celebrates the ways that those efforts bring hope to others. I am incredibly honored by the award and know that in receiving it, I carry the responsibility to represent people like Isabel, who are also “torches” of hope in their own way. It is hard to be a lone voice advocating for change, and it’s harder when you’re taking on something as big as an ultra-rare disease, or the United States government! Isabel is absolutely an individual inspiration to others and I think her story will stay with me for a long time — one person, committed to a cause, can make a change, and can light the way for others.
Are there three things the community/society/politicians can do to help you address the root of the problem you are trying to solve?
There are three main themes that help address the needs of people living with and caring for people with rare diseases — innovation, funding and partnering.
Innovation means working together to quickly and creatively remove as many barriers as possible to scientific progress. When policies allow scientists to innovate and to do so quickly, effectively and safely, research becomes attractive and exciting. We should incentivize research and as a society celebrate the scientific advances for rare diseases like we do for the progress we make that benefits larger populations. All patients are important and there’s room for everyone to be celebrated.
Funding is directly controlled by policymakers but critically impacts rare disease communities. For example, there are frequent conversations about cutting funding for the National Institutes of Health (NIH), but those cuts directly impact the small grants that individual researchers in academia rely on. Small government grants like those from NIH enable researchers to do bench work on rare diseases, often long before drug companies are interested. Without those grants, that research might not happen. For example, Dr. Emil Kakkis — a man who helped change life for MPS1 patients, who — would tell you that the funds he received from government grants were absolutely essential to turn his hypothesis into a reality.
Partnering means bringing together the entire community of 30 million Americans impacted by rare diseases as a collective voice and working with industry, academia and policymakers. We’re all in this together and we can’t do it alone or in silos. The reality is we all rely on one another to innovate and to fund the efforts, to share information, and to participate in the process, which puts partnership at the core of all we’re doing. Without one party working with the others, innovation halts and barriers can reappear. All voices must collectively be at the table and be heard.
How do you define “Leadership”? Can you explain what you mean or give an example?
For me, I think leadership means being hopeful and having empathy. Leaders must have the courage to make tough decisions that can change the future. You also have to be humble enough to walk the journey at times by following in someone else’s steps. If you can’t see or feel another person’s experience, you can’t champion meaningful change.
What are your “5 things I wish someone told me when I first started” and why. Please share a story or example for each.
- Don’t ever be embarrassed to ask questions. When you’re jumping into something you know nothing about, you have to figure it out in your own way. When I was learning about my son Ryan’s diagnosis with MPS I, I used to copy pages out of a medical dictionary at the library (breaking all the rules!) to make sense of everything. And, when I had the chance, I asked the researchers questions. I’m so grateful for their patience.
- People can’t see your journey unless you tell them (in a kind way). You can’t assume that people understand what you’ve experienced. It can be hard to relate to life as a rare disease patient. When you share your journey, you help people see why you need their help.
- You’re going to get a lot of no’s. It’s okay. You’re not wasting your time. I look back at all the people who told me “no” and those were the experiences where I learned the most about myself. I used to take vacation days and knock on doors to fundraise. It took me a while to understand why people could tell me — the dad of a child with a rare disease — no when I asked for donations. Being told “no” can be just as empowering when you learn from it. I once got a great piece of advice. When people say no it’s not because they don’t want to help, they just can’t right now.
- People can be too patient. I can understand the perseverance that scientists must have to put their effort and life’s work into advancing medicine. It moves so slowly! But as a dad with a sense of urgency for my son, sometimes I felt things didn’t move quickly enough and questioned whether this person would be acting with the same speed if it was their son. Don’t be so patient that you don’t push for progress.
- With a uniting cause, there are still times your objectives won’t match up. For example, in my experience scientists and those working in academia who are passionate about rare diseases can be most motivated by their desire to publish versus continuing the amount of research needed to lead to a treatment. I believe we must find common ground — the objective can still be to publish the work, but the mission should be to treat. I believe we would be able to help even more patients and do so more quickly if we thought more collectively about our objectives.
You are a person of enormous influence. If you could inspire a movement that would bring the most amount of good to the most amount of people, what would that be? You never know what your idea can trigger. 🙂
We are living in the movement. Being recognized recently for being an individual who inspires others to work towards change has been very validating! From the early 1990s to today, I have watched as those who were once overlooked gained a voice. People living with rare diseases or conditions are empowered to have conversations with their doctors about their health. The internet has helped them find information about their condition, connect with other patients, and get advice from experts. People living with or caring for someone with a rare disease started this movement — they used their voices to change the world. There are children who have grown into adults and adults who live long, successful lives because they were empowered to demand change. There are bills that have been passed, regulations that have been changed and treatments that have been developed all because of these voices. A person empowered to talk about their health really can change the world — not only for themselves but for others. That’s how the “rare” movement has grown from a handful to thousands, and, hopefully someday soon, millions.
Can you please give us your favorite “Life Lesson Quote”? Can you share how that was relevant to you in your life?
Never, never, ever give up. My son Ryan now has this quote on his desk. When doctors explained Ryan’s diagnosis nearly 30 years ago, they sucked all the hope out of the room for me and Jeanne. We almost gave up. We found that our persistence led to answers. We kept telling our story, networking with others and it led us to one person who shared our passion, Dr. Emil Kakkis. We never gave up.
A few weeks ago, we watched home videos of Ryan when he was little. It was like bringing this mantra to life. He has the same spice today as he did in those videos. It also reminded us of all the difficult things he has fought through, the time in the hospital, and having to do it all with our extended family more than 900 miles away. He has so much inner strength and he never, never, ever gave up.
Is there a person in the world, or in the US with whom you would like to have a private breakfast or lunch with, and why? He or she might just see this, especially if we tag them. 🙂
While she is not one who many would consider “famous,” I certainly do. I’ve always been enamored with Dr. Elizabeth Neufeld. Dr. Neufeld is the 92-year-old retired chair of UCLA’s Department of Biological Chemistry, and the pioneer who demonstrated the link between basic science, applied investigation and complex rare diseases, specifically lysosomal storage disorders. One time I was in Brazil at a conference for lysosomal storage disorders and I was able to speak with her. I learned she and her family emigrated to the United States as Russian Jewish refugees in the 1940s. She faced many challenges as one of the few female PhDs in her field. Yet, she overcame so much in her life personally and accomplished so much professionally and stayed so incredibly humble and approachable. I believe she is the reason for much of the progress in the field of genetics. She has a remarkable story and I would love to share a meal to ask her more about her experiences and, of course, her feelings on where science needs to go next!
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This was very meaningful, thank you so much. We wish you only continued success on your great work!