Don’t fall for the “this is the way we’ve always done it” conversation. There were a couple of Duchenne organizations in existence before we started CureDuchenne. In fact, we worked hard to support them without any intention of reinventing the wheel. I believe we wasted precious time trying to get their buy-in as they were stuck in an esoteric model that was not moving the science and research being conducted into drug development. After starting CureDuchenne, we reached out to a few of those organizations to help fund some of our projects, but they were unwilling to help saying “that’s not the way we do things.” So, these organizations did not participate in funding the first drug approved for Duchenne and they passed on co-funding a start-up CRISPR company, Exonics Therapeutics, that we co-founded and sold to Vertex Therapeutics. CureDuchenne was able to benefit from this sale, but the Duchenne community would have realized many millions of dollars that could have been redeployed for research had they participated in the original funding. Instead, the profit went to VC firms that shared the same belief and vision as CureDuchenne. The moral of the story is collaborate with like-minded people who share the same vision and sense of urgency that you do.
As a part of our series about strong women leaders, I had the pleasure of interviewing Debra Miller.
In 2003, Debra Miller’s life was interrupted by her son Hawken’s diagnosis: Duchenne muscular dystrophy. To fund research for a cure, Miller founded CureDuchenne and employed an innovative “Venture Philanthropy” strategy. CureDuchenne’s investments accelerated next-generation exon-skipping, gene-therapy and gene-editing, along with new modulation therapies, by funding proof of concept research that attracted over 2 Billion dollars to these research companies from biopharma and venture capital funds. CureDuchenne’s early funding led to the first 3 FDA-approved Duchenne drugs with many more currently in clinical trials.
Thank you so much for doing this with us! Before we dig in, our readers would like to get to know you a bit more. Can you tell us a bit about your “backstory”? What led you to this particular career path?
Well, it’s an interesting path, but one I’m very proud of. I started my career in advertising. My husband Paul and I believed we had the perfect life and everything was playing out as planned, especially finding out about having our son, Hawken. When Hawken was four-years-old, he played soccer and we noticed he couldn’t keep up with the other kids. My husband recollects a time when Hawken had trouble roller blading home from lunch, so we noticed some developmental issues that weren’t quite right. A friend suggested Hawken might have Duchenne muscular dystrophy. At the time, we had never heard of the term and I immediately went full Mom mode in search of a doctor to confirm. We had no family history of the rare disease and were left with an empty feeling and a lot of questions. In November 2002, the Duchenne muscular dystrophy diagnosis was confirmed and threw a massive wrench into our lives, but we were going to fight to do everything in our power and whatever it took to find an answer for Duchenne. From there, after not having any experience in philanthropic or 501c3 work, we started CureDuchenne to give our son and other children the best chance at beating this rare disease.
Can you share the most interesting story that happened to you since you began leading your company?
My husband and I didn’t have any tangible experience with nonprofit organizations, science or medicine. When starting CureDuchenne, we entered into each project with a heightened sense of insecurity and a ton of humility. Soon after CureDuchenne funded its first Venture Philanthropy investment in 2004, the company — Prosensa — received around 12M dollars in VC funding. This was a massive stress relief as we were vindicated that our initial investment proved to be “smart money” by the VC firms. The investment validated our business model, which we still use to this day. The kicker of the whole thing being, when we mentioned the sense of relief felt when the funding went through to the CEO of Prosensa, he laughed and said one of the reasons the VC’s invested in the company was because of CureDuchenne’s investment.
This is when the light bulb went off and truly accounts for the jumping off point of the organization. It was an enlightening experience to realize how powerful a focused patient organization and its advisors can be in the drug development ecosystem.
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?
When Hawken was still quite young, he was granted an opportunity to work with Make-A-Wish, which was to meet Bill Gates. Make-A-Wish is a great organization which I respect and appreciate greatly.
MAW flew us to Seattle, along with two other families, to tour part of the Microsoft campus and have lunch with Gates, followed by an individual meeting. In my past life, I met Gates while I worked at PC Magazine, so I thought he might remember me, but to my surprise, my typically very shy son broke the ice at lunch, asking him what he likes to do when he’s not working. Gates went into explaining his foundation, the medical research conducted by his organization and their commitment to global health. Without skipping a beat, Hawken chimed in saying “my mom has a foundation,” which he again mentioned when we had our one-on-one meeting with Bill.
I was absolutely tormented the entire afternoon with Bill. I have so much respect for the Make-A-Wish foundation and what they do, and this was about Hawken, not CureDuchenne, so I was determined to not interject CureDuchenne’s work into the conversation. Hawken continued innocently mentioning “his mom’s foundation,” and I continued to have an internal battle about the right thing to do. Ultimately, I did not mention CureDuchenne, which still to this day, makes me wonder “what if.”
I’ll never know if it would have made a difference, but at the very least, I’d love to have been able to keep him apprised of our progress in research and drug development over the years. My lesson is; as long as the motive is noble (finding a cure is), don’t hesitate to take every opportunity to discuss your path and progress with others, especially others of significant influence.
None of us are able to achieve success without some help along the way. Is there a particular person who you are grateful towards who helped get you to where you are? Can you share a story about that
There are many people responsible for the success of CureDuchenne, but if I had to pick one person it would be my son, Hawken. He was the reason my career path was redirected and the inspiration behind CureDuchenne. Throughout the entire process from diagnosis when he was 5-years-old to graduating High School to earning a degree in Communications from USC, he’s been the guiding light and provided incredible positive reinforcement that has allowed me, my husband and the entire organization to push forward. Hawken has given me the latitude to run this organization, while being a Mother, which I’m eternally grateful for and cherish every day.
In my work, I often talk about how to release and relieve stress. As a busy leader, what do you do to prepare your mind and body before a stressful or high stakes meeting, talk, or decision? Can you share a story or some examples?
I’ll provide two tangible examples on what helps me release and relieve stress. The first is exercise and living an active lifestyle. Physical activity through daily exercise has many proven benefits, including increased overall health, mental clarity, better sleep habits and, especially for me, creating an environment where I live with a positive attitude. The release of endorphins and dopamine during physical activity is something that provides a springboard into my busy weeks. The other critical stress reliever is time management. It’s a basic life skill that many find difficult to achieve, but with so many different moving parts within CureDuchenne, it’s an absolute must and I’m always working to block out chunks of time throughout the day/week/month to focus on upcoming meetings, panel discussions or discussions with partners.
As you know, the United States is currently facing a very important self-reckoning about race, diversity, equality and inclusion. This may be obvious to you, but it will be helpful to spell this out. Can you articulate to our readers a few reasons why it is so important for a business or organization to have a diverse executive team?
This is an extremely hot-button topic with everything going on in the world today, but it’s important to understand that Duchenne doesn’t care about your age, race, gender, ethnicity or socioeconomic status. It’s a rare disease that affects thousands of families around the world and we’re here to help provide a positive reinforcement and guiding light in their lives. We’re in an interesting position as we are a predominantly female workforce with great diversity coming from all walks of life, but with one common goal, to find a cure and improve the lives of Duchenne patients. It’s especially important to reach families that are not easy to find or may not even know there is help and support out there.
As a business leader, can you please share a few steps we must take to truly create an inclusive, representative, and equitable society? Kindly share a story or example for each.
Boys affected by Duchenne ultimately end up in a wheelchair, which sets them apart from society and can have them feeling left out. As leaders, we must take a step back and observe what creates these barriers and take action to course correct feeding into a fully inclusive environment. At CureDuchenne, we’ve adapted all of our content, materials, educational tools and workshops with the launch of CureDuchenne en Espanol, creating inclusivity for families with a Spanish background. Additionally, we’ve diversified our staff to represent our community’s families who live with Duchenne and can represent our community firsthand. To create an equitable society, we offer all of our content and resources free of charge in order to provide an equal opportunity for people of all socio-economic backgrounds to access the latest data and information related to Duchenne helping to improve their quality of life as much as possible.
Ok, thank you for that. Let’s now jump to the primary focus of our interview. Most of our readers — in fact, most people — think they have a pretty good idea of what a CEO or executive does. But in just a few words can you explain what an executive does that is different from the responsibilities of the other leaders?
As CEO, it’s my responsibility to have a clear vision for the organization. It’s truly the CEO’s responsibility to ensure all efforts align with the organization’s core values, purpose and mission. It’s my duty to empower a wide breadth of teams and lead with fearlessness to make decisions and take responsibility for our success and failures. A key differentiator from other leaders within the organization is that my main responsibility is jumping from one program, department or project to another, ensuring each leader has the necessary tools to drive their responsibilities forward. Implementing our vision means having the broad understanding and responsibility of the entire entity, versus subject-specific responsibilities.
What are the “myths” that you would like to dispel about being a CEO or executive. Can you explain what you mean?
A myth that I’ve heard in the past is that CEO’s don’t like to relinquish control, which is quite funny because as CEOs, it’s our responsibility to hire talented leaders and staff that will take our organization one step closer to our goals. I started CureDuchenne on my own, as a mother looking for a cure for my son, never aspiring to be a CEO. Throughout my career, I’ve learned the “ins and outs” of a nonprofit along the way, and as much as I love doing it all, it was critical that I learned to step back, hire help and allow my teams to flourish in their roles — which ultimately allows us to grow and expand as an organization.
In your opinion, what are the biggest challenges faced by women executives that aren’t typically faced by their male counterparts?
It’s been a unique experience and I’ve been thrilled to see, during my time as CEO of CureDuchenne, society evolving into a culture where women support and empower other women to embrace executive positions on top of motherhood and all the other roles we have in our lives. When I began my career in the tech space, I was one of the few women in my field. To power through it all, I put myself in the same role as my male counterparts and didn’t allow myself to be treated any differently. My personal motto is, “if you expect equality, you will receive it,” and that’s proven to be true in my career — hard work and dedication has gotten me to where I am today.
Of course, I’ve faced challenges since my son’s diagnosis with Duchenne. Juggling work with CureDuchenne and my duty as a mother always presents its obstacles, but with the support system I’ve built, including my husband and the organization, I’ve been able to adapt and stay focused on my son and the mission of our foundation.
What is the most striking difference between your actual job and how you thought the job would be?
I’ll start by saying, my son’s diagnosis and the lack of answers received early on due to very little research advancement in the Duchenne space, thrusted me into this position. In my wildest dreams, I never imagined the work I’d be doing, how many people I’d be responsible for, what my actual job would be and the families we’ve helped since starting CureDuchenne.
Prior to becoming a CEO, I thought they were more distant and less directly involved in projects and programs. As it turns out, because we are a growing organization, I insert myself and am more involved in key projects. But, when working with medical research and pharmaceutical companies with compliance issues, I’m ultimately responsible, not only for the success of the organization, but to make sure we act with integrity and professionalism.
Certainly, not everyone is cut out to be an executive. In your opinion, which specific traits increase the likelihood that a person will be a successful executive and what type of person should avoid aspiring to be an executive? Can you explain what you mean?
The type of person that will succeed as an executive will be one who has a clear vision and is determined to reach their goal, no matter what it takes. It takes an unwavering and relentless mindset that doesn’t acknowledge quitting as an option and, at times, needs to be competitive and make sacrifices in order to achieve the mission at hand.
In my case, being challenged to find a cure for a rare genetic disease is a hurdle we face every day, as a team, but our inspiration is our family. Personally, it’s all about my son Hawken, and the community that is counting on CureDuchenne to help provide guidance and support for their sons. I wake up every morning determined to continue pushing forward and work extremely hard to achieve our goal. With science, research and technology, we’re getting closer to finding a cure, but the work is not done and I won’t stop until we’re able to support every boy and families affected by Duchenne.
What advice would you give to other women leaders to help their team to thrive?
Just be a leader and break down those barriers. Be the best. Learn and do whatever you have to do to know your market, your organization, your constituents, which will help you become the best at what you do. Possess more knowledge, more skills, and continually hone your craft and strive to do better, while putting the right people in the correct positions to thrive.
How have you used your success to make the world a better place?
I really hope I’m using my success to make the world a better place. It takes a lot of dedication, determination, focus, and sacrifice. I self-reflect often to gauge if what I’m doing is worth the time of my team, the generous donors, and the families who have put their hope and faith into what we’re doing as an organization. While I can’t give you a definitive answer as this story is still incomplete, It’s important to conduct reality checks with yourself and your organization to see if what you’ve done and are endeavoring to do is really making a difference in the world. I’ve seen the significant progress CureDuchenne has made over the last 17+ years in getting close to a cure for a genetic disease, and don’t know exactly what the future holds, but in my heart, I can honestly say we’ve made a difference in the Duchenne community and the rare disease space, which makes me proud.
What are your “5 Things I Wish Someone Told Me Before I Started” and why? (Please share a story or example for each.)
- How absolutely ridiculous it is to expect nonprofits to operate with an 80% profit margin while still growing. The traditional accepted expense ratio of 20% revenue going toward operating expenses is unrealistic for a growing organization, especially one in the rare disease space that’s lifeblood is medical research. It’s important to understand that to make an impact on a disease you need to hire the best and brightest in all necessary fields (scientists, business strategists, etc.).There are so many nonprofits that fail because they don’t invest in the right talent. In spite of that, we have maintained that ratio, but if I did it over, I’d focus on the impact and not stress about the expense ratio.
- Expect more out of people. When I first started CureDuchenne, I lacked management experience and felt guilty asking my employees to do the work I hired them to do. Is that a female thing? I’m not sure, but, I’m now bolder in expecting my team members to have ownership on the outcome of their projects, I expect Duchenne parents to step up and help us raise funds to bring treatments to their kids, and I expect more of our researchers and board members to be fully engaged.
- Don’t fall for the “this is the way we’ve always done it” conversation. There were a couple of Duchenne organizations in existence before we started CureDuchenne. In fact, we worked hard to support them without any intention of reinventing the wheel. I believe we wasted precious time trying to get their buy-in as they were stuck in an esoteric model that was not moving the science and research being conducted into drug development. After starting CureDuchenne, we reached out to a few of those organizations to help fund some of our projects, but they were unwilling to help saying “that’s not the way we do things.” So, these organizations did not participate in funding the first drug approved for Duchenne and they passed on co-funding a start-up CRISPR company, Exonics Therapeutics, that we co-founded and sold to Vertex Therapeutics. CureDuchenne was able to benefit from this sale, but the Duchenne community would have realized many millions of dollars that could have been redeployed for research had they participated in the original funding. Instead, the profit went to VC firms that shared the same belief and vision as CureDuchenne. The moral of the story is collaborate with like-minded people who share the same vision and sense of urgency that you do.
- Go where the puck is! My husband is a hockey player and that has become our mantra. Medical research is interactive, and we are far from the final treatment solution for Duchenne. As soon as you fund one project, you need to be thinking several steps ahead. We learned to plan for success with each step, while also being aware of the many future steps it will take to cure this disease. For example, our gene therapy funding project with Bamboo Therapeutics, which was acquired by Pfizer, is soon beginning it’s Phase III trial. Even though we have great hope for this therapy, we have already begun funding next generation gene therapy that could potentially deliver the full length dystrophin protein that is needed to truly cure this disease for all patients.
- Random lessons — Have a single focus; for us it was finding a cure for Duchenne. Hire people smarter than you; it’s reasonable to expect them to be loyal. Build the plane while you fly it; you’ll never have perfect knowledge, and technology changes so fast you can’t possibly keep up. Separate your emotions from your logic; hard to do with a seriously sick child, but you have to do it. Take risks!
You are a person of great influence. If you could inspire a movement that would bring the most amount of good for the greatest number of people, what would that be? You never know what your idea can trigger.
There are 30 million people in the United States with rare diseases, mostly fatal childhood diseases. So far, approximately 150,000 in the US have died from COVID-19, a very disturbing loss of life. Almost all children born with rare, genetic diseases will die. Long after COVID is a distant memory, which I hope happens quickly, children will still be dying every day from rare diseases.
Let’s take the technology, the accelerated regulatory pathways, the funding, and, most of all, the will and the grit to save the lives of these children that will continue to be born with rare diseases. This is my commitment and I welcome the help and support from everyone who believes we can do this!
Can you please give us your favorite “Life Lesson Quote”? Can you share how that was relevant to you in your life?
My favorite quote is “if you want to see God laugh, tell him your plans.” This quote was true for me even before CureDuchenne was founded. As I mentioned earlier, my husband and I thought we had our lives planned out and then Hawken’s diagnosis changed all of it. Our original plans were so far from what our reality turned out to be. This is relevant to CureDuchenne and any other nonprofit as sometimes science projects don’t work out, while there are others that out of the blue just happen. What I’ve learned from this quote and applying it to my own life is that you can work hard and prepare, but you have to be pragmatic. What I mean by that is you can’t be so attached to an outcome of a situation that you don’t have a “plan b.” It’s important to have a plan and work hard towards your goals, but also be able to pivot that plan when the outcome isn’t what you anticipated.
We are very blessed that some very prominent names in Business, VC funding, Sports, and Entertainment read this column. Is there a person in the world, or in the US with whom you would love to have a private breakfast or lunch with, and why? He or she might just see this if we tag them.
Elon Musk; He gets the next to impossible done. He thinks big. He can cure Duchenne.
Thank you for these fantastic insights. We greatly appreciate the time you spent on this.