This is the first of a new series of interviews we’ll be sharing to help bring our readers closer to others involved in the Duchenne community.
I was honored to have the opportunity to ask Debra Miller a few questions about her experience as the President and CEO of CureDuchenne. She was very generous with her time and candid with her thoughts.
CureDuchenne’s Mission: “Our mission is to save this generation of children and young adults with Duchenne muscular dystrophy.” CureDuchenne is recognized as the global leader in research, patient care and innovation for improving and extending the lives of those with Duchenne. We operate with integrity through compassion, and empowering the community nationally with positive solutions. Our actions fuel hope for families, enable progress for drug development, and extend ambulation for patients in collaboration with pharmaceutical companies, medical and healthcare professionals, our scientific advisory board, and our board of directors.
You have made a tremendous impact in the Duchenne community through your work with CureDuchenne, Debra. I think your story is amazing and I’d like to know, how you have managed to obtain so much support for this cause?
I’d like to say that this has been a quick and easy road, but it’s been a long and arduous process. We started off with a small group of supporters. My motto has always been “integrity first,” so we built a strong reputation of not making promises we could not keep and we were able to encourage these supporters to expand our donor base. Our strength is innovation. As a mom of a Duchenne boy, I have no patience for bureaucracy and I am acutely aware of what is needed in this community. When there was very little research being funded, CureDuchenne stepped up and took the risk of funding early scientific projects. When academic researchers were publishing papers, CureDuchenne hired drug development experts to push the science through to clinical trials. Having seen the positive impact that a knowledgeable physical therapist made on my son’s condition, we created CureDuchenne Cares, so that Duchenne patients around the country could benefit by trained physical therapists and learn about research and clinical trials no matter where they lived. I love the fact that our team can change on a dime and create new programs out of a vision. Science changes fast and we are dedicated to keeping up with it.
How do you decide where to invest CureDuchenne’s precious resources including funds, time and effort?
As the CEO, the final decision of which projects and programs to fund is mine, but I welcome robust dialog and different opinions. We become aware of new research either through reading or through meeting with scientists and biotech companies. CureDuchenne has two very knowledgeable professionals on board. Jak Knowles is an M.D. with extensive drug development experience. Michael Kelly is a Ph.D. that more recently has been evaluating science for a large venture capital firm. Both men have excellent analytic skills and view science through the lens of, “Can this molecule or therapy actually make it into an approved treatment?” instead of, “Is it interesting science that can be published?”
We take a top down view and search for drugs that can treat the whole disease, since so many organs are affected by Duchenne. When we have a project of interest, we turn to our scientific advisory board and engage the members that have the most expertise in that area. The final test is the mom filter. It has to be something that I would be willing to give my son and something that will move the needle.
Speaking of investment in research; will you please tell me about CureDuchenne Ventures?
Raising money for a rare disease can be really difficult. Biotech companies are doing much of the research now and academic institutions are earning lucrative licensing fees for their research. It did not make sense to us that our donor money would end up enriching a biotech company or academic institution. Then we would have to go back to those same donors and ask for more money. Instead, we use a venture philanthropy model where CureDuchenne Ventures invests in a biotech company. If the research is successful and there is a return on investment, CureDuchenne is able to share in the gain with the biotech company. 100% of the funds from these successful investments is then redeployed into future Duchenne research. Our first successful exit contributed the seed financing for six additional research projects.
There are a few organizations involved in the DMD space. Does that cause any problems? How do you view collaboration with other organizations?
Our first goal is to bring beneficial therapies to market. My belief in collaboration is working together on a project that gets buy-in from different groups. Too many times organizations have attempted to bring other organizations together, but it’s been just for the sake of bringing everyone together. Collaboration for collaboration’s sake doesn’t work. Each organization is unique and created by people that want to be independent. If the collaboration is focused on a project external to all the organizations, then everyone is working on this independent goal and it works much better. I have learned over the years to say no to any organization that wants to take my time with anything other than measurable and impactful projects.
CureDuchenne runs very effectively and efficiently. I believe that develops from great leadership. How do you bring your staff onboard to be as devoted to the mission as they appear to be? Do most of them have a personal connection to Duchenne?
My husband and I are the only CureDuchenne staff or board members affected personally with Duchenne. My style has been to hire good people and let them run with it. Individuals that want to grow, create, and work really hard do well at CureDuchenne. Those that see this as a 9-5 job and need a lot of direction don’t do well in our environment. I travel a lot so they have to be self-motivated. My strength is not in my management, but in my vision…my ability to see what needs to be done. I drive my team crazy often. When I come up with ideas and goals, they have panic in their eyes. But, they are slowly learning to trust that magic can occur if we think about the problem differently and if we believe there is a solution to every problem. I expect a lot from my team, but the reality is that they work as hard as they do for the boys. They have gotten to know Duchenne families very well and they are in the fight for them.
I believe that CureDuchenne will fulfill its mission and deliver a cure for this generation of children and young adults with DMD. I truly believe that and I’m fully committed to that exact same mission for the good of all. I began on this road because of my son’s DMD diagnosis, but it’s now developed into working for the good of everyone affected by DMD. As CEO of CureDuchenne, how do you delineate between Mom choices and CEO choices? I mean, as parents, we’ll give anything for our kids’ well-being, but has that ever conflicted with a “business decision”? How have you handled that very difficult situation if it has occurred?
At first, I just wanted a cure for my son. Then I met a few other Duchenne boys and they had a place in my heart. I’ve evolved to the point that I see such an injustice with the lack of funding, focus and education in Duchenne, it drives me to care about all the boys, and those few girls. When I was young, my mom always called me her pragmatic kid. I was always really good at finding plan B, when plan A didn’t work out. I’ve had extreme heartbreak that my son lost an opportunity to have a drug developed for his specific mutation, mostly because of politics. I don’t see my work bringing therapies to other kids as being in conflict with hoping and working on a drug for my son. I also feel a tremendous responsibility to the families and the donors. I feel that God blessed me with extreme determination and a cause. I’ve accommodated this by focusing my life on very few things; God, my son and husband, and my work. I’m probably the most boring person in the world unless you are interested in Duchenne research.
Your focus, commitment, and determination have led to great progress toward fulfillment of CureDuchenne’s mission, Debra. It might not seem possible, but I promise you, your mission means just as much to me as it does to you. My son is also one of, “…this generation of children and young adults with Duchenne muscular dystrophy.” On behalf of everyone in the DMD community, thank you for all you have done to provide care, comfort, and hope to our loved ones.
You can stay connected with Debra’s work with CureDuchenne by following them on Facebook, Twitter, LinkedIn, YouTube, and Instagram