Debra Miller, CEO of CureDuchenne, co-founded the organisation along with her husband, with the aim of helping her son and others like him. She talks to RARE Revolution about the importance of combining passion with strong business acumen in order to advance research and achieve their ultimate goal of a cure for Duchenne CEO series: meeting the beating hearts behind the RARE brands
What made you want to move into the wide world of rare disease, and then specifically CureDuchenne and what did that journey look like?
My entry into the world of rare disease was a result of our son, Hawken, being diagnosed with Duchenne muscular dystrophy at the age of five. I had no intention of founding a non-profit, but there were no organisations focused specifically on finding a cure for Duchenne. My husband and I took the first step forward and have not looked back in the last 17 years.
What do you anticipate will be the biggest challenges and opportunities for your organisation in the next two years?
We have no shortage of quality research and clinical programs to fund. Almost every day we come across impactful projects that could accelerate progress toward identifying a cure. Funding is the greatest challenge we have. Approximately 10,000 families are affected by Duchenne, it’s not a large disease community, so we have to find ways to educate and engage the general public in our mission of curing this disease.
What is your proudest moment in your career thus far?
A career highlight so far has been when CureDuchenne searched for the best scientist to develop CRISPR for Duchenne. We partnered with Dr. Eric Olson at UTSW and co-founded a company alongside him called Exonics Therapeutics. We committed to an investment that was large at that time for our organisation, but we were counting on other Duchenne organisations to help us raise the funds. Unfortunately, none of them contributed, however, we worked hard and were able to fund this project alone. Within two years, Vertex Therapeutics acquired Exonics for $245 million and committed vast resources to do their best to bring this experimental, yet hopeful therapy to Duchenne patients. If anyone can bring a CRISPR treatment for Duchenne, Vertex can. This project demonstrated the commitment of the CureDuchenne team and truly validated our Venture Philanthropy model.
What and who are your personal and professional inspirations and why?
My biggest inspiration is my son, Hawken! He is an amazing 23-year old young man that lives life to the fullest, with Duchenne. He never complains, he looks to the future and has an amazingly positive attitude. He graduated with honors from USC, he is working as a writer for the Washington Post, along with several freelance writing gigs.
I’m proud of his desire to help other rare disease patients navigate this journey and share his faith with so many people. In the Duchenne community, Hawken is advocating for the disease through his passions in gaming, streaming, writing and recording podcasts.
What advice would you give someone considering working in the rare disease space?
To succeed in the rare disease space, you need to be powered by passion. There is no other way. It is very challenging to cure a rare genetic disease and giving up is just not an option. I believe it needs to be a life’s work, otherwise, you will not be able to deal with the valleys of despair and the pinnacle of successes.
Also, you need to be prepared to apply strong business practices to your passion. There are thousands of non-profits that had great intentions, lots of passion, but no plan and not enough financing to succeed.
Do you think the government does enough for the rare disease community at a local and central level, and what gaps do you see currently or emerging?
I recently presented at a Fireside Chat with Peter Marks, FDA Director of Center For Biologics Evaluation and Research (CBER), and Christopher Austin, Director, National Center For Advancing Translational Sciences (Ncats), National Institutes of Health (NIH). We discussed the need to memorialise the will of the people and the government to find a cure and treatments for COVID-19, after we have treatments and vaccines for this virus. There is much more to be done for rare disease, and it rests on all of us addressing rare disease as an emergency, just as we have treated COVID as a national emergency. As Dr. Austin said, “it is not that we can’t do it, it’s that we won’t”. We need to change the will not too, to can do. The government has many mechanisms to speed access to therapies for rare disease; from funding to working with rare disease organisations on scientific projects to flexible trial design.
What would you say are some of the biggest motivators for your employees?
Without a doubt, a dedication to finding a cure for Duchenne and serving our families are the biggest motivators for the CureDuchenne team. We have been blessed that several of our funding projects have resulted in many clinical trials, approved drugs, and a very successful venture philanthropy model. They are able to point to our progress proudly which attracts donors, scientists, investors and rare disease families.
What are the toughest parts of being a CEO, and conversely what are the most rewarding?
Obviously, the sense of responsibility to the families we serve. I do not want to lose any of the boys or young men we have become so close to. There are so many facets to serving a disease community; finding and funding the best research, developing clinical programs and care education to improve the lives of these families, thinking far ahead of our current projects and forecasting what will be needed if the current projects succeed and if they fail. Every day, I feel a bit of whiplash toggling between all of this. And, of course, raising money is a constant priority since it fuels all of our programs. Fortunately, I have a dynamite team of experts that are self-driven, innovative and they teach me so much.
What would be your one wish for CureDuchenne for the year ahead?
We have at least two gene therapy programs entering phase III trials this year. My wish is that they are dramatically effective for the boys and that we can find solutions to the limitations of these therapies which will enable older Duchenne patients to participate in these treatments. My son included, his clock is ticking loudly and we need to move these ahead at warp speed.
If you weren’t CEO of CureDuchenne, what was Plan B? What did your 10-year-old self want to do as a job?
Before our son’s diagnosis, I was heading down the path of computer publishing. There are a lot of similarities to the hay days of the PC industry and today’s biotech industry, I most likely would have stayed in technology, unless I discovered biotech through a different means. I have always been good at raising money, so this has obviously been a strength for CureDuchenne. As much as I wish my son did not have Duchenne, he saved me from living a life with little meaning chasing financial success. Every single day I am aware of the blessings of my family, my community and my country. My son Hawken has given me the deep desire to help others.