Name: Alison Rockett Frase
Title: President, co-founder, and patient advocate
Organization: Joshua Frase Foundation for Congenital Myopathy Research
Disease focus: Myotubular myopathy, a X-linked genetic condition that affects skeletal muscles and occurs almost exclusively in boys. People with this condition have muscle weakness and decreased muscle tone. The condition impairs the development of motor skills, such as sitting, standing, and walking. Often people with the condition need assistance breathing and suffer abnormal bone development. About half will die within the first 18 months of life, and most will not live past early childhood.
Headquarters: Ponte Vedra Beach, Florida
How did you become involved in rare disease: In 1995, when our son was born with an ultra-rare genetic disorder, they told us to bring him home, and “if he makes it to his first birthday, bring him back and we’ll reevaluate him.” There was very little research being done at the time—only 6 research papers published in 30 years on myotubular myopathy(MTM). Neither NORD nor MDA had any information on MTM. We knew we needed to do something to make a difference. Before our son was a year old, we filed for a 501(C)(3). We decided to use my husband’s platform in the NFL to raise a lot of money.
Previous career: I was in the music industry and the fashion industry. I worked as an assistant personal manager for a big-name rock and roll band.
Education: BS in Science (telecommunications), Oral Roberts University.
Organization’s mandate: Fund cutting-edge research for centronuclear and myotubular myopathies, and to support families on the day-to-day journey with patient advocacy and financial assistance for unmet needs.
Organization’s strategy: Implementing structural integrity. We have several advisory boards, and these advisory boards help us run smooth operations– a scientific advisory board, a translational advisory board (which was key), and an educational advisory board, which is very active with parents who have children who are currently alive or have passed. We educate new families as they are born into this horrible disease.
Funding strategy: The first two years we partnered with MDA and raised 400k. From there, we used our connections on Wall Street, the NFL, and music industry. We raised $6.3 million in next ten years. When the market crashed in 2008 the funding strategy changed (our last event was 2009). We were heavily weighted in Wall Street and it hurt us. Five of our best years we averaged 850k in one night. To that point, we had incredible preclinical data. Things slowed down considerably for the fundraising, but we had a mouse and a canine animal model with robust pre-clinical data. The science attracted entities like the NIH, and GENETHON, a company from France. They were excited about the progress we had made.
What’s changing at your organization in the next year: Our research focus is shifting from myotubular myopathy to other related myopathies as MTM has entered clinical trials. There has been a shift as Audentes Therapeutics is moving our gene therapy program into clinical trials. The first child was treated the week of September 20, 2017. We will continue to search out advancement’s in the field.
Management Style: We have a scientific advisory board, a translational advisory board, and an educational advisory board. Our board of directors is our weakest point. They are not currently engaged at the level we would like them to be. You are as successful as your board is engaged. That is where your strength lies. To bring change and hope to the world requires that we trust our gut instinct when we have no logical reason to walk the path that we’re walking. It requires that we get comfortable being uncomfortable.
Management philosophy: I am eager to be mentored by others who have had success in the niche, and I’m consistently reaching out to key opinion leaders. I encourage others to be the catalyst to initiate change, while engaging others to champion your cause.
Guiding principles for running an effective organization: Trust and pursue your vision. Have fortitude, perseverance, and resolve. A researcher recently said, “You know, your signature on your email for 15 years has been “vision, fortitude, and resolve.” Be flexible in terms of change and setbacks. You have to be able to adjust to unexpected circumstances because that is the nature of the beast in the rare disease space. A friend passed this list on to me recently; it is relevant of the mindset we all must have in order to initiate change. To choose the “UN” in your approach, an UNstoppable attitude, an UNconventional approach, an UNbreakable spirit, an UNtraditional perspective, an UNcommon degree of courage in order to make change.
Best way to keep your organization relevant: I think it is important that foundations keep this mindset as they push science forward for their indication. My thoughts are it’s difficult to keep your organization relevant in the rare disease space if you have advanced to clinal trials and the efficacy of your science has been proven in preclinical work. It’s difficult to convey to donors how much work there is “left” before commercialization. It’s an easier “ask” if you haven’t found a cure yet. My advice and lesson learned is to continue to press through, remain steadfast in your pursuit of finding other treatment options for your community, even if pharma is in the picture. Be willing to adjust with advancements and setbacks because one drug might not be the end-all answer for these children.
Why people like working for you: I’m solution-oriented. I’m results driven. I never accept “no” for an answer. When I commit to a project, I become emotionally invested. I am very passionate about my work.
Mentor: That’s a long list. Let’s start with Dr. Anthony Atala. He is now the director of the Wake Forest Institute for Regenerative Medicine. Dr. Alan Beggs, a world-renowned geneticist at Boston Children’s Hospital. Mostafa Analoui, who is currently executive director of UCONN Ventures. He was head of healthcare and life sciences at Livingston Securities. He’s been very instrumental in approaching pharma. Barry Byrne, of the University of Florida. He is the director of the Powell Gene Therapy Center.
On the Job
What inspires you: The biggest inspiration in my life has been my son. He taught me what true bravery and courage means. He valiantly faced each day with perseverance. He had an unexplainable will to live. He was quick-witted. He never asked, “why.” In each adverse event, in life-and-death situations, he always had something profound to say. It was mind boggling. His dream was to find a cure for this tragic disorder. He was studying biotechnology and planned on working for the Wake Forest Institute for Regenerative Medicine. He passed on Christmas Eve, 2010, 40 days shy of his sixteenth birthday. I am fulfilling his dream to find a cure for this disorder. I will carry the torch until this dream is realized. My inspiration today comes from his peers. These children are precious. I have hundreds of other MTM children who inspire me. I have a beautiful, extended family. The other inspiration in my life was my mom. She would speak words of encouragement through the darkest of times. She would say, “Honey, this is the hardest battle you will ever face, but you have to get back up to fight for your son.” Those words have played over and over in my mind for two decades.
What makes you hopeful: The data from our preclinical. It’s been so robust. Our scientists say they’ve never seen data like this. Our hope is that it translates to these children. We’ve entered the clinic and we are anticipating the same outcome in our children. That’s what gives me hope.
Best organization decision: The initial structure and the scientific team of the foundation’s beginning work. The collaborative mindset our scientists had, and their altruistic motivation put us where we are today. I have seen it is not the same for other indications where there’s a willingness to collaborate arm-in-arm.
Hardest lesson learned: The protection of the foundation and institutions behind the science when dealing with VC. Our goal from day one was to protect our scientists and foundation. Should there be a windfall, we would be able to be part of that windfall. This would allow us to go on to other indications and be able to save more lives. We made the mistake of trusting VC without fully protecting our science and foundation. We gave them everything—15 years of work tied in a nice bow. That’s been a tough lesson.
Toughest organization decision: We were 14 years into pushing this guarded dream. We were heavily into stem cell and gene therapy—regenerative medicine research. Which do we move into the animal model? It was a team decision.
Biggest missed opportunity: Goes back to the hardest lesson learned—not having the right legal counsel in place for the translational medicine component.
Like best about the job: I love getting to know these families and children from all over the world—all from different walks of life.
Like least about the job: The loss of these precious children. Receiving those calls. I’ve had four phone calls in the last three months. We’ve lost nine children this year. Walking parents through such tremendous loss is the most devastating piece of this whole journey.
Pet peeve: Lack of follow through.
First choice for a new career: I would seriously consider a professional patient advocacy position in the Life Sciences. I’d like to share my knowledge learned from 22 years of successful engagement of patient communities. The first journey was so impactful; it would have to be something where I knew I could make a change.
Most influential book: The Bible. For me, to talk about this journey, you can’t separate the spiritual aspect in rare disease.
Favorite movie: Lorenzo’s Oil. The first time I watched it was two weeks before my son was born. So many times during this journey I’ve gone back to that movie, when I was at a crossroad. The movie had a deep impact on my journey.
Favorite music: Classical, but I’m an old-time rock and roll girl.
Favorite food: Italian. It’s in my DNA
Guilty pleasure: A great bottle of French wine.
Favorite way to spend free time: Yachting, skiing, or vacationing somewhere in the mountains.