Name: Marianne Clancy
Title: Executive Director
Organization: Cure HHT
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Hereditary Hemorrhagic Telangiectasia (HHT), also known as Osler Weber Rendu Syndrome is a genetic blood vessel disease that creates a direct connection between arteries and veins without intervening capillaries. It occurs in 1 in 5,000 people and is autosomal dominant. There are 70,000 Americans affected and 1.4 million people worldwide. It is more common than hemophilia, cystic fibrosis and ALS yet is highly unknown. There is a 27 year lag from symptoms to diagnosis. It is often misdiagnosed for years, which is why we call it “The Great Masquerader.” These direct connections between arteries are known as arteriovenous venous malformations (AVMs). They are most commonly found in the brain, lungs, and liver. The AVMs can spontaneously hemorrhage in the lung and brain and lead to sudden death or stroke in children and adults. Liver AVMs lead to heart failure. About half of people with AVMs are anemic. Chronic bleeding from the nose and gastrointestinal organs lead to iron infusion and blood transfusion dependence. There are no FDA approved therapies for the condition.
Headquarters: Monkton, Maryland
How did you become involved in rare disease: My family has suffered as I lost a sister at the age of 14 suddenly and my mother died from complications from HHT when I was just 21. My brother had part of his lung out when he was 17 and that is when we heard of this disease. After seeing well-known physicians in Boston, Washington, D.C., and Dallas, I was told I did not have this rare disease. I was diagnosed after having my third child and was shocked. My physician and a small group started this foundation in 1991 and asked me to join the board of directors. I became a volunteer president for one term, and then they asked me to become the executive director and grow the organization. I was the first paid employee back in 2001.
Previous career: Manager of professional education and research programs at Oral-B Laboratories. I also had a pharmaceutical consulting business focusing on transdermal and transoral drug delivery. Additionally, I worked in academia as a dental and dental hygiene adjunct faculty member at Forsyth Dental Center, Boston University, and Tufts University Schools of Dentistry.
Education: Certificate in dental hygiene from University of Pennsylvania, B.S. in health administration from University of Pittsburgh, Masters in Public Administration from Northeastern University
Organization’s mission: The mission of the HHT Foundation International is to find a cure for HHT while saving the lives and improving the well-being of individuals and families affected by HHT.
Organization’s strategy: We fund research to find better treatments and a cure. We educate families and physicians about HHT so that awareness of crucial diagnosis and available treatments prevents needless disability and death. We provide linkages between people affected by HHT. And we collaborate with multidisciplinary HHT Treatment Centers worldwide while advocating for patient access to these Centers. In addition to that, we advocate for and support those with HHT while increasing public, private, and governmental awareness of the disorder. Finally, we engage the scientific and medical community so that talented individuals dedicate efforts toward advances in HHT screening, diagnosis, treatment, and research.
Funding strategy: Our organizational funding has primarily come from patient donations. The board of directors and a small group of generous donors have made HHT a top funding priority, which has led to breakthroughs unheard of just a few years ago. We do not receive any funding from pharmaceutical or medical device companies for research. We hope to grow partnerships with biopharma and device companies as physicians are treating patients with therapies manufactured by these corporations. We fund the best science anywhere in the world that will have the most impact. Additionally, we do not have funding going to high overhead as we want precious patient donations going to the research as opposed to university overhead. Our mechanistic work has resulted in novel partnerships creating groundbreaking discoveries. Our work is all about collaboration among HHT Centers of Excellence and bringing basic science and the patient voice into the priorities and development of proposal strategies. Cure HHT has funded the international treatment guidelines that are evidence-based. These guidelines are critical for diagnosis, treatment, and management of HHT for all families. They set the standard of care.
What’s changing at your organization in the next year: We are a small but mighty organization with great aspirations. We are excited about 2020 and this new decade. Our focus will be on building capacity to grow our research and clinical network within North America and globally. New Centers of Excellence will be launching and incorporated into our community. HHT families everywhere deserve expert care and treatment near their homes. Our staff consists of five full-time employees, one medical director, and three part-time employees. We all wear many hats and rely on our volunteers and board members to assist in initiatives. The board is a working board. We are currently recruiting for a manager of development and a technical program manager.
Management philosophy: I have been fortunate to have had a career in academia, business, pharma, and non-profit management. My style is as a visionary; I am an optimist and embrace change. I believe in collaborative work and am a consensus builder. Coaching employees to see their potential and then getting out of the way allows people to take ownership of achievements. Positivity is so important. My philosophy is to hire intelligent people who strive for excellence. Experience in an area is important; however, some skills cannot be taught. One can be trained in an area—having core values and teamwork are critical to keeping an organization growing. The Board sets the overall organizational goals and priorities. We set the strategies and together ensure the goals align with the strategies. Employees are empowered to utilize their own creativity to achieve results. Working in teams is encouraged and utilization of technology streamlines tasks and increases efficiencies. Goals and plans are reviewed weekly through staff meetings and all employees understand what help is needed on specific projects. We monitor and adjust as needed. Flexibility is important. We work until the job is complete and we are overachievers.
Guiding principles for running an effective organization: Clear communication is paramount. Our organization has a set of values that includes a high degree of ethics, responsiveness, compassion, results-oriented, patient and family focus, and collaboration. We ensure everyone is treated with respect and are transparent with all of our activities. Empowering patients to become their own advocates and to ask questions is crucial to moving the care and treatment paradigm forward. Living with anemia and transfusions is not okay and we want to ensure our patient community they can increase their quality of life. We believe that diverse groups are better problem-solvers than homogenous groups. We embrace the patient voice into our scientific and patient and physician conferences. The personal stories have a profound impact on scientists and physicians who attend. Patients ask leading and provocative questions and can move conversations from the esoteric science into real problems that need solving.
Best way to keep your organization relevant: The most important way to keep our organization relevant is to listen. Our community struggles through years of misdiagnosis: HHT symptoms are silent; the AVM’s are in internal organs; we need to know if patients are experiencing migraines, shortness of breath, long standing anemia and low iron. We go to where our patient community is whether it be social media, through email, or patient forums. It is amazing to me that in 20 years, there are still 800-1000 people per year who contact our organization because they either self-diagnose, experience a life-altering event in the family, or are referred to us by a physician. The number of undiagnosed people in our population is unacceptable. Getting the message out to the public through sharing of stories to the local and national media, educating physicians at their professional meetings, and increasing awareness is so important. There are 7,000 rare disesases. HHT is one that is under the radar and is a “common” rare disease.
Why do people like working with you: We are passionate, committed, and willing to think outside of the box. Our patients, physicians, and scientific community collaborate and we leave the egos at the door. Our mission is to make the most impact with limited resources. We lead and continue to advocate in Congress, NIH, FDA, CDC and hospital administrations. We set strategies and are not afraid to push through boundaries. We keep our promises and are an open organization. We want everyone to have a voice and are willing to take risks and incorporate new ideas wherever they arise.
Mentor: I have been fortunate to have several mentors in my life. In academics, I had a boss who was a world renowned dental researcher, Ralph Lobene. He gave me the freedom to push boundaries and lead a department at the age of 23. At Oral-B Laboratories, I had a mentor who was a champion for my team, Bob Perry. He taught me the importance of business strategy and to hold onto passion for doing what you believed in. At Cure HHT, Robert White, a physician who formed the Foundation with a small group of patients and brought me onto the board of directors, helped lead our organization in the early years through the mid-2000s. I have been fortunate to work with our first medical and scientific directors, Jim Gossage, Marie Faughnan, and Chris Hughes, who are my role models. I learned to push beyond limits and boundaries, and advocate for our priorities, and understand the importance of collaborations globally. Their passion and dedication to patient care motivates me to conquer this disease. Finally, it is the patient community—the newly diagnosed patient and their families, who have suffered loss needlessly like I have. Those who are living with this chronic illness and have lost jobs and their zest for life drive everything I do in leading this global organization to accelerate our research and advance therapies now.
On the Job
What inspires you: The resilience of our patient community and knowing what is possible to transform this disease inspires me. I have personally seen how a community of committed professionals, patients, and our organization can set the course forward and create breakthroughs. We have made genetic testing possible through our collaborations, set international standards through our guidelines, ensured our patients have access to expert care through growing our centers of excellence, and driven awareness through patients stepping forward and out of the shadows.
What makes you hopeful: This next decade will be transformational. I already see the pace of change with the advent of new therapies, and our first multi-center clinical trial funded by NIH will occur this year. Everything we have built has led us to this moment where personalized medicine is possible, as well as gene therapy.
Best organization decision: Forming a task force to get the patents for our genes released to make genetic testing possible. This was the first priority when I became executive director.
Hardest lesson learned: The pace of change is slower in working with a non-profit organization. I am driven and have come to understand that people are volunteers and have full-time responsibilities. It took some time for me to adjust from working at a for-profit organization to a non-profit with a reliance on volunteers. I had to learn, and I am still learning, how to manage my expectations.
Toughest organization decision: Recommending research funding for a few projects that were high risk high reward. The results provided the biggest impact for patients and have led to large investments in therapies for the future.
Biggest missed opportunity: Early in my career, I had several meetings with leaders in the U.S. Senate. They were in the minority party and the environment was not one of bi-partisanship. I should have been more assertive in communicating “the ask” and taking advantage of having a hearing on HHT with Congress. We need this disease to come out of the shadows and into the public conversation.
Like best about the job: Each day is different. Some days I work on medical and scientific research with the investigators. Some days I work with hospitals on the processes for bringing on new centers. Other days involve planning communications for our patient community, or working on patient conferences, or meeting with philanthropic donors to discuss their investment in the foundation and our projects. I also work on legislative advocacy with government agencies.
Like least about the job: We lack the number of people to accomplish everything we want to do. Our staff wears a lot of hats and we are hampered by the size of our organization.
Pet peeve: The phrase “we have always done it this way.” Insular thinking can inhibit progress.
First choice for a new career: Federal prosecutor—I love law and I am a crime mystery fanatic
Most influential book: Gift from the Sea by Anne Morrow Lindberg. I read it every year. It teaches about life and cycles in relationships and family. It also teaches me to have patience and faith.
Favorite movie: The Sound of Music—discover who you are and overcome your own inhibitions.
Favorite music: Bruce Springsteen, Eagles, and Joni Mitchell
Favorite food: Lobster (pizza is a close second)
Guilty pleasure: Relaxing for a full day into the sunset on a beach with a great book, white wine, and cheese
Favorite way to spend free time: Traveling to new places and hiking with my three golden retrievers