Rare Leader: Cher Bork, Executive Director, Alagille Syndrome Alliance

The Basics
Name: Cher Bork

Title: Executive director

Organization: Alagille Syndrome Alliance

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Disease focus: Alagille syndrome is a rare disease, an autosomal dominant, genetic disorder associated with liver, heart, eye and skeletal abnormalities, facial features and more. People with the condition may have any combination or severity of these symptoms, making it a difficult rare disease to diagnose and treat.

Headquarters: Portland, Oregon

How did you become involved in rare disease: In 2010, after my second child was born, I was diagnosed with a rare cancer. That journey launched my desire to move from nonprofit volunteering to a dedicated career helping others with medical challenges.

Previous career: Casting director for film, tv and print

Education: Christian Brothers University, Memphis, Tennessee

The Organization
Organization’s mandate: The Alagille syndrome Alliance (ALGSA) is a nonprofit organization working to find a cure for Alagille syndrome (ALGS), a rare genetic disease that affects an estimated 10,000 individuals worldwide. We work closely with scientists, researchers, clinicians, pharmaceutical companies, and other industry partners to cultivate interest in ALGS discovery and encourage the development of better treatments. We connect with ALGS families offering financial, educational and other support while involving them in engagement with stakeholders to keep the patient voice at the forefront of all we do. We are committed to collaboration with all stakeholders from scientists to clinicians to families to solve the mysteries of ALGS, leading to new studies and treatments and improving those currently in existence. Central to everything we do, our mission inspires us to strive for excellence in all facets of our work within the ALGSA and in the wider world.

Organization’s strategy: We let the patient voice be the driving force behind all we do—collecting experiences, concerns and data and turning that information around in a useful way to bridge knowledge gaps with clinicians and researchers. Communication between Alagille families and scientists is critical, and we provide opportunities for knowledge sharing with all of our stakeholders. The complexity and variability of Alagille syndrome is misunderstood, and the Alagille Warriors themselves can open the doors to new information about daily life and the nuances of the syndrome. We strive to increase our funding so we are able to support scientific research while also meeting the needs of our families.

Funding strategy: We aim for a variety of revenue streams from individuals, grants, corporate support and other fundraising campaigns, but many rare disease families are struggling financially and find it difficult to give on a regular basis. Our industry partners understand those hardships, and those partnerships with biotech/pharma are the backbone of our organizational funding. We rely on grants for larger programs and capacity building initiatives. We truly exist because of a culmination of corporate support, individual donations, and grants.

What’s changing at your organization in the next year: This is an incredible time of growth for the organization. We have launched an international ambassador program with working groups in several countries. This program will aid us in having a comprehensive focus on the patient voice. We are also in the midst of a focus on ALGS adults. Historically, Alagille syndrome research and care has been focused on pediatric needs. Our ALGS adults have many different challenges and need a standard of care that includes their growing concerns with pain, nutrition, fertility, and reproduction. Through our surveys and our support groups, we have been able to pull together strong data about life-limiting bone pain in Alagille syndrome, and we are working toward new research in that area. More succinct disease education is a priority for us especially in the areas of fertility, pain and genetics. We will also likely see the first FDA approved drug to treat the debilitating symptom of itch within the year.

Management Style
Management philosophy: Collaborations are key. One of the most impactful things we have done is a roundtable meeting where all of our stakeholders were represented at each working group table. Providing those opportunities for families to sit at the table with heads of pharma companies, leading scientists and ALGS experts is so important to us. When we look at the things that need to be accomplished, we try to stay focused on the tools and resources that will help us accomplish our goals. That includes building great relationships with mentors and stakeholders, turning the patient needs into actionable, measurable programs, fundraising to support programs and capacity building initiatives for our Board of Directors, and keeping an answer driven focus on what’s at stake for our families.

Guiding principles for running an effective organization: Our philosophy revolves around that multi-stakeholder collaboration. Listening to patient needs and the needs of researchers, offering support to both, and continually providing ways for stakeholders to interact. Interactions include providing platforms for conversations, roundtable discussions and committing to being adaptable to meet the ever-changing needs of the community – families, science and care. We are not afraid to ask the tough questions, dig until we find answers, accept help when we need it, and offer help when someone else is in need.

Best way to keep your organization relevant: The complexity and variability of Alagille syndrome comes in to play again here. We adjust to meet the needs of families/scientists/care teams by consistently measuring their need. We utilize needs assessment tools and surveys to ensure we always provide the support the stakeholders actually need. As mental health concerns become more prevalent in our community, we are adding programs to support our families like never before. We focus on the most relevant and day to day impactful symptoms and hardships, but we are not scared to adapt and change. Having strong relationships with our families, our experts and our scientists gives us the opportunity to be a hub of information and a trusted place to turn. 

Why people like working with you: Our community is hurting and struggling. The family community as well as the greater science community needs compassionate leaders who can relate, who understand the need for collaboration, who can take an idea and turn it into a measurable program, who are open to mentoring and being mentored and who consistently show dedication. To know the ALGSA today is to feel all of those things. I think people quickly see our incredible progress thanks to family engagement and dedicated partners. People want to be a part of something that creates impactful change for those who are hurting.

Mentor: There are so many people in the rare disease space I have turned to and continue to turn to for knowledge and ideas, advice and best practices. From other nonprofit leaders to leaders in the pharmaceutical industry to our families themselves. There is always someone in the rare disease space to offer mentorship! It’s an incredibly giving community.

On the Job
What inspires you: Every one of our Alagille families inspires me to keep pushing forward. I celebrate with them, and I mourn with them. The community is like an extended family, and you would do anything for your family. They are the reason we are here doing this work. One of our Alagille kiddos sent me a painting recently, and I keep that hanging right above my desk as a constant reminder of those we are helping.

What makes you hopeful: As our relationships deepen with researchers and scientists, we see the work being done and the new discoveries in Alagille syndrome. The science behind both treatment of symptoms as well as the root cause of the disease gives me hope of a day without suffering in the future.

Best organization decision: In the past we were not as focused on supporting the science of Alagille syndrome but rather aimed our attention more on just being there for families. Shifting our focus has opened up so many doors to the organization. We have been able to offer support to researchers that in turn has allowed us to implement truly impactful family programs.

Hardest lesson learned: I don’t think it’s Alagille specific but rather rare disease in general, but there is a continual feeling we are not doing enough because Alagille Warriors are still suffering and still dying. It’s frustrating and exhausting to work so diligently on the mission but still face loss in the community. The daily lesson is not to let it stop or trip us up. We have to keep going forward. Our motto is “stay strong, press on,” and that’s appropriate not just for our families but also for those of us behind the scenes.

Toughest organization decision: The best decision was also the toughest. Our organization has always “been there” for our families, but we are small. Devoting so much time to being there was hindering us from progress in other areas. We had to find a balance, but it wasn’t easy. Alagille families are our top priority, but we had to make the tough decision to devote more time to implementing truly impactful programs (like financial assistance) and more time spent supporting science initiatives. At the end of the day, what our families need are better treatments and we want to help make that happen for them.

Biggest missed opportunity: No question our biggest missed opportunity was not receiving the Chan Zuckerberg Rare as One Grant. We made it to the final round and learned an incredible amount from the application process alone. We cannot imagine the knowledge that program would have brought us as rare disease nonprofit leaders, teaching us valuable lessons in working more efficiently and effectively to bring better treatments more quickly to the community. We are grateful the model of CZI is one of collaboration and knowledge-sharing so we can still benefit from what others are learning.

Like best about the job: When a newly diagnosed family turns to us, they are scared and overwhelmed. To be a trusted source of information and someone they can confide in is so rewarding. I love getting to know our families and spending time with them.

Like least about the job: Watching a family endure the ultimate pain and pushing forward when you just want to grieve along with them.

Pet peeve: The ticking of the clock. We feel there is not enough time to get things done, to find treatments fast enough, to help uncover the answers before tragedy strikes our community again. Time is not always on our side in rare disease. 

First choice for a new career: Complete truth – I am doing exactly what I want to be doing! I feel so fortunate for the path leading from my rare disease to Alagille syndrome advocacy.

Personal Taste
Most influential book: Most recently David Fajgenbaum’s Chasing My Cure: A Doctor’s Race to Turn Hope into Action. I had the opportunity to meet David. Learning how he approaches the research needs of Castleman opened my eyes to new ways of supporting our scientists. He is both influential and inspirational to anyone in rare disease. His book was so moving for me, and I guess it showed! My son chose the book for his 7th grade biography report.

Favorite movie: My husband and I often have family movie nights with our 10- and 13-year-old kids. We love any movies that keep us on the edge of our seat while making us laugh at the same time!

Favorite music: My playlist is very eclectic. It ranges from Dean Martin to rock to country. I like any music that evokes memories from certain times in my life.

Favorite food: As a life-long vegetarian, I love the Mediterranean diet’s focus on healthy eating.

Guilty pleasure: A glass of red wine around the fire pit. Oh, and dark chocolate covered espresso beans.

Favorite way to spend free time: Family game nights, family movie nights, family geocaching, and family reunions. I love the traditions as well as making new memories with family.