Rare Leader: Christine Waggoner, President and Co-Founder, Cure GM1 Foundation

The Basics
Name:   Christine Waggoner

Title: President and co-founder

Organization: Cure GM1 Foundation

Disease focus: GM1 gangliosidosis is an inherited lysosomal storage disorder that damages nerve cells in the brain and spinal cord. Researchers have classified this condition into three major types depending on the age the symptoms start appearing. These major types differ in severity; however, their features considerably overlap. Some researchers believe that because of the overlapping features of the disease, it represents a continuous spectrum instead of just three types. Type I, or the infantile form, becomes evident at the age of six months and is the most severe. Affected infants generally appear to be normal until their development slows down and muscles used for movement weaken. They eventually lose previously acquired skills and develop an eye abnormality called “cherry-red spot,” as well as having distinctive “coarse” facial features, enlarged gums, and an enlarged and weakened heart muscle. Affected children usually do not live past early childhood.

Headquarters: Albany, California.

How did you become involved in rare disease: I became involved the same way a lot of people do, because their child, or someone they love, was diagnosed with a rare disease. In this case, my daughter Iris has GM1. We decided we wanted to fundraise, and we set a modest goal. We initially didn’t have a nonprofit. The generosity of our friends exceeded our expectations beyond our belief and we set up a nonprofit to address the amazing support.

Previous career: Simulation supervisor/technical director at a computer animation studio

Education: B.A. in in visual art from Brown University.

The Organization
Organization’s mandate: Our organization’s mandate is to fund medical research and drug development for GM1 gangliosidosis and help contribute to bring treatments forward.

Organization’s strategy: Our strategy is to look for promising programs that will advance possible treatments. We’ve been particularly focused on gene therapy, but we’re open to multiple approaches. Our strategy has been a lot of social media and a lot of grassroots fundraising.

Funding strategy: Every little bit makes a difference, whether it is a big donation or a lot of little donations. We work to spread messages to as many people far and wide as possible because we believe that no one can disagree with the importance of the cause when children’s lives are at stake. It’s very non-controversial asking for treatments for dying children. It is something just about anyone can embrace. We want to put a face on the disease and let people know about the unmet need.

What’s changing at your organization in the next year: The next year we expect to reach an amazing milestone when we have our first gene therapy clinical trial. We hope there will be a future of multiple shots on goal for treating GM1 gangliosidosis.

Management Style
Management philosophy:  A little bit can make a big difference. We are extremely lean, and we have an extremely low overhead. Our philosophy has been to put as much as possible toward funding research.

Guiding principles for running an effective organization: Once people see that you have passion and you outline all the initiatives you are taking, people will become interested. It is leading by example. It is also just letting people know about the severity of the disease and the unmet need.

Best way to keep your organization relevant: We are constantly trying to form a broader community. What’s been amazing is that whenever we talk to biotech companies, they are impressed that we are in touch with more than 200 families. One of the pillars of all patient advocacy organizations, especially for an ultra-rare disease, is finding lots of patients. When you tell them, you are in touch with that many families for an ultra-rare disease, it’s impactful. Once you start forming that community, you can start working with that community to build up the organization.

Why people like working with you: I’m a really hard worker and super passionate. In this case, the disease is very well understood. It’s been around a long time. It is ultra-rare. There are great animal models. The thing that was missing was a community and funding. What’s amazing to me is the importance of advocacy and the importance of grassroots contributions to helping.

Mentor: I have lots of mentors. One of the most influential people who helped me is Ken Bihn, the former president and founder of the Cure Tay-Sachs Foundation. His daughter Dakota had juvenile Tay-Sachs, which is very similar to GM1. He has a lot of war stories and a lot of experiences to share that have helped. We were also fortunate to meet an adviser named Mark de Souza. He founded eleven-plus rare disease companies. We met him at the pool at the YMCA.

On the Job
What inspires you: The children inspire me. When you see this disease in particular primarily affects children. There are a few adult-onset patients, but it’s mostly a pediatric disease. Seeing how hard they fight and how cute they are and how important their lives are inspires me.

What makes you hopeful: It’s an extremely hopeful time in medicine right now with all the developments in genomics, and gene therapy, and gene editing. I’m hopeful that more rare diseases will be able to be addressed. Also, our donors make me hopeful. We’ve been extremely fortunate, but what I’ve learned is that if you tell a compelling story, there are so many people who are willing to help, even if they haven’t been impacted by the disease themselves. The support we’ve received has made us hopeful.

Best organization decision: Our best decision was to get the message out there—to not be afraid to go to conferences, travel, meet with people, and spread the message. It’s just so important. At the most basic level I thought that fundraising was the core. It is one of the big pillars and it is extremely important, but one of the most important things is just putting a voice out there. If there is no advocacy, no one out there, and no one asking—it’s extremely important for companies and researchers to know there is someone there on the other side.

Hardest lesson learned: One of the hardest lessons in this rare disease fight is that there comes a point because of the complexity of drug development that these therapies and the fight might not be for your own child. There’s a point at which it has to be for the community at large. That’s really hard as parent advocate. It’s a hard battle to move in the face of an extremely degenerative disease.

Toughest organization decision: One of the toughest decisions we’ve had is knowing our limits. We have been successful in fundraising, but it has not been enough for us to stand completely alone. While it might be attractive to think about spending every penny on a single program, one of the toughest decisions was to decide to diversify and mature as an organization and seed multiple programs versus putting all of our eggs into one basket.

Biggest missed opportunity: We are struggling with the balance of keeping this as an all-volunteer organization versus hiring and growing. That’s something that we’re thinking about a lot and that our board discusses and will need to decide.

Like best about the job: What I like best about working with Cure GM1 is that there’s no question about the importance of the social mission and helping advance treatments for such a severe disease. It feels like that mission is so incredibly meaningful. Everyday you know that work is incredibly so important.

Like least about the job: Knowing that not everyone will be able to benefit immediately from clinical trials. There will be patients who don’t meet the criteria, or whose disease has progressed too far. I wish there was a safe and efficacious way for everyone to have a chance.

Pet peeve: My pet peeve is naysayers, or people who think they can’t make a difference. People who think that donating $5 is not worthwhile. The more people who do a little can amount to a lot. I much prefer when there are people who might be willing to try, even if they don’t regard themselves as extremely wealthy people. You don’t have to be fabulously rich to advance a cause.

First choice for a new career: There was a time where I was in high school when I was really interested in medicine. I never knew this whole rare disease fight would be my future. I love to be hands-on. There’s a part of me that wishes I had pursued medical school

Personal Taste
Most influential book: The Cure, by Geeta Anand, about John Crowley’s experience as a patient advocate and his pursuit of a cure for his children.

Favorite movie: Ratatouille

Favorite music: U2

Favorite food: Sushi

Guilty pleasure: Romantic comedies

Favorite way to spend free time: With my family

December 28, 2018