Rare Leader: Sue Kahn, Executive Director, National Tay-Sachs & Allied Diseases Association

The Basics
Name: Sue Kahn

Title: Executive director

Organization: NTSAD – National Tay-Sachs & Allied Diseases Association

Disease focus: The organization works in a number of lysosomal storage disorders and leukodystrophies including Tay-Sachs, GM1, Canavan, and Sandhoff. All are caused by genetic mutations and are progressive, neurological disorders caused by an enzyme deficiency.  All the diseases are fatal in children and progressively debilitating in adults.

Headquarters: Boston

How did you become involved in rare disease: I became involved when I was working in business development at Genzyme Genetics, which provided genetic testing services to physicians.  I was based in Europe for a year where I met colleagues in the therapeutics side of Genzyme, which led me to connections with NTSAD.  In parallel, I had been doing volunteer consulting work for non-profits through Community Consulting Teams and decided I wanted to transition my career to the nonprofit word.  At that time, I was fortunate that NTSAD was seeking a new Executive Director.  On a personal note, I also have a second cousin who had a child a number of years ago affected with Tay-Sachs, but it was a different time and no one in the family talked about it. Unfortunately, I never met their son before he passed away.

Previous career: I worked in business development and finance positions for Genzyme Genetics and a medical diagnostics company.

Education: Bachelor’s degree in applied math and economics from Brown University, MBA from the Tuck School of Business at Dartmouth College.

The Organization
Organization’s mandate: Our mission is to lead the fight to treat and cure Tay-Sachs, Canavan, Sandhoff, and GM1, and support affected families and individuals in leading fuller lives.

Organization’s strategy: We have goals in each of our focus areas—family services, research, and education. In family services, our goal is to provide comprehensive services to affected families and individuals. We do that in a variety of ways, including an annual family conference. The goal of that is to empower people, engage them, and provide some support and healing. In order to support families, we cultivate a strong family network. They learn a lot from each other. The key thing is not to be isolated. We also have dedicated staff for our family services programs.  In research, our goal is to direct, fund, and promote research to develop treatments and cures. Right now, all of our diseases are fatal in children and progressively debilitating in adults. There are no disease-modifying treatments. We look to fund research to see if there are programs where our relatively small dollars can make a difference in advancing it. We are also looking to fund areas that are unique to NTSAD and where we can involve families, such as gathering fundamental information that is critical for getting to clinical trials, funding natural history studies, or engaging families in registries. The third area is around education because our legacy is around carrier screening in the Ashkenazi Jewish community. We still want to help get the word out about prevention and screening to our families and anyone who could benefit from seeing a genetic counselor to learn about their risks.

Funding strategy: It’s diverse. NTSAD is over 60 years old, so we have a broad supporter base. As is typical, 85 percent of our funding is from the generosity of individuals, including family foundations. The balance is from corporations and foundations. We have our annual appeal outreach plus our annual “Imagine & Believe” event here in Boston (which was held just and was a big success). In addition, we hold our annual Day of Hope when our families hold fundraisers primarily for research and raise awareness in their communities. The events can be anything from a lemonade stand to having a walk. We have restricted family funds if someone wants to start a fund in memory or in honor of someone. We will soon be launching a planned giving program. People who have a long history with NTSAD often want to remember a loved one and some have approached us recently about giving through their estate plans or retirement plans.

What’s changing at your organization in the next year: There are several possibilities for clinical trials in the coming year for the first time ever, which is beyond exciting. Some of our longtime families never believed there could possibly be treatments in their lifetime.  There are now a few companies either working in our diseases or related diseases where they are hoping to leverage their therapies for one of our diseases. Assuming that clinical trials come to fruition, it will change the role of Diana, our family services director, who will be communicating the relevant information to our families. Naturally, there’s a lot of pent-up demand. Once we have news to share, we will have to figure out how to best communicate it so that we can continue to be seen in the role of trusted communicators involving trusted experts.

Management Style
Management philosophy: It’s important to have good working relationships with everybody that I’m connected to. Along with a small staff, I work closely with a large team of volunteers: the board of directors, the scientific advisory committee, and a corporate advisory council.  With a small staff, each person plays a key role and also has opportunities to play multiple roles, which includes the need to be flexible.  I try to make sure that we’re all informed and working closely as a team. I’m supportive, so people can learn and grow, and at the same time get their work done. It’s always a balance.

Guiding principles for running an effective organization: For this organization, the families are at the center of what we do. If we think about any of the elements of our work, we need to understand how it benefits the families. They are really our key constituents. I think the spirit of cooperation and having people feel valued are essential.

Best way to keep your organization relevant: It’s both building on the legacy of NTSAD, as well as looking forward to what’s changing and how we can address families’ needs. NTSAD has a long history as a trusted brand. It takes a long time to build that trust. It’s essential to be relevant so we can maintain those trusted relationships. Then we can find out what’s valued, make plans to act on what’s important and what the families need. Going forward, as there are going to be clinical trials and potential treatments, how does NTSAD’s role change and how can NTSAD play a meaningful role? As part of our upcoming strategic planning process, we’ll be reaching out to all our constituents—families, physicians, scientists, industry and labs – to understand what they’re thinking.

Why people like working with you: I’m encouraging, supportive, and communicate what’s going on. My staff knows what I expect, and we share priorities so that we can work happily and effectively as a team. We are in it together.

Mentor: When I look back, some of the former bosses I had were mentors. I think that I learned from every one of the NTSAD board chairs I’ve worked with—and I’ve worked with six—and they learned from me.

On the Job
What inspires you: When I see the ways that families in our community support each other and do the big things and small things that can help each other out, it can be very inspiring.  Also, because of my background in biotech business development, I look for opportunities to help move along NTSAD’s research program.  I might do that by connecting people who haven’t previously met, but who could benefit from knowing each other, e.g., our scientists, companies and / or clinicians.  I’ve been fortunate to involve some people in NTSAD from my past corporate life, which has led to opportunities to work with consultants and other experts to inform our understanding of research opportunities.

What makes you hopeful: When I see how dedicated the people are who we work with—our families that are passionate about making things happens, or doctors or scientists, or donors who are really committed and believe in change and believe things are possible—that creates hope.

Best organization decision: We launched a corporate advisory council to complement the work of our scientific advisory committee, and to involve experts in bringing therapies to market as we move closer to clinical trials. This group of people has opened up my eyes to NTSAD’s potential and new ways of thinking about our approach to research.

Hardest lesson learned: It’s a lot harder to partner with related disease organizations than I would have thought, having come from a business development background. It’s very different in business than in patient advocacy. It’s more personal. It’s more cultural. Even though in the long run we have the same goals, I don’t think people always see it that way.

Toughest organization decision: One of the challenges is that we have a pretty broad scope (i.e., family support, research) and multiple diseases, which can be different. It’s great that we have such a big scope, but it’s a challenge to prioritize. I think we’ll get some better clarity because we will be working on our strategy.

Biggest missed opportunity: One of the things I think about is having a chief scientific officer. One year I had a science communications intern as an experiment. The question is, could we take a step up in our ability to impact research if we had someone on staff who was both knowledgeable about the science as well as experienced in drug development?

Like best about the job: I like working with committed families and scientists. I like the diversity of things that I need to do, but sometimes there’s also the challenge of what to do first. I like working together as a team. I draw on my previous corporate expertise and contacts to work on research and strategy. I enjoy pulling those skills out of my toolkit and being able to use them in this role. Hopefully the work we are doing helps a family and makes a difference in the quality of life of the family because of the connections, support, or education they have gained through NTSAD.

Like least about the job: The flipside is that there is so much to do. For a small organization, there are a lot of administrative pieces—the necessary but not enjoyable things.

Pet peeve: People who are negative; lack of communication.

First choice for a new career: This goes back to when I was a kid.  I wanted to be in the Tommy Bartlett water ski show in the Wisconsin Dells.  It was probably never realistic, but always fun to think about.

Personal Taste
Most influential book: Just Mercy by Bryan Stevenson. I read it over the summer, and I keep thinking about it. There’s too much injustice in our world; we must do better.

Favorite movie: To Kill a Mockingbird

Favorite music: Aaron Copland’s Fanfare for the Common Man. Even though I was a woodwind player, I love the brass instruments.

Favorite food: Mediterranean

Guilty pleasure: Ice cream—ginger molasses.

Favorite way to spend free time: On the hiking trails at Acadia National Park in Maine

November 15, 2018