Rare Leader: Emily Milligan, Executive Director, Barth Syndrome Foundation

The Basics
Name: Emily Milligan

Title: Executive Director

Organization: Barth Syndrome Foundation

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Disease focus: Barth syndrome is a rare, life-threatening, genetic mitochondrial disorder primarily affecting boys.  Our Barth boys and young men may suffer from heart failure, muscle weakness, and infection (caused by very low white blood cell counts, or neutropenia). Typically the individuals who have Barth syndrome are “short and skinny”, as the disease is characterized by growth delay, impaired lipid metabolism, and cardiolipin deficiency. Most people with Barth syndrome also suffer from persistent and extreme fatigue. In some individuals affected by Barth syndrome, the symptoms can be very severe, sometimes resulting in heart transplant, potentially lethal infections, and even death. Barth syndrome is X-linked, usually transmitted from mother to son. A mother who is a carrier of a Barth syndrome mutation passes on a defect in the tafazzin gene—also called TAZ or G4.5. Mothers do not have any signs or symptoms of Barth syndrome and are often unaware that they are carriers. There is a 50 percent chance that a boy born to a female carrier will have Barth syndrome, whereas girls born to a carrier have a 50 percent risk of being carriers themselves. Sadly, the death rate for young children is very, very high. Some 85 percent of boys with Barth syndrome will die before their fifth birthday.

Headquarters: Larchmont, New York

How did you become involved in rare disease: I was head of global research operations of JDRF, which is the leading global funder of research for type 1 diabetes. It was a big remit overseeing the finances and research operations of a $500 million portfolio. In 2017, I moved to Boston where I started the JDRF T1D fund, a venture philanthropy fund dedicated to catalyzing commercial investments in early-stage companies developing therapies in type I diabetes. After the fund was staffed and we closed several key investments, my job was done and I was bored. I decided to seek a new challenge. At that time, I was approached by a headhunter to explore an opportunity in Barth syndrome. Naturally, it was the first time I had heard of it. But the formula was simple. I have boys, and it’s an X-linked mutation that primarily affects young men and boys. The search committee was looking for a fun mom or dad who could connect with the families as well as understand R&D and how to run a nonprofit. The stars aligned. They picked me to lead BSF, and I began my career in rare disease.

Previous career: Research and operations in academia and nonprofits

Education: Bachelor’s degree in international relations from the University of Florida and a Master’s in Public Health from Columbia University

The Organization
Organization’s mission: Our mission is to save lives through education, accelerating treatments, and finding a cure for Barth syndrome. We do that in three distinct ways. First, we fund research by seeding innovative ideas. We also help families in crisis navigate the healthcare system through our family services program. The third pillar of our mission is advocacy, which is includes working with regulators and advocating in a nonpartisan way for therapies or policies that could improve quality of care for our families.

Organization’s strategy: The strategy is multipronged. It follows the three pillars of our mission–advocacy, family services, and research. With research we’ve seen a big shift. I commend Matthew Toth, who is the director of science for BSF, for catalyzing our discovery program that has led to more than 100 publications relating to Barth syndrome. Our historical strategy has been to seed novel ideas and innovative approaches that could advance potential therapies for Barth syndrome. For family services, it’s building community. We do that through many different ways so that families do not feel alone no matter where they live. Frequently our families may be the only diagnosed family in their city, state, or country. It’s a global union of families that would otherwise never have been brought together.

Funding strategy: Our foundation is going through a bit of a shift. When I joined, BSF was primarily supported by the philanthropy of affected families and people within their social networks. We want our families to continue to contribute, but as you can imagine, it’s difficult to ask families already burdened with high medical expenses and the challenges of caring for children who are very sick to support research in any meaningful capacity. Clinical trials can cost millions of dollars and are a necessary step in any R&D program towards a path to market access. We are continuing to lean on families, but we also want to explore strategic collaborations with pharma and biotech. We’ve seen a shift over the last year and we’ll see that shift continue going forward.

What’s changing at your organization in the next year: We are going to be hire a new director of science. That’s a big change. Matthew Toth has been with the organization for 13 years. Kudos to Matt for the success of building the research program we have today. We’re sad to see Matt go and need to fill a big gap that his exit will create. But in rare diseases, we learn to turn lemons to lemonade and we’re excited to have a new team member join us. From a strategic side, we’d like to see a launch in a gene therapy trial next year. That can’t come fast enough for our families. The technology and advancements that are coming out almost on a daily basis are changing the landscape of doing R&D in rare disease. It’s our job to matchmake and propel potential new therapies by translating preclinical science into human models.

Management Style
Management philosophy: Surround yourself with good people—capable, ethical, principled, talented, committed, and compassionate people—and empower them to do the best job possible. I abhor micro-management. I like self-starters and collaboration. When we share and bounce ideas off each other, we get stuff done.

Guiding principles for running an effective organization: Do the right thing for the right reason and you will sleep well at night. You have to always follow your principles in everything you do. Running an effective organization is sometimes tough. You can’t personalize things. You need to be mission-centric and sometimes make difficult decisions. If you do it for the right reasons, I find it will work out.

Best way to keep your organization relevant: Be a contributor to the landscape. There’s an element of reciprocity. Go to conferences. Share with others what you’ve learned. Don’t just absorb what others have to tell you. You need to be at the table. Exchange ideas and be open and receptive to input from others.

Why people like working with you: I get the best out of them possible. They like working with me, but that comes after they hate working with me. I push them. I see the talent. It may be uncomfortable for them to explore new areas they may not feel confident about, but I have confidence in them, and I will teach them how to do it. I give them feedback and I celebrate them. I am not a so-and-so person did this. I am a WE-did-this person. It’s a group effort and a group success. I make people great and, subsequently, the organizations we work for, exceptional.

Mentor: I don’t have one mentor. I have thousands of mentors. I believe each person I encounter I can learn from. I don’t care if it is the intern who is here for six weeks, or my chief medical officer. There’s something you can learn from everybody. You just have to be open to what that is and appreciate it when it crosses your path.

On the Job
What inspires you: The reason I get up every morning is that I want my kids to be proud of what I do. I want them to be proud that I am helping families. My children drive me, the children I work for motivate me, and BSF’s families inspire me.

What makes you hopeful: Hope is both rational and emotional. Rationally, I’m hopeful the advancements I referenced earlier in technology are unprecedented. The cohesion across rare disease groups is unprecedented, and it’s only going to grow. I see coalitions and stronger partnerships forming and that gives me hope – wherever there is collaboration, there is opportunity. But I also see technological advancements on the horizon that could bring therapeutic hope to people. On the emotional side, I just had a mom in my group whose son is under two years of age and just had a heart transplant. This mom is so honest and sincere and giving of her thoughts—her hope for her son—with the community during the direst of circumstances. Her hope is contagious. It is my oxygen. It is why I do what I do for BSF.

Best organization decision: It is healing division and finding commonality, bridging divides to unify people around a common goal. Whether it’s working on contracts, collaboration, creating community, connecting the internal team, it’s about creating space and dialogue. Take the time to talk and heal.

Hardest lesson learned: If it’s not in writing and signed, it means diddly-squat. Don’t trust people at face value. This is not on a relationship level, but on a business level. When doing deals, everything must be in writing. Signatures. Don’t settle for good faith.

Toughest organization decision: Letting go of good people when the job no longer exists in the organization. It could be there is a restructuring, or that we eliminated a group because of budget constraints, and you need to consolidate resources. It’s really tough to have to let people go, especially people who have been high contributors to an organization, but the organizational needs have changed. No matter what, though, take care of and be fair to good people – this core value is paramount.

Biggest missed opportunity: There was a time when I was promoted to lead a new division. I didn’t feel I had the experience or knowledge to lead the team, and I subsequently didn’t do it to the extent of my ability. I feel I let my staff down. I have learned since then that it’s okay to have self-doubt. That usually means you have to dive in, figure it out, have humility, be open to others’ input. You don’t need to have all the answers. At that stage of my career, I thought if you were the chosen leader, you needed to have all the answers. I have since changed my philosophy on that metric. What you need is vision.

Like best about the job: I like being able to solve a lot of different problems every day, although I’d like to not have so many problems to solve. There’s a degree of autonomy and decision-making authority to get stuff done every single day. I don’t have to be pigeon-holed into a smaller mindset. I can explore the boundaries of what is possible in my job. I can be opportunistic where others may not have considered possibilities. To draw on the analogies of astronomy, I like that my board empowers me to constantly question the boundaries of what we know to find a new universe of hope and potential.

Like least about the job: My ambitions are not matched by resources. I need to fix the resource problem, not scale back my ambitions.  

Pet peeve: I can’t stand a robotic mindset. People who say, “That’s the way we’ve always done it,” don’t tend to last long on my team. We need people who always ask, “How can we do it better? Smarter? Faster?”

First choice for a new career: Why would I want a different job? BSF is where I want to be.

Personal Taste
Most influential book: My favorite book is The Road by Cormac McCarthy. I find inspiration in stories of adversity that celebrate that bond between a parent and child. Also, Lexicon: A Novel by Max Barry. It’s a completely different vibe, but as a linguist, I celebrate the power of language over psychology.

Favorite movie:  The Thin Red Line – Anything by Terrence Mallick, sign me up. Poetry, realism, nature combined in visual artistry. It is entertainment bliss.

Favorite music: 90’s rock

Favorite food: Mediterranean – grilled octopus. Anything that is chargrilled, I eat. Try me.

Guilty pleasure: Guilt is a useless emotion.