Rare Leader: Emily Kramer-Golinkoff, Co-Founder, Emily’s Entourage

The Basics
Name: Emily Kramer-Golinkoff

Title: Co-founder

Organization: Emily’s Entourage

Social Media Links:

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Disease focus: Cystic Fibrosis (CF) is a progressive genetic disease that affects the lungs and digestive system as well as many other organ systems. It causes thick, sticky mucus to build up in the lungs, which leads to life-threatening lung infections. There are approximately 30,000 people with CF in the United States and 70,000 worldwide.

Headquarters: Merion Station, Pennsylvania

How did you become involved in rare disease: I have Cystic Fibrosis. I became more actively involved in the community back in 2011 because of a number of forces. There was this new hope in the CF community that was being driven by these game changing new breakthroughs that were coming down the pike that targeted the root cause of CF rather than just the symptoms. But at the same time, with two copies of a nonsense mutation, none of these revolutionary breakthroughs were going to help people like me. The added element was that with advanced stage disease, time was of the essence. I realized that the trajectory of drug development was beyond my lifetime. If we wanted realistic hope for me and tons of people who were in the same boat as me, we were going to have to disrupt the field and the pace at which drug development was done. That’s when I took on this new role as a foundation leader and an advocate.

Previous career: Manager of Strategic Initiatives and Operations, Penn Medicine’s Center for Digital Health.

Education: B.A. in communication from the University of Pennsylvania; MBE in bioethics from the University of Pennsylvania; Certificate in clinical ethics mediation at the University of Pennsylvania School of Medicine

The Organization
Organization’s mission: Our mission is to speed research and therapeutic development for nonsense mutations of CF.

Organization’s strategy: We see ourselves as the hub for nonsense mutations and our strategy involves building an infrastructure and a pathway so development of therapeutics for nonsense mutations can be a straight shot by removing all of the operational barriers that can stand in the way. We provide connections to people, to ideas, to resources, and to funding. We provide impetus and urgency. And we also provide partnership to fund creative, out-of-the-box ideas and promising opportunities and usher them through the pipeline as quickly as possible. We also partner closely with industry to provide useful resources, influence their pipelines, and get them focused on nonsense mutations.

Funding strategy: We raise money through a lot of different channels. We have two major special events every year—one in Philadelphia in December and another New York that happens in the spring. We have grassroots digital campaigns and we’re in the middle of our annual drive, which is all digital. We have a major gift program called Catalyst for the Cure. We have corporate programs. We have a fitness arm called Team EE for people who participate in fitness activities to raise money and spread awareness. We have a variety of different revenue streams.

What’s changing at your organization in the next year: We are rapidly growing. We now have a staff of four full-time members. We’re recruiting a director of operations. At the end of the day, we’re a research organization. Our grants are making progress. The science is advancing and that’s incredibly exciting. Our role in the scientific community is rapidly expanding as well. Our opportunity for impact is far greater than ever before. We have invested heavily in building an infrastructure, developing cell models, research resources, animal models for nonsense mutations. Those elements are all now in place, which we think is a critical part of building that highway to breakthroughs. We have been fortunate to have an amazing Entourage that has rallied behind us and that believes in what we’re doing. There is now an FDA-approved, revolutionary new therapy for 90 percent of the CF population that is transforming their lives, giving them a second lease on life. It is remarkable, but that outlying 10 percent includes those with nonsense mutations. That’s why we started Emily’s Entourage — because we saw that they were being left behind. That’s come to fruition. That 10 percent is still living with the same killer of a disease where we are still just treating symptoms as opposed to the root cause of the disease. 

Management Style
Management philosophy: We’re incredibly goal oriented. We are fortunate to have an amazing cadre of supporters, volunteers, and staff members who are deeply committed to our end goal. They describe themselves as “lifers.” This isn’t a charity of the week for them. They are in this for the end game. We’re very fortunate to have that kind of passion and commitment. We’re all about outcomes and about achieving our goals. That guides a lot of what we do. As a patient family-led foundation, a unique asset is that there is not a second of the day that goes by that we are not painfully aware of what is at stake and why we’re fighting. That passion to save the lives of people with nonsense mutations sits firmly on our chest all the time. That is a real motivator for everyone who’s involved in Emily’s Entourage. We describe ourselves as a startup nonprofit. We have an entrepreneurial approach, a disruptive approach. We’re resourceful and we’re hustlers. That is also part of our culture.

Guiding principles for running an effective organization: We take ethics and transparency and, and honesty seriously. That’s a value and a character trait of who we are and that is embodied in Emily’s Entourage through and through. Our philosophy from conception has been multi-stakeholder collaboration. We believe that patients and their families are uniquely poised to make transformative change in this area and that their leadership is critical to ushering in this new era of patient advocacy and impact. We believe in these multi-stakeholder collaborations with patients and families in leadership roles side by side with industry, clinicians, researchers, academic researchers, supporters and the whole realm of stakeholders. That is an important part. We also believe in the power of the patient community to make change. A lot of what we do is around infusing their voices—bridging the gap between drug developers, researchers, and the patient-family communities. We think they share a lot of aligned goals and we seek to bring those groups closer.

Best way to keep your organization relevant: With game-changing new targeted drugs recently approved by the FDA for 90 percent of the CF community and yet nothing for the final 10 percent, unfortunately, we don’t have to try hard at being relevant. We are talking to members of the CF community every day that are on these revolutionary new drugs. I can’t even begin to articulate the immediate, transformative impact on their lives. It’s astonishing. And yet for 10 percent, there’s still nothing. This is a disease that doesn’t wait. Months, days, and moments matter, and so there’s a ton of timeliness, relevance, and urgency.

Why do people like working with you: You can’t touch Emily’s Entourage without feeling the deep, overwhelming passion and commitment of this community. The Entourage is made up of the most amazing people in the universe. I think everyone who interfaces with us in any way immediately feels that. People are incredibly kind and have big hearts and really want to be part of change. It’s up to us to present opportunities for them to do that. That’s what I think Emily’s Entourage does. Finally, especially in this time, political climate, and world we’re living in today, I think Emily’s Entourage embodies an opportunity to show what can happen when you see injustice in the world, or something rubs you the wrong way, or doesn’t feel right, or makes you angry. It shows our power to do something about it. That is something that the world is thirsty for and in need of right now. What we’re doing is showing that you don’t have to accept the status quo—that we are infinitely more powerful than we realize and it’s up to us to leverage that power to make transformative change. People want to be part of impactful change and that’s what Emily’s Entourage offers them. They have witnessed the incredible progress that we have experienced in a short period of time. That personal and direct opportunity for impact is really motivating.

Mentor: I don’t know that I have one specific mentor. There are a lot of people who have inspired to me. I recently spoke at our gala about how during a year of significant health challenges, entrepreneurship through Emily’s Entourage became a lifeline for me in a number of ways. The way that I learned about entrepreneurship is primarily through podcasts. I became obsessed with them. I listened to them every minute of the day This opportunity to create something transformational out of nothing has been invigorating at a time when there was a lot of bleakness in my life due to my health. I’ve been inspired by a lot of different entrepreneurs.

Beyond that, I have different mentors in different facets of my life. The patient community, the rare disease community, they’re not communities you would choose to be a part of, but they are communities I feel honored to be part of. And I look around at so many other advocates and people living with awful diseases and leaders of foundations who are just the most adaptive and resilient, strong, and visionary people in the universe. I’m constantly humbled by that.

On the Job
What inspires you: First and foremost, I believe so deeply that everyone with CF deserves realistic hope for the future and for a deep breath. I’m endlessly driven by this desperate goal to bring that to them quickly. I realize that time is the difference between life and death. That is on my mind with every single breath I take. That is by far my biggest motivation.  The second one is that I am constantly blown away by the outrageous kindness and generosity of our Entourage. People have stepped up in amazing ways. There are countless people from across the globe who we have no connection to us, some of whom have no connection to CF, and who have become just these unbelievable supporters. Witnessing that kind of next level kindness and devotion has renewed my faith in humanity in powerful ways. I was never cynical before, but I never dreamt of this level of kindness.

What makes you hopeful: There’s a lot to be hopeful about. I feel hopeful about the kindness and solidarity of people. I feel hopeful about science. We have seen the latest treatment that was approved by the FDA on October 21st and the impact is unbelievable. When the researcher from Vertex was first talking about this idea that small molecules could address the CFTR protein at a CF conference years ago, she was laughed off the stage and now that is saving thousands and thousands of people’s lives. I have endless hope and faith in the CF community. Now, the critical factor right now is just about time—time for therapeutic development for the final 10 percent. I have endless faith that we will get there. The question is just how long and that’s a matter of people’s lives.

Best organization decision: The best decision was to start Emily’s Entourage as an independent organization. We got counsel not to do so. We considered it and after a lot of thought, we listened to our gut. That was our first leap of faith in ourselves. We thought we were in a unique position to make impactful change. That was an early decision, but it was one of the best.

Hardest lesson learned: You can do everything right, everything under the sun. You can have the best medical care. You can have the most love and support. You can be educated and informed and take the best care of yourself. And yet, with a disease like CF, nothing is enough to halt the progression. It’s a losing proposition and it is constantly crushing. In almost any other realm of life, if you do the right things and are persistent, strategic, and smart, you will succeed. CF is an exception to that.  You can do everything right, and it’s still not enough. That’s a tough pill to swallow.

Toughest organization decision: The toughest decision was making the plunge to directly fund labs. It has paid off in dividends.

Biggest missed opportunity: In 2015, I got invited by President Obama to the White House. It was shocking and exhilarating and came out of nowhere. I wanted to make sure I was the only CF patient that was going to be at the event because people with cystic fibrosis can’t be together due to the threat of cross infection. It turns out, another person with CF was attending and to make a long story short, I ended up being disinvited from the White House event. I was crushed! My boss at the time said to me. “Good work gets recognized in time. This isn’t the end of the story.” A month later, we were named precision medicine initiative “champions of change,” invited to speak at the White House and participate in the ongoing program. It was a powerful lesson that if you stay focused and work hard, it pays off in due time.

Like best about the job: I feel honored to have a job with such an important purpose. There’s such an important role for us here. I feel grateful that people have the confidence in us to allow us to do it. To have this kind of platform to bring about the changes that we believe in so deeply is an enormous gift.

Like least about the job: I wish my life wasn’t on the line. It would be so much more exhilarating and amazing to do what we do if my life didn’t depend on it and if I weren’t very sick with CF myself.  

Pet peeve: Making the same mistake twice.

First choice for a new career: My dream is to cure CF and move on to another rare disease.

Personal Taste
Most influential book: When Breath Becomes Air by Paul Kalanithi and The Year of Magical Thinking by Joan Didion

Favorite movie: Dirty Dancing

Favorite food: Lobster, raisins and lattes (not together)

Guilty pleasure: I follow a number of different influencers on social media and read a bunch of different fashion and lifestyle blogs.

Favorite way to spend free time: I love to travel and experience new places, spend time with my family, and be in nature around dramatic, beautiful scenery.