For Most People with Cystic Fibrosis, Life Expectancy Has Increased by a Decade. This Nonprofit is Fighting for Those Left Behind

Emily Kramer-Golinkoff is one of about 30,000 people with cystic fibrosis in the United States and approximately 70,000 people worldwide. While there is no cure for cystic fibrosis, there are treatments. Except, Emily’s condition falls into the roughly 10% of the population that does not benefit. So, amazingly, Emily’s friends, family, and the scientific community rallied around her to help! We spoke with Emily to learn more about Emily’s Entourage, and to find out how you can help too!

A CONVERSATION WITH EMILY KRAMER-GOLINKOFF

How and why did Emily’s Entourage get started?

My name is Emily and I am a 38 year old with advanced stage cystic fibrosis (CF), a fatal and progressive genetic disease primarily affecting the lungs and digestive system. There is no cure for CF. Currently, I have 30% lung function and spend 4 to 5 hours a day doing medical treatments to try and delay the progression of my disease. If that were all it took, I would not complain for a moment. Unfortunately, CF is a vicious beast of a disease that resists control despite my most valiant attempts.

Back in 2011 we were seeing game-changing breakthroughs coming down the pike for the roughly 90% of the CF population that has the most common CF genetic mutations. However, due to my rare mutations of CF, I happened to fall in the outlying 10% that wouldn’t benefit. Without any hope we could grasp onto, it was an incredibly difficult place to be. On top of that, given the advanced state of my disease, we knew we didn’t have time to wait and most importantly, that there was a whole group of people like me that weren’t going to benefit from the forthcoming advances and that did not have time to wait—so we started Emily’s Entourage (EE) to speed lifesaving breakthroughs for the final 10% of people with CF.

It was that collision of hope and desperation that sparked the formation of EE. My family and friends decided we couldn’t just sit back passively, and so we dove in head first, launching EE to speed lifesaving breakthroughs and a cure for the final 10% of the CF community. Since then, we have come a long way! We’ve raised over $13 million dollars, funded 32 research grants with top investigators from around the world, and secured over $42.8 million dollars in follow-on funding. We’ve also launched a now-acquired CF gene therapy company that is about to begin clinical trials, developed a patient registry and clinical trial matchmaking program to accelerate clinical trial recruitment, and led worldwide efforts to drive high-impact research and drug development.

What do you want people to know about Cystic Fibrosis, especially about the type of CF?

Thanks to the brilliance of researchers, companies, and the CF community, we now have revolutionary mutation-targeted therapies that are radically changing the trajectory of this deadly disease for 90% of people with CF. They are not incremental advances, they are true life-savers, giving people a second lease on life. They’re taking people off transplant lists, letting them go back to careers and grad school, start families and actually plan for futures that they believe in. We’ve even had a pregnancy boom among people with CF that started these new treatments.

However, for 10% of us with rare genetic mutations of CF, there’s still — nothing. As a progressive illness, CF gets worse with each passing day. We are waiting with bated and fading breath, working our hardest while our diseases rage on.

Here’s a powerful video about two women and one fatal disease. Their stories were the same until one day, they weren’t.

In addition, one of the craziest parts of CF is that it is an invisible illness. Just looking at me, you would have no idea that I have only 30% lung function (on a good day) and that I am teetering on the verge of end-stage disease. As sick as my lungs and other organs are on the inside, on the outside I look like a healthy, vibrant adult at the prime of her life. This is a blessing and a curse. The downside is that it is often hard to appreciate how sick we feel and how difficult everyday tasks are because we look so deceivingly healthy on the outside. But on the flip side, it’s nice not to wear your disease on your sleeve, so to speak, so people see more than just your disease when they look at you.

I also want to note that while EE is focused on the final 10% of the CF population, our work has the potential to extend far beyond. The research and drug development we are advancing can benefit not only people with CF, but also millions of people living with genetic diseases as well as those combating antibiotic-resistant infections for whom there are no existing treatments.

How does a condition like this affect your mental health and how does having friends and family rally around you make a difference?

Life with CF and especially advanced stage CF is incredibly hard. I spend countless hours a day tethered to medical devices, and that’s when I’m healthy! When I’m sick, I go into the hospital for weeks-long courses of IV antibiotics. And yet, that is not to even mention the enormous cognitive and emotional burden of living with a fatal illness that continues to progress despite working so hard to stop it.

Also, CF steals so much from us—our breath, our friends, our dreams of a career and a family, our hope, and our very future. It forces us to keep adjusting to new normals. And, while our everyday reality dictates that we must be tough and strong, there’s a limit to resilience. There’s only so much our heart and our spirits can take. There’s also a unique pain associated with losing your friends with CF at the hands of your own disease. You grieve the loss of your friend, but you also feel angry at your own disease and scared because it’s a glimpse into what lies ahead for you, too.

My motivation for enduring and working so hard to survive is my family. I am the oldest of four and being able to stick around for the big moments (like officiating at my brother and new sister-in-law’s wedding this summer) but also the small, everyday ones matters so much to me. It’s because of them that I can continue to live such a rich and full life despite the advanced stage CF. I’m also incredibly fortunate to be surrounded by a remarkable group of friends who have stuck by my side through thick and thin and lifted, loved and supported me in ways that defy my wildest dreams.

Finally, our “Entourage” of scientists, CF community members, clinicians, and supporters is a lifeline. They give me hope. They have taught me what it means to come together, envision a better future, and not stop until we get there. They make me feel joined and powerful and like we can achieve our goal of fast, lifesaving breakthroughs for the final 10% of the CF community — because we are. I am forever humbled by their devotion and grateful for their support.

How can we help raise awareness and help Emily’s Entourage?

I feel so incredibly lucky to be surrounded by a mighty Entourage of people from around the world who have believed in EE, shared our conviction that those in the final 10% deserve better, and poured their wholehearted support to securing that future fast! I am eternally grateful to everyone who has supported EE in any way over these past 12 years and truly so deeply moved and humbled by their generosity and devotion.

At EE, we are firm believers that every single person has something critical to offer. There are lots of ways people can be helpful!

— You can donate at www.emilysentourage.org/donate. At the end of the day, it is money that fuels research, and research that fuels breakthroughs and cures.

— Follow EE on social media (Instagram, Facebook, and Twitter), encourage your friends and family to follow too.

— Sign up for our newsletter here and visit our website.