Interview with Emily Kramer-Golinkoff of Emily's Entourage
We sat down with Emily Kramer-Golinkoff, Co-Founder of Emily's Entourage and internationally recognized patient advocate and speaker. We chatted about her organization, her accomplishments, role models in the CF community, and the future of Emily's Entourage.
Emily's Entourage is a 501(c)(3) organization that speeds research and drug development for those with nonsense mutations of CF who are part of the 10% that don't benefit from the revolutionary new treatments for CF. At their core, they are a research organization; they bring life-saving breakthroughs to patients and do it quickly. In order to do that, they have to raise money to drive the research. As a result, they work hard to raise money, raise awareness, advocate, and help fund the research that will drive tomorrow's breakthroughs.
Interview with Emily from Emily's Entourage
What does Emily's Entourage look like day-to-day?
As a research organization, we have funded 21 research grants to academic institutions around the world. We launched a CF gene therapy company called Spirovant, and they're advancing two different gene therapy candidates towards clinic and we're incredibly excited about that. We also maintain a CF nonsense mutation patient registry where we partner with different companies to help speed clinical trial recruitment and also provide patients with relevant opportunities to participate in studies and trials.
In addition, we have developed a whole array of different research resources using cell and animal models to help expedite drug discovery and development. We're really lucky to have an incredible scientific community and community as a whole. We have a number of different fundraising efforts but it's really all for the singular goal of accelerating research and drug development. While 90 percent is remarkable, when you're part of the 10 percent, you know, it's not good enough. We believe that we can do better. That's why EE exists, so nobody is left behind.
The breakthroughs for the 90% are such a powerful proof of concept of what's possible in the CF community and beyond. We've been burned by science before, but this was proof that science is capable of amazing things that can be total game changers and lifesavers.
You have obviously achieved so much with Emily's Entourage, but if you could pinpoint one accomplishment that you're the proudest of so far, what would it be?
This is kind of meta, but the thing that I'm most proud of is what Emily's Entourage represents about humanity and about people - how much people want to be part of change, and how much people care about other people. Being on the front lines and seeing how many people have no other connections to CF but hear our story, have it touch their hearts, and join our team, that is just really the most moving thing in the world.
A lot of times change feels impossible; you spot injustice or something that feels wrong in the world, and the thought of changing it feels insurmountable. What I'm so proud of is what Emily's Entourage represents: How change starts with allowing yourself to remain open and vulnerable enough to see something in the world that feels wrong, and believe that you could be part of the change.
You're clearly very passionate. Were you always like this? Were you always a leader and an advocate for yourself or did that develop over the years?
I'm actually an introvert and was never very public about my story - it's not a particularly comfortable place for me to be. The reason that I started telling my story publicly is that I felt like there was a need. The more I did it the more I realized the impact that stories have - they're what touch peoples' hearts and motivates them to take action. I saw the effect of my story and other stories, and that’s what has kept me sharing.
I think I have always been a pretty passionate and goal-oriented person. I've always held my values close to my heart. I am not scared to be disruptive or do what it takes to achieve my goals. The other thing is that I believe really deeply in that final 10 percent of the CF community. They're people with dreams and families and important things to do in the world, and I believe they deserve better.
Who would you consider to be your biggest role model in the CF community?
I honestly don't think I could identify just one person. I will say, with the disclaimer that I'm very biased, that I think the CF community is made up of the most resilient, mountain-moving people in the universe. I am constantly blown away by the strength, positivity, and character of the CF community. It's a group I feel very proud to be part of.
The other heroes, and often unsung heroes, are the family members of those with CF. CF is a family disease. I couldn't do even half of what I do if it weren't for the unbelievable support from my family, my parents and 3 siblings. I know there are tons of CF families out there and I stand in eternal admiration of the depth of their strength, love, and devotion. They’re the ones living and breathing all the high and low points of this disease right by our side, and they are our reasons for working so hard to endure.
What is in the future of Emily's Entourage?
We have an FDA “Patient Listening Session” coming up in July where we will be meeting with the FDA to educate them about the final 10% of the CF community and advocate for the urgent unmet treatment needs that remain. We are really looking forward to that and we see that as the start of a building relationship with the FDA.
We are also developing our Patient Registry Program, trying to get people with one or two copies of a CF nonsense mutation to join our registry. We have a whole queue of different companies that are interested in working with us to use our registry to help recruit for their trials. It's sort of a clinical trial matchmaking service. We do not share or sell any of the data to companies - we hold it and we're the matchmakers between clinical trial opportunities and patients. We're trying to get as many people with one or two copies of a nonsense mutation to join our registry and grow our partnerships with different clinical trials. We just formed a Clinical Research Review Committee with clinicians and CF community members to help evaluate the different proposals that come in and make decisions on it.
We're also just getting ready to release our sixth round of grant funding. We provide grants to academic research institutions around the world.
In December we host our big, signature annual event - the Evening with Emily’s Entourage gala. This year it's going to be a hybrid gala/virtual event. It's an incredibly inspiring event; we often say it's not your average gala, because it's not a stuffy sit-down dinner. It's very mission-driven and educational - one of those events where you can walk away knowing what you raised money for. This year will be our 10th anniversary of Emily's Entourage, which is a big milestone event for us.
We also have a team that is running in the Philadelphia marathon, which we do every single year. We have charity bibs for Emily's Entourage and we usually have a really big and spirited team. Those are some of the big things on the horizon!
Have you (or a loved one) been experiencing any negative side effects from Trikafta?