Emily Kramer-Golinkoff is an internationally recognized patient advocate and speaker. She has a master’s degree in bioethics and certification in clinical ethics mediation from the University of Pennsylvania, where she also completed her undergraduate degree. Emily was named a “Champion of Change” for President Obama’s Precision Medicine Initiative and is the recipient of the 2020 Philadelphia Magazine Luminary Award and the 2016 Global Genes Rare Champion of Hope for Advocacy Award. As an adult with cystic fibrosis, Emily is both the inspiration behind and co-founder of Emily’s Entourage.

The Pulse: The theme of our conference this year is the empowered healthcare consumer, so I thought we could start with your experience as a patient. Could you give an overview of cystic fibrosis (CF) and the challenges you face on a day-to-day basis? 

Emily Kramer-Golinkoff: I have advanced stage CF. I am 38 years old and have about 30% lung function. While there have been groundbreaking, revolutionary advances in CF treatment in recent years, these life-saving therapies only benefit about 90% of the CF population. Unfortunately, I am part of the 10% that doesn’t benefit from these new treatments. 

Because of the advanced stage disease, my daily routine involves spending four or more hours on medical treatments, including wearing a vest to vibrate my lungs and clear mucus, taking pills, and taking inhaled medications. I have CF-related diabetes, which requires me to use an insulin pump. I also spend an hour a day exercising to keep my body strong. And this is all in the best of circumstances. When I get sick, I have to go to the hospital for weeks-long courses of IV antibiotics. Then there are a million other complications that come with the disease, so it’s a constant rollercoaster ride.

It’s really hard, but even with all of that, I wouldn’t complain if it was enough to stop the disease from progressing. I’m a hard worker, and I never miss my treatments. But despite my best efforts, and despite doing everything under the sun, the disease keeps progressing. There is no way to stop it, and everything is just a means to delay the progression as much as possible.

The other challenging thing is that I have been in strict isolation since March 2020 because my medical team feels that catching COVID is too risky for me given the advanced state of my disease. I don’t go in any public spaces except the hospital for essential medical appointments and tests.

On a more positive note, I’m excited about the advances that are finally starting to happen for the final 10% of CF patients. I have a lot of faith in these therapies, and I am proud of the critical role Emily’s Entourage is playing in advancing them. I see this time in isolation as a period of incubation for me while I wait for these therapies to advance. I’ve worked hard to get to this point, and I’m not willing to let anything get in the way of benefiting from these exciting treatments in the future.

The Pulse: How can the power of patients be harnessed, especially as it relates to clinical research and therapeutic drug development?

EK: Patients have been the most underutilized resource in medicine, including in therapeutic development. After all, who is more invested in finding cures than those whose lives depend on it? This is especially important in the era of precision medicine and the ongoing revolution in genetics. We’re learning that diseases that were once grouped together are actually distinct, and that some previously unconnected diseases have more in common than we previously realized, including the potential to be treated with similar therapies.

In this new era of genetics and personalized therapies, there is a unique opportunity — and need — for patients and families to be a driving force behind research. The therapies needed for rare diseases are typically not going to be “blockbuster drugs” that address common issues such as heart disease and diabetes. So, to get these rare diseases on the research radar, we need champions who can push for progress and recruit researchers to develop the necessary resources. Unfortunately, this often falls on patients and families who are desperate for progress. They realize that the only way to develop therapies that might come in time to save their lives is to dive in headfirst themselves.

The Pulse: To that end, I’d love to hear about the origin story for Emily’s Entourage. What motivated you to form the organization and how has it evolved over time?

EK: Emily’s Entourage was born in 2011 out of desperation. Like I said, my family and I realized that the game-changing therapies that were helping 90% of the CF community were not going to benefit 10% of patients like me, and, on top of that, I had advanced-stage disease. We were literally running out of options. More importantly, we saw a whole community of people in the same boat and knew we had to do something.

At first, we raised money and gave it to other organizations. As we evolved, we began vetting, evaluating, and giving funding directly to labs ourselves. Now, we have a robust grant and venture philanthropy program and have made huge progress in advancing treatments. Getting these therapies to the finish line requires focus, leadership, coordination, and support, including from private funders like Emily’s Entourage. We aim to usher therapies from the lab through clinical trials to regulatory approval to the bodies of people with CF, whose lives depend on them.

The Pulse: How have you been able to overcome the challenges of starting a non-profit focused on complex scientific research and drug development?  

EK: Starting a nonprofit was not in our grand plan, but we were driven by fear and a desire for hope, so we dove in headfirst. Even though we had no idea what we were doing, and we didn’t have backgrounds in science, fundraising, or non-profits, we had a crystal-clear vision: to create fast breakthroughs for the final 10% of the CF community. That clarity of vision has been our superpower.

Looking back, it’s been an incredible journey. The craziest part for me is seeing the direct impact that Emily’s Entourage has had in creating hope and opportunities for people like me. We never would have dared to dream of the progress that has been made, and the results have been far beyond our wildest imagination.

The Pulse: Could you give an overview on how Emily’s Entourage’s funding approach and venture philanthropy model allows your organization to make such a large impact?

EK: As an organization, our model has allowed us to invest in high risk, high reward research and to be agile and goal-oriented in our funding mechanisms. Achieving our end goal as expeditiously as possible is what truly matters to us, which allows us to finance things in creative ways and let need and opportunity dictate our approach.

We have a robust grant program and have given out 31 different grants, including nine in the past year, to top academic institutions around the world. These projects have spun out into successful companies and are progressing in ways we’re thrilled about.

In addition to our grant program, we also support research through our venture philanthropy model. In 2016, we made a strategic investment and launched a gene therapy company called Spirovant Sciences (formerly Talee Bio). Time is our biggest threat and our hope was that a company could develop a therapy faster than an academic lab. We identified a management company, connected them with the key players in the CF community, funded their exploration of a few different therapeutic approaches, and ultimately gave them the seed funding to launch Spirovant, a company focused on developing gene therapy.

Spirovant is currently pursuing two gene therapy programs for cystic fibrosis, and one has advanced far enough to be approaching the clinical trial phase. It’s incredible to think about this therapy reaching people with CF in the near future, and it wouldn’t be possible if it weren’t for Emily’s Entourage and our initial impetus, organizing power, and seed funding.

The Pulse: What are some of the potential breakthroughs or developments that you are most excited about?

EK: I’m most excited that there are multiple promising therapies and different therapeutic approaches supported by impressive data. It’s the first time we have seen such a wide range of potential treatments, from gene therapies to mRNA therapies, and even more out-of-the-box solutions like molecular prosthetics.

At Emily’s Entourage, we started a clinical trial matchmaking program with a CF nonsense mutation patient registry that helps connect patients with the right clinical trials. This is particularly important for rare mutations and rare diseases where recruitment can be a major challenge and a significant obstacle in drug development. Our program is the only direct-to-consumer registry in the space, which not only facilitates faster, more targeted recruitment for clinical trials, but also empowers the CF community with the tools and information to pursue clinical trial opportunities directly, without relying on their clinicians or other gatekeepers. We are working with many companies to help them expeditiously recruit patients for their trials. Patient recruitment is absolutely critical for the success of these programs, and it also helps prove to companies that there is a market for these treatments as well as to promote diversity and representation within clinical trials.

Many clinical trials fail, but to now have multiple shots on goal feels like the greatest gift. This diversity in therapies offers multiple opportunities for success for patients. I am realistic, but I can’t help but feel optimistic about the future. The hardest part was when there was nothing to hang our hope on, but now we have a lot to be hopeful about and more is on the way.

The Pulse: A lot of our conversation has focused on overcoming challenges. Could you discuss how you have been able to channel these challenges into such a positive impact on the world? 

EK: In 2015, I was doing digital health research at Penn and loving every moment of it while spending my nights working on Emily’s Entourage. Then I was diagnosed with a terrible bacteria in my lungs that is associated with a very high mortality rate. The doctor did a full assessment and said that the thing that worried her most was how much I was working. I was literally working myself to the ground, and she said that I needed to quit my job the next day.

I was 30 years old and am a hard-working, overachieving, ambitious person who had always cared a lot about my career. I felt like I was retiring at the age of 30, and it was a really sad and scary time for me. It felt like not only was I losing a big part of my identity, but also that my potential was dimming.

I eventually realized that you take your potential with you wherever you go. It wasn’t my plan, but by quitting my job, I was able to devote more of my time and energy to Emily’s Entourage. And when you look at the trajectory of the organization’s fundraising and impact, a steep acceleration started in 2015. I was able to take all of that potential that I put towards my career and direct it to Emily’s Entourage, which has become my life’s work.

We’re on a mission to help thousands of people just like me, with parents, spouses, siblings, and children that love them and that need them. They are people that have such important things to do in the world and deserve better. We see what’s possible, and this feels like the homestretch. Pouring ourselves into something this important is the best way to channel negative feelings into productivity.

The Pulse: How can our audience best support you and your mission at Emily’s Entourage? 

EK: Anyone interested in Emily’s Entourage should visit our website and follow us across social media. We are a fundraising organization that runs on donations, so we appreciate anyone who is able to donate.

Also, people in the Wharton community are so smart and driven, so we appreciate every connection we can make. We are entrepreneurial, and our guiding philosophy is that every single person has something critical they can offer. We would love to have people get involved in any way possible, so please reach out if you are interested. We have met some of the most remarkable people in the world that are driven by the same mission. That is the power of Emily’s Entourage and we invite you to join us on our urgent quest to secure a better future for the final 10% of the CF community — and do it fast.

Interviewed by Tommy McGrath, January 2023.

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On Feb 16-17, 2023, Wharton is excited to feature more expert perspectives at our annual Wharton Health Care Business Conference. This year’s theme is ‘The Empowered Health Care Consumer’. Conference details and tickets are available here.