By Emily Kramer-Golinkoff
Back in 2011, the collision of hope and desperation led my family, friends and I to start Emily’s Entourage (EE). We saw life-changing advances coming for 90% of the Cystic Fibrosis (CF) community and felt unprecedented hope and energy building in the CF community.
And yet, we realized that those with two nonsense mutations and other hard-to-treat mutations fell in the unlucky outlying 10% that would not benefit. Even worse, research was severely lagging for that final 10%. It became clear that there was an urgent need for focus, coordination and leadership, and that we had to move fast.
And so, EE was born.
I was reminded of those extreme, opposing forces more than ever on October 21, 2019 as
Trikafta was approved by the FDA for 90% of the CF community, giving so many a second lease on life.
And yet for that remaining 10%, including those with nonsense mutations of CF like me, there is still nothing.
I am overcome with happiness to see so many members of our community and so many dear
friends experiencing profound transformations as a result of the modulators. My happiness is
pure and overwhelming. But I cannot deny that it can be hard to read about these enormous
changes (purges! no more coughing! huge FEV1 boosts! delaying lung transplant evaluation
and listing! gains and upward trends! realistic hope!) from the other side, especially as I face
advanced-stage disease with roughly 31% lung function and know that there are many others in the same boat as I am. I am thrilled for those experiencing the life-changing transformations; I just want those same transformations for everyone in the CF community, for us all to share in that same real optimism for a future that so many are feeling for the first time with Trikafta.
What has kept me afloat—and focused on the future—has been the overwhelming recognition of the outlying 10%, the incredibly moving, heartfelt expressions (and actions!) of intense commitment and solidarity, and the growing progress we are seeing in the scientific and biotech communities.
My enduring belief, and what has become our driving mission at EE, is that 100% of the CF
community deserves realistic hope for the future, no matter one’s genetic mutation, and that no one should be left behind. But, reaching the goal of 100% alone is not enough. With a cruel, progressive disease like ours, time is the difference between life and death. CF waits for nothing, least of all for science and medicine to catch up.
That is why we are creating a foundation of the future at EE, shepherding in a new era of
medical research and drug development that changes the way—and the pace at which—drug development is done. EE takes a disruptive, entrepreneurial approach to identifying, advancing and financing high risk, high reward initiatives. We have laid the groundwork for rapid drug discovery and development and cultivated a community of the world’s top CF scientists and biopharmaceutical partners.
The co-existence of hope and urgency remains, more strongly opposed than ever, and it
continues to propel our crystal-clear mission every single day. With that, I am proud to share
EE’s 2020 video that captures the exciting work that we are spearheading and our tireless,
impassioned commitment to extend the remarkable hope and possibility to 100% of the CF
the community in meteoric speed—with nobody left behind.
We at EE believe that leadership from patients, families, and communities with unique expertise and vested interest is critical for fast progress. Our focus is on harnessing collaboration between patients and families, academia, and industry to progress therapeutics through the pipeline rapidly. And we appreciate the critical importance of doing it with unprecedented speed, especially for the remaining 10%.
Thanks to amazing support from our Entourage over the years, we have made significant
progress in our quest to speed breakthroughs for CF. Since 2011, EE has:
● Funded 17 research projects at top academic institutions worldwide
● Awarded over $4M in research grants, leading to over $6.25M in follow-on funding
● Spun out a CF gene therapy company that was recently acquired by a pharmaceutical
company to develop 2 potentially curative, mutation-agnostic gene therapies
● Built and brought together a research network of the most preeminent scientists,
clinicians and biotech companies
We have come a long way in the CF community and now is our chance to extend the truly
remarkable progress to the final 10%, and to those around the world who are eligible for the
modulators but still lack access. I believe that if there is anyone in the universe who can do it, it is the CF community—small, but made up of the most mighty, resourceful, fierce, and effective mountain-movers and change-makers I know.
I may be biased, but I have always felt that there was something special about the CF
community. I feel honored beyond measure to be among a group of the most resilient, strong, compassionate, committed, community-oriented, and adaptive people in the world. We are all in this together until we put an end to this awful disease for good and for all, and it has been deeply meaningful to see that devotion play out when put to the test.
I encourage you to consider sharing the video with your friends and family and on your social
media to get the word out and help close the gap for the final 10% quickly!
If you are interested in learning more about our work or you want to get involved, please
connect with us on social media (Facebook, Instagram, Twitter, LinkedIn, blog) or reach out via email at email@example.com! Also, if you or your loved one has one or two copies of a nonsense mutation, we ask you to consider joining our CF nonsense mutation patient registry.
To learn more about Emily’s Entourage, please visit www.emilysentourage.org.
About the Author: Emily Kramer-Golinkoff is Co-Founder of Emily's Entourage, a 501(c)3 foundation that accelerates research and drug development for nonsense mutations of Cystic Fibrosis (CF), and an internationally recognized patient advocate and speaker who has advanced-stage CF.
Emily 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. She has given talks at The White House, TEDx, University of Pennsylvania's Annenberg School for Communication and Masters of Bioethics Commencement, American Thoracic Society, Stanford University's Medicine X Conference, and more. She was named a White House “Champion of Change” for Precision Medicine and was the recipient of the Global Genes Rare Champion of Hope for Advocacy Award and Philadelphia Magazine Luminary Award.