I had the privilege to be part of the Cystic Fibrosis Foundation strategic planning committee in 2012. We discussed many issues, including mental health, pain management, specialty doctors and lung transplantation -- all things my son, Mitchell, had already experienced. While the Foundation has long been ahead of the curve in the creation of effective treatments for CF, the sale of its royalty rights in 2014 provided the opportunity to accelerate its mission like never before. These resources, as former CEO Dr. Bob Beall noted, allow the Foundation to “'dream big' in ways we never could have imagined.”
Since then, the Foundation has accelerated the implementation of the strategic plan, addressing these very important facets of CF care. Timing is everything.
Treatment for cystic fibrosis has advanced to the point where the life expectancy is around 40 years. The Foundation's primary focus over the past 10 years has been to develop drugs that can correct CF at its genetic root. With the Food and Drug Administration's (FDA's) approval of ivacaftor (Kalydeco®) and lumacaftor/ivacaftor (Orkambi®) and the Foundation's drug development program focused on developing the second generation of these types of medications, the Foundation is now ready to address other areas that affect people with CF, such as the needs of those whose health is at the point where these medications will not make them better and their only option for life is a lung transplant.
My family knows about this. Mitch has undergone two double-lung transplants and is currently approved, but not activated, for a third. I have watched Mitch's journey with CF; certainly not a straight road, but one with many twists and turns. Somehow, Mitch and our family have survived all these challenges and obstacles put in our path and have remained diligent in fighting this disease with every breath we take. Today, Mitch fights not because of me, but because of his wife and 12-year-old twin boys.
Through Mitch's transplant journeys, it became apparent to me that the process is lacking in several crucial areas. I noticed little communication among transplant centers, no best practices in place, no new anti-rejection medications on the horizon and little financial support and resources for the family members of the person with CF who have to make so many changes in their lives. As I spoke with other parents, I realized many had similar experiences and others did not know where transplant centers were located. They lacked information on when to get evaluated for a transplant and what to expect. There was also a lack of ready information regarding the transplant process for people with CF and their families.
After voicing these concerns at strategic planning meetings and talking with others at the Foundation, the Foundation announced in early summer 2015 that a Lung Transplant Summit meeting would be convened in the coming months. The meeting, held in February, brought together expert clinicians and research scientists to help the Foundation develop a strategy to improve the outcomes of lung transplant for people with CF and provide greater support for lung transplant care and research. Because treatment options for people with end-stage CF lung disease are limited and considered an area of unmet need for people with CF, lung transplantation has become a higher priority for the CF Foundation.
At this meeting, I was amazed that all the researchers and doctors in the room shared the concerns our family faced. Their comments validated my perceptions and my family's experiences regarding the transplant process for people with CF and their families.
These important issues were being addressed.
Implementing a centralized database, determining best practices and sharing this information among the various transplant centers is monumental but possible. We can improve the outcomes of lung transplants just as we can improve the overall health outcomes of people with CF. It will take time, talent, money, determination and dedication. Thankfully, the CF Foundation has these resources.
Obviously this will not all happen tomorrow and we can't change the past. But as we move forward, we can ensure that our children with CF have a brighter future with fewer challenges than the Mitchells of the world today.
As the chosen representative for the CF community at the summit, my question to the panel was the same that I asked the transplant doctors before Mitch's “do over.” “What are we going to do differently tomorrow to ensure a better outcome for our children and adults with CF?”
Thankfully, the CF Foundation is up to accepting this challenge.