Gayle Florio

Family and Friends: 

We are gearing up for the Great Stride Walks beginning in North Carolina, Florida, Alabama and making my way back to Kansas.  I thought I would update you not only on the girls and their health but also what is happening with the Cystic Fibrosis Foundation.  

Let’s start with the girls.   This year with their move to Montgomery, Alabama, the kids attended school.   Going from homeschooling for 2 1/2 years to a school environment was an adjustment, however, everyone adapted very well.  For the past 3 years neither of them have been hospitalized.  Much of it has to be credited to Jackie’s diligence with the girl’s treatments and self care.  Their lung function is always in the high 90% at their clinic visits.  Activities have included swimming, basketball, pickle ball, badminton and now soccer.  

CF Updates: 

Kalydeco is now available for Children 4 months and older with certain mutations.  This is the miracle drug the girls have been on since 2015.  It certainly has made their life very normal and healthy. 

Orkambi is available for Children 1 year old and older with two F508del mutations.

Symdeko is available for Children with CF 6 years and older with certain mutations. 

Trikafta is available for Children with CF 6 years and older with one F508del mutation.  

There are 12+ treatments available including 4 to address the underlying cause of Cystic Fibrosis.  There are 35 major industry research programs genetic therapies including three for mRNA therapies which are already underway or will begin in the next 18  months.  

Today there are now more than 40 treatments in the drug development pipeline, including new and potentially better modulator therapies, as well as genetic-based therapies.  

With this commitment in mind, the Foundation launched our Path to a Cure, an ambitious research initiative to accelerate treatments for everyone with CF and ultimately deliver a cure. We intend to allocate a half billion dollars to the effort through 2025 and have already seen an 180% increase in funding for this area of research from 2018.

It is also critical to continue to develop new and improved treatments for complications from the disease. We are building on the current momentum, funding an innovative research portfolio and collaborating with top scientists from around the world to deliver the next generation of transformative focused on early-stage genetic therapies.  There are up to 6 clinical trials for breakthroughs in CF. To ensure the advancement of potential therapies, we provide research funding and expertise to draw the best scientific minds and technologies into CF. Last year we held more than 230 meetings with over 120 companies — 61 of which were new to CF — and are currently funding nearly 50 different industry programs.

Despite the many challenges posed by the pandemic, in 2020 the CF Foundation funded approximately $248 million in laboratory research, preclinical drug development, clinical and real-world research, and high-quality, specialized care and training — more than at any other time in the history of the Foundation and nearly a third more than just the previous year.

Without YOU none of this would be possible.  Our thanks is not enough, but just know that YOU have played a major role in our grand daughters lives and we are eternally grateful for your donations and prayers.  WE will Fight Until WE Find a Cure and I honestly believe it will be in our lifetime,  

Love to you all, 

Mike & Gayle