Hello friends and family! This year’s letter comes with some very exciting news! This past October, the FDA approved Trikafta. Yep, it’s another drug to treat the underlying cause of CF…but this one is completely changing the lives of those who take it! In trying to figure out how to convey our excitement over this tremendous breakthrough, we decided Caroline had already done the work for us=) She did an excellent job presenting her research project on Cystic Fibrosis and Trikafta for a class project. She has several quotes from Dr. Francis Collins, the man who discovered the CF gene way back in 1989, that will help you understand how groundbreaking this moment is for the CF community.
“It has been 30 years since we’ve been hoping and dreaming for a day like this, where you could look at the data and just absolutely-your jaw drops because it is so impressive and so good. Now we are at a point with this triple therapy where 90% of people with Cystic Fibrosis are going to have substantial and amazing benefit from the drug therapy that looks as if it will convert what has been otherwise a very threatening and potentially fatal disease into a chronic illness that’s going to require treatment but which should allow people to live much more normal lives. And waiting 30 years and having this happen, yeah, I’m overjoyed.”
“I think this is more in a place where you could say this is becoming a manageable chronic disease like diabetes or arthritis.”
“It is amazing. You might have thought people would have after a while just said, oh, heck, it’s not going to work. Nobody ever said that. For 30 years this journey has been pursued by thousands of scientists from all sorts of different perspectives. All of us are having a pretty big celebration.”
Each one of you is a part of this incredible milestone. Your generosity helped drive the research to create Trikafta. Thousands of mothers, fathers, daughters & sons have now shifted from surviving to thriving! They are looking forward a future they never thought they’d get to see.
In the words of our sweet Caroline: “It is now possible for us to dream about the future. People living with Cystic Fibrosis can now hope to live normal healthy lives. They can go to college, have a family, travel, and have an improved quality of life. Other medicines will also come out in the future. One day CF will stand for Cure Found! It is definitely possible when you look at how far we have come in the last 30 years.”
As you can imagine, it warms our hearts to hear our child think about her future with such hope and optimism. She may never have to experience the sacrifices so many before her have had to make. Her world is full of laughter, adventure, chalk pastels, slime, and her best 4 legged friend-Bumble the guinea pig!
This June 17th, Caroline will take her first dose of Trikafta on her 12th birthday! What an incredible gift you have all given her=)
Team Toole-Katy, Justin, Caroline, Mya, Ben