Dear family and friends,
like in many past years, I am hoping you will again support my fundraising efforts for cystic fibrosis. I am reaching out a bit later than usual, but my family will again participate along with the CF Foundation's lab team in the Great Strides walk on May 21 in Boston. As you all know, I have been involved in cystic fibrosis research for over 22 years, almost 14 of those working directly for the CF Foundation. During that time we have seen great successes, going from no disease-modifying medicines to currently 4 approved treatments. The latest and most efficacious medicine, Trikafta, which was first approved in 2019 for people with CF age 12 and older, is now available for children as young as 2 years old (as of April 26) ! This means around 90% of all people with CF are now eligible for life-changing medicine!
Unfortunately, that also means about 10% of patients with different disease-causing genetic variants are impatiently waiting for their turn and therapies that will help them. Most of the remaining patients without treatment options will only be treatable with some form of genetic therapy, which could be mRNA, gene replacement, or eventually gene editing. We are busily working to drive these new approaches forward. In the last 18 months, between our mission-related business development team and our scientific team we have evaluated over 300 companies with new and relevant technologies. At the same time the scientists in our lab are working on the "blueprints", the proof of principle for new approaches.
Already, there are two mRNA and one gene replacement therapy in early clinical testing, but even if these new therapeutic approaches prove efficacious and without untoward side effects, it will take several more years before they will be available as therapies for people with CF. As exciting as success in a genetic therapy will be, these first approaches are only targeting the lung. Much more work and progress is needed before we will be able to treat all organs and tissue affected by cystic fibrosis, which includes the pancreas, the GI tract and the liver. And it will take a lot more money before we can deliver on our mission to find a cure for all people with cystic fibrosis.
I know that all of you have many good causes to choose from that are asking for your donations, and I very much appreciate your support of the cause that is near and dear to my heart. Knowing that our progress in genetic therapies for cystic fibrosis will inform these types of therapies for many other diseases makes me feel good when I reach out to you.
Thank you so very much!
PS: For those of you interested, I am linking a Facebook post from a mother with a 13-year-old boy with cystic fibrosis. I met them recently in Oklahoma City when I spoke to the CF community there. While eligible for the most efficacious available medicine the boy is very much hoping for treatment that will better help with his sinus and GI issues.