Monday, May 13, 2019

Week 299: Math games

Happy belated Mother's Day to all my fellow moms out there. We had a great Mother's Day here, getting things off to a solid start with a 4:52 a.m. wake-up (thanks, Lime) and a gift of a barrette found abandoned on the school playground (thanks, Lemon). Papa Bear took the kids out in the early morning for bagels, so I was able to enjoy a few extra moments of early morning solitude, and he got me a bunch of very thoughtful gifts as well (and nothing that he found on the ground anywhere).

Since this is the final post before our Great Strides walk this coming Saturday, let's talk about research, a topic near and dear to my heart and to the hearts of all CF families. The CF Foundation has done an amazing job of driving forward the development of therapies for CF. You can see their whole drug discovery pipeline by clicking here. Of course, the drugs that have been developed that treat the underlying cause of CF (Kalydeco, Orkambi, and Symdeko) get most of the attention in the media. That makes sense, since they are absolutely revolutionary therapies, and we have high hopes for the next generation of these drugs that should help patients like Lemon who only have one copy of the common F508del CF mutation.

But, breakthrough therapies that treat the underlying cause of CF are only part of the CF Foundation's portfolio. They invest a tremendous amount of funding to develop things that sound less glamorous, but are just as important. These include medications to help clear mucus from the lungs, two of which are already approved for use in patients and that almost all CF patients use daily: hypertonic saline and Pulmozyme. There are many new drugs in this category already in Phase II clinical trials, which could help keep patients' lungs even clearer of the mucus where bacteria like to live and cause devastating lung infections.

Speaking of lung infections, the CF Foundation also has a big portfolio of new antimicrobial agents, many of which are already available to patients (including Cayston, our go-to intervention plan). There are many more exciting drugs coming up through the pipeline in this category, too, which are urgently needed as the bacteria that infect the lungs of CF patients often become resistant to the first-line therapies. The Foundation is also helping to develop anti-inflammatories, since there  is a growing understanding that inflammation in the lungs, not just infections, is a cause of lasting lung damage.

The CF Foundation also recognizes that the lungs are just one part of CF, and that managing the nutritional issues associated with CF is a key part of any care plan. They participated in the development of specialized vitamins and pancreatic enzyme products that almost all patients with CF, including Lemon, use every day to help maintain their nutritional status.

I hope this impressive research portfolio, combined with all the other things  the foundation does, is enough to convince you that the dollars you donate to the foundation are put to great use. We are SOOOO close to hitting $5000 for our team this year, thanks to the incredible generosity of so many of you loyal readers. Who hasn't donated yet and wants to be the one to push us over the $5000 mark? You? Excellent. Click right here and thank you so much!