This week, we had the announcement of the exciting results from Vertex's latest Phase III clinical trial. This was a test of a combination of two of their drugs (Ivacaftor and Lumacaftor) in patients with two copies of the F508del mutation (the most common mutation that causes cystic fibrosis; about 50% of patients have two copies of this mutation). After 24 weeks on the drug, patients saw a statistically significant improvement in an important measure of lung function. Although the magnitude of the effect was small, I still think this is a tremendously exciting result since this combination of drugs hits the root cause of CF, and doesn't just treat the symptoms or downstream consequences. This combination of drugs will almost certainly not help Lemon directly, since he only has one copy of the F508del mutation and one copy of a different mutation. But, Vertex has other drug combinations in the pipeline that could help him. I'm really hopeful that by the time he's old enough to use any of the drugs (right now, the youngest patients using them are 12), there will be a good cocktail ready for him to take. It's important to point out that the CF drug development at Vertex is directly supported by the Cystic Fibrosis Foundation, to the tune of $75 million, so for those of you who have supported our various fundraising efforts, this is your money at work--it is gratifying to see such concrete results, I think!
Here is Lemon, using a brand new skill acquired this week to show everyone what he thinks of the Vertex results:
Also this week, our dear friend Sheryl visited us from Boston. Having two little boys of her own, she knows exactly what to do with a baby boy, and that was evident from the moment she walked in the door. Lemon immediately gravitated towards her and wanted to watch her and talk to her the entire time she was here.
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We also had a very fun belated Mother's Day brunch with Nona, courtesy of Uncle Jared who gave us a nice gift certificate. After brunch we still had time for a visit to the nearby park, where we played one of Lemon's favorite games, in which Lemon swings and Mama Bear pretends to be afraid that Lemon is about to collide with her. Hysterical laughter follows.
On Sunday, we went to a work event at a local park that has a splash pad. This is basically the super upgraded, on steroids version of the running through lawn sprinklers that Uncle Jared and I did as kids. Lemon was absolutely beside himself with delight, since it combines two of his favorite elements, fountains and watching other kids. After about 15 minutes at the splash pad, he was so overwhelmed that he fell asleep in the car on the 8 minute ride home, slept an additional 90 minutes after we brought him into the house, woke up, ate something, went back to sleep and slept a further 90 minutes, woke up again, ate something, and went back to bed for the night within the hour.
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Speaking of splash pads, check out what the wacky weather of the Midwest did to our street this evening! Our basement got a little wet and Papa Bear got trapped at work for an extra hour, but otherwise we are unscathed (knock on wood, since the thunder is still rolling out there as I write!).
