Tuesday, September 11, 2018

And now, for some good CF news

This afternoon, I got home from running errands and opened up Facebook. The post I was greeted with is, honestly, possibly life-changing.
Last week, Vertex Pharmaceuticals announced data from Phase 3 clinical trials for the triple combination CFTR modulators is expected to be reported in late 2018 and early 2019. Based on the results, a new drug application is expected to be submitted to the U.S. Food and Drug Administration for review for people with one F508del mutation and one minimal function mutation by mid-2019.
We anticipate every person with at least one F508del mutation – regardless of their second mutation – to benefit from the triple combination modulator.
I couldn't believe my eyes.

Up until this point, I have not had the right combination of gene mutations to qualify for the CFTR modulators Vertex has created. Until now.

Yes, my friends and family, this post is talking about ME! I have one copy of f508delta—the most common CF mutation—and one mutation that is considered a "nonsense mutation." And when I pulled up the list of mutations Vertex included for this, there it was: mine. Staring right at me. I'm on the list.

I hadn't even clicked to read the article, I just went off of the last paragraph in there saying "regardless of their second mutation", but I called Jarrod as soon as that bit of text above had sunk in. I read it to him verbatim, and started crying as I did. (I let him know ahead of time I had news that was GOOD, so he wouldn't be worried something was wrong.) And I think I continued to shed tears for the next hour.

Oh wow. Now September 11 can be associated with something good (for me), it was the day I found out that the drug, the one I've been waiting for, the big one, the one that could really change the game for me, is showing promise!

There's still another year of hoops and whatnot (not to mention, finishing the Phase 3 trial) for them to get through before this drug is actually available and to patients, but it's so amazing to hear that it's on its way. I knew this trial was going on, but originally I didn't think it was for me. I thought that another drug aimed at nonsense mutations (with approval possibly another 5ish years away) was what I was waiting for. So this is amazing.

I feel like I have yet another reason to fight, to kick this cancer and get better. I start round 2 of chemo on Thursday, let's do this!

Link to article about all this here.

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