SOCIAL MEDIA

16 December 2020

A vaccine is amazing, but that's not the medicine I'm anxiously awaiting

For nine months, the world has ached and longed for a vaccine for COVID19.  It has been thrilling this last week to watch the distribution of a medicine that has the potential to change the world back to the way it's supposed to be.  Along with so many others, I look forward to the day when I, too, can have access to this drug.

But, for me, the vaccine, as remarkable as it is, pales in comparison to the drug I'm waiting for.  

The drug I'm waiting for is one I've waited for for over a decade.  It's a drug that has the potential to change my son's world forever and to extend his life expectancy.  The drug I'm waiting for is Trikafta.

Trikfata is the first ever drug to treat the underlying cause of cystic fibrosis to which Bennett will have access.  It's not a cure but it's considered "a game changer."

Any day now, Vertex, the pharmaceutical company who makes Trikafta, is expected to submit paperwork to the FDA for approval for it's triple combination modulator therapy.  At one point several months ago, Vertex said they expected to do so by end of Quarter 4.  I've been on the countdown for months. 

As of today, there are only 10 business days left in Quarter 4.  (Do you see me pacing the floor?!) 

No one outside of Vertex knows when they will submit their application to the FDA.  COVID19 and other issues may force Vertex to wait to submit in 2021.  

Even after they file with the FDA, it will likely be another few months before the FDA approves the drug giving us access.  I have no choice but to be patient.

But knowing access to Trikafta is *so close* but still so far away can feel intolerable for me as a mother of a child with CF.  Like a toddler given permission to have candy but forced to wait until it's unwrapped, waiting at this point feels excruciatingly difficult.  Every day we wait is one more day Bennett is at risk for further lung damage.

It's bittersweet to listen to the stories of people with CF currently on Trikafta (it's already available for adults with CF with qualifying mutations).  Their stories of weight gain, no more coughs, fewer hospitalizations, less treatments, before and after pictures are just amazing.  It's super exciting.  I want my child to experience it too.

Of course, nothing is for certain.  Just like the COVID vaccine isn't 100%, neither are we guaranteed that Trikafta will work for Bennett.  We won't know until we give it a good try.  But all signs seem to point to this drug greatly benefit Bennett.

Bennett will still have CF and there are a lot of parts to CF that Trikafta won't be able to fix.  But it will hopefully stop this progressive disease in its tracks, slowing it's progression and giving Bennett a better quality of life overall.

In March, I watched the world become preoccupied with social distancing, trying to stay well from a respiratory disease.  The world began doing many of the things we in the CF community had been doing for years.  I felt thankful to no longer feel so alone in it.  

Ironically, I feel the same sense of solidarity. Maybe others are looking at the vaccine and I'm looking at Trikafta.  But, either way, there's a sense that we're all in this together: each of us is patiently waiting for our own access to a medicine that offers us hope.