Monday was a holiday, so we’re talking about my Tuesday morning thoughts…
I was in California for the past week, which you’ll hopefully see as my reason for not blogging much. We’re a small time operation here. I can only do so much at once!
I had the chance to join the Global Genes RARE Patient Advocacy Summit, which was a meeting of advocates (some of them patients like me!) from all over the rare disease community. It was full of a number of informative talks ranging from setting up a non-profit, to developing a patient registry in an illness community. From the outside looking in, the “rare disease” community is where we were with cystic fibrosis 20-25 years ago. Many of these groups don’t have specific medications and are kind of just getting off the ground…BUT that’s why they have these conferences.
Cystic fibrosis is very much an outlier in the world of medicine. We’ve had THREE disease-specific medications go from the test tube to the patient in the past few years. That’s unheard of in healthcare. If anything, I hope that our experience and success in creating change community-wide is something that inspires the rare disease world.
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I had the tremendous opportunity to share the main stage at the @globalgenes RARE Patient Advocacy Summit with none other than Mike Porath, the founder and CEO of @themightysite !! We spoke about “stories with impact” inside the rare disease community and the importance of patient-centric leadership. Thanks to Global Genes for the amazing opportunity! #cysticfibrosis #cffighter #raredisease #RAREis #cysticfibrosisawareness #2018GGSummit #spoonie #chronicillness
I had the chance to speak on the main stage with Mike Porath, the CEO of The Mighty. We spoke about the importance of sharing stories within the chronic illness community and how we can use each other’s experiences to help guide us through our own lives. My favorite part of the session, though, was recognizing my “CF mentor,” Amy before hundreds of rare disease community leaders.
(You probably know who Amy is if you’ve been reading her Drug Development Wednesday posts).
Behind the scenes, I had been conspiring with Mike and Global Genes to recognize my friend Amy. Amy has done so, so much for me over the past 10 years we’ve known each other. She’s the person that I have gone to (and will continue to go to) with questions about CF care and community needs.
You can take a minute to read about the moment in my Instagram post below!
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Since people with cystic fibrosis can transmit Multidrug-Resistant Bacteria between each other, this is what it looks like when two people with CF meet – we’re masked up with N95 grade respirators and beyond the minimum safe distance for respiratory droplet transmission. 😷😷😷 As a result of what we call “cross-infection” many of us have very deep Internet friendships. Amy has been my “CF mentor” for about 10 years, and we finally had the chance to “see” each other at the @globalgenes RARE Patient Advocacy Summit. Amy is 37 years old with CF and was one of @boomeresiasonfoundation’s first scholarship winners! She’s been through a lot in her life and has made it her responsibility to make sure I have been prepared for the realities that cystic fibrosis has thrown my way as I’ve gotten older. 🤧🤧🤧 What Amy didn’t know was that behind the scenes I had been conspiring with Global Genes to have her recognized at the conference during my talk before a room of hundreds of rare disease leaders. It was one of the greatest moments I have ever been apart of. 👏🏻👏🏻👏🏻 Amy has done more for me than she’ll ever know, and is a huge reason why I’m still alive today! So, although this is as “close” as we’ve ever been… she will always have a place inside my heart. #cysticfibrosis #cfirl #chronicillness #raredisease #cffighter #2018GGSummit #cysticfibrosisawareness #infectiousdisease #infectioncontrol
The Vertex Labs
The Vertex building (which actually looks like a set of lungs!) in San Diego is the most amazing place on Earth – it’s where the magic happens. It’s where the people (a hell of a lot smarter than me) discover the compounds that create Kalydeco, Orkambi, Symdeko and whatever else hopefully comes next.
I talk a lot of about drug development and bringing medications from the test tube to the patient, but prior to last Thursday, I had never actually seen what the process looks like outside of a clinical drug study.
Let me tell you… It is impressive.
You know the feeling when you walk through your grandparents house and your parents tell you not to touch anything? That’s basically what it was like to walk though the lab. Just about everything in there looked like it would break into a million pieces even if the slightest breeze passed through the place.
In fact I actually broke down in tears just talking to a number of the scientists because I was finally looking at everything that we have done for the past 25 years. The experience at Vertex served not only as some validation for the years of blood, sweat and tears, but also as a motivator to keep pushing forward – to keep pushing until we have effective life changing medications for everyone living with cystic fibrosis.
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It’s not often you get to meet your heroes… yesterday I did. *** In our line of business there is a lot of false hope. The “next big thing” or the “golden ticket” to alleviate suffering often turns out to be nothing at all. The median age of death for people with CF – 30 years – has hardly moved over the past several years, and as I’ve gotten closer to that mark, I’ve begun to feel the wrath of the beast living inside me. *** What Vertex is doing for people with cystic fibrosis is nothing short of a miracle. *** Yesterday I had the absolute honor to meet the researchers who are striving to end the suffering that comes with a CF diagnosis, and as one of them said to me, “our late hours at the lab, our hours away from our own families are worth it… so that you get to have more time with your family and friends.” *** We’ve been at the fundraising game for a long time – $140 million in 25 years – and to see how it is finally paying off with Vertex’s line of successes brings tears to my eyes. Our visit to Vertex’s lab was more than I could have ever hoped for, and I am so grateful for what they are trying to accomplish. *** They asked for some advice, and so I said, “go faster”
…and that’s what I’m think about this morning.