FDA-approved treatment for ADPKD gives patient community hope and drive

We reached out to Board of Trustees member and long-time PKD Foundation supporter Bill Brazell to get his thoughts on how the news of our first treatment will impact the PKD patient community:

Tuesday, April 24, was a spectacular day for American PKD patients: The U.S Food and Drug Administration (FDA) approved JYNARQUE™ (tolvaptan). We can finally join our fellow PKD patients in Canada, England, Japan, and the EU in saying that we, too, have access to a treatment that will slow the growth of our cysts.

We are grateful to the PKD Foundation for funding the early studies of tolvaptan and then for helping to find PKD patients who could test it — and to all the patients who participated in those studies. And we all ought to thank Otsuka Pharmaceuticals, the company that put great effort and funds toward creating and testing a treatment that we wouldn’t have without them. Thank you!

We are grateful today, too, to the late Dr. Jared Grantham and Joseph Bruening, who started what was then known as the PKR Foundation in 1982, laying the groundwork for years of research, advocacy and support. And we appreciate as well the early leadership of researchers and the generous donors who supported that research. So many people worked so hard and gave so much to bring us to this day. And we’re not finished yet.

Many of us in the PKD patient community are also remembering loved ones who we wish had lived to see this day. I’m thinking of my cousin Michael, who died at 35, and his father Dick, who died at 64, both of PKD complications. I’m also thinking of my paternal grandmother, my Nana, whose husband, Dick’s father, dropped dead of a heart attack right in front of her when he was 44. She didn’t know he had PKD. (Neither did he.) And of those who are alive today, but for whom this treatment has come too late.

Now, there’s a reason to hope that for our families and for ourselves, the future will be not just different, but better.

When I would tell my Nana that I was raising money for the Walk for PKD, she used to say, “Bill, I hope that Foundation appreciates what you do for them.”

I would laugh, and remind her, “Nana, it’s not for them. It’s for us — for our family — for me, for my siblings and surviving cousins, for our kids. That’s who we do this for. The Foundation is made up of thousands of families just like ours. We’re not working for a Foundation. The Foundation is working for us. We’re all contributing as well as we can to find a treatment.”

She said, “That’s all great, Bill. But I do hope they appreciate you.” Well, that’s what grandmothers are for, isn’t it?

I wish my Nana, my cousin and uncle, and your own late loved ones, were here today to see how much the PKD Foundation appreciates us: So much so, that it helped give us the very first treatment for PKD. We’ve all taken a giant step toward a day when a PKD Foundation is no longer necessary.

We’re not there yet. Not even close. But now we can all see what we can do when we work together. So stay with us! Even better days are ahead.

Questions about JYNARQUE™ (tolvaptan)? Be sure to follow our FAQs page for the latest news & updates: pkdfoundation.staging.wpengine.com/jynarque.

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