Something happened in Boston…

Posted by Karen - October 30th, 2016

Just this week, an amazing event took place in Boston, USA. Yep, it was to do with fibrodysplasia ossificans progressiva (surprise surprise). As a board member of the International FOP Association (IFOPA), I could have attended, but I had a significant work conflict which couldn’t be avoided. Then, just a few weeks beforehand, the conflict disappeared, but I found out that my long-awaited carpal tunnel release surgery was to be scheduled for a few days prior, and of course I would need recovery time. Long story short? I couldn’t attend the FOP event – but wow, I so wish I had been there.

So what happened? It was, in fact, the second ever IFOPA Drug Development Forum (DDF). This was a 2 day long conference hosted by the IFOPA in Boston, and the invitees were scientists, physicians, and representatives of pharmaceutical companies, all of whom are working on (or planning to work on) developing medications to treat FOP. In addition to the science folks, there was also a select number of people with FOP and/or their parents who took part in patient panels, the purpose of which was to introduce the attendees to the perspectives of patients.

A friend of mine, Wendy Henke, attended the DDF. She has a child with FOP, and here’s what she had to say about the event:

Over 150 FOP researchers, clinicians, and pharmaceutical partners from more than 30 institutions in 13 countries(!) shared their insightful discoveries (some not yet published), welcomed challenging peer-review, and sought input from patients.

Awesome, incredible, amazing, encouraging….like a Halloween bucket full of all the best candy, words cannot describe how deliciously, deliriously SWEET the taste of progress and HOPE tasted.

What a great description! It sounds like it was just an astounding conference (thanks Wendy for these terrific words). Along the same lines, I also communicated with one of the scientist attendees – he said he had never seen so much pharmaceutical interest in such a rare disease, and he felt it was extremely promising.

This is all so good, and it makes my heart sing.

If you’re interested in seeing the subjects presented at the DDF, you can read the agenda here: . The whole thing looks great to me, but what would I most like to have sat in on? Presentations and discussions on repurposing existing drugs for FOP, since that would likely be our quickest route to slowing or stopping the progress of FOP. “WHAT DO WE WANT? A MEDICINE FOR FOP! WHEN DO WE WANT IT? YESTERDAY!” (LOL, but so true)

Anyway, why so much interest among pharma companies in a drug for FOP? There is no doubt in my mind that any researcher meeting someone with symptomatic FOP would be extremely moved by the patient’s plight, and want to do whatever’s necessary to end the scourge. However, given how rare FOP is, your average scientist is not likely to ever even see a person with FOP outside of a conference like the DDF. That desire to help is only going to have developed after they have entered the door and experienced an FOP patient panel. Long before that point, most of them were hooked by something else… In all probability, they became interested because a treatment for FOP has significant potential to treat many common bone disorders such as osteoarthritis, ossification following surgeries and amputations, heart valve calcification, and more, conditions which affect millions of people. To be frank about it, this is a great opportunity – so why not get in on the ground floor? (Aside – I am perfectly A-OK with this being the initial motivating factor. Whatever gets us where we want to be is just fine with me…)

My daughter Miranda, who is 11 and has FOP. She is why this is so important to me. There's nothing I want more than a way to stop her FOP.

My daughter Miranda, who is 11 and has FOP. She is why this is so important to me. There’s nothing I want more than a way to stop her FOP.

I am so looking forward to learning more about FOP drug development spurred by researchers who attended the DDF. On your mark, get set…. GO!


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