Wait, what? What’s that about FOP and Facebook…?

Posted by Karen - September 6th, 2015

OK, get this – earlier this week, fibrodysplasia ossificans progressiva (FOP) was actually TRENDING ON FACEBOOK! Never in a billion, gazillion years would I have ever thought anything like that could happen. I mean, come on, this disease is a 1 in 2 million occurrence, and yet people talked about it so much this week that it became a “thing” on social media.┬áTo be honest, I didn’t actually see myself that it was trending. However, a handful of my Facebook Friends posted that they had seen this (I think it was trending as Stoneman’s Syndrome, a title I never like, but whatever), and some even had photos of their computer screens as proof. Amazing!

Aside – just in case you don’t know what “trending” means… See, on a social media app such as Facebook or Twitter, it’s possible to enter a search term which the app will then use to pull up a bunch of stories about that subject. A topic is described as “trending” if a large number of people are posting things about that subject. In Facebook, you can find out that something is trending by looking at the list of trending subjects posted on the side of the Facebook screen – on mine it’s on the right side, just beneath notification of event invitations and Friends’ birthdays.

So what caused this sudden explosion of interest in the ultra-rare disorder which affects my 10 year old daughter? Well, fabulously, it was a research finding which could potentially be very important. A pharmaceutical company called Regeneron published an article this week in which it announced a major finding. In short, Regeneron figured out that the FOP version of gene ACVR1 reacts to a substance secreted by the immune system (“Activin A”) in the face of inflammation, and that substance causes the gene to go haywire and produce a bunch of unwanted bone (the non-FOP version of ACVR1 doesn’t react this way to Activin A). Even more importantly, Regeneron has developed a substance which was shown in the lab to block Activin A in mice with FOP and to stop the rogue bone formation. This news was reported all over the place, generally as some version of “Scientists find possible cure to Stoneman’s Disease”, as in this photo…


Now, of course I must point out that this finding ISN’T actually a cure for FOP. For one thing, the new potential drug has yet to be tested on people, and though the scientists are optimistic that the human gene will behave the same way it does in mice, they don’t know for sure that it will. There’s still lots of testing to do, and the drug will have to enter clinical trials. For another thing, even if the new medicine does work on people, it will treat FOP rather than cure (eliminate) it. Also, this isn’t even the first really promising finding in FOP which is leading to a drug trial – right now, as we speak, Clementia Pharmaceuticals is testing a potential FOP drug which blocks cartilage (pre-bone) formation.

Regardless of any inaccuracies in how it’s being reported, the new finding is actually quite significant scientifically, because it may possibly explain the elusive and poorly understood connection between the immune system and the FOP gene. This really is a major finding, though I think the explosion of social media interest is probably not due to the scientific merit of the issue but rather the opportunity to trumpet a “cure” for a “bizarre disease” (sigh). Anyway, whatever the reason, we’ll take it! Talk about spreading awareness of FOP… Very cool.

Actually, this really was an incredible week for FOP… Just recently, Clementia Pharmaceuticals announced that it’s opening up its current ongoing testing of the potential FOP drug Palovarotene to children age 6 and up. Any child in that age range with the typical FOP mutation (which affects 95% of FOP patients) who suffers a new FOP flare-up on a shoulder, arm, hip or leg may be eligible to enrol in the drug trial. This is really exciting. While nothing has been announced about the efficacy of the drug in adults, I can’t help but think that the drug trial wouldn’t have been opened to kids if the findings so far were negative (yes, this is pure speculation on my part, but still). Since the time of the announcement, my daughter hasn’t yet suffered an eligible flare-up, but if she does, you can bet I’ll be getting the ball rolling to see if I can get her in on the testing. If she does enrol, she won’t be the first child… This past week, a 9 year old boy L became the first child, or one of the first, to enrol. I and my family wish him the very, very best of luck with the testing.




2 Responses to “Wait, what? What’s that about FOP and Facebook…?”

  1. Susie says:

    I think it is wonderful that it was trending. The more we spread the word on FOP the better!

  2. Jonathan says:

    Make sure to get your daughter in the trial before she has a flare up! That way they have all your info and things set up. You have limited time once it starts and you don’t want to waste time filling things out.

Leave a Reply