The latest on the drug trial (!)

Posted by Karen - April 5th, 2015

Hi friends, and happy Easter! On this fine Easter Sunday in Vancouver, the sun is shining and it’s looking like a beautiful day. We’ve got a nice big Easter ham in the oven, and later on we’ll have one of those awesome holiday feasts with my mother-in-law. My kids are enjoying their Easter treats, and my husband and I are sipping our fave coffees from a certain Seattle-based, mermaid featuring coffee company… Ah, feeling like a great day.


Chocolate cupcakes with Easter-inspired colours and designs which my kids and I baked yesterday.

To top it off, earlier this week we got some good news from Clementia Pharmaceuticals, the company which is currently testing Palovarotene, a drug to hopefully treat fibrodysplasia ossificans progressiva. We are keenly following this issue, because there is nothing, hands down nothing, which would mean more to us than finally having a medicine to treat our Miranda’s FOP.

At age 10, Miranda can still walk, which is great. But beginning in September of 2013, about a year-and-a-half ago, Miranda started having her first FOP flare-ups on her legs – and since that time, she’s had 2 (possibly 3), so the clock is seriously starting to tick on her mobility. That medicine can’t come fast enough for us.

So anyway, back to that good news… It seems that Clementia has now successfully completed the first stage of its first ever Phase 2 clinical testing of Palovarotene, and based on the results of the first stage, they’re carrying on with the second stage. Yay! Here’s Clementia’s announcement:

By way of background – and please keep in mind that I am no scientist, so this is based entirely on my layperson understanding – Clementia was able to skip Phase 1 tests (tests to determine basically if the drug is toxic in humans) because such testing had already been successfully completed by another company testing the drug for a different disease. Clementia was in the lucky position of being able to proceed straight to Phase 2, which has the purpose of determining whether or not the potential drug is actually treating the disease condition and having the desired effect. A company may do a number of different Phase 2 tests, depending on the need for different purposes. In any event, the current Phase 2 clinical test is about Palovarotene’s effectiveness on adults age 15 and over who are suffering from an FOP flare-up to a shoulder, arm, hip or leg. Clementia was hoping to get 8 people enrolled in the first stage of this Phase 2 test, and in fact I hear it ended up with a few more than that (all good).

Pill photo


Now, if, say, the first stage of this Phase 2 testing had revealed unforeseen major problems or no evidence of treatment efficacy, presumably the testing would have been halted. That apparently didn’t occur, as Clementia announced that it’s moving on to the second stage. Boo-ya! Not only that, but the company has also indicated separately ( ) that it’s working toward hopefully expanding the current Phase 2 test to children age 8 and over within the next several months. In all, I understand that Clementia is aiming for at least 16 people to take part in the second stage of this Phase 2 test.

Expanding the testing to kids would be so amazing. I can definitely tell you this – if Miranda were to develop another arm or leg flare-up after becoming age-eligible to take part in this testing, we’d be hopping on the plane to the nearest testing site so fast it would make your head SPIN! While I appreciate that participating in drug tests is not something everyone wants to do or can do, I can see no reason why we wouldn’t get Miranda involved. It’s not like it would even cost us anything – Clementia is covering travel costs to one of the test sites for an FOP person and a necessary attendant, even if the person needs special travel arrangements due to FOP physical limitations. Right now there are 3 test sites, 1 in Paris, France and 2 in the USA (Philadelphia and San Francisco). For us on the west coast, the roughly 2 hour plane flight to San Francisco would definitely be the way to go. My family is definitely interested in doing everything we can to move this research forward.

As regards that travel, by the way, I believe that a few short-term trips to 1 of the 3 testing sites is required (but again, all travel costs are covered).

If you have FOP and are suffering from a new flare-up to a shoulder, arm, hip or leg, and you want to know how to get involved in Clementia’s testing, check out information at the website of the International FOP Association here:

Now, does any of this mean Palovarotene is destined to become a drug to treat FOP, and it’s only a matter of time before that happens? No, not at all. The drug could yet be proven to lack effectiveness, or to be too toxic or have too many side effects – any of these things could happen. But even if that does occur, the fact remains that we’re now in an incredibly exciting era, a time when medicines targeted at the microbiology of FOP are actually being tested, and this in and of itself is a wonderful thing. Even if it turns out Palovarotene doesn’t work as hoped, there are other labs working on alternative treatments, and one of those could well move to clinical testing in the not too distant future. This is truly an amazing time.

How did that old song go? “The future’s so bright, I gotta wear shades…”

Miranda on our recent Spring Break trip. We would LOVE to see a treatment for FOP, so that she can continue having fun adventures like these.

Miranda on our recent Spring Break trip. We would LOVE to see a treatment for FOP, so that she can continue having fun adventures like these.

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