Takeaways from Boston

After speaking  at two amazing conferences this past fall (Raina and me in Boston and Raina and Mike in St. Louis) among many more hours of listening to other FOP families, researchers, clinicians and board members, we walked away with new heroes and so much more hope in our united fight for the cure.

We were blown away when we saw how everyone involved is going above and beyond to work together and do all they can to share knowledge, insights, questions and experiences in our unified battle against the devil that is FOP.

There has been so much promising knowledge gained and explored over the last year - surely more than ever before - and we learned in Boston that there are an astounding number of universities (including Harvard, Oxford, Penn, and ~25 more), pharma & biotech companies, and other research organizations that have entire TEAMS actively researching and exploring a multitude of paths towards an effective treatment.

And they are collaborating and working together so efficiently, which is astounding to see (especially for pharma). 

Here are our key takeaways:

This has all been happening at an unbelievable rate since the landmark discovery of the FOP gene in 2006 by the team highlighted here, all three of whom were present at Raina’s clinical appointment in St. Louis and at both conferences. These people are masters of their work and heroes of the entire FOP community. And they are incredibly kind, devoted and ultimately responsible for the hope we continue to speak of for Raina and everyone in this community.

boston girl 2.jpg

The rate of research, development and collaboration has increased exponentially in the last 17 months thanks to the breakthrough discovery highlighted here. Aris Economides, the hero in this story, was present and passionate at both conferences. Rumor has it that he works overtime weekly out of persistence towards the “curative FOP treatment” his team is working on, which is drug #2 below.

It only took until dinnertime at the conference in St. Louis, 2 weeks after the Boston conference when he introduced himself to her, for Raina to finally talk to him (he tried several times but has a Greek accent that threw her off). Once she gave in she clearly won his heart with a hug and a high-five. He is a parent, too, and I think his time with Raina helped to further inspire his career’s devotion (over 25 years) to beating FOP. Here’s his bio if you'd like to know more.

Aris with Sienna Otto, Raina's buddy who also has FOP

Aris with Sienna Otto, Raina's buddy who also has FOP

Which leads us to...

Drug #1: Palovarotene 

This drug is considered safe and shows potential for FOP. They just need to fine-tune dosages, how it will work acutely vs. chronically (and long-term), and a few other things to fill the FDA requirements. Clementia, the company who is pushing it to market, was formed with the primary focus of bold, efficient progress towards a solution / treatment / cure for FOP. This company is dedicated to changing the world of FOP as we know it. They are very close to getting it to market - so much so that there has even been mention of a possible surgical trial - something that is typically considered dangerous for anyone with FOP. The link above tells their story and that of Palovarotene very well.

Drug #2: The Anti-Activin A Antibody (REGN2477)

We’ve got most of our money on this one. Read about the accidental breakthrough discovery that led to the swift development of this drug here, here, here and here

There’s not much press or articles out about the antibody itself yet, but it has actually been to human trial already - in healthy human volunteers (adults with no risk, disease or pregnancy). There were no problems or safety issues found, so the first trial in FOP patients is expected to occur by mid-2017.

There has been amazing promise with the antibody in animal testing showing its effectiveness on HO (bone growth) in FOP. Paloverotene is exciting and promising, as well, but if this antibody works, it could be the “curative treatment” everyone has dreamed of…and Aris might finally be able to get some sleep.

On top of all these heroes and hope we found in Boston, we also have tear-jerking memories of cupcakes.

When Raina and I were on stage telling our story, it just happened to be her 5th birthday and did not go unnoticed. The entire conference sang Happy Birthday to her as members of the IFOPA brought her a cupcake and lit candle that she quickly blew out and enjoyed right there on stage. 

Raina was ecstatic to share her birthday with the new IFOPA executive director, her "birthday buddy" Michelle Davis, who gave her two presents at the conference. When we saw her again two weeks later at the IFOPA Midwest Gathering in St. Louis, Michelle was her "best buddy" and she looked for her everywhere we went that weekend.

There is so much more to say, and I have pages and pages of notes on a ton of studies, breakthroughs, data and questions from the Drug Development Conference. It's a lot of science that I'm happy to provide to other nerds like me upon request.

But I’ll leave you with the following two quotes that stuck with me the most after the two conferences. These words basically sum up the hope, status and promise of progress in the global battle with FOP, and we are beyond grateful that we have entered this inspiring and motivated community at a time like this:

“The first FOP Drug Development Forum (in 2014) felt like everyone was finally joining together and heading down the same path. This time it’s a sprint to the finish line.” Boston (IFOPA Research Board Member Eric Otto, whose daughter Sienna has FOP)

“Very few diseases this rare have ever received as much attention, effort or momentum as FOP. Unlike most of them, FOP has a characteristic that has become increasingly clear since the gene discovery: it’s very druggable.” St. Louis (Aris Economides) 

I’m in tears yet again over FOP. In a good way.