Hope on the horizon…

I swear I am trying to get better about blogging.  Each time I post, as I click the “publish” button sending my ramblings into cyberspace, I make a promise to myself that I will blog more often.  Then I procrastinate, followed by total avoidance.  When I do finally get down to business, I obsess over every word.  I worry that nothing I write will be eloquent enough, or profound enough that I should ask you to read it.  Today I realized that even worse than the risk of boring you with an excess of insignificant and clumsily worded blathering is the risk that you will assume the infrequent posting is attributed to a lack of exciting advancements to report, which could not be further from reality.  So here goes…

THE TIMES THEY ARE A CHANGIN’

Stasia and I headed down to DC a couple weeks ago to attend the most comprehensive annual DMD conference in the world.  We participated in two intense days of scientific presentations as well as visited with the health liaison for our Senator, Chuck Schumer.  I’ve been to this conference six times since Javier was diagnosed.   My first time there, there was just one drug in human clinical trials – a drug that won’t work for Javier’s genetic mutation.  A lot of the parents I met complained that nothing was changing, that the scientists seem to say the same thing year after year.  Fast forward to today and that same drug has received conditional approval in Europe and will be submitted to the FDA for use in the US in 2016, there are over 25 studies actively recruiting patients.  We take this as a strong signal that hope is on the horizon.  

WHAT THIS MEANS FOR JAVIER…

A lot of promising clinical trials is what we asked for and finally!  We are getting what we have been asking, begging and praying for.  You know the saying, “be careful what you wish for”?  The downside to the increasing number of trials – and it kills me to say anything negative about the auspicious position this patient community now finds itself in – is that we have to choose very carefully.  As of a week ago, Javier was enrolled in a clinical trial that he was to start next week.  Then Stasia and I watched a biotech’s presentation of a drug that appears to be doing amazing things (disclosure: Hope for Javier has invested in this drug’s development).  They are recruiting two new cohorts of patients for what will be an intense trial, not for the faint of heart (well technically it is for the faint of heart – and everything else, but you know what I mean).  If Javier were to start the other trial he was committed to, he would be made ineligible for any new trials – including this very promising drug.  It’s a long shot we will even get into the new trial (there are just five slots to fill, only two of them for boys who no longer walk – and 7 kids ahead of Javier) – but a long shot is better than none.

THE ASK

Whatever god you pray to – and even if you don’t normally pray – we need your positive thoughts and prayers that a space in this promising clinical trial – a space made just for Javier – opens up.

OUR BOLD ENDEAVOR – THE HEALTHCARE INITIATIVE…

Since Javier was diagnosed, we’ve traveled to Cincinnati every year for care.  Hard to believe since we live here in NY, the epicenter for healthcare, right?  That is all about to change!  Our plans to transform care for NY / NJ / CT area boys with DMD has taken a giant leap forward and continues to move ahead at breakneck speed.  We are working hard, collaborating with an amazing team from Stony Brook Children’s Hospital and will be updating you very soon on this exciting project!

That’s it – I’m stopping now because I don’t want to “laundry list” you any more than I already have.  And I know myself too well to end with a solemn promise to blog at least once a month. Instead I ask that you think of Hope for Javier the next time you need a gift, the next time you have a birthday, or like our board member Natasha Kardos (née Pirogovsky), who to Javier’s dismay just married someone else, someone a little older – and asked her guests to donate in honor of their joyous occasion.  Congrats Natasha + Philip!