A Glimmer of Light

By June 16, 2013October 30th, 2016Tracy's Blog

Last Thursday started and ended with depressingly similar phone calls, a pair of dreary bookends on either side of a rainy day.  The morning call came from London and the evening call was from California.  But despite the geographic chasm and the distinct accents, the dialogue was eerily parallel.  First I spoke to a Duchenne dad whose  11 year old son has rapidly deteriorated in the past few weeks.  The boy was remarkably mobile until a couple of years ago.  Then subtle signs of decline started to appear and now, rapid loss of function.  He is almost exclusively using a wheelchair and is having trouble with daily tasks he could do independently just weeks ago: getting into the car, walking from class to class in school.  Our conversation ranged from the practical – what kind of car can be adapted for a wheelchair and doesn’t cost an arm and a leg?  To the deeply emotional – the young man desperately wanted to stand on his own two feet at his bar mitzvah, but now he is closing in on 12 years old and fighting tooth and nail to take a few steps.  It was a gut-wrenching call.

Just before bedtime I caught up with a friend in Los Angeles whose son is also on the brink of losing his ability to walk.  Despite having tried every supplement and alternative treatment available, this family could not stem the tide.  Duchenne is not one of those diseases where you can beat the odds.  Where you can get better.  Where you can control the outcome with discipline and diet and supplements and physical therapy.  It is predictable, it is powerful, it moves very very fast.

How fitting that the clinical trial we are now funding is referred to as “Halo,” because it really does provide a glimmer of light in a terrifyingly dark situation.  The first trial participant, a 13 year old from the midwest, has successfully passed the screening and will be dosed in two weeks.  “Halo” (technically known as HT-100) has the potential to prevent and possibly even reverse fibrosis, a major component in the downstream pathology of Duchenne.  To date, 17 nonprofit foundations have supported the development of this hopeful new medicine.  Who says our community is fragmented?!  We are united in the push to get this drug through clinical testing to determine if it can slow the rapid muscle wasting wrought by Duchenne.  Thank you to all the families who are signing up to enroll their boys in this clinical trial at five hospitals across the US.  The faster we enroll the 30 boys, the faster we will have answers.  And the better shot we will have at getting out in front of this disease that has so far been unstoppable.  We are gaining on Duchenne – it’s time to break out into a sprint and decisively pull out ahead.