Seismic Shift Underfoot

By August 15, 2015November 27th, 2018Tracy's Blog
Charley hanging out at a friend’s pool (Thank you Silvermans for always having us over to swim — love you guys!)

A couple of weeks ago, I spoke to a woman from Miami whose 6-year-old son was recently diagnosed with Duchenne. We talked for a while, and when I hung up the phone, I realized that a seismic shift is underway.

When this family went to see a neurologist, he did not tell them, “There is nothing we can do.” He did not tell them that their son’s muscles will weaken to the point of complete paralysis, and that he will die in his teens or 20s. He did not tell them that there is no medicine to treat this awful disease.

Instead the doctor explained that their little boy is eligible to try a new treatment that produces dystrophin, the protein missing in kids with Duchenne. He told them that in just a few months’ time, two new medicines will be reviewed by an FDA advisory panel and — hopefully — recommended for approval. He told them that several clinical trials are underway to test new medicines that aim to slow muscle degeneration, improve lung function, and protect the heart. He told them that their child — like children all over the world — can consider participating in these cutting-edge treatment trials. I remember what the neurologist told me the day Charley was diagnosed, and I realize that the world has changed, right before our very eyes. We are making Duchenne history.

Charley is testing an anti-fibrotic drug that is designed to decrease scar tissue and enable healthy muscle tissue to grow. Six-year-old Pietro just started a personalized medicine that induces the body to produce dystrophin. Earlier this year, a boy named Jack was injected with a gene therapy approach called follistatin. These are incredible advancements. But a few kids at a time is not fast enough. We will not be satisfied until all children have access to safe and effective new medicines to manage the disease.

Incredibly, as we have pushed the envelope to move the research even faster, our supporters have stuck with us. You have Doubled Down, upped the ante, brought in friends, and stepped up your game. I am eternally grateful for that increased commitment from so many of you. The 2015 Charley Ride was our best cycling event yet, record breaking on every count: number of attendees, amount of money raised, level of enthusiasm. Even the weather beat out past years! Special mention must go to our corporate sponsors, also a record number this year. Of note, three of our top sponsors this year — Biomarin, Sarepta, and Summit — are all companies developing multiple new treatments for Duchenne. Our 2015 Race Against Time is coming up in October, and we’re hopeful that Racers around the country will sign up to help us extend this record-breaking streak.

We all know how progress can change lives. Remember typing your college thesis and using white out to correct the errors? Remember using a card catalog at the library to find an information source because there was no such thing as the internet? That type of historic change is happening right now in the world of Duchenne therapeutics. One day soon we will look back and say, “Can you believe this medicine didn’t exist?” It’s a revolution, and you are driving the historic change. If we keep this up and finish what we started, one day Charley will tell his own children all about it.