D-Day. We all know it, of course, as a pivotal moment in world history. The day in June 1944 when the Allied forces stormed the beaches at Normandy, precipitating the beginning of the end of World War II.
Have you ever wondered what the “D” in D-Day stands for? I’m thinking Normandy, France, Nazis, Allied forces attack, nothing even remotely related to that historic day starts with a D. Turns out, the “D” in D Day stands for “day.” It serves to emphasize the fact that this is a Day with a capital D; a day on which an important operation is to begin or a change to take effect.
Well, last Tuesday Duchenne families had a D-Day of our own.
Early results from a gene therapy trial were released. The boys in the trial were injected with micro- dystrophin, a shortened form of the protein kids with Duchenne are missing. The micro-dystrophin was stuffed into a viral vector and injected into the blood streams of young boys with Duchenne. Three months after the injection, biopsies showed that each one of the boys is producing significant amounts of the missing protein. Also something we’ve never seen before: In all four boys, an enzyme that signals the level of muscle destruction (creatine kinase) dramatically plummeted.
Finally, it really and truly feels like the beginning of the end.
While D-Day went down in history as the decisive turnaround in the effort to defeat the Nazis, it is important to remember that the war did not end on that day. ““D-Day was hailed with a sense of relief all over the world,” The Nation reported on June 17, 1944. “But people were quick to realize that a new period of tension lay ahead.”
The early results released last Tuesday are incredible, but ultimate victory over Duchenne is still pending. With this advancement, our community faces a host of new challenges. Will these early results translate to lasting, meaningful improvement for the boys who have been treated? Will this promising data bear out in a larger, more diverse group of people battling Duchenne? Will this treatment arrest disease progression in older boys who are desperate to save their arms, their hearts, and their lungs? How can we ensure that the regulatory process moves as quickly and as efficiently as possible? Will manufacturing holdups slow the drug development process? We have our work cut out for us.
It’s also critical to remember that while gene therapy looks like it could make a huge difference in the lives of many sick children, a multi-faceted attack is required to ultimately defeat Duchenne. That’s why we’re keeping up the pressure on various fronts. The storming of the beaches at Normandy is famous and historic, but it was not a standalone event. It was part of a “grand strategy” including an offensive in Italy, operations in the Balkans, and a push by the Soviets on the eastern front. Ultimate victory required all hands on deck, attacking the enemy from every side.
As I can imagine must have been the case on June 6, 1944, last Tuesday widespread jubilation was tempered by the experiences of families whose sons have been fighting this disease with everything they’ve got for years and even decades. Too many have lost their lives. For others, Duchenne has wreaked havoc on their bodies, leaving them badly wounded. At 17 years old with significant muscle weakness, Charley is among the injured. If we keep our heads down and do the work that’s laid out for us, if we keep up the pressure on all fronts, we will hasten the ultimate defeat of Duchenne. This work could not be more urgent. We need to save Charley and so many boys like him from having to make the ultimate sacrifice.
June 19, 2018 will most likely figure prominently in the Duchenne history books. Even so, the road to a decisive, resounding victory is fraught with challenges. It won’t be easy, but I’m more confident than ever before that we will get there.
“We must be prepared to withstand hard knocks, to suffer setbacks,” The Nation warned its readers two weeks after D-Day. “…but we have arrived at the beginning of the end.”