Duchenne muscular dystrophy (DMD) nonprofit CureDuchenne is looking to spark public action through a new national public service announcement (PSA) campaign shining a light on what birthdays look like for patients with the progressive degenerative disease.
CureDuchenne has so far raised over $50 million in funding for research, education and care in its 20-year history and has invested $28 million in scientific advancements, supporting companies responsible for six of the eight FDA-approved therapies for the disease. However, the array of marketed treatments still falls short of a cure for the 300,000 people worldwide impacted by the disease, the organization explained in a Wednesday press release.
“As a mother, I know what it feels like to watch your child grow up and want nothing more than to protect their future. For families living with Duchenne, every birthday carries that love, and the need for a treatment that will stop this disease's relentless progression," CureDuchenne’s founder and CEO Debra Miller commented in the release. "What stands between today's research and tomorrow's cure is funding, and we're calling on the public to join us in closing that gap.”
The “A Cure Can’t Wait” PSA, which launched July 8 across broadcast, radio, digital, streaming, and out-of-home channels, showcases a montage of several different iterations of a child’s birthday party as his disease progresses over the years. After his first birthday post-DMD diagnosis at age five, the scenes continue to switch to birthday celebrations during his progressively non-ambulatory teenage years as a voiceover explains that each birthday for parents of children with DMD is “a reminder that the clock is ticking, and I will outlive my child.”
By the end of the spot, the child’s mother stands alone holding a birthday cake before on-screen text appears, explaining that “historically, those with Duchenne muscular dystrophy have not lived past their 20s. CureDuchenne is changing that.”
The campaign is dedicated to Andrien Joshua Quintero, a 23-year-old DMD patient who appeared in the PSA as he “believed deeply in the power of awareness to change lives,” CureDuchenne said. Quintero passed away in May of this year, shortly after filming the campaign.
“We carry his memory forward with urgency, because a cure cannot wait,” Miller added.
Independent agency Horizon Media is also helping the cause by donating media placement services to bring the PSA to a wider audience.
"At Horizon Media, we believe that media has the power to move people, and this campaign is a powerful example of why that matters," Horizon Media CEO and founder Bill Koenigsberg said. "When we heard what CureDuchenne was trying to accomplish, and the story behind it, we wanted to do everything we could to make sure as many people as possible see it. We are honored to play a role in this campaign."
DMD therapies have historically had a rocky road to commercialization, with Sarepta Therapeutics’ Elevidys most recently receiving a label restriction to ambulatory patients four-years-old and up and a new boxed warning after safety concerns threatened the drug’s commercial future. Elevidys was the first drug to win FDA approval for DMD treatment in 2016 and benefited from CureDuchenne’s help in clinical trial development “very early on” in the program, former Sarepta chief Ed Kaye, M.D., has said.
Meanwhile, positive developments in the DMD treatment landscape are also emerging with Satellos’ six-month data showing its phase 1a/b candidate SAT-3247’s benefits in adults, which could “re-imagine the ... therapeutic landscape” if replicated in children, Guggenheim Securities analysts pointed out.