On his sixth birthday, Pietro Scarso, the young boy who is the poster child for Pietro’s Fight against Duchenne Muscular Dystrophy (DMD), learned that Sarepta Therapeutics had announced the opening of enrollment for 20 four-to-six-year-old children diagnosed with DMD in its trials of a drug that is hoped to be a breakthrough in treating the disease.
“Our journey has just begun, but the light at the end of the tunnel is certainly getting brighter,” said Dayna, Pietro’s mom, of the news that her son would be eligible for the clinical trial of Eteplirsen.
The Eteplirsen trial’s initial consistent favorable results in older boys led Sarepta Therapeutics to open the door for younger children with DMD, a fatal degenerative disease that weakens muscles at an aggressive pace.
Most children with DMD are confined to a wheelchair by the time they are 12 years old and die before they turn 20 from respiratory complications.
Upon receiving Pietro’s diagnosis when he was three, his parents, Dayna and Manni, started Pietro’s Fight with the purpose of raising funds for research that will find a cure for DMD and ensure that children with DMD have the opportunity to enjoy a long, full life. There are currently no medications for this condition, and Pietro’s Fight invests in translational research to focus on moving science from the lab into human clinical trials.
“Pietro’s Fight has funded numerous amounts of drug companies and Sarepta Therapeutics is one of them,” said Samantha Altilio, a spokesperson for Pietro’s Fight.
After traveling across the country to multiple destinations in Florida, Iowa and California from his home in New York and enduring blood work, physical therapy testing and an MRI, Pietro successfully completed the prescreening process and was selected for the 96-week trial.
In Iowa City, Pietro underwent a muscle biopsy and port placement. Pietro’s port becomes an easier connect for his weekly intravenous Eteplirsen treatment instead of searching for his vein every week.
“Though the port was shocking at first to see it, is now his ‘Green Lantern’ pack and gives him the strength he needs to get infusions once a week,” said Dayna, Pietro’s mom.
On July 8, 2015, Pietro successfully received his first infusion of Eteplirsen. Wednesday, July 15 was his second treatment.
“He has no side effects, whatsoever,” Altilio said. “The older kids that are on it aren’t showing any side effects either.”
Pietro’s Fight has four major funding events throughout the year. Its next event will be a 5k run on Saturday, September 19 at the 69th Street Pier at the 9/11 Fireman’s Memorial.
Pietro’s supporters are optimistic.
“This is a miracle drug, and I just think Pietro is going to be the miracle boy for this community,” Altilio said.