Experimental drug gives Bay Ridge boy hope
Pietro Scarso, 6, has Duchenne muscular dystrophy
A Bay Ridge boy bravely battling a rare and deadly form of muscular dystrophy is finding new hope in an experimental drug he is taking as part of a clinical trial 3,000 miles from home.
Pietro Scarso, 6, suffers from Duchenne muscular dystrophy, a disease that affects a patient’s motor skills and hampers the ability to breathe. Pietro and his parents, Manni Scarso, a broker of private jet sales, and Dayna Scarso, a stay-at-home mom, are currently in California, where Pietro is receiving once-a-week infusions of Eteplirsen, a drug that has shown great promise in previous clinical trials.
Pietro is receiving the infusions at the University of California at Los Angeles (UCLA) Medical Center.
Most children afflicted with Duchenne muscular dystrophy wind up in wheelchairs by age 12, and many die before they reach their 30th birthdays, according to medical experts. The vast majority of patients are boys. The disease affects one in every 3,500 children.
Pietro was diagnosed at the age of three.
Since May, the Scarsos have traveled to hospitals as far away as Iowa, Florida and California so that Pietro can undergo pre-screening for the clinical trial.
Dayna and Manni are relentless in their determination to help their son and other boys like him and have waged a public awareness campaign to alert people about Duchenne muscular dystrophy. A few years ago, the Scarsos formed a nonprofit organization called Pietro’s Fight to raise funds for research into drugs to help patients.
Since its founding, Pietro’s Fight has raised more than $750,000 through events such as dinner-dances, 5K races, children’s amusement park visits and pajama days at schools.
“The community has really rallied around the family and supported Pietro’s Fight,” Samantha Altilio, the organization’s executive director, told the Brooklyn Eagle. “Pietro is a miracle boy. He’s going to beat the odds.”
The Scarsos had always been hoping to get Pietro enrolled in an Eteplirsen clinical trial being run by Sarepta Therapeutics, a private drug company. But until this year, the company had not conducted clinical trials with kids as young as Pietro. Previous trials involved adolescents.
All of that changed on May 14, when Sarepta Theraputics suddenly announced an enrollment period for a new clinical trial in which Eteplirsen would be administered to younger children.
There was a catch, however. Only 20 children in the entire country would be selected to take part in the clinical trial.
But the Scarsos had determination and perhaps a stroke of luck on their side.
“May 14, 2015 was also Pietro Scarso’s sixth birthday,” the Pietro’s Fight organization said in a statement.
The family’s first stop was UCLA, where Pietro underwent an initial pre-screening process.
“Everything went great, and the first part of the process was done. They did basic physical therapy testing, blood work etc. Pietro met the standards. Next stop, we traveled to Gainesville, Florida to Shands Children’s hospital for an MRI. The hospital allowed for two-and-a-half hours for the MRI. Pietro was able to complete it in 45 minutes. The staff was amazed at how cooperative he was. With that, Pietro successfully completed a second round of pre-screening,” the parents said in a statement.
The Scarsos’ third stop was the University of Iowa Medical Center, where a port was placed in Pietro so that the drug could be administered to him more easily.
Next, it was back to UCLA Medical Center, where the infusions of the drug would take place.
“Eteplirsen is given intravenously to Pietro once a week. The port becomes an easier connect instead of searching for his vein every week,” the Scarsos said.
Pietro successfully received his first infusion of Eteplirsen on July 8.
Dayna Scarso said she realized that the clinical trial had brought out the best in her son.
“Earlier that day I told Pietro how proud I am of him and how strong he is, and he replied, ‘Ma, I’m not strong; that’s why we are here.’ I said, ‘The contrary, my dear boy. God made you with a strong heart and a strong mind.’ And I thank him for that every day,” she said.
The infusion of Eteplirsen began what will be a 96-week clinical trial.
“Our journey has just begun, but the light at the end of the tunnel is certainly getting brighter,” the Scarsos stated.
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