Mountain Climbers From Manalapan Cited For Duchenne Fundraising

MANALAPAN, NJ — Three people, including two Manalapan residents, actually climbed a mountain to raise funds for a new clinical trial to treat Duchenne muscular dystrophy, a progressive disorder that affects about 300,000 children worldwide.

Jim Raffone and Joseph Russo of Manalapan, as well as Matthew Scarfo, climbed Mount Everest for the cause earlier this year and they were recognized in a flag presentation by Rep. Chris Smith, R-Manchester, on World Duchenne Awareness Day on Wednesday in Washington, D.C.

The new, clinical-stage treatment supported by the Raffone Family's nonprofit organization JAR of Hope uses umbilical cord lining stem cell therapy, which is derived from cord blood that has proved efficacious in treating over 70 diseases, including leukemia, sickle-cell disease and lymphoma, a news release from Smith's office said.

"Chris Smith first introduced us to the idea of the healing effects of umbilical cord stem cells, and we remain determined and optimistic that this unique study will help us reach breakthroughs for kids suffering from Duchenne," said Raffone, who founded JAR of Hope in 2013 when his then 4-year-old son James Anthony was diagnosed with Duchenne muscular dystrophy.

On Wednesday, Smith and the climbing team reached the summit of the U.S. Capitol Building on a Dome tour led by Smith, in addition to receiving American flags flown over the Capitol in their honor, Smith's office said in the news release.

"The launch of this new clinical trial harnessing the power of stem cell therapy brings new hope for kids with Duchenne as we continue to strive to cure this devastating disease," said Smith, who authored the Stem Cell Therapeutic and Research Act of 2005 that created, for the first time ever, a nationwide umbilical cord blood stem cell program.

At Wednesday's meeting, Smith and Raffone also discussed next steps to secure more funding for the study, including meetings with appropriate federal agencies such as the National Institutes of Health, as well as pursuing funding through the congressional appropriations process.

Duchenne muscular dystrophy - which currently has no cure - causes a loss of both muscle function and independence with a 100 percent fatality rate occurring usually in the late teens or early 20s, Smith's office said.

Raffone has taken in part in all sorts of challenging fundraising efforts - such as walking to Washington and boxing in Atlantic City and running marathons. He's completed some of the hardest races on the planet, ranging from a seven-day, 171-mile run through the desert to a 12-day trek throughout New Zealand and Australia. He's the owner of six world records in his age group, all to support raising funds to find a way to fight Duchenne, say news articles about him.

Smith's Stem Cell Therapeutic and Research Act established the nationwide umbilical cord blood stem cell program - known as the National Cord Blood Stem Cell Inventory - designed to better collect, derive, type and freeze cord blood units for research and transplantation into patients in order to mitigate and even cure serious disease, Smith's office said.

Smith, who has worked with the Raffone Family for years to raise awareness and increase federal funding for Duchenne treatments, said the long-awaited stem cell trial "marks a major leap forward toward potential life-saving care for patients as scientists continue to study and better understand the regenerative effects of cord blood cell therapies."