A father in New Jersey is leading the charge to cure a rare genetic muscle-wasting condition that affects about 20,000 children in the United States, nearly all of whom are male—including his own son.
“Six years ago, my son, Jamesy, was diagnosed with Duchenne muscular dystrophy. The doctors told me ‘Go home and love your son, there is nothing we can do for him.’ They said there is nothing more to do medically to reverse this premature death sentence,” said Jim Raffone, 49, founder of the West Long Branch, NJ- based nonprofit JAR of Hope. “I have declared war on Duchenne muscular dystrophy.”
Last month, Raffone entered his 10-year-old son into a privately funded treatment administered in New York City that is infusing four young boys suffering from Duchenne (DMD) with a compound that could potentially alter the trajectory of their life-threatening disorder. The compound, “JAR 914,” was created in a laboratory in California.
“There is no other greater ‘skin in the game’ than having your own child do something experimental that’s never been done before,” Raffone said of the JAR 914 medical treatment. “This is historic. So anybody who wants to get involved can really change the whole history of this disease going forward,” he says in a media release.
The treatment is being administered to the boys quarterly by Dr Anthony Salerno, with the next round of JAR 914 infusions scheduled for January.
“JAR 914 is really a novel approach to the muscular dystrophy such that we can delay the onset of symptoms, and by doing so we can afford more time for research to cure the disease,” Salerno states.
While JAR 914 is intended to delay symptoms of DMD, Raffone is also raising funds for a drug to potentially cure the disease. That drug, named “JAR 913,” was developed at University at Minnesota and has passed through a 13-month Phase I study at Biomere, a preclinical contract research organization based in Worcester, Mass, focused on drug discovery. The results were favorable enough to warrant further study of the drug, but the next phase will cost millions of dollars which will take time to raise.
“Over the years we’ve been blessed to raise the funding to get a potential cure, JAR 913, through a Phase I study. But to get it to a Phase II, it’s going to take years and millions of dollars,” Raffone comments. “Right now, if we come together, we can fund JAR 914 for thousands of dollars a month and potentially create a reaction in these children that will delay the symptoms of their disease while we seek more funding to continue the work on JAR 913.”
Raffone has named his current campaign to fund the JAR 914 treatments for his son and three other boys “Operation: Lifeline.” To convey his message that he is at war with the disease, he has taken to wearing a flak jacket during his video fundraising appeals. Anyone who donates to the cause receives an Operation: Lifeline patch with the normally light green JAR of Hope logo altered to a military camouflage style.
DMD is a terminally progressive condition that causes muscle atrophy and most affected individuals require a wheelchair by their teenage years. Serious life-threatening complications may ultimately develop, including disease of the heart muscle and breathing difficulties. DMD affects 1 in 3,500 boys and approximately 1 in 50 million girls. To date there is a no cure, and no one survives.
After Jamesy’s diagnosis in September 2013, Raffone and his wife Karen, started their own fight against the disease which ultimately became JAR of Hope, a 501(c)(3) nonprofit that raises awareness and funds research and clinical trials for potential DMD drug therapies.
“JAR” are the younger Raffone’s initials, which inspired the name of the charity. The drug JAR 913 is named after the month and year of Jamesy’s DMD diagnosis, with JAR 914 following in sequence to symbolize “the day after.” Though much of Raffone’s efforts are personally inspired by his own son, the JAR of Hope mission is to help everyone affected by DMD.
Most of the organization’s efforts are through sponsored participation in races, marathons and other athletic events. Earlier this year Raffone and JAR of Hope team members participated for the second year in an extreme 200-mile, 7-day Alps 2 Ocean Ultra race across rugged New Zealand. The money they raised through sponsorships helped to pay for a wheelchair-accessible van donated to the family of Eddie and Liat Horton in Miami, whose two boys — Ariel and Jacob Horton, ages 11 and 9 — are diagnosed with DMD.
One day after the New Zealand race, Raffone flew to Australia to walk 100 miles in 5 days to visit schools that have students with DMD.
“For the last four years I’ve been traveling the world and I’ve been using the instrument of a push-up as a way to communicate to people how blessed they are. Because if you’re in any sort of a fitness world you might do push-ups to warm up,” Raffone said to his audience of Australian students. “Well, children who suffer from Duchenne muscular dystrophy will never be able to do one push-up.”
With a 235-pound muscular build, Raffone may have an easier time than some doing push-ups, but he jokes that he pays for his Clydesdale-build with every long-distance race he participates in, including his fourth run in the recent New York City marathon.
“We came across JAR of hope. We really loved the approach they were taking, some real novel treatments, all sorts of things we haven’t heard anyone else use,” shares Stephen Gunther of Harrisburg, Pa, whose son Drew is among the four boys receiving the JAR 914 treatment. “As a parent, I’m excited because I’m fully expecting to see real benefit from Drew and the other boys.“
[Source: JAR of Hope]