W. Long Branch Dad Declares War on Son’s Incurable Disease
This parent will do whatever it takes for his child.
Duchenne Muscular Dystrophy (DMD). Could new medical infusions slow this rare disease?
DMD is a terminally progressive condition that causes muscle atrophy and most affected kids 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 no cure, and no one survives.
A father at the Jersey Shore is leading the charge to cure a rare genetic muscle-wasting condition that affects about 20,000 children in the U.S, nearly all of whom are male, including his own son.
Jamesy was diagnosed nearly 6 years ago with Duchenne muscular dystrophy. The doctor's advice? 'Go home and love your son, there is nothing we can do for him.' They couldn't reverse what dad, Jim Raffone, calls 'a premature death sentence.'
So Jim created the nonprofit JAR of Hope to declare war on Duchenne muscular dystrophy.
Last month, Jim entered his 10-year-old son into a privately funded treatment administered in NYC 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.
This experimental treatment, which has never been done before, is being administered to the boys quarterly by Dr. Anthony Salerno, with the next round of JAR 914 infusions scheduled for January.
According to Dr. Salerno says, "JAR 914 is 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.”
While JAR 914 is intended to delay symptoms of DMD, Jim 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.
Jim says that getting this drug to Phase II testing could take years and millions of dollars. He is hoping the community will come together to fund JAR 914 so that the children suffering from this awful disease could delay their symptoms until a cure is found.
Jim 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. Jim has taken to wearing a flak jacket during his 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.
Here is Jamesy's (And JAR of Hope's) story so far:
After Jamesy’s diagnosis in Sept. 2013, Jim 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 Jamesy'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 Jim'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 Jim 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, 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 pushup 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,” Jim 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, Jim may have an easier time than some doing pushups, 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.
Another parent, Stephen Gunther, whose son Drew is among the four boys receiving the JAR 914 treatment, says, “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. As a parent, I’m excited because I’m fully expecting to see a real benefit from Drew and the other boys."
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