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Natick - Local Town Pages

Funding and Finding a Cure

By Sean Sullivan
In “Lorenzo’s Oil,” parents of a young child afflicted with a rare disease go to extraordinary lengths to find a cure.
The 1992 film is based on the true story of Lorenzo Odone, who was diagnosed with adrenoleukodystrophy after his health began to deteriorate. Told their son likely had less than two years to live, Odone’s parents embarked on an epic quest to formulate a remedy.
Their efforts were met with skepticism and resistance from a medical community that saw them as irrational actors, out of their depth in the realm of medicine in which they had no prior expertise or training.
Yet Lorenzo’s parents pressed on, and their persistence paved the way for development of a treatment for their son’s affliction (if not a cure). Dubbed Lorenzo’s Oil, the medicine had mixed results. It sometimes slowed and in some cases seemed to reverse the physical degeneration caused by the disease that had been debilitating him.
The Dion family might find fellow travelers in the odyssey of that story.
Two of their three young children were diagnosed with a rare form of muscular dystrophy, one for which there currently is no cure. And like Lorenzo’s parents, the mother and father of the Dion children embarked on a mission to save their kids.
“We kind of noticed he was having trouble with stairs,” and other activities, said Courtney Dion. She’s mom to Peter, who started showing symptoms a few years ago of a disease that very few people have likely heard of.
Limb-Girdle Muscular Dystrophy is an “ultra rare” form of MS, a condition that deteriorates motor and organ function.
His parents Courtney and Joseph had Peter tested and received the unwelcome news. And because MS is a genetic disorder, the Dions had their two other children evaluated as a precaution. Those tests revealed that Peter’s younger sister Maggie also evinced the mutation which causes the disease.    
Yet unlike the Odones of Lorenzo’s Oil lore, the Dion family would leave the quest for a cure largely to professional researchers. They decided their energies were best spent raising funds and awareness – two precious commodities in a race against time and an obscure disease.
“That’s when we decided to make the foundation,” said Courtney Dion. That organization bears the family’s surname, and its mission is to shine a light on Limb-Girdle Muscular Dystrophy and resource a cure for it. Family and friends serve on the Dion Foundation’s board.
“We wanted to move the needle a little bit.”
The Dions and their new foundation began a nationwide search for specialists with expertise in their specific form of MS, no easy task owing to its rarity.
To “get more players in the game for this drug development,” said Dion, a crucial facet in any strategy to find a cure.
There are government programs that incentivize research for rarer ailments like Limb-Girdle MS, which often come in the form of rewards for drug companies that develop effective treatments. These financial prizes are often far in excess of what a pharmaceutical company could earn by selling their new drug directly to the public, an attractive enticement for smaller and start-up firms. The reward can also keep the eventual cost of the drug affordable, whose makers would otherwise price the treatments to recoup their investment in research and development.
The foundation’s fundraising efforts have come in the form of a 5K race, golf tournament, and a Boston Marathon team. Its lobbying efforts include the family traveling around the country to garner support from legislators and advocates.
In the few years since their diagnoses, Peter has turned 11 and his sister Maggie 8. The family favors stay-cations, as travel can be taxing even under the best of circumstances. They reside in Marshfield and summer at local beaches. 
“For them, a school day is really challenging,” said their mom, adding that the two often come home exhausted after classes are out. “We feel like we have a clock ticking against us.”          
“They were really shocked at this diagnosis,” said Jocelyn Sage Mitchell. She works for the Dions in the family’s chain of local stores (one of them a longtime Natick staple), and is a board member and ambassador for the foundation. “That was the impetus behind them starting the organization.”
The Dions have donated an annual sum of the company’s profits to fund their foundation and help find a cure.
Further efforts to raise awareness have included lobbying Congress for support over the past few years – again, a race against the clock. Some afflictions carry with them the prognosis of a prolonged decline, perhaps many years or even decades. They’re slower moving and better known to the medical community, and thus can be treated or even cured.
“This is not one of them,” said Sage Mitchell.
Peter and Maggie were diagnosed with the disease two years ago. And theirs is counted among one of the more aggressive forms of Limb-Girdle Muscular Dystrophy, which can implicate the lungs, heart and muscles. It threatens to shorten lifespan, and can consign victims to a wheelchair by their teenage years.
“That’s an insane thing to find out,” said Sage Mitchell. “They’re really being brave putting their story out there.”
“Time is muscle,” is a refrain used among the relatively small community of people touched by the disease. In the race against time, Limb-Girdle MS saps its victims of strength and the freedom of movement so many of take for granted.
“Every day,” said Sage Mitchell, “their children are deteriorating before their eyes.”