The 21st Century Cures Act has attracted researchers, physicians, advocates, and patients to engage in a discussion about how to unleash the next generation of cures and treatments, taking advantage of the incredible promise of science and research. Throughout this multiyear effort, the patients have served as an inspiration to continue fighting. Matthew Dugas, a resident of Ypsilanti, Michigan, is no different.
Matthew’s fight against mitochondrial disease, a metababoli disorder, and hemophagocytic lymphohistiocytosis (HLH) ended too soon, at the young age of 10. The Detroit Free Press reported that Matthew and his sister, Adrianna, wanted to be doctors when they grew up, in hopes of becoming a “medical research duo" that would combat diseases and search for better treatments. Adrianna continued, “we both planned on going to school together and finding a cure for his disease so he could live with me forever."
Kids like Matthew and the thousands of others suffering from rare diseases need more research, better treatments, and faster cures. The 21st Century Cures Act fights for Americans so that all patients can get the cures they deserve.
October 6, 2015
The fight continues for Ypsilanti boy who died trying
Matthew Dugas wanted to become a singer someday, he told his mom. But if that didn’t pan out, he said he’d like to become a doctor to research the diseases that were slowly robbing him of his life.
He and his 14-year-old sister Adrianna dreamed of becoming a medical research duo, living and working together to find better treatments and, ultimately, a cure for mitochondrial disease, a metabolic disorder, and hemophagocytic lymphohistiocytosis, more commonly known as HLH. It occurs when the body’s immune system goes awry, and infection-fighting cells attack healthy tissue, causing organ damage.
But Matthew didn’t get to grow up. Though he fought for his own life, he didn’t get the chance to fight for the lives of other kids who struggled as he did.
Matthew died April 20 surrounded by his sisters, brothers and parents at the University of Michigan’s C.S. Mott Children’s Hospital. He was 10 years old.
Fighting for the good guys was something he loved to do, said his mother, Erica Dugas.
When he and his family went to Walt Disney World with the Make-A-Wish Foundation in 2013, she said his wish was to fight the Dark Side, going up against Darth Vader in a light saber duel at Hollywood Studios.
“He was amazing," said his sister Adrianna Dugas, a 14-year-old freshman at Milan High School. “He was the best brother you could ever have. If you told him something, he would keep the secret. He would never tell.
“Me and my brother were always close, but when we really bonded was on the Make-a-Wish trip. As time went on … we both planned on going to school together and finding a cure for his disease so he could live with me forever."
The Ypsilanti boy loved superheroes, “Star Wars" and playing with Legos. But from the day he was born, Erica Dugas said Matthew was almost always sick.
“He was a small baby, but full-term," she said. “Right away, from the very beginning, he was having respiratory problems and trouble eating."
Matthew was termed failure to thrive by the time he was 3 months old. He couldn’t gain weight, couldn’t keep down his formula.
After years of recurrent hospitalizations and testing, he was diagnosed by a specialist in Georgia at age 6 with a genetic mitochondrial disease.
One day, Matthew woke up and had lost the ability to walk or crawl. Then his hands began to atrophy. But it wasn’t until last year that doctors first identified HLH.
It can be triggered by a virus or an autoimmune disorder; some forms are rooted in genetic problems. Many times, said Dr. Jim Connelly, an assistant professor of pediatric bone marrow transplant at C.S. Mott. Children’s Hospital in Ann Arbor, doctors are unable to pinpoint a cause.
It’s most commonly treated with steroids, chemotherapy and sometimes a bone marrow transplant, even though it isn’t cancer. The idea, he said, is to wipe out the overactive immune system and replace it with a new, healthy one. But it doesn’t always work. And there are very serious risks that come with such treatments.
“Our treatment is really based on slowing down or shutting down the immune system," said Connelly, who treated Matthew at Mott. “The most common regimen that we use was actually developed over 20 years ago. So even though we have a lot of brand new drugs we could potentially use to treat HLH, the standard therapy regimen was built over 20 years ago."
