WASHINGTON – Congress marked the last day of February with a resolution calling attention to debilitating but rare diseases.
Some federal lawmakers, led in part by U.S. Rep. Cathy McMorris Rodgers, want something more.
The Pediatric Research Network Act seeks to improve communication among far-flung research centers and funnel clinical trial data from 20 or so large research institutions to smaller laboratories. McMorris Rodgers, a Spokane Republican raising a child with Down syndrome, said the goal is to break down the walls that often grow up around those studying rare genetic disorders.
“I just think there’s huge potential,” McMorris Rodgers said.
The plan draws cautious support from researchers wary of government funding cuts and bureaucratic red tape, while others say it ignores the current problems facing families with these disorders.
Jeffrey Chamberlain heads the University of Washington’s Paul D. Wellstone Muscular Dystrophy Cooperative Research Center in Seattle. There, he and his research team use state-of-the-art technology to test new treatments for a type of muscular dystrophy that afflicts 1 in every 3,600 boys born in America.
Chamberlain said creating a mechanism to share resources would free him to spend more hours pursuing treatments and a potential cure for Duchenne muscular dystrophy, rather than spending time searching his own records for published articles and clinical results.
While research breakthroughs have the potential to help future cases, Audrey Burr, a Spokane counselor who works with families of children with neuromuscular disorders, questions what’s being done right now.
“Families who live with a child with a significant health condition struggle every single day,” Burr said. “They have issues with health insurance, with finding care and with services in schools. Research doesn’t help that a bit.”
For many Washington children, including 50,000 in Spokane County, the state health insurance program known as Apple Health for Kids helps address those issues, Burr said. In 2010, McMorris Rodgers led an effort to establish untaxable savings, known as ABLE accounts, for families dealing with disabilities.
“More and more children with disabilities are now able to live on their own,” McMorris Rodgers said. “Maybe with some support, but more are getting into the workforce and are becoming independent. They may need some extra support.”
Meanwhile, the pediatric research proposal would encourage wider access to data conducted at large research centers like Chamberlain’s, leading to more coordinated breakthroughs in treatments and cures, McMorris Rodgers said. The congresswoman used the example of research on Down syndrome, the disorder her son has, as being helpful to treat Alzheimer’s.
A government shift from providing services to prioritizing research can present its own set of problems, Chamberlain said. While research depends on government funding, selecting which projects to fund should fall to the National Institutes of Health, not the U.S. Congress, he said.
“The NIH sees the big picture,” Chamberlain said, noting the prevalence of congressional intervention into the organization’s activities in recent years. “They fund everything. They need to be given wide latitude.”
The research-sharing proposal provides no new money for research and keeps spending authority in the hands of NIH. But the law calls for labs conducting clinical trials on treatments for four specific illnesses, among them Down syndrome and Duchenne, to receive priority when establishing the network.
Though government spending on health care is a touchy subject in the House, the pediatric research network has received bipartisan support in both chambers on Capitol Hill in recent years. No solid plan has been agreed to yet.
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