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July 2008
Just for children: advocating for research specific to kids
Once upon a time, what was good and true for adults was thought to be good and true for children, just on a smaller scale - halved, usually. A full dose for adults, a half-dose for kids, and so on.
But that was old-school thinking. These days, we know better.
Children can't be treated as if they're small adults, says John Raymond, MD, vice president for academic affairs and provost of MUSC.
"There are differences in their metabolisms, and medications and treatments can interfere and interact differently with the hormonal aspects of their growth development," says Dr. Raymond.
Research specifically for children helps determine these nuances, he adds.
Yet historically, we've shied away from children's research, concerned about the risks.
"Unintentionally, this has hurt children. Adult studies don't necessarily help determine what works for children. We can generally assume they'll be effective for kids, but we don't really know.
"The effectiveness of therapies for children has been under-investigated," he notes.
Research - and advocates for children's research - are hurrying to make up for lost time.
In response, the National Institutes of Health has been making a concerted effort to include children in studies where they've traditionally been excluded.
It's an effort long overdue for those lobbying for children's research.
"Children are our future," says Dr. Raymond. "They have the longest life span ahead of them, so it makes sense to preserve the quality and length of their lives by maximizing the effectiveness of their therapies."
Yet he cautions that children have special ethical needs: "They may not have the cognitive capacity or life experience to make informed decisions about treatment options.
"We have to be absolutely clear about the risks and benefits of these therapies. These are the people who most desperately need our help."
As one of MUSC's chief academic leaders, Dr. Raymond places importance on difficult-to-treat diseases. He references the new treatment for childhood brain tumors that Dr. Bernie Maria is investigating.
"Because the biology of the developing brain is different from the adult brain, it's vital to have this research, and to be able to get closer to curing these diseases without damaging the child."
It takes compassionate, dedicated physicians like Dr. Maria, notes Dr. Raymond.
"Treating these children and driving this research is so important. These researchers need to be frontline pediatric physicians, in my opinion - they are the healthcare providers who interact most closely with children and their families, so this makes them better advocates and better able to balance the risks with the possible benefits of these new treatments."
Nowhere is this balance of care and advocacy for children more at work than in the DCRI. "It's one of the few places in the country where such a broad array of outstanding basic scientists are working side-by-side with pediatric clinicians to target serious childhood diseases, including diabetes, heart disease, rheumatological disorders - all of which are different in children who are still growing than they are in adults."
Children are our future, he notes. "And that's a very good reason to advocate for research specifically for them."
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Inderjit Singh, PhD
Scientific Director
Darby Children's Research Inst. |
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Bernard L. Maria, MD, MBA
Executive Director
Darby Children's Research Inst. |
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