As someone with first-hand experience of a promising new medication for her illness, Springfield High School student Rachel Burns was happy to add her voice Friday to a bill in Congress to promote the need for more research into juvenile diseases.
Rachel, 15, who has cystic fibrosis, joined U.S. Sen. Sherrod Brown (D., Ohio) at a news conference at Toledo Children’s Hospital, along with Rachel’s family, caregivers, and hospital officials, to highlight his bill to require the National Institutes of Health to spend a bigger part of its budget on the illnesses that strike young people.
“Children make up 20 percent of the U.S. population, yet not much more than 5 percent of research dollars coming out of NIH and other research dollars, government and other, go into pediatric research,” Mr. Brown said.
“We know that some of the greatest minds, some of the most skilled minds, are doing pediatric research. We know that the emphasis has not been focused enough on pediatric research,” Mr. Brown said.
Cystic fibrosis is a genetic illness that causes thickening of the victim’s mucus that impairs breathing and invites infection.
Four years ago, Miss Burns participated in a drug trial that improved her condition, but it ended after two years and the drug never came to the market because its average benefit was not strong enough to justify the program, Miss Burns said.
“I was a little upset when it got discontinued. Every time I come up to this hospital center, I always bug Kelly [Houser, nurse and cystic fibrosis research coordinator at Toledo Children,s Hospital,] whether there’s another research or drug that I can help with,” Miss Burns said.
The National Pediatric Research Network Act would require NIH to identify up to 20 centers across the country to coordinate research in a “hub and spokes” way.
The bill also seeks a reasonable proportion of pediatric research grants for rare diseases, which disproportionately affect children.
According to NIH, it funded more than $3.2 billion in pediatric research in fiscal year 2011, including more than 90 research networks and centers programs with a pediatric focus. Additionally, NIH said funding toward broad disease-specific research advances knowledge of pediatric diseases as well.
NIH funded more than $79 million in cystic fibrosis research in fiscal year 2011.
Toledo Children’s Hospital has one of 80 cystic fibrosis care centers in the nation that are funded and accredited by the Cystic Fibrosis Foundation. The center has 169 patients, 90 of them under 18 and 79 who are adults.
The hospital carries out drug and therapy trials paid for mostly by the foundation and by drug companies themselves.
Rachel manages the illness with a medical regimen that starts at 4 a.m., plus softball and volleyball practice while maintaining a 4.1 grade-point average.
On the outside, she said, she looks and acts like a normal teenager.
“On the inside there’s a different story. I have CF. CF has affected my life a lot but the CF research center and the people who put their time and money into finding ways and medicines to find cures have dramatically helped not only me, but everybody out there,” she said.
Rachel’s mother, Heather Burns, said her daughter's daily care involves an hour-long regimen two times a day involving an aerosol and a vest that loosens the mucus in Rachel’s lungs.
Mrs. Burns said her daughter was contacted by the hospital's cystic fibrosis care enter about the event and was sent a copy of the bill for her to read.
“Rachel is a very outspoken person, and she’s very much in charge of her medical condition,” Mrs. Burns said.
Rachel’s father, Ray Burns, was also on hand.
Mr. Brown said he doesn’t know if the increased focus on pediatric research will cost the government more money or mean reallocating money that’s already budgeted.
Dr. Pierre Vauthy, medical director of pediatric pulmonary medicine and research coordinator at Toledo Children’s Hospital, said that because of pediatric research, cystic fibrosis patients have improved their average survival from 5 years to 38 years and researchers are now close to finding medications that may totally control the disease.
Mr. Brown said similar legislation has passed the House, and the bill is supported by a variety of medical entities, including the American Academy of Pediatrics.
Contact Tom Troy at: email@example.com or 419-724-6058.