Attending Thursday's Congressional Pediatric Cancer Caucus were elected officials, cancer specialists and a 9-year-old girl.
Mollie Ward, the 9-year-old fourth grader from Cherry Hill, N.J., was there to help Dr. Peter Adamson of Children's Hospital of Philadelphia push lawmakers to encourage pharmaceutical companies to research and develop more drugs for children with cancer.
She was diagnosed with a rare cancer in her pancreas before she turned 2.
"I had to have a lot of surgeries and X-rays and [get] a lot of checkups," Mollie said. "You get a lot of shots and you have to go through a lot of things."
Her father, Kevin Ward, said that when Mollie was diagnosed with pancreatoblastoma, they went through the traditional courses of treatment, surgery and chemotherapy, which did not work. After several relapses, her parents were faced with two choices: take her home and prepare to say goodbye or try to find the rare clinical trial.
The Wards traveled to the National Institutes of Health in Maryland for an experimental drug.
"We got the opportunity to try this experimental Phase 1 drug," he said. "It's six or seven years later. ... She is living proof [of] the true essence of the drug, of experimental therapy and the miracle of what she is today."
Cancer No. 1 Disease-Related Killer of U.S. Children
There are 12,500 new cases of cancer in children every year and 2,500 children die of cancer each year.
Childhood cancers have increased by nearly 30 percent in the last 20 years. Though pediatric cancer kills more children in the United States than any other disease, it makes up only 1 percent of all cancers, said Dr. Howard Katzenstein of the Aflac Cancer Center in Georgia and Emory University.
"When drug companies are developing new drugs, they're really trying to target the widest population, where they're gonna make their money," he said.
Katzenstein said that drugs are first tried in adult patients and then are adapted for children.
The problem? That process can take 10 years and billions of dollars, frustrating doctors and the parents of children with cancer.
The other problem is that there are no medicines that target pediatric cancers, which are different from their adult versions. Because of this lack of new medicines, the cure rate for pediatric cancers is plateauing.
"One of the struggles we deal with is that all the improvements we've made over the last 50 years and that's going from a zero percent cure rate to a close to 80 percent cure rate is that we haven't seen much progress in the last five or 10 years," said Dr. Keith August, a pediatric oncologist at Children's Mercy Hospital in Kansas City, Mo.
One Cancer Drug Approved for Children in 20 Years
Since 1980, only one new cancer drug has been approved for children; 50 have been approved for adults. Most children are given adult treatments in child-size doses.
Adamson, chair-elect for Children's Oncology Group, said the good news was that four out of five children with cancer were cured.
"We're doing that with drugs that were discovered in the '50s, '60s and '70s and we've gotten about as much mileage out of these drugs as we're going to get," he said.
He said researchers and the pharmaceutical industry needed to invest in finding the cause of childhood cancers and developing better treatments with fewer side effects.
Doctors and experts are focusing on those side effects, which include developmental delay, growth problems, infertility, hearing loss, kidney and heart problems and the possibility of a second cancer developing, Katzenstein said.
"Children are not just small adults," he said. "They're different. They handle drugs differently. They have different side effects."
"One of the key things we hope for is that Congress acts to put incentives in place that will allow industry to bring their expertise to the table," Adamson said. "There's not going to be increased money. ... [Just] incentives that help level the playing field."
"There are too many children who are in a similar situation to Mollie, where the outcome isn't what it needs to be and we're at a point where we're going to have tremendous discoveries," he said. "We need to turn those discoveries into better treatments."