Hope for Kids with Cancer

>No doctor ever wants to tell parents that their child has cancer. It's one of the hardest discussions many will face.

But it can be just as hard telling parents that there are few treatment options, says Donald Miller, M.D., Ph.D., director of the James Graham Brown Cancer Center at UofL.

"While cancer treatment has improved at near-lightning speed for adults, the medical field is years behind when it comes to children," he says.

The majority of cancer drugs used to treat children were developed for and tested on adults. A 2005 study by the Institute of Medicine found a "near absence" of research into pediatric cancer drugs, and about half of the drugs currently in use were developed more than 20 years ago. In fact, between 1948 and 2003 the FDA approved 120 new cancer therapies. Of those, only 30 have been shown to be effective in children and just half of those acquired any FDA labeling for use against childhood cancer over the same 55-year period.

Researchers at UofL now are working to change that situation, thanks to the help of Kosair Charities, the region's largest children's health charity. In August, Kosair announced that it would give UofL $12 million to create the Kosair Charities Pediatric Cancer Research Center at the Brown Cancer Center.

"Children have no better friend than Kosair Charities," says UofL President James Ramsey. "It's clear that we need to transform the way that new drugs are developed for kids with cancer, and we are going to do it right here in Louisville, Ky."

Lenny Sewell, president of Kosair Charities, says the new research center is "a great fit with our mission of 'Helping Kids'."

"This gift will be a permanent source of funding, a true investment in the children of this and future generations with potential impact here and throughout the world," he adds. "We look forward to watching the progress of this exciting project."

Researchers at the center will focus specifically on developing new therapies and drugs to address children's cancers, with the goal of becoming an international leader in the field.

The center will complement other strengths that UofL has helped to build in the community, according to Larry Cook, M.D., executive vice president for health affairs.

"First, in partnership with Norton Healthcare, the Department of Pediatrics has created an excellent center for cancer treatment at Kosair Children's Hospital," he says.

"Second, Kosair Charities and Norton Healthcare both supported the Kosair Charities Pediatric Clinical Trials research unit, led by UofL pediatrics professor Dr. Jan Sullivan. We have nationally recognized expertise conducting clinical trials of new drugs in children, supported by a pediatric proteomics core that was funded in part with the help of Sen. Mitch McConnell. This means we have the infrastructure in place to take drugs from the lab to kids who need them."

The clinical trials unit, located at Kosair Children's Hospital, is a state-of-the-art clinical facility dedicated solely to conducting inpatient and outpatient pediatric clinical pharmacology studies. It is one of 13 sites in the Pediatric Pharmacology Research Unit network, a national network of clinical pediatric pharmacology research sites in the United States funded by the National Institute of Child Health and Human Development.

UofL pediatrics faculty and staff working in the unit have studied medication for a wide range of illnesses and disorders and have the expertise needed to test new cancer drugs for kids.

"The Brown Cancer Center is becoming a leading-edge center for cancer drug development," Cook notes. "This means that all of the pieces are in place to make Louisville the next center for pediatric drug development -- and we wouldn't be this far along without the support of Kosair Charities and the partnership we have in pediatrics with Norton Healthcare."

Miller says Brown Cancer Center researchers already have tremendous strengths in target drug research, with more than a dozen promising drugs in the development pipeline.

"Thanks to Kosair Charities and the talented researchers we already have, we think we can bring some of the leading experts in the field to Louisville to work with our physicians and scientists to move new therapies for kids from the lab to the patient," he says.

This targeted drug development -- and the assistance of Kosair Charities -- is very important because this kind of work is being done in very few places, says Salvatore Bertolone, M.D., chief of the Division of Pediatric Hematology and Oncology at UofL.

"Drug companies are reluctant to develop new therapies for use in children for two reasons," he says.

"First, because of market forces. The number of children with a particular form of cancer is relatively small and drugs are expensive to develop. Second, the drug companies have a natural reluctance to develop drugs targeted for children because of safety concerns."

Bertolone, who works daily with kids with cancer, is eager to collaborate with the new center.

"The ability to work with the breadth of researchers at the Brown Cancer Center and bring a pediatric focus to the work is fabulous," he says.

Miller and his colleagues are currently assembling a scientific advisory board that will guide the center's direction and will begin recruiting promising researchers this spring.

Both Miller and Bertolone say that it is difficult to express the depth of their excitement and gratitude to Kosair Charities for supporting their research vision.

"It's phenomenal," says Bertolone. "It really sets the stage and says we care about children."

"Every single one of the $12 million dollars will make a difference for the future of kids with cancer, and every dollar is appreciated," Miller adds.

Ramsey agrees.

"This center will enhance the quality of life for children and their families, creating new treatments that are more effective and less toxic than those currently available," he says.

"We want to give more kids a fighting chance against this horrible disease. That's what Kosair Charities is all about and that's what the University of Louisville is going to do with the charities' help."