Group forms to test new cancer drugs in children

University of Florida physicians have partnered with eight other academic medical centers to form a national consortium aimed at testing new cancer therapies in children who fail to respond to traditional treatments.

The Pediatric Oncology Experimental Therapeutics Investigators Consortium, or POETIC, launched its first clinical trial Monday, when doctors gave an investigational anticancer drug to an 11-year-old North Central Florida boy who has battled bone cancer for the past three years.

“The importance of the consortium is that there haven’t been a lot of opportunities for children with cancer to access drugs in the earliest stages of development,” said Stephen P. Hunger, M.D., chief of the division of pediatric hematology/oncology at UF’s College of Medicine. “There is nowhere else in the state of Florida where such trials are available. This gives patients throughout Florida and elsewhere in the Southeast opportunities that didn’t exist otherwise. This particular trial is the first of a number of trials the consortium will conduct.”

Each year, an estimated 12,000 patients under the age of 20 are diagnosed with cancer in the United States, Hunger said. Of those, about 20 percent do not respond to first- and second-line therapies, he said.

During the past 25 years, many major breakthroughs in the treatment of childhood cancer have arisen out of the collaborative efforts of research groups across the United States. UF, for example, is one of the statistical centers for the international Children’s Oncology Group, a federally supported consortium that pools resources to design, conduct and analyze studies of pediatric cancer therapies worldwide. Clinicians worldwide funnel their data to statisticians at UF and the other COG statistical and data centers who analyze findings and monitor research initiatives.

Most COG studies focus on research protocols to test established, widely available chemotherapy drugs. In contrast, POETIC focuses on newer agents being tested in people for the first time, Hunger said. In addition to the UF Shands Cancer Center, other members of POETIC are Johns Hopkins University, Memorial Sloan-Kettering Cancer Center, M.D. Anderson Cancer Center, Emory University, the University of Colorado Health Sciences Center, Vanderbilt University, the University of Arizona and Phoenix Children’s Hospital. Together researchers at these institutions will lead phase 1 and phase 2 trials of cancer drugs that target the underlying biologic mechanisms of various malignancies in children.

A phase 1 trial seeks in part to determine the safety and pharmacologic effects of escalating doses of a drug in the first stages of development. A phase 2 trial establishes whether a drug works for its proposed purpose and evaluates doses, adverse effects and drug metabolism. POETIC trials also include detailed studies to determine if the drug being tested is working as predicted from preclinical studies.

The consortium is in the process of developing a half-dozen other trials slated to open in the coming year, Hunger said. The goal will be to have four trials open at any given moment so there’s always something available for a family to consider, he said.

For the study that began this week, researchers will enroll about two-dozen children with various forms of recurrent cancer who have not responded to other therapies. They will test an intravenous drug called 17-AAG, supplied by the National Cancer Institute.

On Monday, a patient of Hunger’s was the first study participant to receive the drug. The young NASCAR fan, who likes to ride his all-terrain vehicle and watch the television show “Unsolved Mysteries,” was diagnosed in October 2001 with a form of bone cancer known as osteosarcoma after experiencing pain in his left arm. His name is being withheld due to patient privacy concerns.

The patient was treated at Shands at UF with conventional chemotherapy and surgery to remove the bone tumor and replace the upper part of the arm bone. However, the cancer spread to his lungs about three months after he finished chemotherapy, eventually forcing doctors to remove his left lung and a large portion of his right lung. Since then, several treatments have been tried, but the cancer has continued to grow. Then his family heard about the current study and returned to Shands at UF for the first treatment.

“This study gives us hope,” his mother said. “We’re glad there’s something else we can try.”

His father said the family is getting desperate.

“It’s at the point now where we’ve got to try other things, even if it means an experimental drug with no history, really, to see if it was going to help him,” he said.

The family discovered the study after searching for alternatives on the computer and calling hospitals across the country. They said they were relieved to learn they could enroll in the trial yet stay close to home.

“That’s what everybody should do, too – never give up,” the boy’s mother said. “Because there may be a doctor who says there’s no more, but you can always find more. And that’s what I would like other parents to know.”