On Dec. 7, Emma Harrison will have her last treatment for acute lymphoblastic leukemia, or ALL, the most common childhood cancer. For more than two grueling years, the St. Petersburg third-grader has endured a combination of chemotherapy agents her mother, Suzan, describes as "unbelievably harsh" but that cures 80 percent of kids with ALL. The chemo ordeal is almost over but, "I'm already worrying about a recurrence," Suzan said. "They can relapse at any point in the process." So she is closely watching a Florida clinical trial, bankrolled by a local charity, aimed at helping the 500 American children who suffer a relapse each year.
Metformin, a widely used drug for Type 2 diabetes, has shown promise as a cancer fighter. But it has never been tried for that purpose in children — though it's used by young diabetics.
"We have good evidence at the University of Miami that metformin actively kills leukemia in the laboratory," said Dr. John Goldberg, a pediatric cancer specialist at the University of Miami Miller School of Medicine.
But money for costly clinical trials usually comes from the federal government, major drug companies or private institutions. Applying for and winning such grants is time consuming, mired in paperwork, and the studies rarely give researchers much local control.
So Goldberg turned to a little-known resource: the Pediatric Cancer Foundation, a small Tampa-based charity founded in 1991 by Melissa Helms and Risa Trammel, Tampa mothers whose infants had cancer.
After 15 years of sending small amounts of money to large institutions, the group became dissatisfied with how long it took to get clinical trials started. So it created the Sunshine Project, to identify and fund teams of hand-picked researchers to conduct clinical trials.
It has launched two clinical trials at 10 hospitals since 2005 and is preparing to launch a third next year, though it's too early to report their final results.
"We wanted to accelerate the finding of new drugs and the only way to do that is to take it back in-house and do it ourselves,'' said Nancy Crane, the foundation's executive director. "Our doctors like it and our donors like it.''
Dr. Damon Reed, medical director of the Sarcoma Program at Moffitt Cancer Center, is a principal investigator with the Sunshine Project.
"There are clear advantages when it comes to working with small groups," he said. "Large groups have done wonderful things for pediatrics, but their clinical trials are often tightly regulated, with no institution in Florida having access to those early-phase trials."
The metformin clinical trial is being conducted at seven medical centers, including three in the bay area. PCF has pledged to raise $274,000 a year to fund Phase 1, which tests the drug's safety in children with cancer. The next phase will focus on how it works against cancer, but children who participate now have the opportunity to benefit.
Without a formal clinical trial that follows scientific protocols, doctors can't experiment on patients, no matter how desperate the situation.
"For a study like this, Florida children would have to travel out of state to participate," said Goldberg, "Under this model we can provide experimental treatment to Florida children, in Florida."
Suzan Harrison's anxiety is somewhat alleviated by knowing the research is under way. "I hope we never have to participate in a clinical trial, but I'm very grateful to think that it would be available," she said.
Contact Irene Maher at firstname.lastname@example.org.