by Laura Scholz
Each year, CURE Childhood Cancer awards more than $1 million in research funds to scientists working directly to discover new treatments, drug therapies and ultimately cures for pediatric cancer. Currently, less than two percent of federal funding for cancer research is directed toward solving children’s cancer. The funds CURE is investing in research is desperately needed and is making a real difference in the fight against childhood cancer.
One doctor leading the charge is Dr. Leslie Kean, Assistant Professor in the Department of Pediatrics at the Emory School of Medicine. A graduate of Emory and past recipient of the university’s Pediatric Fellow of the Year award, Kean is part of the Aflac Cancer Center of Children’s Healthcare of Atlanta’s Pediatric Bone Marrow Transplant Team (BMT). The Aflac Cancer Center is a national leader among childhood cancer, hematology and bone marrow transplant programs and treats more than 350 new pediatric cancer patients each year. Since its inception in 1985, the BMT program has performed more than 800 pediatric bone marrow transplants per year, making it one of the largest pediatric BMT programs in the country.
CURE has awarded Kean a $100,000 research grant to begin testing and clinical trials for a new treatment to prevent Graft vs. Host Disease (GVHD). GVHD is a deadly complication many children experience after receiving a bone marrow transplant, a common treatment for combatting pediatric cancers such as certain forms of leukemia and neuroblastoma.
About half of all bone marrow transplant patients develop this complication, which occurs when the newly transplanted materials, or graft cells, form pathogenic t-cells which attack the recipient’s body. Kean believes that in order to prevent GVHD, t-cells need to be treated and changed so they do not attack the recipient’s body.
With grant money from CURE , Kean started a clinical trial which tests a new treatment called Abatacept, a novel-therapeutic target for GVHD. This targeted therapy is a co-stimulation drug which blocks the t-cells causing GHVD. The Aflac Cancer Center is the only place in the country where patients can participate in this type of clinical trial.
“The money from CURE is absolutely essential in preventing and treating GVHD,” said Kean. Because of CURE ’s grant, Kean has been able to test her research on humans for the very first time.
For Kean, who started this research ten years ago, it’s rewarding “watching an idea blossom into something greater that can help thousands of children.”
The Abatacept clinical trial began six months ago with five leukemia patients, all over the age of twelve and in need of a bone marrow transplant. Patients receive the drug during the bone marrow transplant, after which Kean and her team run tests to determine the effectiveness of Abatacept in preventing GVHD. As the trial goes on, Abatacept will be tested against other drugs on the market to compare side effects, effectiveness and other variables.
So far, those in the trial have responded well to the treatment. Full results will be available within the next six months. Soon, Kean will begin testing on children as young as six, and eventually, she envisions a much larger nationwide clinical trial.
“Our long-term goal is a multi-center trial with the Aflac Cancer Center as the main site,” said Kean. “This larger trial will help test Abatacept head to head against the present standard therapy drugs in order to discover any side effects, but more importantly, to prove that it is more effective in treatment of HVGD than anything else on the market. That way, we can make bone marrow transplants safer and more successful for thousands of pediatric cancer patients as we work toward our ultimate goal: a cure.”