University of Vermont

An often-fatal cancer that affects very young children, neuroblastoma is a solid tumor that develops in the cells of the sympathetic nervous system. Only 30 percent of children with high-risk disease survive five years, and of those children who are treated and relapse, long-term survival is less than five percent. Physician-researcher Giselle Sholler, M.D., assistant professor of pediatrics at the Vermont Cancer Center (VCC) at the University of Vermont (UVM) and Fletcher Allen Health Care, has made it her life's work to change these odds.

During a fellowship at Brown University, Sholler made a serendipitous discovery. One of her neuroblastoma patients had contracted a parasitic disease through a blood transfusion and the therapy for that — a drug called nifurtimox only available through the Centers for Disease Control — had an unexpected outcome. Coupled with the child's other treatment, it shrank the tumor, a result other therapy regimens had been unable to achieve. Thus began Sholler's research crusade to develop FDA-approved clinical trials for treating relapsed neuroblastoma, which have attracted international attention from dozens of families, in addition to pediatric cancer research specialists from across the United States.

On Thursday, May 21, Sholler and the VCC hosted a gathering of national experts for a symposium titled "Developments in Neuroblastoma Research." The event brought together more than 125 attendees, including neuroblastoma researchers, families, parent advocate foundations and UVM faculty and medical students. In addition to investigators from UVM/VCC, featured presenters included keynote speaker Nai-Kong Cheung, M.D., Ph.D., from Memorial Sloan-Kettering Cancer Center, and members of a national research consortium spearheaded by Sholler from the University of Texas M.D. Anderson Cancer Center, University of Hawaii, Rady Children's Hospital in San Diego, Cardinal Glennon Children's Medical Center in St. Louis and Van Andel Research Institute at Michigan State University. The consortium is making innovative Phase I and II treatments available not only in Vermont, but at these cancer research institutions as well.

The symposium also offered insight into the patient family perspective. Andy Mikulak, who lost his son, Max, to neuroblastoma in 2008, and Neil Hutchison, whose son Sam is battling this disease, are two neuroblastoma parent-advocates who provided remarks at the opening and close of the symposium. Hutchison and his wife Margot, along with parents John and Catherine London, established The Penelope and Sam Fund for Neuroblastoma Research at the VCC in 2006.

Among the parent-advocates attending the symposium was Patrick Lacey of Braintree, Mass., whose four-year-old son Will was diagnosed with neuroblastoma at age seven months, and has been receiving therapy from Sholler at Vermont Children's Hospital at Fletcher Allen since July of 2008.

"Dr. Sholler is treating relapsed neuroblastoma patients with a goal of curing them as her mission in life," says Lacey. "She solidly has fixed her gaze on the goal and is marching toward it with steadfast determination with my son in tow."

In addition to participating in a speaker panel on therapeutic decision-making in neuroblastoma, Sholler presented on the collaborative work of the consortium in a session titled "Targeted Trials within the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC)."

The symposium is supported by the Lake Champlain Cancer Research Organization and the UVM Department of Pediatrics. For more information about the event, visit Neuroblastoma Research Symposium.