The Neuroblastoma and Medulloblastoma Translational Research Consortium Tuesday announced the opening of a first-of-its-kind genomic-based clinical trial to treat and study pediatric cancer — specifically relapsed and refractory neuroblastoma, a deadly tumor that forms around nerve cells in children.
The 11-member NMTRC, housed at Van Andel Research Institute in Grand Rapids, is a nationwide network of pediatric cancer clinical trial sites that includes the National Cancer Institute, universities and children’s hospitals that will begin patient enrollment immediately in the Food and Drug Administration approved trial.
“This trial offers new hope to those children facing the worst of all pediatric cancers,” said NMTRC Chair Giselle Sholler, M.D. “We are confident the Genomic-based personalized medicine approach, often defined as ‘the right treatment for the right patient at the right time,’ will provide the necessary data to validate this approach as the new standard of care in the 21st Century.”
In the United States, a child dies of neuroblastoma every 16 hours. Neuroblastoma is a particularly deadly pediatric tumor of the peripheral nervous system, usually diagnosed in children age 6 and younger. Current standard treatment for neuroblastoma includes chemotherapy, surgery, bone marrow transplants, radiation and antibody therapy. Patients diagnosed with Stage 4 neuroblastoma have a less than 40 percent 5-year survival rate.
Genomic-guided therapy leverages next generation sequencing and gene expression technologies to identify subtle differences in an individual’s genetic makeup that provides a clearer picture of the disease state, and could conceivably single out an individual protein or other molecular drug target for therapy. This analysis involves important collaborative efforts including Grand Rapids-based Intervention Insights and the Pediatric Oncology Branch at the National Cancer Institute.
Five NMTRC member hospitals are now enrolling patients, including Drs. Giselle Sholler, Helen DeVos Children’s Hospital, Grand Rapids; Joel Kaplan, Levine Children’s Hospital, Charlotte, N.C.; Don Eslin, M.D. Anderson Cancer Center, Orlando, Fla.; Javed Khan, National Cancer Institute, Bethesda, Md.; and William Ferguson, SSM Cardinal Glennon Children’s Medical Center, St. Louis, Mo.
Additional participating NMTRC centers include Children’s Mercy Hospital and Clinics, Kansas City, Mo.; Connecticut Children’s Medical Center, Hartford, Conn.; Oregon Health & Science University, Portland, Ore.; Rady Children’s Hospital, San Diego, Calif.; and the Medical University of South Carolina, Charleston, S.C.
The NMTRC clinical trials are based on research from a group of closely collaborating investigators, some of which derive from a personalized medicine process and software developed at VARI to permit near “real time” processing of patient tumors and prediction of best drugs for that patient. Craig Webb, Ph.D., and Dr. Sholler, co-directors of VARI’s Pediatric Cancer Translational Research Program are the study’s architects.
“This is the next phase in a five-year process that began with proof of concept trials involving 14 pediatric patients at Helen De Vos Children’s Hospital in 2006,” said Dr. Webb.
Dr. Sholler’s work over the past five years received support from a number of sources of funding, including parents organizations and private foundations such as the Witmer Foundation, Owen Moscone Foundation, Friends of Will, Max’s Ring of Fire, The Ishan Gala Foundation, Lillie’s Friends Foundation, NB Alliance, Melina’s White Light, Ethan’s Rodeo, Solving Kids Cancer, Hyundai Hope on Wheels, St. Baldrick’s Foundation, and the Pappas Foundation.
“Much of the funding for this important work has come from parents of pediatric cancer patients desperate for a breakthrough for their children,” said Dr. Sholler. “Their support has been a source of critical funding and inspiration, and it is my hope this trial will validate their support of our work.”
VARI President and Research Director, Jeffrey M. Trent, Ph.D., said the NMTRC study would have an immediate impact.
“This effort speaks to the sense of urgency shared by all those involved,” said Trent, “This initiative promises to have a profound impact on pediatric patients across the country, and in the coming years, around the world.”