Saint Louis University School of Medicine and SSM Cardinal Glennon Children's Medical Center are participating in a first-of-its-kind genomic-based clinical trial to treat and study pediatric cancer, specifically relapsed and refractory neuroblastoma.
|William Ferguson, M.D.|
SLU and Cardinal Glennon are a part of the 11-member Neuroblastoma and Medullobloastoma Translational Research Consortium (NMTRC), housed at Van Andel Research Institute (VARI). NMTRC is a nationwide network of pediatric cancer clinical trial sites that includes the National Cancer Institute, universities and children's hospitals. Five hospitals, including Cardinal Glennon, are currently enrolling patients in the Food and Drug Administration (FDA) approved trial.
According to William Ferguson, M.D., principal investigator and director of pediatric hematology and oncology at SLU, the goal of the study is to develop a way to tailor cancer treatments on an individual basis, similar to how doctors treat other diseases like strep throat or urinary tract infections.
"When a patient has strep throat, for example, we identify which strain of the disease the patient has before prescribing penicillin or amoxicillin.
"With this study, we hope to develop a technique to use the tumor's unique genetic makeup to identify individualized treatment plans. We chose to focus on neuroblastoma tumors because there are few conventional treatments for patients who relapse. But eventually, this technique could be extended more broadly for treating other forms of cancer. This is cutting edge treatment and right now, with a few exceptions, no one is doing this to treat cancer," said Ferguson, who is the principal investigator of the study at SLU.
Neuroblastoma is a particularly deadly pediatric tumor of the peripheral nervous system, usually diagnosed in children age six and younger. Neuroblastoma accounts for 15 percent of all pediatric cancer deaths in the United States, and is a disease for which children who relapse have no curative therapies today.
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 five-year survival rate.
Genomic-guided therapy leverages next generation sequencing and gene expression technologies to identify subtle difference in an individual tumor's genetic makeup to provide 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.
"This trial may offer new hope to children facing the worst of all pediatric cancers. We hope this study will validate this new ‘right treatment for the right patient and the right time' approach as the new standard of care," Ferguson said.
Hospitals Now Enrolling Patients
Five NMTRC member hospitals are now enrolling patients, including Helen DeVos Children's Hospital, Grand Rapids, Mich.; Levine Children's Hospital, Charlotte, N.C.; Anderson Cancer Center, Orlando, Fla.; National Cancer Institute, Bethesda, Md.; and 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 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 Webb.
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 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."
Established in 1836, Saint Louis University School of Medicine has the distinction of awarding the first medical degree west of the Mississippi River. The school educates physicians and biomedical scientists, conducts medical research, and provides health care on a local, national and international level. Research at the school seeks new cures and treatments in five key areas: cancer, infectious disease, liver disease, aging and brain disease and heart/lung disease.