June 14, 2010
Contact: Rachel Salis-Silverman, Public Relations, 267-426-6063
Pediatric oncologist John M. Maris, MD, describes the current state of the science in combating neuroblastoma, the most common solid cancer of early childhood, in his article in the June 10, 2010 New England Journal of Medicine, "Recent Advances in Neuroblastoma."
In the article, Dr. Maris reviews the field's latest research knowledge -- much of it based on efforts by Maris and his colleagues at The Children's Hospital of Philadelphia. Dr. Maris directs a laboratory at CHOP in collaboration with the multicenter Children's Oncology Group (COG) using tissue samples from 5,000 patients -- the world's largest sample collection for neuroblastoma.
Neuroblastoma is a cancer of the sympathetic nervous system, neuroblastoma most commonly occurs as a solid tumor arising from the adrenal gland in the abdomen. It remains one of the most puzzling of childhood cancers, ranging from cases of widespread but benign tumors in infants that spontaneously and completely disappear, to high-risk subtypes in older children that are relentlessly aggressive.
Neuroblastoma research discoveries at The Children's Hospital of Philadelphia
Dr. Maris is the chief of Oncology at The Children's Hospital of Philadelphia and director of the Hospital's Center for Childhood Cancer Research. Just three years ago, he wrote a review article on neuroblastoma for The Lancet. Since that time, he and his lab team have explored the genetic landscape of neuroblastoma, producing a stream of discoveries:
- In 2008, Maris led the team that identified common DNA variations on chromosome 6, the first time researchers found the cancer's genetic origin.
- In late 2008, Yael Mosse, MD, collaborating at CHOP with Maris, reported that mutations in the ALK gene were the main cause of inherited neuroblastoma and also played a role in many cases of non-inherited neuroblastoma.
- In 2009, Maris published two more gene studies, identifying common variants in the gene BARD1 and copy number variants on chromosome 1 that raised the risk of neuroblastoma. The latter study was the first ever to find a specific copy number variant that predisposes to a human cancer.
Translating neuroblastoma research discoveries into treatments
The ultimate goal of basic research at CHOP is to translate findings into more effective treatments for children. Noteworthy clinical trials include:
- A clinical trial of an ALK inhibitor for children who have relapsed after neuroblastoma treatments, lead by the Cancer Center at CHOP
- A national immunotherapy trial using monoclonal antibodies and cytokines to selectively target neuroblastoma cells, led by the Children's Oncology Group
- An experimental treatment using MIBG, a radioactive isotope that zeroes in on neuroblastoma cells, developed at CHOP and other centers
- Federally sponsored collaborative programs designed to identify new drugs that will interrupt the key cell signaling pathways that drive neuroblastoma