Precision Cancer Medicine for Pediatric Patients
At Columbia University Medical Center, all pediatric cancer patients now have access to genome sequencing of their tumors. Using technologies that have until now been largely restricted to research use and have taken many months to process, the Precision in Pediatric Sequencing—PIPseq—program provides results to a patient’s doctor in less than three weeks.
“We recognize that a diagnosis of cancer or a blood disorder is a frightening prospect that affects not only the child, but the entire family,” says Andrew Kung, MD, PhD, chief of the pediatric hematology, oncology, and stem cell transplantation division and the Robert and Ellen Kapito Professor of Pediatrics. “There have been dramatic advances in the way we manage pediatric cancers and blood disorders, and today patients are reaping the benefits of decades of research.”
While tremendous progress has been made in the treatment of pediatric cancers, oncologists are still challenged by cancers that fail to respond or develop resistance to treatment. The key to success in the battle against these cancers lies in the biology of each child’s cancer. What mutations are driving its growth or rendering it invulnerable to the standard treatments?
PIPseq strives to identify the molecular drivers of each patient’s cancer and use that information to personalize treatment with novel biologically targeted investigational agents. “With next-generation sequencing,” says Julia Glade Bender, MD, medical director of PIPseq, “we can delve deeper into the genetic basis of cancer to pinpoint novel therapeutic targets.”
In early 2014, with support from the Herbert Irving Comprehensive Cancer Center, the division started performing genome sequencing of tumors in patients with high-risk or relapsed cancers, and the sequencing is now offered to all pediatric cancer patients. “No other pediatric cancer program is applying precision medicine to the problem of childhood cancers the way we are,” says Maria Luisa Sulis, MD, head of hematologic malignancies for PIPseq. “We are one of the only programs prospectively sequencing cancers and using the results to make clinical decisions for our patients.”
As part of the PIPseq program, investigators also use biopsy or resection samples from patients to grow the tumors in mice, creating what are termed patient “avatars.” One centimeter of tumor tissue can be used to generate 50 to 100 avatars. Coupled with the identification of gene mutations through PIPseq, the avatar models can be used to determine whether the identified mutations contribute to the cancer’s development. With that information, clinician-scientists can identify target drugs that might be relevant for treating a particular child’s tumor. “Columbia’s program is rooted in science,” says Dr. Kung, “and the rich connection between basic science and clinical care allows us to provide cutting-edge care for children with cancer. We are using tomorrow’s technologies and leveraging advances in genomics, molecular biology, and cancer biology to identify more precise methods to treat patients today.”