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. Our focus on the connection between basic science and clinical programs allows us to provide cutting-edge care for children with cancer.

Source: the 2013 annual report of the Columbia University College of Physicians & Surgeons