NYP/Morgan Stanley Children’s is the only center in New York City routinely applying this technology to pediatric cancers. As part of the PIPseq program, our investigators are creating patient-derived xenograft models (human tumors propagated in mice) using biopsy or resection samples from young patients with solid and hematologic cancers.

One centimeter of tumor tissue can be segmented and implanted in multiple mice, generating 50 to 100 avatars. Coupled with the identification of gene mutations through the PIPseq program, the avatar models can then be used to determine whether the identified mutations contribute to the cancer’s development.

For example, the Columbia researchers created an avatar for a toddler with aggressively metastasizing infantile fibrosarcoma, which did not have the genetic changes commonly known to be associated with this disease. As a result
of sequencing efforts through the PIPSeq program, they identified and cloned a novel gene associated with the tumor’s aggressive behavior. Insight offered by genomic information and creation of an avatar model of this tumor have allowed the clinicians to choose and test an efficacious chemotherapy regimen. Researchers are also using such avatars to evaluate new drugs, with each avatar acting as its own miniature “clinical trial” of a single drug or drug combination, including investigational agents.