NCI Cancer Bulletin: A Trusted Source for Cancer Research News
NCI Cancer Bulletin: A Trusted Source for Cancer Research News
March 18, 2008 • Volume 5 / Number 6 E-Mail This Document  |  Download PDF  |  Bulletin Archive/Search  |  Subscribe

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A series of paintings created by pediatric cancer patients who were treated at NIH
Technology Drives Search for Childhood Therapies

Children's Inn at NIH

A photo of the Children's Inn at NIH, which is a large, two-story, gray-stone building that is surrounded by lawn and large trees. The Inn, which can accommodate 59 families, features several common-area living rooms, laundry facilities, a communal kitchen, television room, library, computer room, and play room.

The Children's Inn at NIH provides lodging, healing services, and programs for seriously ill children and their families who are treated across the street at the NIH Clinical Center. To learn more, go to

Childhood cancers are biologically different from those that arise later in life. Cancers in children are more likely to involve developing organs, for instance, or to begin in the prenatal environment.

But whether a cancer occurs early or late in life, the path to more effective and less toxic therapies is essentially the same - it starts with knowing the genetic changes underlying the disease. The tools for discovering these changes may also be the same in childhood and adult cancers, as an innovative public-private initiative hopes to demonstrate.

The initiative, sponsored by NCI and the Foundation for the National Institutes of Health, is called TARGET (the Childhood Cancer Therapeutically Applicable Research to Generate Effective Treatments). The goal is to identify and validate therapeutic targets for childhood cancers using the latest genetic and genomic tools, which are the same ones used to develop targeted drugs for adult cancers.

"This project was inspired by the powerful technologies that are available for identifying genetic lesions in cancer cells," said Dr. Malcolm Smith of NCI's Cancer Therapy Evaluation Program and a leader of TARGET. The initiative grew out of a 2005 workshop among cancer specialists from the public and private sectors. A consensus emerged that available technologies could advance research on childhood cancer therapies if sufficient resources were dedicated to the challenge.

A pilot phase of TARGET involving acute lymphoblastic leukemia (ALL) and led by researchers from the University of New Mexico, St. Jude Children's Research Hospital, the Children's Oncology Group, and NCI has produced interesting early results. Profiling gene activity in patient samples revealed a previously unknown subtype of ALL associated with a poor prognosis. Additional studies are underway to search for genomic abnormalities such as extra copies of genes that may underlie the abnormal gene activity detected in the patient samples.

NCI's Division of Cancer Prevention sponsors research to reduce treatment side effects, including mouth sores and nausea and vomiting due to chemotherapy, and hearing loss in children treated with cisplatin. Search the NCI Cancer Research Portfolio using "pediatric research" as a special interest category for other examples.

With the diversity of data, the researchers expect eventually to identify the underlying genetic changes in this high-risk subgroup of children with ALL. "The challenge is to identify the genetic lesions that in one way or another are driving the leukemia," noted Dr. Smith.

The initiative is benefiting from lessons learned and tools developed during the implementation of The Cancer Genome Atlas pilot project, which is cataloging genomic alterations in three adult cancers. Both projects will share, for instance, software designed to make the results easily accessible to researchers via the Internet.

The integration of different types of data will be an ongoing process in TARGET. But the researchers believe that by studying relatively large numbers of patients, using consistent research methods, and sequencing between 100 and 150 genes per disease, they will be able to identify critical genes in these cancers that can lead to more effective treatments.

The pilot project is also working on neuroblastoma, with plans to expand to rhabdomyosarcoma and other cancers in the future.