What research is NCI currently doing on childhood cancer?

The NCI is funding a large portfolio of studies (http://fundedresearch.cancer.gov/) looking at the causes of and the most effective treatments for childhood cancers. Ongoing investigations include:

Studies to identify causes of the cancers that develop in children: The Children’s Oncology Group (COG) (http://www.childrensoncologygroup.org Exit Disclaimer) is evaluating potential risk factors for a variety of childhood cancers. Very large studies have been completed of childhood acute lymphoblastic leukemia, acute myeloid leukemia, non-Hodgkin lymphoma, primitive neuroectodermal tumors of the brain, astrocytoma, neuroblastoma, and germ cell tumors. One large study, the Childhood Cancer Survivor Study, is evaluating the risks of second cancers related to radiation therapy and chemotherapy received by survivors of childhood cancer as part of treatment for their primary cancer (see below).

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COG has also established a Childhood Cancer Research Network that creates a national registry of children with cancer. This initiative builds upon the unique NCI-supported national clinical trials system for treating children with cancer.

Monitoring of U.S. and international trends in incidence and mortality rates for childhood cancers: By identifying places where high or low cancer rates occur, researchers can uncover patterns of cancer that provide important clues for further in-depth studies into the causes and control of cancer.

Studies to better understand the biology of childhood cancer, with the hope that this understanding will lead to new treatment approaches that target critical cellular processes required for cancer cell growth and survival: The Childhood Cancer Therapeutically Applicable Research to Generate Effective Treatments (TARGET) Initiative was established by the NCI and the Foundation for the National Institutes of Health to identify and validate therapeutic targets in childhood cancers. The first TARGET project focuses on targets for high-risk acute lymphoblastic leukemia and the second TARGET project focuses on neuroblastoma. More information about the TARGET Initiative can be found in the article “Initiative TARGETs Childhood Cancer” at http://www.cancer.gov/NCICancerBulletin/NCI_Cancer_Bulletin_112106 on the Internet.

• Preclinical studies (animal studies) of new agents to identify promising anticancer drugs that can be evaluated in clinical trials: The NCI-supported Pediatric Preclinical Testing Program (PPTP) systematically evaluates new drugs and substances using animal models (animals with a cancer similar to or the same as a cancer found in children) to find the drugs most likely to have significant anticancer effects in clinical trials. The program is based on a large amount of research showing that animal models imitate the effects of proven anticancer drugs and can be used to prospectively identify new drugs that are effective against childhood cancers in subsequent patient studies. More information about the PPTP is available at http://pptp.nchresearch.org/ Exit Disclaimer on the Internet. Questions concerning the PPTP can be addressed to the PPTP Project Officer, Dr. Malcolm Smith ([email protected]).

Projects designed to improve the health status of survivors of childhood cancers: The NCI funds the Childhood Cancer Survivor Study (CCSS), a study coordinated by St. Jude Children’s Research Hospital. The CCSS has over 25 sites across the country at medical institutions with doctors specializing in long-term care for children and young adults. This study was created to gain new knowledge and to educate cancer survivors about the long-term effects of cancer and cancer treatment. Information about the study is available at http://ccss.stjude.org/ Exit Disclaimer on the Internet.

Clinical trials to identify superior treatments for childhood cancers, thereby leading to improved survival rates for children with cancer: Each year about 4,000 children enter 1 of approximately 100 ongoing clinical trials sponsored by the NCI. The following groups are conducting these trials:

The COG, with support from the NCI, conducts clinical trials devoted exclusively to children and adolescents with cancer at more than 200 member institutions, including cancer centers of all major universities, teaching hospitals throughout the United States and Canada, and sites in Europe and Australia. COG was formed in 2000 by the merger of four children’s cancer cooperative groups to accelerate the search for a cure for childhood cancers and to make it possible for children with cancer, regardless of where they live, to have access to state-of-the-art therapies and the collective expertise of world-renowned pediatric specialists.

The Pediatric Brain Tumor Consortium (PBTC) (http://www.pbtc.org Exit Disclaimer) includes 10 leading academic institutions with extensive experience in the design and conduct of clinical trials for children with brain tumors. The group’s primary objective is to rapidly conduct phase I and II clinical evaluations of new therapeutic drugs, treatment delivery technologies, new biological therapies, and radiation treatment strategies in children up to age 21 with primary central nervous system (CNS) tumors. Another objective of the PBTC is to develop and coordinate innovative neuroimaging techniques. Results from PBTC studies are made available to large international collaborative groups for confirmatory phase II and multiagent phase III clinical trials.

New Approaches to Neuroblastoma Therapy (NANT) (http://www.nant.org Exit Disclaimer) is a consortium of university and children’s hospitals funded by the NCI to test promising new therapies for neuroblastoma. NANT members constitute a group of closely collaborating investigators linked with laboratory programs where novel therapies for high-risk neuroblastoma are being developed. The group conducts early clinical trials to test new drugs and new combinations of drugs so promising therapies can be tested nationally.

The Pediatric Oncology Branch (POB) (http://pediatrics.cancer.gov) of the NCI’s Center for Cancer Research conducts basic, preclinical, and clinical studies of childhood cancer at the NIH Clinical Center in Bethesda, MD. Basic studies include analyses of genetic and biological characteristics of childhood cancers, as well as the study of immune system interactions with these cancers and the effects of chemotherapy on the immune system. Preclinical studies by the POB identify new drugs and types of immunotherapy (treatment to boost the immune system’s ability to fight cancer), as well as agents to control infectious diseases that occur in childhood cancer patients. An active clinical trial program includes phase I and phase II studies of new agents to treat childhood cancers, with a focus on molecularly targeted therapy and immunotherapy, as well as bone marrow transplantation and the development of immunotoxins (antibodies linked to a toxic substance that bind to cancer cells and kill them) to treat childhood leukemia. The POB also develops and tests new treatments for tumors associated with genetic predisposition syndromes such as neurofibromatosis type 1 and multiple endocrine neoplasia.

Evaluations of new drugs that may be more effective against childhood cancers and that may have less toxicity for children: The COG Phase I/Pilot Consortium is a major component of the NCI’s pediatric drug development program. The primary objective of the consortium is to develop and implement pediatric phase I and pilot studies to promote the integration of advances in cancer biology and therapy into the treatment of childhood cancer. The consortium includes approximately 20 institutions that carefully monitor the drugs for toxicity and safety. After their initial evaluation for safety in children by the consortium, the agents and regimens can then be studied within the larger group of COG institutions to determine their role in the treatment of specific childhood cancers.

Selected References

1. American Cancer Society. Cancer Facts and Figures 2007. Atlanta, GA: American Cancer Society. Retrieved December 26, 2007, from http://www.cancer.org/downloads/STT/CAFF2007PWSecured.pdf

2. Ries LAG, Melbert D, Krapcho M, et al. SEER Cancer Statistics Review, 1975–2004. Bethesda, MD: National Cancer Institute. Retrieved December 26, 2007, from http://seer.cancer.gov/csr/1975_2004.

3. Smith MA, Freidlin B, Ries LA, Simon R. Trends in reported incidence of primary malignant brain tumors in children in the United States. Journal of the National Cancer Institute 1998; 90(17):1269–1277.

Source: National Cancer Institute