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Cancer

Research

Leukemia/Lymphoma

Translational research-bringing basic science discoveries to bedside care-is key to developing new drugs that target pediatric leukemia. Our researchers are evaluating new approaches that might be effective in the treatment of T-cell lymphoblastic leukemia-a cancer that accounts for approximately 15 percent of lymphoblastic disease in our pediatric patients. While this aggressive form of leukemia will sometimes respond to more intensive therapy, the treatment can be very toxic to the patient. Our researchers are studying the molecular basis of T-cell lymphoblastic leukemia to identify potential targets for therapy that will lead to more effective, less toxic treatment.

In addition, new technologies derived from the completion of the human genome are becoming increasingly important in studying T-cell lymphoblastic leukemia. Our basic scientists are using genomic tools to analyze thousands of genes at a time, providing an enormous amount of information to explore this cancer in depth. By looking at an individual's genetic profile, researchers are hoping to predict outcome and potential toxicity of the treatment to help tailor therapeutic agents to each patient.

Our physicians are also interested in the effects of nutrition on leukemia treatment side effects and outcomes, and they are collecting prospective nutritional data on more than 500 children with acute lymphoblastic leukemia. In collaboration with the Dana Farber Cancer Institute, we are investigating metabolizing enzymes, dietary intake, toxicity and the event-free survival in children with acute lymphoblastic leukemia to determine if a gene-nutrient interaction exists.

Solid Tumors

Our Pediatric Cancer Foundation Solid Tumor Clinical Research Program develops and tests novel treatment strategies for pediatric solid tumors, including clinical trials for patients with unusual solid tumors or hard-to-treat disease. These therapies call on the research findings of the pediatric tumor biology laboratory, a joint effort with the Hospital's Division of Pediatric Surgery. Our researchers have demonstrated that pediatric tumors make an essential protein, vascular endothelial growth factor (VEGF), and that treatment with anti-VEGF antibody, bevacizumab or the novel VEGF blocking agent, VEGF-Trap, can dramatically inhibit growth in experimental models for neuroblastoma, Wilms' tumor, and hepatoblastoma. As a direct result of this research, our Division's scientists led the first COG pediatric clinical trial with bevacizumab for patients with hard-to-treat solid tumors.

Our solid tumor research also focuses on angiogenesis-the process by which new blood vessels are formed. Tumor angiogenesis is the growth of blood vessels from surrounding tissue to a solid tumor. In the presence of a solid tumor, angiogenesis can occur as a result of the release of chemicals by the tumor. Some cancer treatments work by blocking angiogenesis thus preventing blood from feeding the tumor. With an overall goal of developing new treatment regimens for pediatric cancer patients, our basic and clinical investigators are involved in collaborative research to identify the mechanisms of angiogenesis, develop methods for inhibiting the blood supply to tumors, and define combinations of angiogenesis inhibitors with the potential to shrink tumors rather than inhibit their growth.

A clinical trial is now under way that combines the anti-angiogenic agents bevacizumab and erlotinib for pediatric cancer to determine the potential for these drugs in combination with chemotherapies to reduce the side effects of chemotherapy while still providing their therapeutic effect.

Neuro-oncology

Clinical research interests of the brain tumor team include the use of high-dose chemotherapy with deferred irradiation for infant brain tumors, as well as supportive care initiatives that involve collaboration with the psychosocial group. Other studies include:

  • Phase I/II study of topotecan by intracerebral clysis-a system that delivers drugs directly to the site of a brain tumor-for treatment of recurrent brain tumors
  • Identification of brain tumor cell migratory motor proteins as targets for investigational therapies
  • Erlotinib/etoposide for recurrent brain tumors

Our physicians are also studying the treatment outcomes in children with germ cell tumor, including chemotherapy for patients with newly diagnosed germ cell tumors of the central nervous system and high-dose chemotherapy with bone marrow reconstitution for patients with recurrent germ cell tumors.

Molecular Epidemiology

The Division's Molecular Epidemiology Laboratory is investigating the root cause of childhood cancer secondary to genetic and environmental interactions. These include the role of folate deficiency and genetic variation in metabolism of folate and the development of sporadic (non-familial) retinoblastoma and the role of prenatal exposures to procarcinogenic PAH during pregnancy on the development of chromosomal aberrations detectable in cord blood.

Contact

Herbert Irving Child and Adolescent Oncology
(212) 305-5808
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