Non-Therapeutic Protocol

NBTP01: Molecular and Histopathologic Characterization of Atypical Teratoid Rhabdoid Tumors, Choroid Plexus Carcinomas, Ependymomas and Medulloblastoma / PNET of the Pediatric CNS

Type of Protocol/Clinical Study

Associated Solid Tumor Studies

Description

Atypical teratoid rhabdoid tumors (ATRT) and choroid plexus carcinomas (CPC) are poorly understood, highly aggressive (grow and spread quickly) tumors of the pediatric central nervous system. These tumors occur most frequently in children less than two years of age.  Medulloblastoma / primitive neurorectodermal tumors (PNET) are the most common type of brain tumors occurring in children. Ependymoma is the third most common brain tumor in children. Chemotherapy alone does not work in all cases. Radiation therapy cannot be used due to the long term side effects associated with radiation given to very young children. Effective treatment options in these tumors are limited. By studying the abnormalities and changes in the tumors of these subjects, we can gain a better understanding of the biology to help to develop new and hopefully better treatment in the future.

Objectives

  • The main goal of this study is to study the characteristics and abnormalities of the tumors of subjects with atypical teratoid rhabdoid tumors (ATRT), choroid plexus carcinomas (CPC), ependymomas  and medulloblastoma / primitive neurorectodermal tumors (PNET); then to learn if these abnormalities are related to the way tumors look under the microscope and the way the tumors behave in subjects.  The tumor will be studied genetically (the study of genes and how they are inherited) and pathologically (how tumors look under the microscope).
  • Researchers will also culture and grow tumors from cells collected from the tumor of research participants in this study so that we can learn more about how these tumors grow and spread, and if the tumors will respond to different drug treatments.

Eligibility

  • Participant has ATRT, CPC, ependymoma or medulloblastoma/PNET of the central nervous system as documented by the local neuropathologist and not the study neuropathologist. 
  • Tumor may be primary, progressive or relapsed.
  • Participant is not more than 21 years of age at the time of diagnosis.

 

Principal Investigator

Amar Gajjar, MD

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