A Study of Vorinostat (SAHA) plus Isotretinoin and Chemotherapy in Infants and Young Children with Embryonal Tumors of the Central Nervous System

Full Title
PBTC-026: A Feasibility Study of Vorinostat (SAHA) combined with Isotretinoin and Chemotherapy in Infants with Embryonal Tumors of the Central Nervous System.

The purpose of this study is to determine the safety of adding two drugs to the standard combination of chemotherapy drugs used to treat medulloblastoma and supratentorial primitive neuroectodermal tumor (PNET). The standard drugs include vincristine, etoposide, cisplatin, and cyclophosphamide. The two additional drugs are vorinostat and isotretinoin, which will be given during the initial (induction) phase of therapy and then later, during the “maintenance” stage of chemotherapy.

Vorinostat is approved for treating a form of lymphoma, but its use for medulloblastoma and PNET is considered investigational. It is a drug called a histone deacetylase inhibitor, which prevents cancer cells from reproducing by interfering with certain signaling pathways. Isotretinoin is a chemical cousin of vitamin A that is most often used to treat acne, but also has activity against brain and other cancers. Both drugs are taken orally (by mouth).

This study is being done as part of the Pediatric Brain Tumor Consortium, a group of eleven pediatric neuro-oncology centers in the United States dedicated to improving the treatment of childhood brain tumors.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have newly diagnosed medulloblastoma or PNET.
  • Patients may not have had therapy other than surgery or steroids.
  • This study is open to children at least 2 months old and under age 4 years.

For more information and to inquire about eligibility for this study, please contact Dr. Kim Kramer at 212-639-6410.

Pediatric Brain Tumors
Pediatric Brain Tumors: Brain Cancer
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