Treatment > Phenylbutyrate


J Neurooncol 2002 Sep;59(3):239-42 (Case Report)
Comment in:
J Neurooncol. 2004 Jan;66(1-2):251; author reply 251.[BrainLife]



Abstract

Complete response of a recurrent, multicentric malignant glioma in a patient treated with phenylbutyrate

Baker MJ, Brem S, Daniels S, Sherman B, Phuphanich S

Neuro-Oncology Program, H. Lee Moffitt Cancer Center and Research Institute, Tampa, FL 33612-9497, USA

Sodium phenylbutyrate is a biological-response modifier that acts as a dose-dependent inhibitor of glioma cell proliferation, migration, and invasiveness in vitro, possibly by inhibition of urokinase and c-myc pathways. 
Despite its biological activity in vitro, there have not been any prior reports of efficacy in the treatment of human malignant gliomas. 
We report a 44-year-old female with a recurrent, multicentric, malignant glioma who experienced a durable remission lasting more than four years. 
The patient initially presented with seizures caused by a biopsy-proven anaplastic astrocytoma of the frontal lobe. 
The patient was treated with radiation therapy and Procarbazine-CCNU-Vincristine (PCV). 
However, the tumor progressed and extended to the corpus callosum with midline shift, refractory to four cycles of continuous 72-h infusion of BCNU/Cisplatinum. 
Additional enhancing lesions appeared in the left frontal and left temporal lobes. 
The patient was started on sodium phenylbutyrate, 18 g daily in three divided oral doses, and reduced to 9 g/day and eventually to 4.5 g/day to eliminate mild, reversible side effects. 
Four years later, the patient has a KPS functional score of 100%. 
Phenylbutyrate is a well-tolerated, oral agent that shows potential for the treatment of malignant gliomas. 
Further studies should be considered to identify a subset of patients that have tumors sensitive to phenylbutyrate, either as a single agent or in combination with radiation therapy or other chemotherapeutic agents.

PMID: 12241121 [PubMed - in process]

Source: http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?cmd=Retrieve&db=PubMed&list_uids=12241121&dopt=Abstract



 

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