Journal of Neuro-Oncology

, Volume 59, Issue 3, pp 239–242 | Cite as

Complete Response of a Recurrent, Multicentric Malignant Glioma in a Patient Treated with Phenylbutyrate

  • Matthew J. Baker
  • Steven Brem
  • Stephanie Daniels
  • Beverly Sherman
  • Surasak Phuphanich


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.

astrocytoma biological response modifier brain tumor differentiation agent glioma phenylbutyrate 


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Copyright information

© Kluwer Academic Publishers 2002

Authors and Affiliations

  • Matthew J. Baker
    • 1
  • Steven Brem
    • 1
    • 2
    • 3
    • 4
    • 5
  • Stephanie Daniels
    • 1
  • Beverly Sherman
    • 1
  • Surasak Phuphanich
    • 1
    • 2
    • 3
  1. 1.Neuro-Oncology Program, H. Lee Moffitt Cancer Center and Research InstituteTampaUSA
  2. 2.Department of NeurologyUniversity of South Florida College of MedicineTampaUSA
  3. 3.Department of Interdisciplinary OncologyUniversity of South Florida College of MedicineTampaUSA
  4. 4.Department of NeurosurgeryUniversity of South Florida College of MedicineTampaUSA
  5. 5.Department of PharmacologyUniversity of South Florida College of MedicineTampaUSA

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