TITLE:
A Case of Sustained Objective Response of Recurrent/Progressive Diffuse Intrinsic Pontine Glioma with Phenylbutyrate and Targeted Agents
AUTHORS:
Stanislaw R. Burzynski, Gregory S. Burzynski, Sheldon Brookman
KEYWORDS:
Antineoplastons, Brainstem Glioma, DIPG, Phenylbutyrate, Targeted Agents, Treatment of DIPG
JOURNAL NAME:
Journal of Cancer Therapy,
Vol.6 No.1,
January
8,
2015
ABSTRACT: Diffuse intrinsic pontine
glioma (DIPG) is the most common type of brainstem glioma and one of the most
deadly brain tumors. DIPG in young adult patients is a rare disease for which
treatment options are limited. Radiation therapy remains the standard-of-care
for newly-diagnosed DIPG, but no established therapies for recurrent disease are
available. This paper describes the results of treatment of a young adult
patient diagnosed with DIPG that progressed after radiation therapy. Therapy
included sodium phenylbutyrate (PB) in combination with the targeted agents:
pazopanib, everolimus, erlotinib, and bevacizumab. The patient achieved a rapid
partial response, which persisted over a year and five months. The patient
opted to discontinue the therapy and thereafter elected chemotherapy, which
resulted in a subsequent rapid progression and death within one month. The
targeted treatment was associated with minor toxicity that included a Grade 2
skin rash and Grade 1 elevation of transaminases. In conclusion, a combination
of PB and currently available targeted drugs may offer extended survival in
patients with recurrent DIPG.