Abstract
Treatment of recurrent glioblastoma multiforme (rGBM) poses a difficult challenge. Therefore, the purpose of this report was to evaluate objective response (OR), progression-free survival (PFS), overall survival (OS), and the incidence of adverse events (AEs) in rGBM patients, age 19-70 years, who were treated with antineoplaston AS2-1 (Astugenal) plus targeted therapy. A retrospective analysis was performed. Tumor response was assessed by gadolinium-enhanced magnetic resonance imaging (MRI). Twenty-nine adult rGBM patients were treated between 9/11/2015 and 06/23/2018. Seven had no prior treatment with bevacizumab elsewhere, had radiologic evidence of rGBM, and had MRI assessment of tumor response. The median treatment time was 101 days (range: 55-208 days). OR was seen in six patients (85.7%) with complete disappearance of gadolinium enhancement in four patients (57.1%) and a 50% or greater reduction in gadolinium enhancement in two patients (28.6%). Progressive disease was seen in one patient (14.3%). The median time to first response was 29 days (range: 22-96 days) while the median duration of response was 141 days (range 55-739+ days). Six- and 12-month PFS was 57% and 19%, respectively while 6- and 12-month OS at was 86% and 54%, respectively. Treatment was well-tolerated with no patients experiencing grade 3 or 4 antineoplaston-related toxicity. Regarding response to treatment and toxicity, AS2-1 plus targeted therapy compares favorably with other reported rGBM therapies. Duration of response was shortened by the ill-advised decision of some patients to discontinue treatment after a tumor response was achieved.
 
  
Highlights
Glioblastoma multiforme (GBM) is the most common and most aggressive primary malignant brain tumor (Ostrom et al, 2018)
Possible responses to treatment included an objective response (OR), complete disappearance of gadolinium enhancement (CR) or ≥ 50% reduction in gadolinium enhancement (PR), i.e., SUM ≤ 50% of baseline SUM, and progressive disease (PD), which was evident when there was a ≥25% increase in gadolinium enhancement (i.e., SUM ≥ 25% of the baseline SUM or ≥ 25% of the smallest SUM achieved during treatment) or the appearance of new measureable lesions
PD, a ≥ 25% increase in gadolinium enhancement (i.e., SUM ≥ 25% of the baseline SUM or ≥ 25% of the smallest SUM achieved during treatment) or the appearance of new measureable lesions, was seen in one patient (14.3%)
Summary
Glioblastoma multiforme (GBM) is the most common and most aggressive primary malignant brain tumor (Ostrom et al, 2018). The prognosis for newly-diagnosed GBM patients is poor with a median survival of 14.6 months from diagnosis despite standard treatment with surgery, radiation therapy (RT), adjuvant temozolomide (TMZ) and bevacizumab (BVZ) (Stupp et al, 2005; Ohka, Natsume & Wakabayashi, 2012). In phase II clinical trials (IND 43742), we have evaluated treatment of GBM and rGBM with antineoplastons, a group of peptides, amino acid derivatives, and carboxylic acids that inhibit the growth of neoplastic cells without inhibition of the growth of normal cells (Burzynski, 1976; Burzynski, 1986; Burzynski, 2004; Burzynski, 2006; SR Burzynski, Janicki, GS Burzynski & Marszalek, 2014, 2051-2061; SR Burzynski, Janicki, GS Burzynski & Marszalek, 2014, 565-577; SR Burzynski, Janicki & GS Burzynski, 2014; SR Burzynski, Janicki, GS Burzynski, Marszalek & Brookman, 2014; SR Burzynski, GS Burzynski & Janicki, 2014). Objective responses (ORs) were achieved in 17% of eligible patients while overall survival (OS) was 65.5% at six months, 56.7% at nine months, 39% at one year, and 4.4% at two, five, and ten years (SR Burzynski, Janicki & GS Burzynski, 2014)
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