Phase II Study of Antineoplaston A10 and AS2-1 in Patients with Recurrent Diffuse Intrinsic Brain Stem Glioma

Abstract

The results of this study compared favourably with the responses of patients treated with radiation therapy and chemotherapy. The study continues with accrual of additional patients.

Complementary and Alternative Therapies for Cancer

“…A joint research effort by the NIH Office of Alternative Medicine and the NCI failed to accrue sufficient numbers of patients, and none of the six patients assessable for response showed tumor regression [45]. Further research at the Burzynski Institute was permitted under an Investigational New Drug permit [46]. The group's preliminary report from a singlearm phase II study of 12 patients showed a 50% response rate [46].…”

“…Further research at the Burzynski Institute was permitted under an Investigational New Drug permit [46]. The group's preliminary report from a singlearm phase II study of 12 patients showed a 50% response rate [46]. Researchers at the NCI and elsewhere continue to investigate phenylacetate, a metabolite of the amino acid 84 Complementary/Alternative Cancer Therapies phenylalanine, which makes up 80% of antineoplastons [47,48].…”

Long-Term Survival and Complete Response of a Patient with Recurrent Diffuse Intrinsic Brain Stem Glioblastoma Multiforme

“…4 A preliminary report on a phase II study of ANP in patients with recurrent diffuse intrinsic brain stem gliomas has recently been published. 5 In a small group of 12 patients, there were 5 patients with objective responses (complete and partial responses), 3 patients with stable disease, and 2 patients with progressive disease who subsequently died. Two patients have been surviving for more than 7 years post diagnosis.…”

Targeted Therapy With Antineoplastons A10 and AS2-1 of High-Grade, Recurrent, and Progressive Brainstem Glioma

“…[4][5][6] New strategies employing a multitargeted approach may offer better results for these tragic tumors. [7][8][9][10][11] Among these new multitargeted agents is the treatment with antineoplastons A10 (A10 I) and AS2-1 (ANP), which are synthetic analogs of naturally occurring sodium salts of phenylacetylglutamine (PG), phenylacetylisoglutamine, and phenylacetic acid (PN). 12 ANP affects the RAS, AKT, TP53, PTEN, INI1, p21, MYCC, and apoptosis pathways.…”

“…The proposed mechanism of action of ANP involves a decrease of DNA instability and amplification of oncogenes through normalization of global methylation of the genes by A10I (PG) and activation of silenced tumor suppressors through inhibition of methylation of their promoters and deacetylation of histones by AS2-1 (PN). 7,12,13 In addition, both PG and PN promote apoptosis of neoplastic cells (Figure 1). Additional mechanisms that are involved include inhibition of farnesylation of the p21 protein of the RAS oncogene by PN, normalization of nuclear transport of the mutated INI1 protein, and deamidation of BCL-X L by PG.…”