A Phase II Study of Antineoplastons A10 and AS2-1 in Patients with Brainstem Gliomas. The Report on Non-Diffuse Intrinsic Pontine Glioma (Protocol BT-11)

Abstract

Inoperable brainstem gliomas (BSG) are among the most difficult to treat malignancies. In the intent-to-treat (ITT) population of the BT-11 study for BSG, forty patients (median age 11.2 years old) were enrolled. Antineoplastons A10 and AS2-1 (ANP) were administered intravenously daily. The median daily dose of A10 was 8.70 g/kg/day and AS2-1 was 0.32 g/kg/day. Efficacy analyses were conducted in two subgroups: recurrent pediatric diffuse intrinsic pontine glioma (RPDIPG, N = 17) and non-diffuse intrinsic pontine glioma (NDIPG, N = 11). This paper reports the results of the study of the efficacy and safety of ANP in patients with NDIPG. The results in the RPDIPG group were reported before; complete response (CR) was 6%, partial response (PR) 23.5%, and stable disease (SD) 17.6%. One year overall survival (OS) was 29.4%, 2 years 11.8%, and 5, 10, and 15 years 6%. In the NDIPG group, there were 36% CR and 27.5% SD. OS at 1, 5, 10, and 15 years was 82%, 73%, 62%, and 50% correspondingly. There was only one serious adverse event (9%) reported in NDIPG represented by hypokalemia, Grade 4. The results suggest that ANP shows efficacy and an acceptable tolerability profile in patients with RPDIPG and NDIPG.

Resolution of clinical signs, a complete response, and long-term survival (> 23 Years) in a 3 and ½ month female with a newly diagnosed diffuse intrinsic pontine glioma treated with antineoplastons

“…Baseline MRI (October 13, 1988), on coronal images, showed an enhancing lesion measuring 10.73 cm 2 while axial and sagittal images showed a non-enhancing lesion measuring 12.58 cm 2 and a 14.70 cm 2 , respectively. The patient was treated with IV ANP therapy per BT-11 [8]. The dosages of A10 and AS2-1 were gradually increased to 9.42 g/kg/d and 0.56 g/kg/d, respectively.…”

“…On October 14, 1998, this 3 ½ month female was enrolled as a special exception, according to protocol BT-11, for administration of IV ANP. BT-11 was a Phase II study of Antineoplaston 10 (Atengenal) and Antineoplaston AS2-1 (Astugenal) for patients with brainstem gliomas [8]. The protocol was designed to determine an objective response (OR) to ANP therapy.…”

Antineoplastons A10 and AS2-1 in the Treatment of Children with Optic Pathway Glioma: Final Report for Protocol BT-23

“…The details of ANP administration have been previously published (Burzynski et al, , 2051(Burzynski et al, -2061Burzynski et al, 2015;Burzynski et al, 2014, 565-577;Burzynski et al, 2014, 977-988). Escalation of the dosage of ANP is based on the results of prior studies carried out to determine patients' tolerance of the infusion of the large volume of intravenous fluids necessary to deliver the higher doses of ANP (Burzynski, 2004).…”

Long-Term Survival Over 21 Years and Pathologically Confirmed Complete Response in Pediatric Anaplastic Astrocytoma: A Case Report

“…ANP A10 capsules contain 500 mg of 3-phenylacetylamino-2,6-piperidinedione, which is metabolized in the body to 4:1 ratio of PG, and isoPG and AS2-1 capsules contain 500 mg of 4:1 ratio of PN and PG [6]. Interim results of the phase II studies have already been reported as well as case reports of long-term survival of HGG and diffuse intrinsic pontine glioma (DIPG) [3,[7][8][9][10][11][12][13][14][15][16]. Here, we discuss a case of very long survival (over 21 years) of PAA with pathologically-confirmed complete response (CR).…”