Phase II study of antineoplaston A10 and AS2-1 in children with recurrent and progressive multicentric glioma : a preliminary report

Drugs R D. 2004;5(6):315-26. doi: 10.2165/00126839-200405060-00002.


Objective: To evaluate the response rates, survival and toxicity of treatment with antineoplaston A10 and AS2-1 (ANP) in the first 12 children enrolled in our studies diagnosed with incurable recurrent and progressive multicentric glioma.

Patients and methods: The patients' median age was 9 years. Six patients were diagnosed with pilocytic astrocytoma, four with low-grade astrocytoma and one with astrocytoma grade 2. In one case of visual pathway glioma, a biopsy was not performed due to a dangerous location. Patients received ANP intravenously initially and subsequently orally. The average duration of intravenous ANP therapy was 16 months and the average dosage of A10 was 7.95 g/kg/day and of AS2-1 was 0.33 g/kg/day. The average duration of oral ANP was 19 months and the average dosage of A10 and AS2-1 was 0.28 g/kg/day. Responses were assessed by MRI according to the National Cancer Institute's criteria and confirmed by PET scans in some cases.

Results: Complete response was accomplished in 33%, partial response in 25%, and stable disease in 33% of patients, and there was no progressive disease. One patient was non-evaluable due to only 4 weeks of ANP and lack of follow-up scans. One patient who had stable disease discontinued ANP against medical advice and died 4.5 years later. Ten patients are alive and well from 2 to >14 years post-diagnosis. Only one case of serious toxicity of reversible tinnitus, of one day's duration, was described. The study continues with accrual of additional patients.

Conclusion: The results of the present study are favourable in comparison with radiation therapy and chemotherapy. We believe that confirmation of these results through further studies may introduce a new promising treatment for incurable paediatric brain tumours.

Publication types

  • Clinical Trial
  • Clinical Trial, Phase II
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Antineoplastic Agents / adverse effects
  • Antineoplastic Agents / therapeutic use*
  • Astrocytoma / drug therapy
  • Astrocytoma / pathology
  • Benzeneacetamides / adverse effects
  • Benzeneacetamides / therapeutic use*
  • Brain Neoplasms / drug therapy*
  • Child
  • Child, Preschool
  • Disease Progression
  • Drug Combinations
  • Drug Therapy, Combination
  • Female
  • Glioma / drug therapy*
  • Glutamine / adverse effects
  • Glutamine / analogs & derivatives*
  • Glutamine / therapeutic use*
  • Humans
  • Infant
  • Magnetic Resonance Imaging
  • Male
  • Neoplasm Recurrence, Local / prevention & control
  • Phenylacetates / adverse effects
  • Phenylacetates / therapeutic use*
  • Piperidones / adverse effects
  • Piperidones / therapeutic use*
  • Survival Analysis
  • Treatment Outcome


  • Antineoplastic Agents
  • Benzeneacetamides
  • Drug Combinations
  • Phenylacetates
  • Piperidones
  • Glutamine
  • antineoplaston AS 2-1
  • antineoplaston A10