Symptoms and General Pathology

All Conditions

Neoplasms

Nervous System Diseases

Rare Diseases

Recurrence

Glioblastoma

Glioma

Brain Neoplasms

Astrocytoma

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Central Nervous System Neoplasms

Nervous System Neoplasms

Brain Diseases

Central Nervous System Diseases

Brain Tumor, Childhood

Anaplastic Astrocytoma

Neuroepithelioma

Childhood Brain Stem Glioma

Antineoplaston therapy (Atengenal + Astugenal) by IV infusion every four hours for at least 12 months. Study subjects receive increasing dosages of Atengenal and Astugenal until the maximum tolerated dose is reached.

Children with a primary malignant brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).

Antineoplaston therapy (Atengenal + Astugenal)

Stanislaw R. Burzynski, MD, PhD

RATIONALE: Current therapies for children with primary malignant brain tumors provide limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of children with primary malignant brain tumors.

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children with primary malignant brain tumors.

OBJECTIVES:

* To determine the efficacy of Antineoplaston therapy in children with primary malignant brain tumors as measured by an objective response to therapy (complete response, partial response) or stable disease.
* To determine the safety and tolerance of Antineoplaston therapy in children with primary malignant brain tumors.

OVERVIEW: This is a single arm, open-label study in which children with primary malignant brain tumors receive gradually escalating doses of intravenous Antineoplaston therapy (Atengenal + Astugenal) until the maximum tolerated dose is reached. Treatment continues for at least 12 months in the absence of disease progression or unacceptable toxicity. After 12 months, patients with a complete or partial response or with stable disease may continue treatment.

To determine objective response, tumor size is measured utilizing MRI scans, which are performed every 8 weeks for the first two years, every 3 months for the third and fourth years, every 6 months for the 5th and sixth years, and annually thereafter.

PROJECTED ACCRUAL: A total of 20-40 patients will be accrued to this study

Antineoplaston Therapy in Treating Children With Primary Malignant Brain Tumors

Phase II Study of Antineoplastons A10 and AS2-1 in Children With Primary Malignant Brain Tumors

Objective response rate per Response Assessment in Neuro-Oncology (RANO) for target lesions and assessed by MRI: Complete Response (CR), disappearance of all disease sustained for at least four weeks; Partial Response (PR), \>=50% decrease in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least four weeks; Stable Disease (SD), \<50% decrease and \<25% increase in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least eight weeks; Progressive Disease (PD), \>=25% increase in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions.

Six months and Twelve months overall survival

Burzynski Research Institute

Eight patients were recruited between March 1996 and November 2011. All study subjects were seen at the Burzynski Clinic in Houston TX

Antineoplaston therapy (Atengenal + Astugenal) by IV infusion every four hours for at least 12 months. Study subjects receive increasing dosages of Atengenal and Astugenal until the maximum tolerated dose is reached.

Antineoplaston therapy (Atengenal + Astugenal): Children with a primary malignant brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).

Antineoplaston Therapy

Age, Continuous

Sex: Female, Male

S. R. Burzynski, MD, PhD

Partial Response

Stable Disease

Progressive Disease

Number of Participants With Objective Response

6 months overall survival

12 months overall survival

All study subjects receiving any Antineoplaston therapy

Percentage of Participants Who Survived

Overall Study

Diffuse Intrinsic Pontine Glioma

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Brain Stem Neoplasms

Infratentorial Neoplasms

Fresh-frozen and fixed tumor samples, correspondent normal brain tissue samples, cerebrospinal fluid, urine, and serum samples from patients affected with diffuse intrinsic pontine glioma or brainstem glioma

Javad Nazarian, PhD

Madhuri Kambhampati, MS

Roger Packer, MD

Suresh Magge, MD

Amanda L Muhs, MD

The purpose of this study is to prospectively collect specimens from pediatric patients with diffuse intrinsic pontine glioma or brainstem glioma, either during therapy or at autopsy, in order to characterize the molecular abnormalities of this tumor.

High grade diffuse intrinsic pontine glioma (DIPG) accounts for approximately 80% of pediatric brainstem tumors and 10% of pediatric brain tumors, and is the most lethal form of brainstem gliomas in children. There is currently no effective therapy to treat these tumors. We hypothesize that this tumor exhibits unique molecular abnormalities leading to altered RNA and protein expression. The aim of this trial is to collect specimens from pediatric patients with diffuse intrinsic pontine glioma including serum, cerebrospinal fluid, urine, brain tumor and other constitutional tissue, during therapy and/or at autopsy. Our goal is to study this tissue to characterize the genetic abnormalities that lead to tumor formation in order to identify key molecules as biomarkers which we can target to design and test new and more effective treatments.

Cerebrospinal fluid, serum, urine, brainstem tumor and constitutional tissue from patients with diffuse intrinsic pontine glioma or brainstem glioma will be collected.

Molecular Analysis of Samples From Patients With Diffuse Intrinsic Pontine Glioma and Brainstem Glioma

Collected tumor and normal samples will potentially be used for RNA genome-wide expression pattern profiling.

The molecular analysis done on collected samples will be validated through whole genome sequencing.

To obtain full characterization of collected samples, proteomic profiling will be done on tumor and normal samples collected.

Collected tumor and normal samples will have the immunochemistry and western blot compared to assess protein expression variation.

To obtain full characterization of collected samples, whole genome sequencing will be done on tumor and normal samples collected.

Collected samples will potentially be used for in vitro analysis and generation of animal models of this tumor.

In an effort to continue to draw knowledge from samples collected, potential benefits and drawbacks from specimen acquisition will be continuously accessed.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Antineoplaston Therapy in Treating Children With Primary Malignant Brain Tumors	NCT00003476	Childhood Brain...	Antineoplaston ...	6 Months - 18 Years	Burzynski Research Institute
Molecular Analysis of Samples From Patients With Diffuse Intrinsic Pontine Glioma and Brainstem Glioma	NCT01106794	Diffuse Intrins... Brainstem Gliom...		- 21 Years	Children's National Research Institute

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