Antineoplaston Therapy for Pediatric Brain Cancer: What Does the Evidence Show? What does the research show about antineoplaston therapy for pediatric brain cancer? Review ANP studies, reported responses, limitations, risks and FDA status.
Certain recurrent, progressive, high-grade, and brainstem pediatric brain tumors can be extremely difficult to treat. Families facing these diagnoses may therefore investigate experimental treatments such as antineoplaston therapy.
The biggest problem: the studies aren’t being interpreted. The page currently reproduces favorable results from three ANP studies. For example, the first reports complete responses in 33% and partial responses in 25% of 12 children; another reports five-year survival of 22% among 18 patients; and the PNET report describes six of 13 children surviving more than five years.
Those results certainly deserve to be discussed.
But the reader also needs to understand: these are very small studies; they aren’t randomized controlled trials; several involve heterogeneous patients and prior treatments; and much of the published ANP literature comes from the treatment’s developer and associates.
There are intriguing reported responses. But intriguing results are not the same thing as proof of efficacy.
Why I write about antineoplastons
My interest in ANP is personal. After several years of conventional treatment for multiple myeloma, including relapses and treatment-related side effects, I underwent antineoplaston therapy from November 1997 through April 1999. I subsequently achieved complete remission.
My experience is one patient’s experience. It cannot establish that ANP caused my remission or predict what ANP might do for another cancer patient—particularly a child with a brain tumor. For that reason, this article separates my personal experience from the published clinical evidence.
Because of my ANP experience, I am frequently contacted by cancer survivors of all types asking me about Dr. Burzynski and antineoplaston therapy. Because Burzynski and ANP are controversial, I have learned to stick to recounting my own cancer experience and offering studies. Several studies regarding pediatric cancer are linked below.
I am both a cancer survivor and cancer coach. Pediatric brain cancer is one of the most feared of all cancer diagnoses. Conventional oncology has little to offer the patient. If you would like to talk to me about ANP and pediatric brain cancer, please scroll down the page, post a question or comment and I will reply to you ASAP.
What is antineoplaston therapy?
Antineoplaston therapy is an experimental, unproven cancer treatment created in 1976 by Dr. Stanislaw Burzynski. It uses synthetic chemical compounds (peptides and amino acid derivatives) originally found in human blood and urine. Major health organizations, including the U.S. Food and Drug Administration (FDA), state it is not approved and lacks reliable scientific proof of effectiveness. [1, 2, 3, 4, 5, 6, 7]
Why do parents of children with difficult-to-treat brain tumors investigate ANP?
Parents of children with difficult-to-treat or terminal brain tumors usually investigate Antineoplaston Therapy (ANP) because they are seeking alternative options when conventional medicine offers no cure. ANP is a highly controversial alternative cancer treatment developed by Dr. Stanislaw Burzynski at the Burzynski Clinic in Houston, Texas. [1, 2, 3, 4, 5]
What pediatric brain cancers have been studied with antineoplastons?
Antineoplastons (A10 and AS2-1) have been studied in phase II clinical trials and case reports at the Burzynski Clinic for pediatric primary brain tumors, including high-grade and low-grade gliomas, brainstem gliomas, and embryonal tumors. [1, 2, 3, 4, 5]
What do the published ANP studies report?
| Study population |
Patients |
Reported findings |
Important limitation |
| Recurrent/progressive multicentric glioma |
12 |
CR 33%; PR 25%; SD 33% |
Very small, nonrandomized study |
| High-grade recurrent/progressive brainstem glioma |
18 |
2-year OS 39%; 5-year OS 22% |
Small phase II population |
| High-risk/recurrent PNET |
13 |
6 patients survived >5 years |
Mixed prior treatment and heterogeneous tumors |
These numbers should not be interpreted as proof that ANP produces these response or survival rates in children with brain cancer. Without randomized or well-controlled comparison groups, it is difficult to determine how much of an observed outcome resulted from ANP, previous treatment, tumor biology, patient selection, or other factors.
Is antineoplaston therapy FDA approved?
No, antineoplaston therapy is not approved by the U.S. Food and Drug Administration (FDA) to treat or prevent any disease. Major health organizations state there is not enough reliable proof that it works against cancer. [1, 2, 3]
What are the potential side effects?
Antineoplaston therapy can cause mild reactions as well as severe nervous system complications. Documented adverse effects range from fatigue and skin rashes to high blood pressure, confusion, and life-threatening seizures or brain swelling. Independent reviews note a lack of sound clinical proof supporting its safety or cancer-fighting benefits. [1, 2, 3, 4, 5]
Questions parents should ask before considering ANP
- What specific type and molecular subtype of brain tumor does my child have?
- Has ANP been studied specifically in this tumor type?
- How many children were enrolled in the relevant ANP study?
- Was there a control group?
- Were the results independently reproduced?
- What treatments had responding patients received previously?
- What adverse events occurred?
- What is the expected outcome with current standard therapy?
- Are clinical trials available through a major pediatric neuro-oncology center?
- What are the financial and logistical requirements of ANP treatment?
- Would pursuing ANP delay an established treatment or another clinical trial?
PeopleBeatingCancer Evidence Rating — Antineoplastons for Pediatric Brain Cancer
| Evidence category |
Assessment |
| Laboratory rationale |
🟡 Limited/moderate |
| Published human studies |
🟡 Yes, but mostly small nonrandomized studies |
| Phase II evidence |
🟡 Yes |
| Randomized controlled trials |
🔴 None published |
| Independent replication |
🔴 Insufficient |
| FDA approved for cancer |
🔴 No |
| Evidence of individual responses |
🟡 Reported |
| Evidence of improved overall survival |
🔴 Not established |
| Overall PBC Evidence Rating |
C — Limited/Preliminary Evidence |
NCI states that no randomized controlled ANP trials have been published in peer-reviewed literature and characterizes the clinical evidence as inconclusive. It also states that antineoplastons are not FDA-approved to prevent or treat any disease.
Feel free to ask any questions about Antineoplaston Therapy.
Thanks
David Emerson
- Cancer Survivor
- Cancer Coach
- Director PeopleBeatingCancer
Burzynski and colleagues reported results from 12 children with recurrent or progressive multicentric glioma. Four achieved complete responses and three partial responses. However, this was a small, nonrandomized study, making it impossible to determine the treatment effect with the confidence provided by a controlled trial.
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”
CONCLUSION:Antineoplastons contributed to more than a 5-year survival in recurrent diffuse intrinsic glioblastomas and anaplastic astrocytomas of the brainstem in a small group of patients.
Complete response was accomplished in 23%, partial response in 8%, stable disease in 31%, and progressive disease in 38% of cases. Six patients (46%) survived more than 5 years from initiation of ANP; 5 were not treated earlier with radiation therapy or chemotherapy. The serious side effects included single occurrences of fever, granulocytopenia, and anemia. The percentage of patients’ response is lower than for standard treatment of favorable PNET, but long-term survival in poor-risk cases and reduced toxicity makes ANP promising for very young children, patients at high risk of complication of standard therapy, and patients with recurrent tumors…”
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