Antineoplaston Therapy for Brain Cancer: Evidence and Clinical Studies. Can antineoplaston therapy treat brain cancer? Review the clinical studies of ANP for glioblastoma, glioma and pediatric brain tumors and what the evidence actually shows.
Antineoplaston therapy (ANP), developed by Dr. Stanislaw Burzynski, has been studied for several forms of cancer, but some of the most frequently cited research involves brain tumors, including gliomas, brainstem gliomas, glioblastoma, and pediatric brain tumors.
This research is also controversial.
Small Phase II studies and case reports published by Burzynski and colleagues have reported complete responses and unusually long survival in some patients. At the same time, antineoplastons have never demonstrated effectiveness in a randomized Phase III clinical trial, and attempts to independently confirm the reported results have not established that ANP improves survival.
So what should a brain cancer patient make of the evidence?
The most accurate answer is:
There are intriguing clinical observations and published Phase II studies involving antineoplastons and brain tumors, including reports of long-term survivors. However, the evidence is not strong enough to establish antineoplastons as a proven treatment for brain cancer.
That distinction is important.
What Are Antineoplastons?
Antineoplastons are chemical compounds originally identified by Dr. Stanislaw Burzynski in human blood and urine. Laboratory-produced versions were subsequently developed as an experimental cancer treatment.
Two formulations frequently appearing in the brain tumor research are:
- Antineoplaston A10
- Antineoplaston AS2-1
The National Cancer Institute (NCI) describes antineoplastons as an experimental cancer therapy and notes that laboratory, animal, Phase I, Phase II and case-report research has been conducted.
However, no Phase III randomized controlled trial has demonstrated that antineoplastons improve outcomes for cancer patients.
That is the central limitation patients should understand when evaluating ANP.
Why Have Antineoplastons Been Studied for Brain Cancer?
Brain tumors present an unusually difficult therapeutic problem.
Depending on the tumor type, location, molecular characteristics and patient age, conventional treatment may involve:
- surgery,
- radiation therapy,
- chemotherapy,
- targeted therapy,
- tumor-treating fields,
- and clinical trials.
Some aggressive tumors — particularly recurrent glioblastoma and certain brainstem gliomas — remain extremely difficult to treat.
This poor prognosis helps explain why experimental therapies such as antineoplastons have attracted interest from patients and researchers.
ANP research has included patients with several different brain tumor diagnoses rather than one single disease. These have included:
| Brain tumor studied |
Type of ANP evidence |
| Glioblastoma |
Case reports and Phase II studies |
| High-grade glioma |
Phase II studies |
| Brainstem glioma |
Phase II studies |
| Diffuse intrinsic brainstem/pontine glioma |
Phase II studies |
| Low-grade glioma |
Phase II reports |
| Pediatric malignant brain tumors |
Phase II studies |
| Primitive neuroectodermal tumors (PNETs) |
Small clinical studies |
This distinction matters because “brain cancer” is not one disease, and results from one tumor type cannot automatically be applied to another.
What Have the Brain Cancer Studies Reported?
1. Recurrent Glioma: The Independent Mayo Clinic Study
One of the most important pieces of ANP research is important precisely because it did not originate at the Burzynski Clinic.
Researchers at the Mayo Clinic conducted a Phase II study of antineoplastons A10 and AS2-1 in patients with recurrent glioma.
The investigators were unable to reproduce the dramatic responses previously reported by Burzynski and colleagues.
This study represents one of the largest weaknesses in the argument that the existing ANP literature proves effectiveness: independent investigators have not successfully reproduced the reported results.
The NCI specifically cites this problem when reviewing the evidence for antineoplastons.
2. Brainstem Glioma
Burzynski and colleagues have published Phase II research involving patients with recurrent diffuse intrinsic brainstem glioma.
Some patients reportedly experienced objective tumor responses, including complete responses, while others experienced stable or progressive disease.
A registered Phase II study of A10 and AS2-1 for brainstem glioma ultimately enrolled 40 patients.
These results are potentially interesting because brainstem gliomas can be extremely difficult to treat.
But the study design creates an important limitation:
There was no randomized control group receiving standard treatment for comparison.
Without a control group, it becomes difficult to determine how much of an observed survival difference resulted from ANP rather than differences in tumor biology, previous treatments, patient selection or other factors.
3. Pediatric Brain Tumors
Antineoplastons have also been studied in children with recurrent or progressive brain tumors.
One Phase II report included children with diagnoses such as:
- glioblastoma,
- anaplastic astrocytoma,
- diffuse intrinsic pontine glioma,
- primitive neuroectodermal tumors,
- and other malignant brain tumors.
Another publication examined 13 pediatric patients with high-risk primitive neuroectodermal tumors.
The researchers reported:
- complete responses in some patients,
- partial responses in some patients,
- stable disease in others,
- and long-term survival in several children.
These results deserve to be documented.
However, these were small, uncontrolled studies, and much of the published clinical evidence came from investigators associated with the developer of antineoplaston therapy.
That substantially lowers the certainty of the evidence.
4. Glioblastoma Case Reports
Perhaps the most compelling ANP publications from a patient’s perspective are reports of individual long-term survivors.
For example, Burzynski and colleagues published a case involving a patient with recurrent diffuse intrinsic brainstem glioblastoma who received intravenous A10 and AS2-1.
The researchers reported that the tumor gradually disappeared on imaging and that the patient achieved a complete response and remained tumor-free for years after discontinuing ANP.
Cases like this are worth studying.
But a case report cannot establish that a treatment caused the outcome.
Exceptional responders occur throughout oncology. Determining whether a therapy produced the response requires larger controlled studies capable of separating treatment effect from tumor biology, previous therapy, diagnostic differences and other factors.
What Does the National Cancer Institute Say?
The National Cancer Institute has reviewed the available antineoplaston literature.
Its conclusion is considerably more cautious than many descriptions of ANP found online.
The NCI notes that:
- Phase I and Phase II studies and case reports have been published.
- No randomized controlled Phase III trials demonstrating effectiveness have been completed.
- Most published clinical studies have come from the therapy’s developer and associates.
- Independent investigators have been unable to reproduce some of the reported results.
- Interpretation of some brain tumor studies may be complicated by previous radiation, chemotherapy and imaging issues.
This does not mean that every reported ANP response is necessarily invalid.
It means that the clinical evidence has not reached the level normally required to conclude that a cancer treatment works.
The Central Problem: Interesting Results vs. Proof
This distinction is essential for patients considering experimental cancer treatments.
Imagine three levels of evidence.
Level 1 — A patient responds
A patient receives ANP and subsequently experiences tumor shrinkage or long survival.
That is an observation worth documenting.
Level 2 — A clinical study reports responses
A group of patients receives ANP and researchers report complete responses, partial responses or prolonged survival.
That is stronger evidence.
Level 3 — Independent controlled trials reproduce the result
Patients receiving ANP are compared with appropriate controls, ideally in randomized trials, and independent researchers reproduce the benefit.
That is the evidence needed to establish ANP as an effective brain cancer therapy.
Antineoplaston research has produced evidence at Levels 1 and 2.
It has not successfully reached Level 3.
That is why patients can encounter apparently contradictory statements such as:
“There are published studies reporting responses to antineoplastons.”
and
“Antineoplastons have not been proven effective for treating cancer.”
Both statements can be true.
PeopleBeatingCancer Evidence Rating
| Question |
Assessment |
| Laboratory/preclinical evidence |
Limited / suggestive |
| Published human evidence |
Yes |
| Case reports of significant responses |
Yes |
| Phase II clinical studies |
Yes |
| Independent replication |
Weak / unsuccessful |
| Randomized controlled evidence |
No convincing evidence |
| Phase III evidence |
None establishing efficacy |
| Established survival benefit |
No |
| FDA-approved treatment for brain cancer |
No |
| Overall PBC Evidence Rating |
2/5 — Preliminary / insufficient clinical evidence |
How I interpret that rating
A 2/5 rating does not mean that ANP has been proven ineffective.
It means something more specific:
The available clinical evidence is insufficient to conclude that antineoplaston therapy improves survival or tumor control in patients with brain cancer.
The published responses and long-term survivors provide a reason for scientific interest, but they do not substitute for independent, well-controlled clinical trials.
What About ANP Side Effects?
Antineoplastons are sometimes described as a nontoxic or natural alternative to chemotherapy.
That characterization is too simple.
Clinical reports have described adverse effects ranging from mild symptoms to potentially serious complications. The NCI reports side effects including:
- anemia,
- dizziness,
- fatigue,
- headache,
- high blood pressure,
- nausea and vomiting,
- numbness,
- fever,
- joint stiffness,
- and swelling near the brain.
Because ANP formulations may involve substantial sodium exposure, electrolyte abnormalities and related complications are also important considerations.
Therefore, ANP should not be interpreted as automatically harmless simply because it differs from conventional chemotherapy.
My Perspective as an ANP Patient
My interest in antineoplastons is not purely academic.
I underwent antineoplaston therapy after being diagnosed with multiple myeloma and experiencing multiple relapses following conventional therapy. My experience influences my interest in ANP and is one reason I have followed this research for many years.
But my experience is an anecdote, not a clinical trial.
That distinction is central to the way I now evaluate cancer therapies on PeopleBeatingCancer. I believe patients deserve to hear about unconventional treatments and unusual long-term survivors. I also believe they deserve to know exactly how strong — or weak — the supporting evidence is.
Those two principles are not contradictory.
Should Someone With Brain Cancer Consider Antineoplaston Therapy?
I would not frame the decision as:
ANP versus conventional oncology.
A better question is:
What treatments have evidence for my exact brain tumor, and what experimental options might reasonably be investigated in addition to those choices?
For someone facing glioblastoma, recurrent glioma, DIPG or another difficult brain tumor, questions worth discussing with a neuro-oncology team include:
- What exactly is my tumor diagnosis and molecular subtype?
- What treatments have demonstrated a survival benefit for this tumor?
- Are there appropriate clinical trials available?
- Has the experimental treatment I am considering been independently reproduced?
- What are the known risks?
- Could pursuing the experimental therapy delay a treatment with stronger evidence?
- How will we objectively determine whether the treatment is working?
Patients facing aggressive brain tumors understandably search beyond standard therapy.
That search should be encouraged — but it should also be evidence-based.
Bottom Line
Antineoplaston therapy occupies an unusual place in cancer research.
There are published Phase II studies and case reports describing responses among patients with difficult brain tumors, including glioblastoma, brainstem glioma and pediatric brain tumors.
Some reported outcomes are remarkable enough to deserve attention.
But remarkable outcomes are not the same as proof.
After decades of investigation:
Antineoplaston therapy has not demonstrated a brain-cancer survival benefit in randomized Phase III clinical trials, and the results reported by its developers have not been convincingly reproduced by independent researchers.
For that reason, I believe ANP should be described accurately as an experimental and unproven therapy, rather than either a proven cancer cure or a treatment that can simply be dismissed without examining the published research.
For brain cancer patients, the most useful approach is to examine the evidence, understand its limitations and discuss both established and investigational options with an experienced neuro-oncology team.
I am both a cancer survivor and cancer coach. Have you been diagnosed with brain cancer? What type? What stage? To learn more about both conventional and non-conventional therapies for you please scroll down the page, post a question or comment and I will reply to you ASAP.
Thanks and hang in there,
David Emerson
- Cancer Survivor
- Cancer Coach
- Director PeopleBeatingCancer
Possible Therapies for Brain Cancer:
To Learn More About Brain Cancer
To Learn More About Antineoplaston Therapy:
To Learn More About Cancer Management:
Brainstem glioma carries the worst prognosis of all malignancies of the brain. Most patients with brainstem glioma fail standard radiation therapy and chemotherapy and do not survive longer than 2 years. Treatment is even more challenging when an inoperable tumor is of high-grade pathology (HBSG)..
The overall survival at 2 and 5 years was 39% and 22%, respectively, and maximum survival was more than 17 years for a patient with anaplastic astrocytoma and more than 5 years for a patient with glioblastoma. Progression-free survival at 6 months was 39%. Complete response was achieved in 11%, partial response in 11%, stable disease in 39%, and progressive disease in 39% of patients. Antineoplastons were tolerated very well with 1 case of grade 4 toxicity (reversible anemia).
“Primitive neuroectodermal tumors (PNETs) are usually successfully treated with craniospinal radiation and chemotherapy; however, difficulties with standard treatment can be encountered in very young children, in adult patients at high risk of complication from standard treatment, and in patients with recurrent tumors. Thirteen children, either with recurrent disease or high risk, were treated in phase II studies with antineoplastons (ANP).
The median age of patients was 5 years, 7 months (range, 1-11). Medulloblastoma was diagnosed in 8 patients, pineoblastoma in 3 patients, and other PNET in 2 patients. Previous treatments included surgery in 12 patients (1 had biopsy only, suboccipital craniotomy), chemotherapy in 6 patients, and radiation therapy in 6 patients. Six patients had not received prior chemotherapy or radiation.
The treatment consisted of intravenous infusions of 2 formulations of ANP, A10 and AS2-1, and was administered for an average of 20 months. The average dosage of A10 was 10.3 g/kg/d and of AS2-1 was 0.38 g/kg/d. 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 study is ongoing and accruing additional patients.
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.”
“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.
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.”
“The patients were diagnosed by pathologists not associated with BC and objective responses were verified by Central Radiology Review. The group of 77 patients (19%) survived over five years from the treatment start. Of particular interest were results in patients with brainstem gliomas. The group of 17 patients with brainstem glioma underwent the treatment and 65% of these patients survived over five years. An additional group of 42 patients diagnosed with diffuse intrinsic pontine glioma (DIPG) have been treated and a total of 19% survived over five years.
The quality of survival is very good and there is no long-term toxicity related to ANP…”
Antineoplaston Therapy for Brain Cancer
