Antineoplaston Therapy FDA Approved or Not?

Short answer: No. Antineoplaston therapy is not FDA approved as a treatment for cancer—or for any other disease.

That answer sounds straightforward. The history behind it is anything but straightforward.

Antineoplaston therapy (ANP), developed by Dr. Stanislaw Burzynski beginning in the 1970s, has been studied and debated for decades. Some patients have reported remarkable responses. Burzynski and colleagues have published clinical studies and case reports. At the same time, the National Cancer Institute (NCI) says that no randomized controlled trials demonstrating the effectiveness of antineoplastons have been published in the peer-reviewed medical literature.

The FDA has allowed antineoplastons to be investigated under clinical research protocols and has granted orphan-drug designation for certain antineoplaston compounds for gliomas.

Neither of those things means the FDA has approved antineoplaston therapy for treating cancer.

For cancer patients trying to understand this controversy, that distinction is critical.

Having said all that, I have to be honest and confess that I understood NONE of this when I traveled to Houston, Texas to undergo Antineoplaston Therapy at the Burzynski Clinic in Noverber of 1997.

All I knew was that my cancer, multiple myeloma, did not respond to conventional therapies prescribed by my oncologist and that I was end-stage. I was young, (36), recently married with no kids and no mortgage. Meaning I had few is any real responsibilities.

I have writted extensively about the risks and benefits surrounding ANP because I am hoping other cancer survivors can learn what I should have learned before undergoing ANP.

Are you a cancer patient or survivor? Scroll down the page, post a question or comment and I will reply to you ASAP.

Good luck,

David Emerson

Cancer Survivor
Cancer Coach
Director PeopleBeatingCancer

Antineoplaston Therapy and Multiple Myeloma – My Story

What Is Antineoplaston Therapy?

Antineoplastons are chemical compounds originally identified in human blood and urine by Dr. Stanislaw Burzynski.

The compounds used therapeutically are now manufactured synthetically rather than extracted from urine.

The best-known antineoplaston formulations include:

Antineoplaston A10
Antineoplaston AS2-1

Antineoplaston therapy has been investigated primarily as a cancer treatment, including for difficult-to-treat brain tumors.

The proposed theory behind ANP is that these compounds may help regulate abnormal cell growth. However, biological plausibility and laboratory findings are not the same thing as demonstrating that a treatment improves survival or other meaningful outcomes in patients.

That is where much of the antineoplaston controversy begins.

Are Antineoplastons FDA Approved?

No.

The National Cancer Institute states clearly that:

Antineoplastons are not approved by the U.S. Food and Drug Administration for the prevention or treatment of any disease.

That includes cancer.

This does not mean that antineoplastons have never interacted with the FDA regulatory system.

They have.

Understanding how they have interacted with that system helps explain why patients sometimes become confused about their regulatory status.

FDA Approval vs. FDA Permission to Conduct Research

One of the most important distinctions for patients to understand is the difference between an investigational treatment and an FDA-approved treatment.

An experimental drug may be administered as part of an FDA-regulated clinical investigation without being approved for routine medical use.

These are fundamentally different regulatory categories.

Investigational status

An investigational treatment is still being studied.

Clinical trials are intended to answer questions such as:

Is the treatment safe?
What dose should be used?
Which patients might benefit?
Does it shrink tumors?
Does it delay cancer progression?
Does it improve survival?
How does it compare with existing treatments?

Allowing a drug to be investigated does not mean that the FDA has concluded that the drug works.

FDA approval

FDA approval generally requires sufficient evidence demonstrating that a treatment’s benefits outweigh its risks for a particular indication.

Therefore:

FDA authorization of clinical research ≠ FDA approval of the treatment.

This distinction is particularly important when discussing antineoplastons.

What About FDA Orphan-Drug Designation?

Here is another source of confusion.

The FDA granted orphan-drug designation to antineoplaston A10 and antineoplaston AS2-1 in 2008 for the treatment of gliomas.

That sounds significant—and it is a legitimate regulatory designation.

But orphan designation is not FDA approval.

The FDA’s own orphan-drug database lists:

Drug: Antineoplaston A10 and Antineoplaston AS2-1
Indication: Treatment of gliomas
Orphan designation: Designated
FDA orphan approval status: Not FDA Approved for Orphan Indication

Orphan-drug designation is intended to encourage development of treatments for rare diseases by providing potential development incentives.

A drug can receive orphan designation years before researchers have established whether it is sufficiently safe and effective to receive marketing approval.

Therefore:

Regulatory status	What it means	Does ANP have it?
Investigational clinical research	Treatment may be studied under regulatory oversight	Yes
Orphan-drug designation	Development designation for a rare disease/condition	Yes, for A10/AS2-1 and gliomas
FDA approval for glioma	FDA has approved the treatment for marketing for glioma	No
FDA approval for cancer generally	FDA has approved ANP as a cancer therapy	No

For patients researching antineoplastons online, this may be the single most important table in this article.

Why Has Antineoplaston Therapy Remained Controversial?

The controversy isn’t simply a disagreement over whether a particular drug works.

It involves several interconnected questions.

1. Patients have reported responses

Antineoplaston advocates point to patients who reportedly experienced tumor regression, long-term survival, or unexpectedly favorable outcomes.

Individual patient outcomes can be important signals.

But they cannot reliably establish whether the treatment caused the outcome.

Cancer outcomes may also be influenced by previous treatment, concurrent therapies, tumor biology, patient selection, and other factors.

This is why controlled trials matter.

2. Much of the clinical evidence comes from the treatment’s developers

The NCI notes that many publications concerning antineoplastons have been authored by Burzynski and colleagues.

Independent replication is one of the foundations of medical science.

If several independent research groups reproduce an effect, confidence in that finding increases.

According to the NCI, other investigators have not successfully duplicated the reported results.

3. Randomized controlled evidence is lacking

The NCI states that no randomized controlled trials demonstrating the effectiveness of antineoplastons have been published in peer-reviewed scientific literature.

That is an important limitation.

Without adequately controlled studies, researchers have difficulty determining whether apparent survival or tumor responses resulted from:

antineoplastons,
previous cancer treatment,
simultaneous treatments,
patient selection,
unusual tumor biology,
or other factors.

This does not prove that antineoplastons cannot work.

It means that their effectiveness has not been established to the standard normally required for an FDA-approved cancer therapy.

Haven’t Antineoplastons Been Studied in Clinical Trials?

Yes.

Antineoplastons have been investigated in numerous clinical studies.

For example, ClinicalTrials.gov contains studies sponsored by the Burzynski Research Institute examining A10 and AS2-1 in different cancers.

But another important distinction applies:

A clinical trial’s existence is not evidence that the treatment works.

ClinicalTrials.gov is fundamentally a registry of research studies. Registration tells us that a study exists or existed—not that the treatment was proven effective.

Some antineoplaston trials were completed, while others were terminated or otherwise discontinued.

The useful question for patients isn’t simply:

“Was there a clinical trial?”

It is:

“What did the trial show, how strong was the study design, were the results published, and have independent researchers reproduced them?”

Those questions lead to a much more meaningful discussion about evidence.

What Does the National Cancer Institute Say?

The NCI’s antineoplaston review is useful because it separates the existence of research from the strength of that research.

Its conclusions include several important points:

Antineoplastons remain experimental.
No randomized controlled trials demonstrating effectiveness have been published in peer-reviewed medical literature.
Reported responses have largely come from studies conducted by the treatment’s developer and associates.
Independent investigators have not successfully reproduced the reported findings.
The evidence supporting antineoplastons as a cancer treatment remains inconclusive.
Controlled clinical trials would be necessary to determine the treatment’s value.

That doesn’t mean every reported patient response should simply be dismissed.

It means the existing evidence cannot establish with sufficient confidence that ANP caused those responses.

Are Antineoplastons Safe?

“Not FDA approved” does not automatically mean “unsafe.”

But neither should “experimental” be interpreted as harmless.

The NCI reports antineoplaston-associated adverse effects including:

anemia,
abnormal blood calcium,
hypertension,
irregular heartbeat,
headache,
dizziness,
nausea and vomiting,
fatigue or sleepiness,
numbness,
swelling,
confusion,
seizures,
and swelling near the brain.

Serious neurologic toxicity has been reported.

The risk-benefit calculation becomes especially complicated for patients with advanced or treatment-resistant cancer because they may reasonably be willing to accept risks that someone with highly treatable disease would not.

That is a personal medical decision—but it should be made with accurate information.

An Important Confusion: Sodium Phenylbutyrate

There is another regulatory distinction worth understanding.

Sodium phenylbutyrate is an FDA-approved drug.

For example, the FDA has approved sodium phenylbutyrate products for certain urea cycle disorders.

But FDA approval applies to a particular drug for a particular indication.

The fact that a chemical related to or involved in antineoplaston discussions has an FDA-approved medical use does not mean antineoplaston therapy is FDA approved for cancer.

This illustrates an important rule when researching unconventional cancer therapies:

Always ask, “FDA approved for what?”

FDA approval of a compound for one disease does not automatically establish its safety or effectiveness for another disease.

PeopleBeatingCancer Evidence Rating

PeopleBeatingCancer evaluates cancer therapies according to the quality and independence of the available evidence—not simply whether positive studies or patient testimonials exist.

Evidence question	Antineoplaston therapy
Laboratory/preclinical rationale	Yes
Human clinical research	Yes
Published reports of tumor responses	Yes
Large randomized controlled trials	No
Independent replication	Limited/No convincing confirmation
FDA approved for cancer	No
Evidence sufficient to establish effectiveness	No

PBC Evidence Rating: Limited / Investigational

This rating does not mean that antineoplastons have been proven ineffective.

It means something more specific:

There is insufficient high-quality, independently replicated clinical evidence to conclude that antineoplaston therapy is an effective cancer treatment.

That distinction is important.

Does Lack of FDA Approval Mean a Treatment Doesn’t Work?

Not necessarily.

FDA approval and biological effectiveness are related concepts, but they aren’t identical.

Some treatments may show promising preliminary evidence years before FDA approval. Other therapies may never undergo the large trials necessary for approval.

Conversely, FDA-approved cancer treatments do not work for every patient.

Therefore, saying:

“Antineoplastons aren’t FDA approved, therefore they cannot work”

goes beyond what the evidence establishes.

But the opposite argument is equally problematic:

“Some patients responded, therefore antineoplastons work.”

The evidence currently supports neither absolute conclusion.

A more scientifically accurate statement is:

Antineoplaston therapy remains investigational, and the available clinical evidence has not established its effectiveness through the type of controlled, independently replicated research normally expected for an FDA-approved cancer therapy.

Questions Cancer Patients Should Ask

If you are considering antineoplaston therapy—or any experimental cancer treatment—consider asking:

Is this treatment FDA approved for my specific cancer?
If not, under what regulatory mechanism would I receive it?
Is treatment part of a registered clinical trial?
What peer-reviewed studies support its use for my cancer?
Were those studies randomized or controlled?
Have researchers independent of the treatment’s developer reproduced the results?
How many patients like me have received the treatment?
What percentage experienced objective tumor responses?
What were progression-free and overall survival?
What serious adverse events have occurred?
What will treatment cost me?
Will receiving this treatment delay or prevent me from receiving another therapy?

That last question can be particularly important.

For someone with an aggressive cancer, the greatest risk of an experimental therapy may not be only its direct side effects. There can also be an opportunity cost if pursuing it delays a treatment with stronger evidence.

So Why Has the Antineoplaston Debate Continued for Decades?

Because it sits at the intersection of several difficult issues in cancer medicine:

Patient autonomy. Experimental medicine. Regulatory oversight. Anecdotal survival. Scientific evidence. Terminal illness. And hope.

Patients with advanced cancer understandably search for options when conventional medicine offers limited ones.

Researchers and regulators, meanwhile, have to ask whether apparent responses actually demonstrate that a treatment works.

Both realities can exist simultaneously.

A patient can experience an extraordinary outcome.

That outcome deserves attention.

But extraordinary outcomes also need rigorous investigation before they can establish that a therapy is effective for other patients.

That is the unresolved issue at the heart of antineoplaston therapy.

The Bottom Line

Antineoplaston therapy is not FDA approved for cancer.

Antineoplastons have been investigated in clinical trials, and antineoplaston A10 and AS2-1 received FDA orphan-drug designation for gliomas. Neither fact constitutes FDA approval.

The National Cancer Institute considers antineoplastons experimental and describes the clinical evidence as inconclusive. Randomized controlled evidence and convincing independent replication remain lacking.

For PeopleBeatingCancer, the most useful position isn’t to tell patients that they must believe either side of a decades-long controversy.

It is to distinguish clearly between:

what has been reported,
what has been studied,
what has been proven,
and what remains unknown.

Cancer patients—especially patients considering experimental treatment—deserve to understand those differences before making a decision.

FAQ Questions

Are antineoplastons FDA approved for cancer?

No. The National Cancer Institute states that antineoplastons are not FDA approved for the prevention or treatment of any disease.

Did the FDA approve antineoplastons for brain cancer?

No. Antineoplaston A10 and AS2-1 received orphan-drug designation for gliomas, but the FDA database specifically lists them as not FDA approved for that orphan indication.

Does FDA orphan-drug designation mean FDA approval?

No. Orphan designation encourages development of treatments for rare diseases. It does not establish that a treatment has been proven safe and effective or approved for marketing.

Have antineoplastons been tested in clinical trials?

Yes. Antineoplastons have been studied in clinical trials, primarily by the Burzynski Research Institute. However, the NCI states that randomized controlled trials demonstrating effectiveness have not been published in peer-reviewed literature.

Does lack of FDA approval prove antineoplastons don’t work?

No. Lack of approval does not by itself prove that a treatment is ineffective. It means that antineoplaston therapy has not met the evidentiary and regulatory requirements necessary for FDA approval.

To Learn More About Antineoplaston Therapy:

To Learn More About Cancer Management:

Research Links

FDA — Orphan Drug Designation: Antineoplaston A10 / AS2-1 for Gliomas
Particularly useful because the FDA record distinguishes orphan designation from FDA approval.

ClinicalTrials.gov — Antineoplaston studies
Useful for reviewing individual ANP clinical-trial records, study design, status and reported results.

FDA — Sodium Phenylbutyrate (Buphenyl/Pheburane)
Demonstrates why FDA approval must always be tied to a specific indication: sodium phenylbutyrate has FDA-approved uses for urea cycle disorders, which does not constitute approval of antineoplaston therapy for cancer.

Our Mission

Is Antineoplaston Therapy FDA Approved? Understanding the Controversy.