Disclaimer: Consult with a doctor before deciding on a treatment plan for cancer or any other disease.
Phenylbutyrate is a substance made up of naturally occurring chemical compounds including amino acids and peptides. They’re found in both blood and urine and through a complex mechanism involving DNA, these compounds have been shown to fight cancer. The presence of phenylbutyrate in urine could help explain why urine fasting has been shown to fight cancer .
Antineoplastons share active ingredients with the metabolites of phenylbutyrate and in fact, phenylbutyrate is a precursor of antineoplastons. In other words, once the phenylbutyrate enters the body, it is converted into antineoplastons . Antineoplastons are found in the blood and urine, but they are also manufactured in the laboratory for research .
Research has shown that sodium phenylbutyrate, a compound that can inhibit deacetylase, can inhibit growth to restore the cell to health or cause the programmed death of cancer cells. Butyric acid and its derivatives have been studied for years to learn about their potential use in treating cancer as well as other diseases. The most extensive research has been done on sodium phenylbutyrate .
The chemistry underlying the success of sodium phenylbutyrate and antineoplastons as a cancer cure has to do with acetylation. Though it isn’t necessary to understand all of the chemical complexities of why phenylbutyrate works, most patients can understand the basic idea: acetylation activates DNA while deacetylation causes DNA to stop transcribing itself. Deacetylation of cancer cells causes the DNA to stop transcribing and therefore stop reproducing itself. This deacetylation therefore inhibits the growth of cancer cells and at the same time causes cancer cells to re-express the genes that have to do with normal, healthy programmed cell death (apoptosis) .
When prostate cancer cells are exposed to phenylbutyrate, their DNA synthesis is reduced which ultimately causes approximately 50-60% of the cells to undergo apoptosis. Sodium butyrate has a similar effect on prostate cancer cells. One study observed the effect of phenylbutyrate on a patient with highly resistant acute promyelocytic leukemia. Twenty-three days after treatment was initiated, all visible leukemia cells had been eliminated from the patient’s bone marrow and complete remission followed shortly after that .
The antineoplaston “landscape” is politically complex, as with many integrative or complementary treatments for cancer. While sodium phenylbutyrate enjoys some acceptance in conventional medicine, antineoplastons are considered to be nothing but hype in conventional circles. But antineoplastons are naturally occurring substances, so that fact alone indicts Big Pharma and its tendency to denigrate non-patentable substances for the sake of elevating patentable drugs that turn a big profit. Dr. Stanislaw Burzynski is a proponent of antineoplaston use for cancer treatment and he markets his treatments as “targeted” at the patient’s unique genetics to achieve such amazing results. While critics (who may very well have a vested interest in making sure that antineoplastons, naturally occurring substances, never achieve widespread popularity), claim that Dr. Burzynski is a quack and his targeted treatments aren’t targeted at all, many patients get so overwhelmed and confused studying the information that’s been carefully crafted by spin doctors that they give up on alternative treatments altogether.
Phenylbutyrate and antineoplastons can help doctors effectively target specific genes involved in the development of cancer . The use of antineoplastons are currently not FDA approved  however, phenylbutyrate is FDA approved for urea cycle disorders .
Dr. Stanislaw Burzynski at the Burzynski Clinic in Texas creates a customized treatment protocol by doing a DNA analysis of tumors to create a customized plan based on the genetic make-up of these cancer cells. He has been touted as a charlatan for his use of targeted therapies that take aim at specific genes and proteins in cancer cells while using sodium phenylbutyrate and antineoplaston therapy. The Burzynski Clinic uses primarily FDA approved chemotherapies in conjunction with non-FDA approved sodium phenylbutyrate and antineoplaston . Despite this, his treatments are described with typical “spin” words used by large pharmaceutical companies to scare the public away from them. Wikipedia, for example, opens with the statement that the medical clinic is “offering unproven cancer treatment” and that Burzynski is a “merchant of false hope” .
According to the National Cancer Institute:
“Antineoplastons are drugs composed of chemical compounds that are naturally present in the urine and blood. They are an experimental cancer therapy that is purported to provide a natural biochemical substance that is excreted and therefore lacking in people with cancer” .
The National Cancer Institute goes on to assert that while antineoplastons were originally isolated from human urine, now they’re synthesized from chemicals in the developer’s laboratory. The synthesis of chemicals that are similar to naturally occurring antineoplastons is cast as a positive thing even though these chemicals are most certainly biologically different than the naturally occurring antineoplastons. As a result, they could have a negative impact on the body. But the public is led to believe that a chemically synthesized treatment is better than one that occurs naturally in nature .
Further, although no randomized controlled trials showing the effectiveness of antineoplastons have been published, the developer’s institute is currently investigating the efficacy of antineoplastons . While Burzynski’s treatments using phenylbutyrate and antineoplastons has been dubbed a source of “false hope”, the National Cancer Institute is working to manufacture a patentable version of a similar but unnatural version of antineoplastons and that can be sold to the public at a profit .
Top ranking articles at Google discrediting Burzynski’s “targeted cancer therapy” using antineoplastons focus on the fact that phenylbutyrate is a drug and antineoplastons are naturally occurring and therefore less therapeutic and not “targeted” according to the definition of “targeted” espoused by conventional medicine . Patients need to know that when naturally occurring substances are chemically produced by pharmaceutical companies, their chemical structure is altered and the final product reacts differently in the body than the original model. Naturally occurring substances with actual medicinal value are often discredited only because they lack profit-potential, not because they don’t work.
Safety and Effectiveness
Sodium phenylbutyrate and antineoplastons have been used to treat the following types of cancer, often in conjunction with conventional chemotherapy agents. Below are diseases that have been treated with sodium phenylbutyrate:
- Prostate cancer 
- Leukemia 
- Diffuse intrinsic pontine glioma (brain stem glioma) 
- Gliobastoma multiforme (brain cancer) 
- Pancreatic Cancer 
- Neuroblastoma 
- Urea Cycle Disorders 
- Amyotrophic Lateral Sclerosis (Lou Gehrig’s Disease) 
- Spinal Muscular Dystrophy 
- Spinocerebellar Ataxia Type 3 
- Esthesioneuroblastoma 
- Non-small cell lung cancer 
Dr. Stanislaw Burzynski uses a combination of sodium phenylbutyrate with conventional chemotherapy agents on hard-to-treat cancers such as brain stem gliomas and brain gliomas. Dr. Burzynski analyzes his patients’ tumors to design a customized treatment plan based on the DNA of the tumor . His studies have shown minor toxicity including the following side effects:
- Skin Rash 
- Elevation of transaminases 
- Diarrhea 
Many of these side effects resolved with a lower dosage of the sodium phenylbutyrate .
How Phenylbutyrate Is Administered
Phenylbutyrate is well-tolerated via both intravenous and oral routes of administration . In one study that examined the use of phenylbutyrate for gliomas (tumors that start in the glial cells in the spinal cord or the brain) the maximum tolerated dose was about 27 grams/day. In this study involving 23 participants, one patient had complete remission. The median survival time from the participants’ enrollment in the study was 5.4 months .
Possible Negative Effects
High doses of phenylbutyrate (about 27 grams/day or more) can lead to reversible central nervous system depression .
Other Important Information
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