Coverage Policy Manual - Arkansas Blue Cross and Blue Shield

Antineoplaston Cancer Therapy

Description:

Antineoplaston therapy is an alternative cancer treatment made up of a group of compounds that occur naturally in the human body, primarily peptides and amino acids originally taken from the blood and urine. Dr. Stanislaw Burzynski discovered this therapy in the late 1960s and presently offers this therapy to treat cancer, HIV infection, and autoimmune diseases at the Burzynski Research Institute in Houston, Texas.

Antineoplaston therapy has been a controversial treatment for many types of cancer. Antineoplaston therapy does not have FDA approval for any indication. In December 2008, the Office of Orphan Products Development of the U.S. FDA granted an orphan drug destination to antineoplaston A10 and antineoplaston AS2-1 for the treatment of gliomas. The Orphan Drug Act is intended to encourage development of products for rare diseases affecting fewer than 200,000 people in the United States. This orphan drug approval has appeared in information on the Burzynski Research Institute website but is incorrect based on a letter from the FDA to Dr. Burzynski dated 10/18/2012. A search of the Orphan Drug Database on 1/30/13 shows requests for an orphan drug determination for the treatment of brain stem glioma in Sep 2005 and all gliomas in Oct 2008 were not approved by the FDA.

The treatment is given orally or by injection into a vein. The duration of treatment ranges from eight to twelve months.

There are no specific CPT or HCPCS codes for antineoplaston therapy.

Policy/
Coverage:

Does Not Meet Primary Coverage Criteria Or Is Investigational For Contracts Without Primary Coverage Criteria

Antineoplaston therapy in the treatment of any indication does not meet member benefit certificate primary coverage criteria that there be scientific evidence of safety and effectiveness in improving health outcomes.

For contracts without primary coverage criteria, antineoplaston therapy in the treatment of any indication is investigational. Investigational services are exclusions in the member benefit certificate of coverage.

Rationale:

There are no published, randomized, controlled trials studying the use of antineoplastons in the treatment of any cancer or disease. There is a lack of scientific evidence that antineoplaston therapy is a safe or effective treatment for any indication.

In 1993, the National Cancer Institute sponsored clinical trials to investigate the antitumor potential of antineoplastons in patients with brain tumors . The trials were closed 2 years later as poor patient accrual precluded conclusions about treatment efficacy. In addition, a Mayo clinic study found no benefit (1). Antineoplastons have also been tested in patients with gliomas, but lack of a rigid scientific approach in these studies has been questioned. A preliminary study of antineoplastons adjunctive to hepatic arterial infusion post-hepatectomy did not improve overall survival, although cancer-specific survival was higher. The status of other clinical trials using antineoplastons as investigational drugs for various cancers remains unknown.

2012 Update

A literature review was conducted using the MEDLINE database. There was no new literature identified that would prompt a change in the coverage statement. According to the National Cancer Institute, no phase III randomized controlled trials have been conducted (National Cancer Institute).

A search of the clinicaltrials.gov website revealed 61 studies involving antineoplaston therapy. Of these 61 studies, all but two of the studies have either been withdrawn or the status of the study is unknown. A randomized controlled trial of combination antineoplaston therapy versus Temozolomide (NCT01260103) was scheduled to begin enrollment in December 2011 at the Burzynski Research Institute; but according to the website enrollment has not begun. Additionally, a phase II study of antineoplastons in patients with malignant melanoma has been completed but no results were found to have been published (NCT00003509).

2013-Jan UPDATE

A literature review was conducted through Dec 2012 using PubMed. There were no articles describing reports of randomized controlled trials comparing the use of antineoplastons with any other therapy for the treatment of cancer or any other indication.

A search of the clinicaltrials.gov website revealed 61 studies involving antineoplaston therapy. Sixty-one (61) trials were listed: 7 were withdrawn prior to enrollment; 1 was terminated for slow patient enrollment; 1 was completed but no results were posted; the status of 50 trials was "unknown" indicating the status of the study had not been verified in more than two years, some going back to 1999.

A randomized controlled trial of combination antineoplaston therapy versus Temozolomide (NCT01260103) was scheduled to begin enrollment in December 2011 at the Burzynski Research Institute; but according to the website this trial is not yet recruiting.

Additionally, a phase II study of antineoplastons in patients with malignant melanoma (NCT00003509) has been completed but, as previously noted, no results were posted on the clinical trial website and no publication of the trial results was identified.

Currently there is a lack of scientific evidence that antineoplaston therapy is a safe or effective treatment for any indication.

2014 Update

A literature was conducted using the MEDLINE database through December 2013. There was no new information identified that would prompt a change in the coverage statement.

2015 Update

A literature search conducted through December 2014 did not reveal any new information that would prompt a change in the coverage statement. The key identified literature is summarized below.

Ushijima and colleagues reported on a study that investigated demethylation effect of the antineoplaston AS2-1 (a mixture of phenylacetylglutamine and phenylacetate in the ratio of 1:4) on various genes in colon cancer cells. (Ushijima, 2014). An HpaII-MspI methylation microarray was used to investigate the methylation status of 51 genes at the promoter region in HCT116 and KM12SM human colon cancer cells before and after treatment of AS2-1. The expression of protein and mRNA of the demethylated genes by AS2-1 in HCT116 cells was evaluated. In 19 of the 34 methylated genes in HCT116 and in 7 of the 8 methylated genes in KM12SM, the methylation status was downregulated after treatment with 2 mg/ml of AS2-1 for 24 h. AS2-1 dramatically downregulated the methylation status of p15 and ESR1 in HCT116 cells and of MTHFR and MUC2 in KM12SM cells. Both mRNA and protein expression of p15 increased in a dose- and time-dependent manner after treatment with AS2-1. The antineoplaston AS2-1 may normalize the hypermethylation status at the promoter region in various genes including tumor suppressor genes, resulting in activation of the transcription and translation in colon cancer.

2016 Update

A literature search conducted through January 2016 did not reveal any new information that would prompt a change in the coverage statement.

2017 Update

A literature search conducted through January 2017 did not reveal any new information that would prompt a change in the coverage statement.

January 2018

A literature search conducted using the MEDLINE database through December 2017 did not reveal any new literature that would prompt a change in the coverage statement. .

2019 Update

A literature search was conducted through December 2018. There was no new information identified that would prompt a change in the coverage statement.

2020 Update

A literature search was conducted through December 2019. There was no new information identified that would prompt a change in the coverage statement.

2021 Update

Annual policy review completed with a literature search using the MEDLINE database through January 2021. No new literature was identified that would prompt a change in the coverage statement.

2022 Update

Annual policy review completed with a literature search using the MEDLINE database through January 2022. No new literature was identified that would prompt a change in the coverage statement.

2023 Update

Annual policy review completed with a literature search using the MEDLINE database through January 2023. No new literature was identified that would prompt a change in the coverage statement.

2024 Update

Annual policy review completed with a literature search using the MEDLINE database through January 2024. No new literature was identified that would prompt a change in the coverage statement.

References:

A Randomized Study of Antineoplaston Therapy vs. Temozolomide in Subjects With Recurrent and / or Progressive Optic Pathway Glioma. NCT01260103. Accessed at www.clinicaltrials.gov. Last accessed June 28, 2012.

Antineoplaston Therapy in Treating Patients With Stage IV Melanoma. NCT00003509. Accessed at www.clinicaltrials.gov. Last accessed June 28, 2012.

Search of trials for antineoplastons. www.clincialtrials.gov. Last accessed 1/30/13.

Ushijima M1, Ogata Y, Tsuda H et al.(2014) Demethylation effect of the antineoplaston AS2-1 on genes in colon cancer cells. Oncol Rep. 2014 Jan;31(1):19-26. doi: 10.3892/or.2013.2839. Epub 2013 Nov 8

Group specific policy will supersede this policy when applicable. This policy does not apply to the Wal-Mart Associates Group Health Plan participants or to the Tyson Group Health Plan participants.
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