Antineoplastons are an experimental cancer therapy that is not approved by the US Food and Drug Administration (FDA) for any indication. To date, there have been no randomised, controlled trials showing efficacy of antineoplastons. However, side-effects of this therapy can include serious neurological toxicity. So, how can such therapy continue to be in use after more than four decades, and be administered to thousands of patients, without any proven safety or activity data?
Antineoplastons were first proposed as a potential cancer treatment in 1976 by Dr Stanislaw Burzynski, a Polish-educated physician, MD, and PhD in Biochemistry from the Medical Academy of Lublin (Lublin, Poland).
He moved to the USA in 1970 and joined the laboratory of Dr Georges Ungar in the Department of Anaesthesia at the Baylor College of Medicine (Houston, TX, USA).1 While at the Baylor College of Medicine, Dr Burzynski isolated compounds from human blood and later urine (aminoacids and peptides), which he called antineoplastons and suggested had antitumour activity. Antineoplastons are a combination of sodium phenylacetate acid and phenylacetylglutamine that can be administered either orally or intravenously.2 Dr Burzynski described this concoction as a non-toxic natural form of cancer protection, and began production of different synthetic versions in his laboratory.3 In the late 1970s, he left Baylor and opened his own clinic, the Burzynski Clinic (Houston, TX, USA), where he and a small team of physicians have now treated thousands of patients with cancer using this unproven therapy, charging thousands of dollars.4 In fact, the Cancer Research UK website suggests that a year’s course of treatment at the Burzynski Clinic costs between US$30 000 and $60 000.5 Recently, the Burzynski Clinic has started requiring patients to provide a deposit before treatment starts, and their website informs patients that they do not accept insurance to pay for any portion of the treatment.6 In fact, patients often fundraise to meet the cost of treatment, as they have to pay for this alternative therapy out of their own pockets.7 How is this possible considering this treatment is not FDA-approved?