The Burzynski Clinic in Houston, Texas has been in the news lately – but not in a good way. They’re offering an experimental cancer therapy called “antineoplastons”.  The therapy costs patients thousands of dollars (not covered by insurance) and has not undergone randomized controlled trials to prove its efficacy over other treatments.

Ever since the discovery that mustard gas (an agent used in chemical warfare) was found to reduce lymphoma cells, the field of oncology was born.  This study of cancer is where you kill bad cells, and some good cells. Ultimately, the patient relies on the research in the field to have the most benefit with least toxicity.  Only, sometimes it doesn’t work out that way. Often the journey of the cancer patient is not a pleasant one, with nausea, vomiting, hair loss, lethargy, and the feeling that you have the flu.  Too often chemotherapy is like carpet bombing- you bomb everything and hope the good survives. When it doesn’t, the collateral damage can be a life – as it was with my brother who died after becoming septic from his first round of chemotherapy for lung cancer.

The holy grail of cancer chemotherapy has been to find the smart bomb that would kill only the cancer cells, or a way to turn the body’s immune system against cancer cells. In the United States hopeful agents that are ultimately paid for have to go through the same process- a trial by the National Cancer Institute (NCI), which happens in three phases:


Phase 1 trials – to determine toxicity of the agent.  Even the most promising discovery in the bench of a scientist can have toxicity in humans.  These are small trials, usually reserved for people who have not had remission from traditional treatments.

Phase 2 trials – once toxicity has been determined, the phase 2 is to determine if the agent works against cancer in people. Does it shrink the tumor at all is the question asked. There are lots of promising treatments in the lab that never make it past this trial because they just don’t work in humans.

Phase 3 trials - compare the new treatment to standard treatments. These are very large trials, hundreds of people. Sometimes they are stopped when early data shows either toxicity not seen in earlier trials, and happily some are stopped when they find it is of much more benefit than the therapy it was up against.

Phase 4 - looking at side effects or long term effects after the agent is on the market.

The Burzynski Clinic states they offer “innovative and cutting-edge personalized gene targeted cancer therapy. Customized for over 50 types of malignancies.”  This is all done and under a trial authorized by the Food and Drug Administration (FDA).  All that sounds great – but here is the rest of the story.

The antineoplastons did undergo Phase 1 and Phase 2 trials. In Phase 1 there were some side effects that were mild.  In Phase 2 trials of brain cancer patients there were some severe nervous system side effects. However, other investigators were not able to obtain the same results as reported by Burzynski.  Some results were seen in Japan, but not the same as Burzynski.  In 1991 the National Cancer Institute conducted trials and by 1995 only 9 people had enrolled. The FDA has not approved antineoplastons for the prevention or treatment of any disease.  The FDA did allow the Burzynski’s clinic to continue treating patients. No randomized trials have been approved, and part of this is because Burzynski chose to not go forward with them.

Recently, the “Burzynski Patient Support Group” threatened a 17-year-old blogger, Rhys Morgan when he questioned the validity of Burzynski’s claims.  Morgan’s wrath came about because of a money raising campaign for a patient. Morgan raised the ire because he doubted the claims that Burzynski’s antineoplastons could cure the tumor.

What remains for this skeptical physician is simply this: Burzynski refuses to work with government-sponsored researchers, to have his treatment evaluated. He charges tens if not hundreds of thousands of dollars for the treatment.  His supporters charge big Pharma doesn’t want him to succeed since cancer is big business. As far as conspiracy about big Pharma- well, seems like Burzynski, while he is making a lot, would make more if his antineoplastons worked and were sold by big Pharma than not.

It is horrible to have cancer, or a loved one with cancer. That disease has caused more people to seek out treatments that have not been tested than almost any other disease today.  

Physicians are trained in science – we want to see things go from the bench to randomized trials. We know things don’t always work out (look at Avastatin, works in the lab, but not in breast cancer) – and yet this is the system that provides a non-bias method of evaluating treatments. When someone says they alone have the answer, but the data cannot be reproduced by large numbers of physicians, or they refuse to work with them – it is every reason to shy away.  Is Burzynski a fraud, over charging a susceptible group of patients?  We won’t know unless his “treatments” are subjected to randomized trials.  

As a physician I applaud when an NCI trial is stopped because the agent tested is better than the agents on the market - and occasionally that happens. That has never happened with Burzynski’s antineoplastons.


Terry Simpson started out life as a basic scientist and decided he preferred people to petri dishes. He is a surgeon in Phoenix, authored several books, and has his musings on (among other blogs)  His view of the world became altered in the 1980's in conversations with of a  patient who was undergoing treatment in Seattle - Carl Sagan.


[Editor's note: Article edited to correct previous errors. Thanks to everyone for bringing this to my attention]