Court Testimony

--- Testimony of Dr. Nicholas Patronas of the NCI before the Texas State Board of Medical Examiners, May 24, 1993 ---

Dr. Patronas' background: 1 year internship, 4 years residency in radiology, and 1 year training in neuroradiology (evaluating regions of the central nervous system); he spent 7 years at the University of Chicago as staff radiologist; he spent 4 years at the hospital of the National Institutes of Health (NIH); he then went to Georgetown, where he became a full professor of radiology; the National Cancer Institute (NCI) contracted radiologic services with Georgetown, then he returned to NIH, where he currently is Head of the Section of Neuroradiology. He is one of 17 radiologists who provide radiological services to the hospital of the NIH. At the NIH, he is involved with patients in research protocols who are admitted to the NIH for experimental treatment. He evaluates the various lesions and assesses the effectiveness of the treatments using imaging modalities such as MRI and CT scans and standard radiology.

Nicholas Patronas, MD, called as a witness by the Respondent, after having been first duly sworn by the Judge to tell the truth, the whole truth, and nothing but the truth, testified as follows:

"..My job [is] to assess the effectiveness of the drugs that are given [at the National Cancer Institute] and to provide the diagnosis at the initial stage, upon admission."

"...I was called as an expert in assessing the images to evaluate, together with the rest, the other five members of that team, to evaluate the effectiveness of his [Dr. Burzynski’s] treatment."

"We spent about 7 hours at the Burzynski Institute, and we reviewed the material that was given to us."

"Initially there was a presentation of the cases by Dr. Burzynski; each individual case was studied separately. We were given the history, the pathology, the previous treatment, and the timing of these treatments, and we have somebody who recorded these data. Then the histological slides were presented to one of our neuropathologists, one neuropathologist who was also a guest consultant in the team. He reviewed the slides and confirmed the histological grade of the tumor that Dr. Burzynski was indicating in his presentation. Then there was assessment of the images, either CT scans or CAT scans, or MRI scans. They were serial studies in any given patient. So we were able to see how the tumor started and how it ended up under treatment."

"We took our notes and we discussed the findings, and there was a report [from the NCI team] that was issued indicating what we had found."

"...The memorandum shows or summarizes our findings for each individual patient.... The basic conclusion was that in five of the patients with brain tumors that were fairly large, the tumor resolved, disappeared."

Q by defense attorney: "Was that just happenstance? I mean, was that just by some miracle of --"

A by Patronas: "Well, since the treatment that was given started after the previous conventional treatments which had failed previously, we took the position that this probably represents the results of this new treatment. And so there was only minimal residual tissue at the tumor bed, which looked like a scar, and had no fissures to suggest that there was a tumor in the majority of the cases. Two of the seven patients did not do very well. One of them [is] deceased. The tumor dissolved at least microscopically; we could see it with the naked eye, but it recurred later, a year later. And the other, there was very, very minimal decrease in the size of the tumor. But the tumor was very big, the last one, the seventh. So the last two cases did not survive, although there was definite improvement in one of the two last cases."

Q by defense attorney: "I guess that would be called an objective response in that these patients --"

A by Patronas: "Exactly, because we were six people, and we all looked at the images and we saw the chronological order. We checked the names of the patients on the films, and the films were obtained at different institutions from the entire country, basically where the patients were located. And we had no reason to believe that these were not the results of the treatments."

Q by defense attorney: "Doctor, based on what you have testified to before about your background and credentials, it’s fair to say, isn’t it, that you have seen a lot of brain cancer patients?"

A by Patronas: "Yes, in fact, we see a lot of these cases."

Q by defense attorney: "An that’s part of what you do at the hospital, is to evaluate treatments on brain cancer patients?"

A by Patronas: "Well, different cancers, but since I am the neuroradiologist I see all brain tumors. And I see a large volume of them."

Q by defense attorney: "Now, with regard to at least five patients, I think you testified that five of the patients had their tumors resolved, they all --"

A by Patronas: "Disappeared."

Q by defense attorney: "...Disappeared. Can you give us some kind of context of that? How often does that happen with any -- with no treatment, just by spontaneous remission, or by whatever it is that you --"

A by Patronas: "I’m not aware that spontaneous remission occurs; I don’t think it does. And the available treatments only rarely produce results like that. The only medication -- the only treatment, which I think is the last resort, is radiation therapy. Chemotherapy has very little to offer unless there is an experimental protocol somewhere. However, conventional chemotherapy is -- provides very little, nothing, basically. Radiation, there are some reports indicating that radiation treatment in children particularly could lead to resolution of the tumors, although I don’t know whether it is a permanent one or temporary. So when this happens it is very rare, and I have seen only isolated reports here and there where that has happened with radiation."

Q by defense attorney: "With one case here or there --"

A by Patronas: "Yeah."

Q by defense attorney: "-- An isolated report, you are talking about on a case-by-case basis?"

A by Patronas: "Yeah. Well, radiation should give these results, if it works at all, the first 2 months after completion of the treatment. In these cases, all the patients had already failed radiation because they were treated months, several months after radiation was given and had failed."

Q by defense attorney: "What happens with these patients? Let’s say they failed radiation; what happens then to the patient with brain cancer?"

A by Patronas: "Well, it depends on the grade of the tumor. If the tumor is low-grade astrocytoma, and we are talking about primary gliomas, if it is low grade, survival for years is possible. If it is an intermediate grade, the anaplastic, the mean survival is 2 years, and if it is high-grade glioma the mean survival is about 12 months. That’s it; they die in 12 months, they disappear."

Q by defense attorney: "Now -- It sounds like you are saying that if someone has already failed radiation, at least, that there’s not too much else --"

A by Patronas: "Nothing to offer, exactly."

Q by defense attorney: "-- And that those people are going to eventually die of their disease, barring any unforeseen event or cure?"

A by Patronas: "Exactly."

Q by defense attorney: "And there is nothing that any -- that you could do at NCI?"

A by Patronas: "Nothing we can do, no; not at the present time."

Q by defense attorney: "All right. What about these five patients that are all basically doing -- how come they lived?"

A by Patronas: "Well, it’s amazing, the fact that they are living and some of them are doing well. They are not -- they are not handicapped from the side effects of any treatment, and side effects of the most aggressive previous treatment are worse than the tumor itself. So these particular individuals not only survived, but they didn’t have major side effects. So I think it is impressive and unbelievable."

Q by defense attorney: "How many times have you ever seen this, in your experience, that someone comes with a drug like this, to have this kind of effect? How often does that happen?"

A by Patronas: "I don’t -- I have not seen it at any time with the medication that is given systematically. We have done -- we have an experimental protocol at the NIH where we inject a chemotherapeutic agent through the carotid artery, the artery that goes to the brain, and we have three survivals with this technique, by providing massive amounts of chemotherapeutic drugs to the brain that harbors the tumor. And we destroy the tumor, but we destroy a large part of the brain as well, and the patients became severely handicapped, and a life that’s not worth living. And so I have three cases with this particular experimental protocol which resulted in killing the tumor but a large part of the healthy brain as well. So overall, the protocol was abandoned and is not any more in effect because of the serious side effects that we witnessed."

Q by defense attorney: "Now let me ask your opinion or advise. Based on what you have seen from these patients -- I mean, I think the opinion actually, or the letter actually concludes that the site team concluded that there was antitumor effect from the antineoplastons. What would happen, let’s say for some reason Dr. Burzynski’s brain tumor patients can’t get the medicine any more and have to go off treatment. What’s going to happen to them, in your opinion?"

Objection by prosecuting attorney Helmcamp -- not relevant.

Response by defense attorney: "I think it’s relevant; I think it’s relevant at least to the level of the facts -- The issue in this case is going to be what, assuming a violation has occurred, what’s going to happen to Dr. Burzynski in terms of his ability to practice medicine. And certainly, based on the prior rulings of Your Honor and Mr. Martin, that’s really the issue we are advocating in this case."

Objection overruled.

A by Patronas: "I think these patients will die."

Q by defense attorney: "One of the patients you reviewed was PM; is that correct? What happened in that case?"

A by Patronas: "The tumor was very large and involved the hypothalamus, a very sensitive part of the brain that cannot be operated [on], and had both cystic components and fleshy components, mass-like. And the lesion disappeared. This patient did not have previous treatment, if I recall, other than -- previous chemotherapy or radiation, and the tumor disappeared under our eyes. It was a low-grade astrocytoma, which is compatible with long survival. However, even those low-grade astrocytomas, when we see them, they don’t go away even though they may permit the person to live for many years. In this particular patient’s case the tumor disappeared, and there was a small, tiny remnant left, small percentage of the original size. And there has been several years since then and the patient is well, I’m told."

Q by defense attorney: ‘So at least for that patient you would not recommend that he go off the treatment, would you?"

A by Patronas: "No."

Under cross-examination:

Q by prosecuting attorney: "What if they [the five patients] had received something else; would that affect your decision or your conclusions about the shrinkage of the tumors?"

A by Patronas: "I would be surprised if anything else has caused that because I don’t know of any active agent that produces these results. And if there was one I would like to know its name, for my own education."

Under redirect examination:

Q by defense attorney: "Now, are the gains that you have documented in these five to seven cases, are they what you would call marginal gains?"

A by Patronas: "No, no, definitely not. When I say marginal gains, meaning a sense of survival. The tumor does not go away with these medications [methotrexate, vincristine, BCNU], with methotrexate and the conventional. It just slows down the growth, and so the patient is allowed to live a few extra months. What we see here with antineoplastons, it was near complete resolution of the tumor and long survival, not marginal survival. So we have a different picture here."

Q by defense attorney: "Now let me just -- just to finalized this point, basically what I’m -- Basically what you are saying is that at least from what you’ve seen in your 20 years of experience, the treatment that you have been evaluating, even the experimental treatments with the BCNU, has anything ever come close, any of these chemotherapeutic agents every come close to what you have seen in these seven cases?"

A by Patronas: "As I said, only radiation treatment has shown some results. These are well known and published. And these chemotherapeutic agents have not brought results close to what I saw without massive complications."