Case Report Series Introduction

By Nicholas J. Gonzalez, M.D.

Conventional medical journals often publish case reports, that is, descriptions of individual patients whose disease might have taken an unusual course in response to some new treatment. Such �anecdotal� evidence, as it is technically called, contrasts with a controlled clinical trial, in which different treatments are given to large groups of patients with a particular illness, and the results compared. Some scientists stubbornly insist that only such rigorous exercises, ideally pursued under the most stringent rules and regulations, can �prove� to everyone�s satisfaction that a new treatment for a disease has any value. They often argue that case reports, these histories of individual patients, though perhaps interesting or entertaining, have little scientific merit.

My mentor Dr. Good, one of the finest scientists of the 20th century and the most published author in the history of medicine, always insisted case reports, if properly written and carefully documented, can teach us much about the potential of a new approach. When I first began to evaluate Kelley�s records, Dr. Good said that if I could find even one patient with appropriately diagnosed, biopsy proven metastatic pancreatic adenocarcinoma who had lived five years under Kelley�s care he would be impressed, since no one else in medicine anywhere to his knowledge had such a case. Dr. Good�s knowledge was indeed extensive, since he was at the time President of Sloan-Kettering and an expert in the disease. A single example might not prove to everyone�s satisfaction that the enzyme therapy had value, but it certainly should grab the attention of any fair-minded researcher.

In terms of cancer, a case report, to have value, must meet certain basic criteria; as a start, the diagnosis must be confirmed by biopsy, and the stage by appropriate radiographic studies or surgical procedures. Then, the unusual response to treatment must be carefully defined, carefully explained and carefully documented. The endpoints of most importance for cancer case reports include objective evidence of improvement in the underlying disease, or unusual prolonged survival.

For patients with the typical solid epithelial tumors, disease regression can be verified by serial radiographic studies, such as PET or CT scans. For blood cell malignancies such as leukemia or myeloma, normalization of blood parameters, such as white count or blood protein, might be the marker followed over time.

Survival, if particularly unusual, can be a valid endpoint with or without evidence of disease regression. If this be the chosen criterion, the patient in question must have lived far beyond the accepted medians and means for the disease. Such information on expected survival can be culled with some effort from a number of sources, both governmental and private, so comparisons can be made. The SEER and American Cancer Society websites, for example, provide survival statistics, including medians and means, for many cancers. However, no precise definition of �significantly� prolonged survival really exists, so it becomes more of a judgement call in each case. When I first presented at the NCI in July 1993, Dr. Michael Friedman, then Associate NCI Director, said that if a patient of mine diagnosed with inoperable pancreatic cancer lived three months beyond the reported mean of six months, he wouldn�t be impressed, whereas survival six months in excess of the standard averages would be meaningful. Of course, absolute values for �significance� will vary from cancer to cancer: six months of extra life might be unusual for a patient with a pancreatic neoplasm, but not so for a woman with metastatic breast cancer. In this case, two years beyond the mean would, to me, indicate an interesting response to treatment.

Traditionally the National Cancer Institute, which sets the standards for all oncology worldwide, hasn�t considered survival as a valid endpoint, only objective response as documented by radiographic or other tests. At the time I presented case reports at the NCI in 1993, for epithelial cancers the NCI experts defined �response to treatment � as a 50% or greater reduction in tumor size that lasted at least four weeks. Unfortunately, as it has turned out, many chemotherapy drugs easily shrink tumors to this degree and within this time span, but the patients live no longer than if they had received no therapy. Tumor reduction, in chemotherapy studies, generally does not translate into longer life for the patient. Though the phenomenon has long perplexed the research establishment � logically, one expects if tumors shrink, patients should live longer - scientists now recognize that chemotherapy may kill the less aggressive population of cells and shrink tumors nicely, but then leave a small drug resistant clone that quickly takes over and proliferates explosively. So, the selection for more virulent cells cancels out the initial benefit. In any event, I have long believed this particular definition of response, 50% reduction lasting four weeks, to be rather meaningless, since patients care more about their length of life, not necessarily the size of their tumors.

With my treatment, I learned early on that at times tumors will reduce significantly or blood parameters will improve, to the great joy of patients, but at other times, the disease does not objectively regress, but instead stabilizes. I find patients in the �stabilized� group often survive as long as those enjoying radiographic or laboratory evidence of benefit, as long as they determinedly stick to their nutritional regimen.

During my 1993 NCI presentation, though I discussed a number of patients from my practice with documented disease reduction on standard testing, I also described several cases with long term stabilization without proof of regression. I argued that in such instances the unusual survival should be considered as a response, regardless of what the radiographic or blood tests showed. I know if I had cancer, and had a choice between ten year survival but no radiographic evidence of improvement, or a six week lifespan but impressive tumor reduction, I would choose the former within about a millisecond. Though I hardly assume my comments influenced anyone�s thinking at the NCI, today the scientists there have reworked their definition of response to include not only radiographic or laboratory evidence of regression, but significantly enhanced survival, with or without correlating �objective� documentation.

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In the following case reports, I describe patients appropriately diagnosed with cancer who have survived far beyond what normally would be expected for their situation. For most, but not for all, I also provide evidence of tumor regression, often with complete resolution of their disease.

I would also like to make a point or two about my practice in general. Over the years, I have repeatedly heard the claim, from any number of sources, that I must be processing and treating thousands and thousands of new cancer patients each year. This is simply not the case, nor will it ever be, for a number of reasons. First of all, our approach to patients is extremely time consuming, requiring me to spend at least four or five hours with each new patient divided into two sessions over two separate days. My patients tend to be very sick people with often very long and complicated stories, so even basic history taking can be laborious. Furthermore, I individualize each protocol, also a time intensive process. Then, I must spend hours with each new patient reviewing the details of the complex therapy, which for most will be very new and oftentimes very bewildering. Few, for example, will have experience with coffee enemas prior to meeting me.

My practice is not local in nature, and in fact most patients live some distance from New York. Consequently, I spend considerable time each day on the phone � in my case at least two hours daily - dealing with the inevitable questions that come up regarding the protocol and its management, as well as the medical issues that my patients encounter as they fight their disease.

Since most of my patients have serious medical conditions, even office visits can run up to 90 minutes - not the ten minutes most conventional physicians might allocate. I do not have a revolving door, assembly line practice, nor would I want to. On the other hand, such time commitments reduce the number of new patients I can possibly see in any given week.

A good friend of mine, a scientist by training and a strong supporter of my work, recently remarked that I must be seeing �350-450� new cases of pancreatic cancer yearly, because I am well known for success with this particular illness. I still do not know how he came up with these numbers, but he was completely surprised when I told him in reality I might see a handful of new pancreatic patients a year, and no more. True, I do get many calls from those diagnosed with the disease, but most have already been heavily � and unsuccessfully � treated with aggressive chemotherapy and radiation. By the time they learn of my work and call the office, most have already deteriorated into the final terminal stages of the disease, at a point I cannot help.

I also want to add that a substantial number of patients, perhaps 30% of my total practice, come to me with non-cancer problems, such as chronic fatigue, multiple sclerosis, environmental illness, and any manner of situations for which orthodox medicine offers little.

So, with those thoughts in mind, I present the following case reports, culled from my files.

Individual Cancer Types

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