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     Our thoughts (mostly questions) on Cancer Clinical Trials:

To begin with, the primary goal of all clinical trials should be to try to save/cure all the patients in the trial. Sadly, the trials seem to concentrate on getting the best possible scientific information and the responsibilities to those patients getting placebo seem at times to be secondary in the trial design.  Pure science is good, but not at the expense of saving patients now.  A natural question is would the doctors who understand the trials participate in them?

The first thing to ask is whether or not the trial is "open" or double blind.  Double blind means neither you or the doctor knows if you are getting no real medicine (no actual treatment.)  If it is double blind, be especially diligent in understanding what follows herein.   It may be wise to exhaust every other possibility before entering a double blind trial.  There are almost always more options if one studies and understands the options on nutrition used with medical treatments.

Trials can also be open or single blind (only the patients doctor knows) and still get good data if the placebo effects is less than the effects of the treatment at that stage. 

A simple truth is that the only time a double blind study is justified is when the benefit is expected to be on the order of a known placebo effect of both the doctor's and patient's response.  Unless the effect of the doctor knowing whether or not the patient is getting placebo or actual treatment is on the order of the expected drug effect, the study should be single blind (only the patient not knowing.)

If there is reason to expect the benefit to be substantial and the risk of toxicity is manageable, there is no reason to have any patient get placebo.

We would ask the question; How strong is the placebo effect in cancer?  Have there been sufficient studies to quantify the placebo effect in cancer?  Is so, and if it is a small percentage effect, why can't that be taken into account statistically with modern software and computers and the placebo group at least made very small. 

How strong is the effect from the doctor knowing whether or not the patient is getting placebo or real treatment?

If sufficient analysis of ongoing health monitoring for bad effects is done, why would equal placebo and actual treatments need to be a necessity?  Statistical analysis does not require two equal groups.

Why not test two groups each with a different actual treatment; no placebo group?

Is there ever any justification for giving a group of terminal patients only placebo?  Do people in such placebo groups actually know they may get no treatment and will almost certainly die if nothing else is done (we believe that there is always hope if nutrition is dramatically changed.)

Is there ever any reason/excuse to continue a trial beyond the point where there is a significant probability that the placebo group is dying at a significantly higher rate?

There should be legally mandated testing (more than just whether or not the patients are living longer in the treated group) and real time data analysis and monitoring by an oversight/disinterested panel.

Are there laws which require a detail and specific set of information to be given to the trial participants which adequately explains the probable deaths of the placebo group?

Clinical trials can be very good and beneficial if done properly.  They do not always benefit all the patients in the trial and you need protection.

NEJM Risks and Benefits of Phase 1 Oncology Trials, 1991 through 2002  " --a response rate of 4.4 percent."  "--death due to toxic events was 0.49 percent."  "--14.3 percent had had at least one episode of grade 4 toxic events."  About 3 times higher risk of a bad toxic reaction than a positive response.  (Positive response does not mean cure.)

Rethinking Phase 1 Clinical Trials  (Read the link and give it some thought.  With modern computers and statistical techniques, there should be no clinical trial which does not have a "good" probability of benefiting the patients.)

The Best Guess Approach to Phase I Trial Design  From the Journal of Clinical Oncology. Not as bad as the title sounds; something to think about.

New Paradigm in Dose-Finding Trials: Patient-Specific Dosing and Beyond Phase I  (Some good thinking.)

Another approach to phase I trials proposed by a university.

IMPROVING CLINICAL STUDIES ON ANTICANCER AGENTS

You need to understand and make your decision on the following as a minimum, in addition to normal question about side effects, possible permanent damage to your body, etc.;

First of all, define to your doctor what your standard of acceptable toxicity (short term health problems and long term health problems) are.  (Are you willing to risk death?  Are you only willing to risk long term disability?  Are you only willing to risk long term pain?  Are you willing to risk extreme nausea?  Are you willing to risk other cancers?  Are you willing to risk accelerated cancer mutation to a more malignant faster growing form?  Or, are you willing to risk no more than hair loss? Make it very clear how far you are willing to push the chemo toxicity.

Then ask;

    1) What is the risk to those who receive a placebo?  How will that risk be controlled?

    2) What will be done to protect those on placebo?  What will happen if the drug is showing a statistical benefit above a certain level?

    3) Will the results of testing and patient condition be monitored in real time (say weekly) with statistical analysis of data to protect those on placebo or for adverse effects of the drug on those receiving it?

    4) Can I terminate my participation if I need on my own initiative?

    5) Will anyone from the outside be monitoring the trial for safety?

    6) Is there any possibility that the treatment can make the cancer worse?

    7) Is it possible that the treatment will make the cancer dead ended (probability of limited lifespan.)

This link is aimed at breast cancer but is a good general list of clinical trial questions.

Before making a decision, be aware that some chemotherapy agenst are so toxic that a special handling system is being introduced to protect the medical works!?  "Even though chemotherapy agents benefit cancer patients, some are known carcinogens. In September 2004, the National Institute for Occupational Safety and Health (NIOSH) issued an alert stating that health-care workers who handle hazardous drugs or work in areas where they are used may be exposed to these agents in the air or on work surfaces, clothing or medical equipment."  We are not arguing against chemotherapy (it is certainly necessary in some form where the tumor cannot be reduced any other way), but we are suggesting that it be really understood, researched, and very carefully considered if it is at all highly toxic.

From the Annals of Internal Medicine: "The long-term benefit of adjuvant, high-dose chemotherapy compared with that of standard adjuvant chemotherapy in patients with breast cancer has not been established."  Notice that the operative phrase is; has not been established.  The intuition that if a small dose kills cancer, a higher dose would be even better is not necessarily true. 

Beware of any clinical trial that is just more of the same. Be sure there is something with scientific data behind it which indicates a probably significant benefit worth the risks.

If you have a choice of trials, and other parameters are near equal, it is probably better to choose one which is the closest to a natural therapy.  For example, there are trials of variants of vitamins (called analogs.)  There are also trials using nutrition to boost the effects and/or lower toxicity of proven agents. There may be trials, however of single nutrients, and while nutrients can be very powerful, single nutrients are probably not going to cure cancer.   There is what is called BRM therapy; "Biological therapy tries to get your own body to fight cancer. It uses materials made by your own body or made in a laboratory to boost, direct, or restore your body's natural defenses against disease. Biological therapy is sometimes called biological response modifier (BRM) therapy or immunotherapy. Biological therapy is being tested in clinical trials."  (Our emphasis.)

The same is probably also true for simple chemo or radiation protocols; cancer almost certainly needs to be attacked on multiple fronts.   "--the greatest and most durable therapeutic benefit will likely be achieved with combination regimens that address several targets."  Integrative Cancer Therapies, Vol. 3, No. 4, 349-380 (2004)

The farther from a path which has some proven element or the more toxic the risk, the harder one should look to be sure there are not safer or more probable benefit options in other trials.

Off-Protocol: Getting New Treatments Outside a Clinical Trial

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