Clinical Trials and Other Experimental Therapies

Key Points

  • These therapies are considered the leading edge of cancer care.
  • Clinical trials involve both conventional therapies and many complementary therapies.
  • An unproven treatment being touted as an alternative cure for cancer is not the same as an experimental therapy that is being studied in a rigorous scientific manner.

Many experimental therapies, categorized as both conventional and complementary in approach, are being investigated in clinical trials for their effects on cancer. These therapies are considered the leading edge of cancer care.

According to the National Cancer Institute (NCI):1

Clinical trials are research studies that involve people. Through clinical trials, doctors find new ways to improve treatments and the quality of life for people with disease...Researchers design cancer clinical trials to treat cancer, find and diagnose cancer, prevent cancer, manage symptoms of cancer and side effects from its treatment. Trials are available for all stages of cancer. It is a myth that they are only for people who have advanced cancer that is not responding to treatment.”

NCI-supported trials are listed at Find NCI-Supported Clinical Trials. 

Major cancer treatment centers often conduct clinical trials, both privately or in conjunction with NCI. The American Society for Clinical Oncology (ASCO) patient information website provides guidance and resources: Finding a Clinical Trial.

Types of Clinical Trials

Clinical trials involve many types of treatments and therapies such as these, some of which are regarded as complementary therapies:

  • Pharmaceuticals
  • Radiation and other energy-field therapies
  • Lifestyle modifications
  • Biologic therapies
  • Metabolic therapies
  • Herbal and other natural product therapies

If you are considering participating in a clinical trial, bear in mind that a trial may not even be asking whether a treatment is effective, but only whether it's safe to use.

Clinical trials typically follow three phases:2

  • Phase I trials investigate the safety of a new drug or treatment or combination of treatments. This phase is not designed to test the effectiveness of the treatment, though the treatment may possibly improve the cancer outcome. The question under consideration is “Is this treatment safe to administer to patients?”
  • Phase II trials look further into the safety of a treatment as well as whether or not it shows an effect for a specific cancer. If a phase II trial shows that the treatment is likely to work and is at least as safe as regular treatment, then doctors can progress to a phase III clinical trial. In a phase II study, the main question is “Does this treatment improve the cancer outcome for patients?”
  • In phase III clinical trials, the clinical trial treatment is compared with the standard treatment regarding effectiveness and side effects. Phase III trials are randomized controlled trials that can take many years to complete. In a phase III study, the main question is “Is this treatment more effective or less likely to produce side effects than one or more other treatments currently used for this disease or condition?”

If you are considering participating in a clinical trial, bear in mind that a trial may not even be asking whether a treatment is effective, but only whether it's safe to use.

Blinded Trials

The practice of “blinding” trials of treatments—so that patients are not aware of which treatment they receive—is held up as a hallmark of rigorous research. Blinding the physicians to the treatment (a “double-blind study”) is considered even better.3

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Study Methods

Studies come in many different formats, each with strengths and weaknesses. Most research studies considered "strong" are randomized controlled trials in which patients consent to be in the trial and then are randomly assigned to one of several “arms” of the research study.

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Experimental vs. Questionable Treatments

An unproven treatment being touted as an alternative cure for cancer is not the same as an experimental therapy that is being studied in a rigorous scientific manner. Questionable “cures” typically have little or no scientific evidence or rationale that they may actually be effective against cancer. They are marketed using pseudoscientific explanations and claims of being “the miracle cure” or “the missing link” in cancer treatment or some similar claim. Promoted using patient “testimonials” and unverifiable stories, they often prey on vulnerable patients who are fearful and looking for any ray of hope. These may also involve great expense for the patient.

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