Cancer Immunotherapy

More About Clinical Trials

New drugs must go through clinical trials before they can be approved by the U.S. Food and Drug Administration (FDA) and other regulatory agencies. The purpose of clinical trials is to test new treatments and evaluate if they are safe in patients and determine if they are more effective than currently approved treatment options (called 'standard' treatment options).


Potential Benefits

There are several potential benefits of taking part in clinical trials.

  • You are guaranteed to receive the most current standard of care. The National Comprehensive Cancer Network (NCCN), a group of the most highly credited cancer treatment centers in the United States, believes that the best management of any cancer patient is in a clinical trial and strongly encourages participation.
  • If you have advanced cancer or have exhausted standard treatment options, a clinical trial may represent the best option for improving the odds of treatment success.
  • You will have access to a new treatment that is not available to people outside the trial.
  • If the treatment being studied is more effective than the standard treatment, you may be among the first to benefit.
  • You are helping to advance new treatments and new knowledge that has the potential to benefit many others in the future.

(Adapted from the National Cancer Institute, "Learn About Clinical Trials.")


Potential Risks

Participating in clinical trials may also entail certain risks.

  • The new treatment may not be better than, or even as good as, the standard treatment.
  • New treatments may have side effects that doctors to not expect or that may be worse than those of the standard treatment.
  • You may be required to make more visits to the doctor than if you were receiving standard treatments, which may involve extra expenses, such as travel and child care costs.
  • You may need extra tests, which may be uncomfortable or time consuming.
  • Even if a new treatment has benefit in some patients, it may not work for you.
  • Health insurance may not cover all patient care costs in a trial.

(Adapted from the National Cancer Institute, "Learn About Clinical Trials.")


Clinical Trial Phases

There are several phases of clinical trials. Because the overwhelming majority of immunotherapies are still in the experimental phase, this focuses on phases I, II, and III trials, the trials that occur before a treatment is approved. Each phase is designed to answer certain questions. As such, there are different factors related to each trial phase to take into consideration when choosing a trial that might be right for you.

Phase I Trials

Phase I trials are conducted to determine safety and to identify the optimal treatment dose. Many phase I trials represent the first time that a particular drug is being tested in patients. Although this entails some risks, there are always guidelines built into trials to ensure patient safety, such as provisions for conservative dose escalation and to terminate trials if there is evidence of serious adverse effects. Phase I trials are small and typically enroll 15 to 30 patients.

Phase II Trials

Phase II trials are larger trials to test for signs of clinical efficacy of a treatment, usually in a more defined cohort of patients, such as patients with a specific cancer type. Phase II trials are larger than phase I trials and may enroll up to 100 patients.

Phase III Trials

Phase III trials include large numbers of patients, often hundreds to several thousands, and are designed to test if a new treatment is better than the current standard of care. Often, phase III trials are randomized, which means that some patients will receive the experimental treatment and others, in the control group, will not. Regardless, all patients will receive the standard of care, whether they are in the treatment or control group.

Mary Elizabeth Williams: Hope, Survival, and Freedom from Cancer

Mary Elizabeth Williams

In 2010, Mary Elizabeth Williams, a young mother of two, was diagnosed with stage IV melanoma. After participating in a clinical trial directed by CRI Scientific Advisory Council associate director Dr. Jedd D. Wolchok, Mary Elizabeth today is in remission.