Clinical trials are the only way to understand what the best and safest therapies available to patients are with any disease. Carefully examined and evaluated by scientists, and then approved by independent ethics committees, these studies are almost always available to patients with any type of cancer.

Once a new idea or novel drug is identified in the laboratory, the FDA requires three levels of clinical testing prior to the approval of that drug for widespread use. These studies are usually referred to as trials in Phase I, II or III.

The focus of a Phase I trial is to determine the appropriate dose and schedule of a given drug and its side effects. Patients enrolled usually have advanced cancers, which have progressed through standard therapy or for which standard therapy does not exist. Sometimes these patients have been through "everything" and still function extremely well. For them to "give up" is just unacceptable. In other cases, patients with advanced cancers do not want to put themselves through the rigors of conventional chemotherapy and are searching for newer treatments.

The Phase II trial applies the Phase I information on dose and schedule and tries to get a hint of efficacy. Twenty to seventy-five patients are enrolled who have the same type of disease and the same stage and degree of prior treatment. Testing after this stage will lead to a decision on whether or not to proceed with further confirmatory trials.

The Phase III trials are then the final set of studies conducted, and usually involve hundreds, if not thousands, of patients nationwide or worldwide. Here, the novel agent is compared to standard therapy, with the goal of showing that longer survival or some other measure of clinical benefit will occur in the treatment of the cancer with the drug tested.