Q. I hear people at the cancer center talking about clinical trials. What are they?

A. A clinical trial is an experiment designed to determine whether a new medical treatment is safe and effective. Clinical trials involve human patients. While the majority of research on any treatment happens prior to bringing in human subjects–pure research, plus trials involving animals–it’s a series of clinical trials that determines whether or not the drug or process will become an FDA-approved treatment.

Q. Sometimes I’ve heard trials referred to Phase I, or Phase III. What’s that all about?

A. A drug or treatment is subjected to three levels of trial before its effectiveness is proved or disproved. Here’s the explanation:

• Phase I trials are the most basic; the most “raw,” if you will. The treatment hasn’t been tested on humans at all; this is the first chance researchers have to see its exact effects, both good and bad, on human patients. Typically, Phase I involves the best way to apply a treatment. For instance, should the drug be given by injection, or by mouth? It also assesses the most effective dosage. How much of the drug can be given, and at what risk of side effects? Since Phase I trials are inherently risky (after all, researchers have no data at all about how the treatment will affect humans), participants are usually those with the most to gain–e.g., terminally ill patients.

• Phase II trials are longer-term, and allow researchers to determine if a treatment actually works– and if so, how. Does the tumor shrink? Do women whose diagnoses points to a 65% risk of recurrence actually see only a 25% recurrence rate? What’s happening to effect these changes (or, why aren’t the desired improvements being seen?) Since Phase II trials are somewhat less risky–they happen only after the drug or treatment has been determined to be relatively safe, at least in the short run–they involve slightly more participants. That said, many if not most of the subjects are still desperately ill.

• Phase III trials are the ones in which most patients are involved. Phase I and Phase II have proven the drug or treatment to be both safe and effective. Now, Phase III will compare it to the current standard of care. In Phase III trials, participants are randomized; that is, some will receive the standard treatment, some the new treatment, and the results compared. Phase III trials can involve thousands of patients, and take place over several years. They’re truly where the rubber hits the road: will this new drug gain FDA approval, or not? Will this new type of intensive one-week radiation replace the standard multi-week radiation regimen?

Q. How do I find out about clinical trials?

A. Ask your doctor. He or she should know about any available trials being conducted through your hospital or cancer center for which you’d be eligible. Also, take a look at TrialCheck, an online service that matches you and your particlular diagnosis with any matching trials in your geographic area.