Becoming a guinea pig to get cutting-edge treatment
By Dr. Mitchell Kahn, M.D.

Clinical trials offer a chance to participate in the development of potentially effective or cutting-edge therapies. But understand that sometimes new treatments are no better than already established treatments, and might even be more dangerous.

Whether or not to participate in a clinical trial can be a complex decision. In order to decide, it may help you to understand more about what clinical trials are and how new treatments are studied and approved for wider use.

Clinical trials are used in all specialties of medicine to test new therapies. They evaluate potential treatments that have had some positive effect against disease in the test tube. These may include new drugs, surgical procedures, medical devices such as pacemakers or stents, radiation therapy or novel approaches such as gene therapy. Trials may also compare two commonly used therapies to see if one is better.

Their goal is to determine if a treatment both works and is reasonably safe. Therapies that prove effective during trials may become the new standard of treatment. Those that don’t are discarded. By comparing smaller groups of people taking different treatments for the same type and stage of disease, researchers can verify that any benefits are due to the treatment rather than to chance or other factors.

Who Participates?
Those who meet the criteria for a particular clinical trial can participate. Researchers usually look for people with the condition the medication is meant to treat. But some trials need healthy people to investigate ways to prevent disease or to determine side effects and effective doses of medicines.

Many people take part in clinical trials because treatments for their condition aren’t available or are not working. If you’ve had no luck with standard treatments, a clinical trial might be the only way to get access to new but unproven therapies. Some people join clinical trials to help people who might develop the same condition in the future.

Each trial lists criteria for participation, including age, sex and type of disease and its progression. Also important is whether you’ve been treated for your condition. If an effective treatment exists, you may not be eligible for a clinical trial of an experimental treatment, although you may become eligible if the standard treatment no longer works for you. Some studies are restricted to people who haven’t started treatment; others focus on people who’ve tried other options.

Your doctor might recommend a clinical trial to you or you might find out about a trial on your own. Often patient advocacy groups let their members know about clinical trials that are recruiting. Newspapers may carry advertisements for clinical trials at nearby research centers. The National Institutes of Health posts listings of thousands of clinical trials online.

Once you find out about a trial that interests you, talk to your doctor. Although your personal physician may not be familiar with a particular treatment, he or she can discuss a clinical trial’s benefits and risks and help you decide whether it is suitable for you.

Phases of Research
Before any treatment is tested in people, it must be proved reasonably safe and effective in the laboratory or in animals in what is called preclinical research. This usually takes one to three years. When a treatment is tested in people, it takes place in distinctly defined phases.

Phase I trials study how much of the treatment to give to get an effect, how much the body reacts to it, how much can be given safely and the best way to give the treatment. It carefully monitors for any harmful effects. Earlier studies have often only been done in animals, so Phase I trials carry more risks for participants. Researchers try to minimize the risks by starting with very small doses, increasing them only if no or minimal side effects occur.

Only a small number of people who would not be helped by existing treatments are included in these trials. About 70 percent of drugs tested in Phase I are successful and proceed to the second phase of research.

Phase II trials focus on determining whether a new treatment works the way it’s intended. For example, does it shrink a tumor or improve the results of cancer-related blood tests? Researchers also monitor common short-term side effects that occur with the therapy. Some things aren’t yet known about the treatment, so risks are still present. Several hundred people may participate in these studies, which take from several months to years. On average, 33 percent of drugs tested in Phase II are successful and move on to the third research phase.

Some unknowns
Some clinical trials may compare a potential new treatment with a placebo. Before you participate in any trial, you’ll be always be told whether a placebo will be involved. If you agree to participate, you must be willing to take a chance that you’ll get the placebo and not the active treatment. Neither you nor your doctor can control which you will receive.

Phase III trials begin only after a treatment shows promise in Phases I and II. At this stage, you’re often assigned randomly to receive either the experimental treatment or the standard treatment. This helps to avoid bias in the results. Phase III trials provide more information about the safety of the experimental treatment and demonstrate whether the standard or the experimental treatment group has better survival rates and fewer side effects. These trials usually include several hundred to several thousand people and often last a few years. Large trials make it easier to estimate what would happen if the experimental treatment were available to everyone with the condition. About 25 percent of drugs tested in Phase III are successful and go on to the government for approval.

Recent advances in genetics and medicine, such as the mapping of the human genome, have fueled an explosion of research into new, targeted therapies that treat more accurately and with fewer side effects than current methods. Participation in a clinical trial gives you the opportunity to help scientific research and gain access to these new treatments. Remember to thoroughly discuss the risks and benefits with your doctor, but don’t be afraid to be a guinea pig.

Mitchell Kahn, M.D. is Assistant Clinical Professor of Medicine at Columbia Medical School and is Director of the Miller Health Care Institute at New York’s St. Luke’s-Roosevelt Hospital.

©Longevity Alliance, Inc. 2006