Clinical trials, with eyes wide open
When Los Angeles resident Lisan Cooper read recently about a potential breakthrough in the treatment of multiple sclerosis, her curiosity was piqued. Her first thought was that the treatment, using high doses of vitamin B-3, might help her sister-in-law, who had been diagnosed with the condition two years ago.
“There are so few options for people with MS. I would love her to have access to something like this,” Cooper says. She might. The treatment, which has shown promising results in animal studies at Harvard Medical School, may soon undergo clinical trials -- a type of scientific study involving humans.
But just because Cooper’s sister-in-law could participate in a clinical trial doesn’t mean she necessarily should.
Treatment trials, such as the one that intrigued Cooper, evaluate innovative methods of treating diseases. (Other clinical trials investigate potentially better ways of preventing or detecting diseases.) According to the National Institutes of Health, thousands of clinical trials are underway in the United States and actively enrolling hundreds of thousands of people.
The decision to participate in this type of study is a complicated one, requiring a clear understanding of the risks and benefits. “I often worry that study participants aren’t adequately educated,” says John Gallin, director of the Clinical Center at the National Institutes of Health. Some participants may believe that they are assured of getting positive results; others might deny there is any downside to enrolling.
Scientists and physicians take extensive precautions to help ensure the safety of treatment trial participants. Researchers must submit a detailed application to a review board at the institution where the trial will take place, outlining precisely how the study will be conducted and how participants will be protected. They must minimize risks as much as possible, and the risks must be reasonable relative to potential benefits. (Trials evaluating a new drug or medical device -- not simply a new use for an existing treatment -- are subject to additional scrutiny.)
In spite of these safeguards, there is always the possibility that the treatment will cause problems. Side effects caused by a new medication are perhaps the most obvious example.
The likelihood of this happening correlates with the stage that the clinical trial is in. The initial phase of testing carries the highest risk. Because it is the first time the treatment has been used in humans, the risks are somewhat unpredictable and can be potentially serious. Safety issues are less of a concern in later phases, although participants continue to be closely monitored.
Some people enter clinical trials because they have exhausted all conventional treatment options. Others decide to participate because the treatment might offer benefits over traditional therapies.
But this latter group especially must remember that there are no guarantees. Not only might an experimental therapy not offer the anticipated benefits, it might not work at all.
The likelihood that a person will benefit from a particular therapy is higher in the later stages of testing than in the earlier stages. Until the first phases of testing are complete, researchers do not have any concrete evidence that the intervention or therapy under study is effective in humans; the benefits are still theoretical and based solely on the animal testing that usually precedes human studies.
Sometimes the benefits are indirect. “When somebody joins a clinical research trial, they have access to the best care available,” says Gallin. “These studies are conducted in outstanding institutions.” In many cases, the care related to the study is also provided free of charge or at very low cost.
For some people, participation serves as its own benefit of sorts. “Some people do it simply because they are altruistic,” says Gallin. “Clinical research is essential for the development of new and better therapies.”
Weighing the risks and benefits of participation is an inexact science. It is researchers’ job to help potential participants in the decision-making process. They have a legal responsibility to educate them about the study -- answering whatever questions they can up front and disclosing new information as they acquire it. But patients have a responsibility too. They should never join a clinical treatment trial based on blind hope -- or refuse to participate in one based on irrational fears.
Nor should they let a lack of awareness of existing trials cause them to miss out on a good opportunity. Disease-specific information on clinical treatment trials can be found at www.clinicaltrials.gov.
Although a vitamin B-3 study might not be the answer for Cooper’s sister-in-law, one of the other clinical trials involving multiple sclerosis might be. With close to 100 of them to choose from, it certainly doesn’t hurt to explore the possibility.
Dr. Valerie Ulene is a board-certified specialist in preventive medicine practicing in Los Angeles. She can be reached at themd@att.net. The M.D. appears the first Monday of the month.
