You don’t have to wait until life is over to donate your beleaguered body to science.
Right now researchers need women with regular periods as well as excessive body hair. They need impotent, overweight men.
They need older teens and young adults with cancer. And even if you’re the picture of health, they need you to act as the “control” specimen.
In Illinois, a research hotbed, 948 federally and privately supported clinical trials are registered with the National Institutes of Health’s Web site, clinicaltrials.gov, hundreds more than any other state. The epicenter, the University of Chicago, has about 250 open trials in cancer alone.
The pay, if any, for participants is often minimal. And people sign on knowing they aren’t guaranteed a lifesaving treatment, or any health benefit for that matter.
But since the mid-20th Century, human clinical trials have been the framework for determining the safety and effectiveness of new drugs, medical devices or biologic treatments, such as vaccines or gene therapy.
Designed to answer scientific questions, the studies have demonstrated everything from the benefits of Streptomycin against tuberculosis, to the heart dangers of the anti-arthritis drug Vioxx.
As the Vioxx debacle showed, access to
the results of clinical trials is critical. The findings are so influential, they can put an entire generation on a certain type of medication. And they subsequently can take millions off the same pills.
“The key issue [in trial participation] is informed consent so people know what they’re getting into,” said Tim Murphy, a professor of philosophy in the biomedical sciences at the University of Illinois at Chicago.
“Desperate people will go wherever hope is to be had, so it’s important researchers know the benefits [of trials] are worth the risks to keep them safe.”
Conducted at university medical centers or hospitals, doctor’s offices or private research institutions, the studies that involve medications are largely funded by drug companies. Studies take all forms but primarily are classified as interventional or observational.
The preventive aspect
Interventional trials examine experimental drugs, devices, behavioral therapies, alternative approaches to surgery or new ways of using previously approved drugs. They also can be preventive in nature and include using medicines, vitamins, vaccines, minerals or lifestyle changes.
For example, an interventional trial might look at the effect of injections of Botulinum Toxin Type A, or Botox, on bladder overactivity. It could compare two different types of stroke medication. Or it could enlist volunteers to try a new vaccine for melanoma skin cancer. All are current trials at Loyola University Medical Center.
To eliminate bias, the best trials are double-blind, meaning neither you nor the doctor knows whether you’re taking the investigational drug or a placebo, a substance with no active medication. Trials also should be randomized, meaning patients have an equal chance of being in any of the groups, like flipping a coin.
Observational trials differ from interventional in that they look at health issues in large groups of people, often in regular settings. These can be as simple as filling out questionnaires to help researchers collect data on disease symptoms or answering survey questions.
In an observational trial at the University of Chicago, professor Elmer Abbo is trying to determine whether advance directives, which tell the doctor the type of care you’d like if you’re in a coma or unable to make medical decisions, need to be revised.
Volunteers first are given Illinois’ traditional advance directive and then a modified version. Abbo’s hypothesis is that living wills, as drafted, haven’t dramatically affected end-of-life care because they rarely apply.
“It’s a study to see whether patients are interested in other options, how living wills are written and whether they’re willing to execute one,” Abbo said.
There’s a downside
Some trials are significantly riskier. The experimental treatment might have unpleasant, serious or even life-threatening side effects. It might flop, as was the case with studies that showed a new class of antidepressants didn’t work any better than placebos. And the process might be more taxing than a regular treatment, including trips to the study site, more drugs or surgeries, complex dosages or longer hospital stays.
But patients can withdraw at any time. And though the seriously ill don’t see it this way, the ultimate aim of clinical research is future, not immediate, benefit.
“Most of the people who get involved feel like they are contributing to society’s understanding of disease,” said Wendy Wolf, director of Northwestern’s NuGene Project, an ongoing clinical trial at the Center for Genetic Medicine that collects DNA for future researchers. “They’re participating for altruistic reasons or because someone in the family has a condition and they feel like they can contribute to lessen the disease burden.”
Meanwhile, for those whose disease has reached a dangerous stage, some trials give patients access to new research before it’s widely available.
California’s Ellis Fleenor, 68, said he was the fifth person in the U.S. to try a new radiation treatment for prostate cancer called CyberKnife, a small linear accelerator on the arm of a robot. Instead of undergoing conventional external beam radiation therapy (which lasts 37 days over nearly eight weeks), Fleenor chose the experimental CyberKnife at Stanford Medical Center, which involves five treatments in one week.
CyberKnife, manufactured by company Accuray, is promoted as a more accurate treatment that allows a higher dose of radiation to be applied each day.
Locally, it’s available at Community Cancer Center in Normal, Ill., Cybertechnologies in Bloomington and at St. Catherine Hospital in East Chicago, Ind. according to the Accuray Web site.
“I’d say at this point I’m happy with the results,” said Fleenor of Palo Alto, who had just marked the 15th month following his treatment. “I’ll have a better feeling on my case at the end of two years when my trial is over and I see if the PSA [or prostate-specific antigen, a protein produced by the cells of the prostate gland] has dropped to 0.5 and whether there are remaining significant side effects.”
Another advantage of clinical trials is that participants can cultivate stronger relationships with their doctors than they could in a typical office setting because of strict consent protocols, said Ruth Ross, director of clinical trials research for Advocate Health Care.
Long-term relationships
Chicago’s Tamica Head, 30, who was diagnosed with polycystic ovary syndrome (PCOS) when she was 18, is on a first-name basis with her doctors, something she attributes to years of clinical trial participation.
PCOS, which strikes between 5 and 8 percent of women in the U.S., appears around puberty and causes irregular periods, excess hair growth and obesity. Head is participating in a study that looks at the link between PCOS and sleep disturbances.
The three-part project, headed by Dr. David Ehrmann, director of the Center for PCOS at the University of Chicago, needs women who have PCOS and sleep apnea (a cessation of breathing for 10 or more seconds during sleep), overweight women who have sleep apnea (but not PCOS) and healthy volunteers who don’t mind having their sleep disturbed for study.
“There is still little known about what causes PCOS or the treatment for it,” said Head, who works for the American Dental Association. “I’d still like to know if causes are hereditary or if there is a genetic mutation or what it may lead to. I’m just as curious as the doctor.”
Critics say problems with the clinical trials system, which include the potential for drug companies to suppress negative results, highlight the need for a mandatory national clinical trials registry and more openness from pharmaceutical drug companies.
There also is an emotional toll for participants. Murphy of the University of Illinois warned that people can have their hopes dashed by trials when a new study drug or device doesn’t work.
But researchers say future medical breakthroughs depend on these experiments, which can sometimes yield unexpected results.
One University of Chicago study designed by Dr. Mark Ratain showed that the drug sorafenib, developed as a treatment for colon cancer, wasn’t effective.
But thanks to the clever trial design by Ratain, associate director for Clinical Sciences at the Cancer Research Center, the drug was found to help with kidney cancer. It’s now in Phase 3 trials, the stage before it receives government approval.
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The phases that make a clinical trial
Clinical trials are conducted in different phases, called Phase 1, Phase 2, Phase 3 and Phase 4 trials. Before a treatment reaches Phase 1, it has been tested only on animals.
Phase 1 trials might be the most unnerving, because it’s the first time a procedure has been used on humans. Phase 1 trials take a small group of people (20 to 80) and look at the side effects and safety of an experimental drug. These trials also study what happens to a drug in the human body, looking at how it is absorbed, metabolized and excreted.
If a drug clears Phase 1, it enters Phase 2 trials, which study whether the drug fights disease. These studies often compare the new drug to the current standard of care, to see which works better. In Phase 2 trials, the experimental drug or treatment is given to a larger group, between 100 and 300 people.
In Phase 3 trials, the experimental treatment is given to large groups of people (1,000 to 3,000) to confirm its effectiveness, monitor side effects and compare it with commonly used treatments. These are randomized studies, meaning a computer decides who gets the standard treatment or a placebo, and who gets the experimental treatment.
Phase 4 trials take place after the drug has been approved by the Food and Drug Administration.
The main goal of these post-marketing trials is to find out as much as possible about long-term risks associated with the use of the drug.
–Julie Deardorff
A sample study
This trial highlights just one type of opportunity for people interested in being part of a clinical trial:
– ANGER MANAGEMENT
Purpose: University of Chicago psychologists Mike McCloskey and Kurt Noblett are looking at how different forms of talk therapy compare in reducing anger and impulsive aggression in people.
Eligibility: Both genders, ages 21 through 55. Participants must have been diagnosed with intermittent explosive disorder (IED), a disorder with frequent and sudden outbursts of anger. Contact: Mmcclosk@yoda.bsd.uchicago.edu.
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On the radio
Would you be willing to take part in a clinical trial to benefit mankind, if not yourself? Share with Kathy and Judy between 9 a.m. and noon Monday. Tune in WGN-AM 720.
jdeardorff@tribune.com
