Last year, when 5-year-old Hailey Platz was diagnosed with Type 1 diabetes, her mother felt helpless.
"You feel like there's not much you can do. There isn't a cure, you just manage everything," said Heather Platz, 24.
A couple of months after Hailey's diagnosis, Platz turned her concern into action by entering a clinical trial at the University of South Florida Diabetes Center along with her younger daughter, Emma. Platz hopes the trial, investigating the genetics of the disease, helps yield answers that might help her girls and other children.
"It was a pretty easy decision," Platz said. "When I was told about the research study, I was all for it to help us understand why (Type 1 diabetes) happens and find ways to prevent it."
Clinical trials are essential. It can take years and cost millions to prove that a treatment is safe and effective and should be approved for use in patients.
The trial Platz and Emma joined is a long-term, international study that aims to enroll 20,000 people a year. Its goal is to find ways to delay or prevent Type 1 diabetes in people at increased risk. Blood relatives of people with Type 1 have a 10 to 15 times greater risk of developing the disease than people with no family history. The study, known as Type 1 Diabetes TrialNet, started in 2001 and has already found that two drugs can delay symptom onset if started in time.
"Clinical trials are the foundation of medicine, and the people who participate are champions," said Dr. Stephen Liggett, vice dean for research at USF Health. "At any given time, we have 250 trials (at USF) in various stages of enrollment or analysis, involving about 1,100 people."
But only a small percentage of eligible people enroll in clinical trials. According to the nonprofit Center for Information & Study on Clinical Research Participation, just 2 percent of Americans get involved in clinical research. Even among people with chronic health conditions, 6 percent participate in trials. Just 2 percent of cancer patients enroll in trials.
Statisticians use complicated formulas to determine how many people must be enrolled in a study to get meaningful results. The further along in the research process, the more participants a trial needs.
"Some trials die on the vine because we can't get enough patients to participate," said Dr. Peter Blumencranz, a Morton Plant Mease surgical oncologist who specializes in breast cancer.
Surgeons used to remove all lymph nodes during a mastectomy, leaving many women with painful swelling of their arms. Research conducted by Blumencranz and others found that it was necessary to take all the lymph nodes only if cancer was found in the first or sentinel node where fluid drains.
"We needed over 5,000 women for that study, and I didn't have a hard time getting patients to enroll. That study set the standard for how breast cancer is treated today across the country," said Blumencranz, whose practice participates in six to eight breast cancer clinical trials at any given time.
"They are so altruistic," he said of his patients who participate in trials. "They stop focusing on themselves and decide, 'If I am going to go through this, then let me do some good for someone else at the same time.' "
People give many reasons for not participating in trials. Some worry they'll be among those given placebos, though without this comparison group, there's no way to be sure what's being tested works. Others fear the unknowns about a new drug.
"They may think, 'Why would I risk getting a treatment we don't know about when there's one we do know works?' " Liggett said. "Every person has to weigh the risks and potential benefits. We lay that all out for them, but in the end, it's the person who has to make the decision."
Clinical trials are run according to strict ethical protocols designed to protect patients. People are not deprived of standard care; instead, the new treatment is tested against the standard. Trials are stopped if it becomes clear that the new treatment should be given to all patients — or to no patients.
If you want to join a trial, stick with reputable research centers and physicians, said Dr. Dan Sullivan, associate center director for clinical investigations at Moffitt Cancer Center in Tampa. Ask about trial sponsors and whether results have been published in respected journals. "There should also be several levels of oversight and independent monitoring of the trial's progress," he said.
Sometimes, patients gain valuable information right away through trials, which generally include free health services related to the trial. Because of her participation in the USF diabetes trial, Heather Platz found out that she carries the genetic trait for diabetes and passed it on to both of her daughters. Because Emma is now considered at high risk for Type 1, she was enrolled in a related trial testing an oral form of insulin, given before symptoms develop with the hope of preventing the disease.
Platz doesn't mind the time and effort she is giving.
"It really does a lot for research and for the future,'' she said. "It may help prevent (diabetes) in Emma and may cure Hailey, so it's definitely worth it.''
Contact Irene Maher at firstname.lastname@example.org.