Research, as long as it is ethical and needed, aims to make the future better. —Dr. Michael Spigarelli, director of clinical trials at the University of Utah School of Medicine
SALT LAKE CITY — It used to be that patients with cystic fibrosis couldn't expect to live very long, yet they are now living into their 40s.
Children with leukemia were in the same boat, essentially issued a death sentence with diagnosis. However, the survival rate for a child with leukemia today is greater than 95 percent.
Mental illness is now classified as a disease that can be treated and dealt with, instead of what once was believed by some to be a result of demonic possession. The consequences of smoking tobacco, poor self-esteem and eating too much salt are also more understood, thanks to years and years of research.
Clinical trials have the potential to make a difference in the world of medicine, as they have over time, and Utah is seemingly a hotbed of activity. Since 1999, nearly 1,500 trials have been conducted at research institutions in Salt Lake City alone.
"I don't know how we'd exist without research," said Dr. Michael Spigarelli, director of clinical trials at the University of Utah School of Medicine. "Our modern world wouldn't be here. We'd be stuck in a world of common sayings and have no true data telling us how to do things."
While Spigarelli, a researcher, physician and pharmacologist, may be biased, much of clinical care in general is built upon comprehensive research. And after detailed study and sometimes animal testing, much of that research relies on volunteer participation by humankind.
In addition to traditional procedures, John Bennett has tried multiple interventions in his ongoing treatment of Stage 4 metastatic prostate cancer. He is hoping any one of them will help to slow down the "runaway train" he said is threatening his life.
"I'm still trying to be as active as I can, to fight it, to slow it down and hopefully allow me to enjoy many more months," he said.
Most recently, Bennett, 59, and his wife began participating in a Qigong course offered to prostate cancer survivors at Huntsman. It is an attempt to research how the calming exercise can improve prognosis of the disease.
"If it weren't for all of these extra things I am doing, doctors said that by August or September this year, I wouldn't be here," Bennett said. The Liberty Park tennis instructor said he'd doesn't have all the answers, but he wants to be involved in cutting-edge research to help with all the hard things he and others have to face every day.
In Salt Lake City alone, thousands of clinical trials and studies are being conducted, to improve how medications and procedures are handed down. Each requires specific numbers of local participants to fulfill testing requirements set by the U.S. Food and Drug Administration.
In addition to earlier access to promising, new medications, patients who choose to enroll in clinical trials often receive expert medical care through the duration of the study, and "have the satisfaction of knowing that they are playing a role in guiding their health care," said Jeff Trewhitt, spokesman for the Pharmaceutical Research and Manufacturers of America. "Patients in clinical trials are making a definite contribution to research."
But companies are routinely having a hard time getting the necessary number of patient participants to further legitimize their studies.
"When biopharmaceutical companies have trouble securing an adequate number of participants for a clinical trial, it slows down drug development and it can even bring it to a temporary halt," Trewhitt said. "That means that patients have to wait longer for treatments they might need."
While the risks might scare some people away, Trewhitt said a bigger problem is that many people just aren't aware that trials are available in their area. He said patients ought to have the opportunity to make an informed decision, weighing the risks and the potential benefits of being involved in any particular study.
A list of the ongoing U.S. National Institutes of Health-sponsored clinical trials can be found online, at www.clinicaltrials.gov. Those being conducted at the U. can be found at www.healthcare.utah.edu/clinicaltrials. Trewhitt said more than 137 trials are actively recruiting in Salt Lake City, 23 in Ogden and about 10 in Provo. Far more are in progress at academic institutions, hospitals and private businesses statewide.
"There is a lot of health care research infrastructure here," he said, adding that pharmaceutical companies seek out the available and dependable patient population, as well as the reliable research base that Utah offers.
"It is clear they are impressed with Utah," Trewhitt added.
Spigarelli's team has recruited more than 800 of the 1,000 subjects needed for a nationwide study on the causes and treatment of pneumonia in kids. They have surpassed the efforts of multiple partners in the project, even helping to redefine parameters of the study.
"People are more willing to help and volunteer here," Spigarelli said. "They're in it to help others."
While risks should be considered, patient protections are built into the process. An independent Institutional Review Board, made up of providers, lawyers, patient advocates and others, must approve each trial "with an eye toward ensuring patient rights," Trewhitt said. Institutions conducting trials, whether they are public or private, are also required to comply with strict regulations set by the FDA.
"We can't have the treatments of tomorrow without doing research today," Spigarelli said, adding that the level of detail involved in a clinical trial is "much higher than what you'd get if you were just seeing a doctor and the doctor was giving you medicine."
Trials are carefully monitored. Instead of visiting a doctor every six months, some studies require weekly visits. And when problems arise, trials are terminated immediately.
"We don't stop being doctors and nurse practitioners or other health professionals," Spigarelli said. "If somebody is having a bad side effect, we stop it. Even if we don't know it was caused by the drug."
He believes all research must seek to improve clinical care in some way. Studies wouldn't be approved "if we didn't think they will work," Spigarelli said. "We can't do a study just because we want to know something."
Unknown reactions are equally important to the research, as the idea is to present specific claims pertaining to each drug, procedure or recommendation.
"We want to be able to say that this drug works for this condition, in this age group, and at this dose," Spigarelli said. "We also want to know how frequently it works," which brings added importance for larger groups of subjects.
In addition to drug testing, institutions also assess surgical procedures, medical efficiencies and even behavioral interventions.
The Huntsman Cancer Institute's Qigong project aims to find ways to decrease fatigue and balance problems that can result from various treatments of prostate cancer in males 55 and older.
"We want to know if exercise makes a difference," said Kathleen O'Connor, research coordinator at Huntsman. For 12 weeks, participants gather twice a week for an hour of the Qigong.
"We think if we can improve physical functioning in this population, we can improve balance and reduce falls, but also help in improving their quality of life," O'Connor said.
The next round of research begins June 5, and anyone interested can contact O'Connor by calling 801-587-4556.
While the tai chi-like exercise seems harmless, there may be risks involved in any type of research, Trewhitt said, adding that it is important to include a person's regular doctor in any decision of clinical trial participation.
"You have to weigh the benefits and the risks, and indeed there are risks, but there are also definite benefits, particularly for chronic disease sufferers who are still seeking the treatment that is right for them," he said.
The drug development and approval process spans approximately 15 years, including three phases of clinical trials to test the safety and effectiveness of new medications and procedures. The average cost of developing a new biotechnology drug (new generation of drugs) is $1.2 billion, Trewhitt said. Between 45 percent and 75 percent of the cost is borne by implementation of the trials, which he said could be a boon to the local economy, depending on the number of studies done here.
The influx of money, however welcome it is at a budget-driven university, doesn't change the way trials are conducted.
"There is no amount of money that would make me hurt people to help any company, Pharma, or anybody," Spigarelli said. "Research, as long as it is ethical and needed, aims to make the future better."
He said the U. doesn't aim to make money on its successful trials, but has a goal to help people and improve overall clinical care.
More and more trials are recruiting children, as fairly new legislation made it OK to involve them with parental consent. Spigarelli said it is making a difference because many of the drugs available today, while they are commonly prescribed to children, have yet to be tested in young people.
Pregnant women are another understudied population that researchers are working on.
Ongoing research is helping to update drug labels, as well as redefine dosage instructions, making new and old medications safer for children and adults alike.
"A university's fundamental jobs are to teach people, expand knowledge and create a more educated work force for tomorrow," he said. "Doing clinical research fulfills all of those goals."
Research, Spigarelli said, "distinguishes the truth from myth and lets us move on to what can really help. The benefit to the public is the health care we have today."