Clinical Research Turns Laboratory Ideas into Reality for Patients

OMAHA - Basic medical research like sequencing the human genome and transforming stem cells into blood, bone and brain tissue makes headlines because the ideas involved are so new, so cutting-edge, so "Gee whiz!" But at research hospitals across the country, including the University of Nebraska Medical Center, it takes clinical research on volunteer subjects to determine whether the latest big idea can help real human beings.

In a five-part series beginning today, StatePaper will tell you about the clinical research that makes up almost a third of the externally funded science UNMC does each year. You'll learn about doctors testing:

• A popular anticoagulant drug (also known as a blood thinner) to see whether it can safely be given in small doses over a long term to prevent recurring blood clots


• "Microbubbles" manipulated with harmless ultrasound waves that can pace the heart, deliver medications with pinpoint accuracy and break up clots


• Patient-specific rheumatoid arthritis treatments which could more quickly halt that debilitating disease's march through the body


• Machines that use pinpoint precision to safely deliver higher levels of cancer-killing radiation than ever before possible


• A vaccine made from patients' own tumor cells to prevent the recurrence of non-Hodgkin's lymphoma

"Clinical research is critically important because it does apply the knowledge gained (through basic research) at the bedside," said Dr. Harold Maurer, chancellor of the medical center.

Dr. Tom Rosenquist, interim vice chancellor for research, explained the difference between basic and clinical research.

"The basic science research addresses sort of fundamental questions about the biology of cells and tissues and genes and DNA those kinds of things that we predict may have some value for disease prevention or treatment. But you don't know ahead of time," Rosenquist said.

Out of that basic research can come ideas for new drugs.

"Most of the clinical research that we do here relates to either drugs or methods of applying the drugs to various diseases," Rosenquist said. "… [R]esearch that you apply directly to human beings to see if you get any improvement in their disorder."

Clinical trials are usually funded by pharmaceutical companies or medical equipment manufacturers, which want to find out whether their latest inventions are useful. Volunteers get the trial treatment and any associated examinations, like blood tests, free of charge from the company. But patients and their insurance companies are responsible for the costs of other care. For example, in the non-Hodgkin's lymphoma vaccine study you'll read about in Part 5 of this series, patients are responsible for undergoing a six-month course of chemotherapy before receiving the trial vaccine.

There are three stages of clinical research:

• Phase I: A very small trial where the drug is given to perhaps a dozen patients who have terminal diseases and are not expected to survive. Patients agree to take the new drug without any expectation that it will help them, although it might. At this stage, scientists want to know whether the drug has harmful side effects. Healthy people may also take very small amounts of the drug to determine its toxicity.


• Phase II: Participating at this stage are a small number of patients, perhaps 50, who haven't had luck with existing treatments. This trial determines not only whether the drug is safe for humans, but whether it's effective at treating the targeted medical condition.


• Phase III: Thousands of patients at sites across the country may participate at this stage, which tests the effectiveness of a drug over a long period of time.

Rosenquist said it's usually easy to recruit volunteers for each phase.

"I think the reasons that the seriously ill patients want to participate are pretty self-evident," he said. "Those who are not convinced they may derive any benefit themselves are doing this out of a sense of duty to their fellow man."

That’s the same motivation for many in Phase II and Phase III trials.

"It makes you feel to good to participate," Rosenquist said.

UNMC values each of the volunteers who work with the dozens and dozens of trials going on at any one time.

"They're indispensable. Absolutely. Nothing can be done to advance the science without them," Rosenquist said.

Maurer agreed. "Without them, without the patients participating, there would be no research," he said.

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This story originally appeared in Nebraska StatePaper on July 23, 2001.