Clinical trials are important in the development of new treatments for diseases such as cancer. They are research studies that help scientists determine the safety and effectiveness of new therapies. For patients like Donald, a clinical trial may be their last, best hope.

“My particular type of cancer wasn’t responding to conventional chemotherapy. The clinical trial was a possible avenue of treatment that nothing else seemed to provide. It made me feel proactive,” he says.

Clinical trials are typically divided into four phases. A phase IV clinical trial tests the long-term safety and effectiveness of a treatment in several hundred to several thousand people. A phase I trial, on the other hand, is the first time a treatment is studied in people—usually a select number of patients who have not had success with other treatments. A rigorous process of government approval takes place before doctors test these new therapies on patients for the first time. It’s a monumental effort, which is one reason phase I trials aren’t common.

Center for Investigational Therapeutics Team

Opened in 2009, Huntsman Cancer Institute’s Center for Investigational Therapeutics (CIT) program streamlines the phase I trial process. CIT scientists test theories about new therapies using computers. They then develop and study these therapies in the lab to see if they actually target cancer cells. When a treatment looks promising, CIT scientists may study it in a phase I clinical trial. The CIT’s long-term goal is developing new cancer drugs based on HCI genetic discoveries. Already, it has opened a record number of early-phase trials for patients who have few options left.

Sunil Sharma, MD, and David Bearss, PhD, are co-directors of the CIT and leaders in the field of experimental therapeutics.

David Bearss, PhD, and Sunil Sharma, MD

“It’s incredibly rewarding to have this program for patients,” says Sharma. “This is unique to Utah, and we now have so many more options for patients.” Bearss agrees. “To bring not just one, but many phase I trials to this state is unprecedented. It’s a real benefit to the people of Utah, and I’m proud to be a part of it.”

In its first six months of operation, the CIT opened five phase I trials. Having the option to try an experimental treatment is something keeping patients like Donald hopeful. “I’ve thought about this a lot. Even if it doesn’t work for me, it will help somebody. The doctors thank me for participating in the trial, but I thank them for the opportunity. I can’t lose one way or the other.”

The Phases of Clinical Trials

Phase I trial: The first step in testing a new treatment in humans. These studies test the best way to give a new treatment (for example, by mouth, intravenous infusion, or injection) and the best dose. Because little is known about the possible risks and benefits of the treatments being tested, Phase I trials usually include only a small number of specific patients.

Phase I/II trial: A trial to study the safety, dosage levels, and response to a new treatment.

Phase II trial: A study to test whether a new treatment has an anticancer effect (for example, whether it shrinks a tumor or improves blood test results) and whether it works against a certain type of cancer.

Phase III trial: A study to compare the results of people taking a new treatment with the results of people taking the standard treatment (for example, which group has better survival rates or fewer side effects). In most cases, studies move into phase III only after a treatment seems to work in phases I and II. Phase III trials may include hundreds of people.

Phase IV trial: After a treatment has been approved and is being marketed, it is studied in a phase IV trial to evaluate side effects that were not apparent in the phase III trial. Thousands of people are involved in a phase IV trial.