Newswise — Bringing new cancer therapies to the broader patient population is a multi-faceted process. Whether it’s a new therapy, revised method of surgery or novel combination of the two, it must always be first tested in humans before becoming approved standard of care. 

The majority of human trials are conducted by the pharmaceutical industry. Institutions across the United States including The University of Kansas Cancer Center support these clinical trials as patient sites, closely following the pharmaceutical company’s guidelines, administering the treatment and then monitoring the patient. All data is sent to the company for analysis.

Not all clinical trials follow this path: a researcher with an idea can also design and initiate a clinical trial. Called an Investigator-Initiated Trial, or IIT, the idea is born and grown at academic medical institutions like KU Cancer Center.

Pharmaceutical clinical trials typically test a new therapy for a specific issue; they do not investigate all potential uses for the new therapy. The goal of an IIT is not always to test a new therapy, but to find new or revised applications for already-approved products. IIT’s expand product knowledge, including safety.

IIT’s are a niche academic area, and it takes a complex support system in order to execute the study conception, quality control and quality assurance, design and data analysis and interpretation.

Qamar Khan, M.D., associate professor of medicine, has led seven breast cancer IIT’s in his career.

“The National Cancer Institute views IIT’s as an indicator of cutting-edge research. With an IIT, we come up with the idea, we design the trial and we obtain our own funding,” Khan says. “It is 100 percent our own.”

Melissa Mitchell, M.D., Ph.D., assistant professor, balances patient care with research. Her interest is in reducing overuse of radiation therapy in certain breast cancer subpopulations. 

“When I was in graduate school, I knew I wanted to conduct research and treat patients,” Mitchell says. “When you are in charge of your own clinical trial, you have the opportunity to change standard of care and therefore drastically change the outcome for an entire group of patients – not just the ones who walk through your clinic.”

Observation leads to innovation

For many physician-scientists, inspiration comes from their patients.

“The research questions always come after my interactions with my patients in the clinic. You find the unmet need for that particular situation,” Khan says.

For example, it is standard treatment for women with estrogen-receptor-positive (ER-positive) breast cancer to undergo a lumpectomy followed by chemotherapy treatment.

“We wondered if these patients would benefit from treatment before surgery. Then we could study the biology of cancer, allowing us to personalize the treatment to the individual patient,” Khan says.

This thought spurred a small clinical trial testing a combination of two therapies in women with ER-positive breast cancer with the hope that they may be able to avoid chemotherapy while still effectively treating the disease.

The trial has revealed encouraging results, and Khan is preparing for the next phase, which will take place at 10 centers across the United States. Siteman Cancer Center, an NCI Comprehensive Cancer Center, will analyze biopsy samples. KU Cancer Center will monitor and conduct the trial.

“This next phase is a completely unique model – a large investigator-led, multi-center trial,” Khan says. “A collaboration of so many NCI-designated cancer centers participating in an IIT is unprecedented.”

For Mitchell, it was remarks made by her patients that propelled her towards one of her investigator-initiated trials.

“Some patients are more fearful of radiation than any other component of their cancer care. Several of my patients who had no cancer left after chemotherapy  and questioned why they need radiation. It is standard of care to give radiation to anyone with breast cancer undergoing lumpectomy, as we know there is a survival benefit.  However, in HER2-positive breast cancer patients, newer targeted agents have drastically improved outcomes, and studies have begun to question whether radiation is actually needed for these patients,” she says.

Mitchell was cautious, but looked closely at the data. She was encouraged by what she saw and consequently initiated a trial that will study the effects of omitting radiation as part of overall therapy.

“This is a first-in-kind trial,” Mitchell says. “No one else is doing this.”

Every clinical trial has a long journey before it makes it to the broader patient population. But KU Cancer Center’s curious, passionate investigators are dedicated to developing the latest in cancer care.

“We ask the questions that could lead to the next cutting-edge cancer therapy,” Mitchell says. “Our patients have the opportunity to receive the latest treatment before it’s even available to the broader patient population.”

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Newswise: Moving Homegrown Ideas into the Clinic


Caption: Melissa Mitchell, MD, PhD

Newswise: Moving Homegrown Ideas into the Clinic


Caption: Qamar Khan, MD