Early-Phase Clinical Trials Assess Efficacy and Lead to Potentially Life-Improving Treatments for Patients

August 12, 2020

Decades of basic (or bench) science has built the foundational knowledge needed to make advancements in the diagnosis, treatment and prevention of diseases, including cancer. That preclinical research has led to early-phase clinical trials and today’s leading-edge cancer treatments at The University of Kansas Cancer Center.

Joaquina Baranda, M.D., leads the KU Cancer Center’s early-phase clinical trial program. Early-phase clinical trials are the first step in taking new therapies from the research lab to the patient. In phases I and II, researchers figure out whether a new treatment is safe, its side effects and ideal dosage and monitor its therapeutic benefit.

“It used to be early-phase trials only tested safety and dosage. As a result, the road to making a new treatment available to patients was much, much longer,” Baranda said.

However, the National Cancer Investigator Handbook published in 2002 made therapeutic benefits a required component of Phase I trials and, now, Baranda said, “Therapeutic intent is always present in Phase I trials, which often means patients can start getting treatment faster.”

Prior to the change, typically 20-30 patients were enrolled in an early-phase trial. Now, if the investigator observes signs of efficacy, many more patients – sometimes thousands – may become involved in the trial. According to Baranda, this means patients don’t have to wait until the trial moves into Phase III for a potentially life-improving treatment.

Early-phase clinical trial efforts were further bolstered – and cancer treatment options for individuals expanded – in 2010, when the Patient Protection and Affordable Care Act was enacted, mandating that health plans cover the routine cost of care for people participating in cancer clinical trials.

Finally, clinical trials continue to evolve as we gain a deeper understanding of cancer itself. Basic scientists expand our awareness of the mechanisms by which cancer develops and progresses, as well as how it responds to treatment. The result is well-informed, highly targeted efforts to develop therapies. Some drugs have received quicker approval based on early-phase data.

“Early approval is a huge deal because it could mean more lives saved,” Baranda said. “With these advancements, the patient benefits. In early-phase trials, patients not only contribute to science but may potentially receive clinical benefit.”

For these reasons, people generally are open to learning more about clinical trial opportunities.

“We now have so much preliminary data to support the ‘why’ behind a clinical trial. When I explain to patients the details of a clinical trial, they are nodding along, eager to learn more,” Baranda said. “Basic lab discoveries make doctors’ work easier.”

Connecting academia with industry

Each year, Baranda and her team host a symposium to convene clinical trial experts in academia and the pharmaceutical industry. A pharmaceutical industry veteran herself, Baranda acknowledges that creating better cancer therapies is achieved through partnerships across the two sectors.

“Everyone has the same goal – to wipe cancer off the face of the earth. While working in  the pharmaceutical industry, I was touched by my colleagues’ passion. When I returned to academia, I realized we are stronger when we all work together,” Baranda said. “This is one of the ways the KU Cancer Center works to bring the latest therapies to Kansas and western Missouri.”

The virtual Translational Oncology in Academia and Industry Symposium is scheduled for Nov. 19, 2020.

More to do

Scientists don’t rest on their laurels. With each new breakthrough, they continue to push for treatments with better results, fewer toxic side effects and improved quality of life. Immunotherapy is a promising new frontier for cancer treatment. Baranda’s team is working to understand why it works for some cancers and not for others.

“Prostate cancer is one cancer type that has not benefitted much from immunotherapy,” Baranda said. “However, research shows we may be able to improve efficacy by altering the tumor microenvironment, allowing immune cells to enter the cancer cells. We’re excited about the early-phase preliminary data.”

View the KU Cancer Center’s prostate cancer clinical trial opportunities.

Great strides have been made in creating new and more effective cancer therapies. Still, Baranda reiterates, there is more to do. Over the next 10 years, cancer diagnoses are predicted to increase 45-50 percent: one in two men and one in three women will be diagnosed with cancer in their lifetime.

“The battle is not over yet. There is still a lot we need to understand about cancer,” Baranda said. “We are on a good trajectory with our early-phase clinical trials. Through these trials, we are advancing science and helping our patients.”