What Is a Clinical Trial?

Alexis Jenkins has had her share of peaks and valleys during her battle with stage 4 neuroblastoma. In the 5½ years since her local pediatrician referred her to St. Jude Children’s Research Hospital, her cancer has returned three times. Yet, through it all, her parents find solace in knowing their easy-going yet resilient 8-year-old has options.

The family’s journey to St. Jude began when Alexis was a toddler having persistent night fevers. Doctors referred Alexis to the hospital after testing revealed low blood counts and masses in her chest. When Roger and Bridgette Jenkins arrived at St. Jude, they learned about the tumors in Alexis’ abdomen, bone marrow, chest and along the tissue near her spine.

Because the cancer had spread to other parts of Alexis’ body, there was a strong chance standard treatment would not result in a cure. Clearly, Alexis needed something different.

From lab to clinic

St. Jude has created more clinical trials for cancer than any other children’s hospital. Many of these are studies that originate from discoveries made in the hospital’s labs.

In a clinical trial, investigators ask important research questions and provide a plan with detailed instructions on how they aim to answer the questions and the results they hope to achieve.

“The collaboration between laboratory scientists and clinical researchers is well integrated at St. Jude. It’s a continuous loop of information where the laboratory feeds ideas for the next generation of clinical trials, and what we learn in the clinic is used to feed the science in the laboratory,” says Victor Santana, MD, vice president for Clinical Trials Administration at St. Jude.

When Alexis’ cancer returned after standard therapy, Roger and Bridgette enrolled their daughter in several clinical trials.

“Before we enrolled Alexis in a clinical trial, we asked lots of questions,” Bridgette says. “We wanted to know what they had learned from the laboratories. Will the benefits outweigh the side effects? We wanted to learn as much as we could so we could determine if it was the right option for our child.”

More treatment options

In early-phase studies involving new agents or existing drugs, the goal is to determine a safe dose of the new agent in a small group of participants. Once a safe dose is established, researchers test to see how well the cancer responds.

Alexis participated in early-phase studies with some good responses, but her cancer inevitably returned. In 2010, her parents enrolled Alexis in another clinical trial using a new antibody therapy to attack her cancer. She responded well, and her disease went into its longest remission. This antibody has now become an integral part of St. Jude neuroblastoma therapy.

“At St. Jude, patients can participate in a multitude of clinical trials. That’s important, because it gives the children access to the latest therapy that could potentially benefit them,” Santana says. “Because they have participated in trials before and we know their history, their participation becomes a rich source of information in terms of understanding how they may respond to future treatment.”

Creating new standards of care

Doctors have used what they learned from Alexis and others in the study to develop a similar clinical trial for patients with newly diagnosed neuroblastoma. If children in that study continue to respond well, researchers will test to see how well the experimental drug compares to standard therapy or whether that drug can be combined with standard therapy.If the response rate is better than the current treatment, then the experimental therapy will become the new standard of care. Safety and review committees at St. Jude carefully monitor each clinical trial throughout each phase to balance the science and the safety of research participants.

“We want to make sure the science is incorporated into clinical trials in a way that we maximize the potential for benefit and minimize the risk for those who volunteer to participate in clinical research,” Santana says.

Partners in care

Research grants and gifts from St. Jude supporters enable investigators to leave no stone unturned when it comes to seeking new treatment options, with fewer side effects, for Alexis and other children with tough-to-treat cancers. Those funds give St. Jude investigators the freedom to focus on what’s best for each child.

Equally important are the partnerships St. Jude has established with other institutions.

“Pediatric cancer is rare. This poses a challenge, because some trials do not have enough patients to open or the trials close early because not enough patients are enrolled,” Santana says. “As pediatric oncologists, we have to join forces with others throughout the nation and the world to develop methodical clinical trials. That helps us get the best answers to our research questions and identify the best treatment options for our patients.”

Looking ahead

“If it weren’t for our decision to enroll Alexis in clinical trials, she would not be with us today,” Roger says.

Alexis received stem cells from her mom as well as from her sister, Hanah, for bone marrow transplantations. Thus far, all of the healthy stem cells donated by Hanah have engrafted into Alexis’ bone marrow.

Doctors will continue to follow Alexis’ progress. Her parents hope that her cancer stays in remission as they rely on their faith and support from family, friends and clinicians at St. Jude to get them through the days ahead.

“It’s comforting to know that St. Jude has so many options for our child,” Bridgette says. “I’m sure the doctors learned a lot from us because we were willing to participate in clinical trials. Somebody has to be willing to try it. If one kid tries it and it works, then that person can make it better for the next child. All of us have to work together so we can continue to have advances in medicine.”

Learn more: stjude.org/clinicaltrials

Editor’s note: Alexis lost her battle with cancer January 19, 2015.

Abridged from Promise, Spring 2014

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