Should I Enroll in a Cancer Clinical Trial?

What you should know about participating in cancer research.

Cancer clinical trials play a vital role in exploring new medical treatments for people. They allow researchers to test innovative therapies and ensure they can benefit patients in the real world. People who participate in clinical trials directly contribute to progress in medical research.

“Our role is to ensure every patient has the support, guidance, and access they need to make informed decisions about clinical trial participation,” says Craig Moskowitz, M.D., the physician-in-chief of Sylvester. “When patients enroll in clinical trials, they’re not only accessing tomorrow’s treatments today — they’re helping us discover more effective, personalized approaches to care.”

Physician-scientists at Sylvester Comprehensive Cancer Center conduct more clinical research than any other institution in South Florida, with more than 550 clinical trials currently underway.

Why join a cancer clinical trial?

“By participating in a trial, one has access to potentially more effective and superior drugs before they get FDA approved. Waiting for a drug to be approved can take several years that some patients just don’t have with the current available treatments,” says Carmen Calfa, M.D., an associate professor of clinical medicine and the clinical research lead of the Breast Site Disease Group.

Dr. Calfa currently oversees the TAPUR study, a first-of-its-kind precision medicine clinical trial that utilizes genetic tests to match patients with new treatments. Many patients who had exhausted all other standard treatments have enrolled in the trial, to great success.

“I’m delighted to say that some patients went literally from the hospice bed to being cancer-free at five years. This is all thanks to research and thanks to our patients who believe in us,” she says.

Patients who participate in clinical trials also often find a better quality of care than with standard-of-care treatment.

“One of the things I think is great about clinical trials is that because there are so many more people involved, there’s actually kind of an enhanced experience for patients,” says Matthew Schlumbrecht, M.D., M.P.H., Sylvester’s assistant director of community outreach and engagement and a gynecological oncology researcher and physician. “Research nurses and clinical research coordinators are checking in on them all the time, and they actually get imaging more frequently than they would with standard of care treatment.”

Rigorous safety standards for clinical trials

José Lutzky, M.D., a triple-board-certified hematologist and oncologist at Sylvester, underscores the safety of clinical trials.

“When you design a clinical trial and when it goes through the Institutional Review Board process, the patient’s well-being is the main concern. Even if there is a potentially great benefit to society and science, if there is a significant risk to patients, it’s not something that will be approved,” he says.

Some patients worry that joining a clinical trial means they won’t receive the usual care for their cancer. But Dr. Schlumbrecht says that’s not only untrue — it would be completely unethical.

“Everybody in a cancer clinical trial will get some treatment. It’s either the standard of care or they get the study drug or some combination of study drugs — we’re not just going to watch the cancer grow,” he says.

How do cancer clinical trials work?

All clinical trials that test a new drug move through several phases.

Clinical trials are an important part of developing new treatments, and the process takes time to ensure everything is as safe and effective as possible. It begins with doctors and scientists studying the treatment in the lab. If it shows promise, they carefully move on to testing it with people — step by step, in phases — always putting safety first and learning more at each stage.

In phase 1 clinical trials, scientists will examine how the body processes the drug and this offers more information to physicians.

“What’s happened over the decades is that a lot of the trials now have better responses, better results. And that’s because we are now selecting the populations a little bit better. We know who’s more likely to respond,” says Dr. Lutzky.

Phase 2 trials test the efficacy of the drug in a small group of participants. Researchers can determine whether the drug is targeting its molecular targets and gain insight into potential side effects, as well as understand the benefits for patients.

By the time a drug goes through phase 3 clinical trials, any risks are well known. The aim of a phase 3 trial is to obtain Food and Drug Administration (FDA) approval. During these trials, the drug may be compared with existing treatments. If a drug successfully completes testing, it is submitted for FDA approval.

How do I find a clinical trial?

Patients interested in finding a clinical trial should first discuss options and potential trials with their providers.

Every clinical trial in the United States must be registered with the FDA. Patients can search the clinicaltrials.gov site, filtering by disease and location to find trials for which they may be eligible.

And then, Dr. Schlumbrecht says, it’s important to start asking questions to understand the benefits.

Questions to ask about available clinical trials:

• What phase of clinical trial is this?
• What is known about this drug going into the clinical trial?
• What are the range of side effects?
• How does this drug work, and why might it be effective in treating my particular type of cancer?
• What happens if I don’t go on this clinical trial? What are the other options?
• How long would I be on the clinical trial?

For many, the risks of signing up for a clinical trial will be well worth it, says Dr. Calfa.

Once the state-of-the-art Kenneth C. Griffin Cancer Research Building, a cornerstone of Sylvester’s vision for advancing cancer discovery and care, opens later this year, clinical trials and collaboration will accelerate innovation in the pursuit of cancer cures. The Griffin Cancer Research Building will stand as a 12-story, 244,000-square-foot, state-of-the-art facility on UHealth’s downtown Miami campus. It will house Sylvester cancer researchers, leading-edge technologies, and clinical trials, encouraging more precise and personalized cancer treatment strategies.

“Taking a leap of faith and following the science can lead a patient to join a clinical trial that can save his or her life or prolong life significantly, allowing for more progress to be made,” Dr. Calfa says.

Written by Jodie Nicotra, a contrinutor for Sylvester Comprehensive Cancer Center.

Learn more about clinical trials at Sylvester.

Tags: Advancements in oncology, Dr. Carmen Calfa, Dr. Jose Lutzky, Dr. Matthew Schlumbrecht, Innovative cancer treatments, Patient participation in clinical trials, Precision medicine in cancer treatment, Sylvester Comprehensive Cancer Center