How Cancer Clinical Trials Work and Why They Matter

Every new cancer medication, therapy or surgical protocol has one thing in common. They were all tested — and verified to be safe — in a clinical trial.

Clinical trials are critical in a doctor’s ability to deliver the most advanced, effective treatment for patients. As Georgia’s only National Cancer Institute-designated Comprehensive Cancer Center, Winship Cancer Institute of Emory University is a regional and national leader in these efforts.

“We’re in a unique position at Winship,” says Manali Bhave, MD, medical director of the Phase 1 Clinical Trial Unit. “Thanks to our team of experts and our partnerships, we offer a wide range of clinical trials for nearly every cancer type from early to advanced-stage disease.”

To understand the value of clinical trials for cancer, it’s important to know how they work and impact cancer care.

Clinical trials are medical studies that help researchers discover future cancer treatments. Patients who participate help determine how well potential drugs, procedures or other therapies perform compared with existing options.

Dr. Bhave, who is also an associate professor of hematology and medical oncology at Emory University School of Medicine, says these trials are the engine behind current and future improvements in cancer treatment.

“Clinical trials offer early access to promising new therapies and novel approaches. They study new treatments that are not part of the standard of care,” she says. “These studies may introduce patients to strategies that we hope are more effective, less toxic and hopefully improve quality of life.”

Researchers conduct these efforts in phases:

• Phase 1 trial: Investigators test the safety of a new medication, device or procedure.
• Phase 2 trial: Researchers investigate the safety and effectiveness of a new treatment, tracking medical benefits and side effects.
• Phase 3 trial: Investigators conduct a head-to-head comparison between the new and existing therapy to determine which produces the best outcomes.

Cancer clinical trials can focus on any type of cancer and include patients with early-stage or advanced disease. There are four types of trials:

• Diagnostic studies evaluate new early detection methods to identify cancer before symptoms appear or when it’s more easily treatable.
• Prevention research explores methods to prevent cancer in people without active cancer who may be at risk for developing the disease.
• Quality of life studies concentrate on symptoms and side effects to improve comfort and quality of life for people with cancer.
• Treatment trials look for better cancer prevention methods or test the performance of new treatments or procedures against existing therapy options.

If you choose to enroll in a cancer clinical trial, you can benefit in several ways:

• Access to more treatment options that aren’t widely available to the public yet.
• Cost of medication may be covered
• Early access to new, promising drugs and cancer treatments
• Support of a dedicated clinical trials team in addition to your cancer care team.
• Potential to help improve your survival, reduce symptoms and side effects, and find better ways to prevent and detect illnesses.

Every clinical trial has its own requirements for participation. Patients are eligible for studies based on cancer type, cancer stage, personal characteristics and other criteria.

Why are clinical trials important for cancer care?

For over a decade, cancer care has been moving toward more individualized treatment options. Clinical trials play a key role in making that possible. They can be particularly beneficial for patients who have tried several existing therapies with little or no success.

“Cancer clinical trials give patients access to new therapies that can directly impact their care, control their cancer and hopefully prolong their life,” Dr. Bhave says.

“We have more opportunities for personalized medicine. By selecting patients based on blood markers and genetic markers, we can give them access to new therapies more tailored to treat their specific cancer.”

Participating in a cancer study also gives patients additional support. Alongside their clinical care team, the trial’s scientific team monitors them closely. Patients undergo more frequent imaging exams and lab tests, she says. Through this extra layer of supervision, researchers continually assess the safety and effectiveness of the investigative treatment.

But these trials do more than boost care for current patients.

“Clinical trials allow us to gain important insights on how we can advance cancer care for the patient in front of us and for future patients,” Dr. Bhave says.

Currently, roughly 1,300 patients are enrolled in one of Winship’s more than 550 ongoing interventional clinical trials. As a National Cancer Institute Comprehensive Cancer Center, Winship is well-positioned to connect you with forward-thinking studies.

“Our biggest advantage is our thoughtful team of medical providers and scientists. We work together to identify key unmet needs among our patients,” Dr. Bhave says. “And discover new ways to help advance the field of cancer care.”

For example, some of our groundbreaking clinical trials focus on biomarkers that will help us understand cancer genetics. Other studies may identify unique pathways that could allow us to target tumors with specific medications or immunotherapies.

Winship takes a multidisciplinary approach to cancer studies. Researchers work closely with the medical, radiation and surgical oncologists on every patient’s care team. They also partner with nurse navigators, patient advocates and social workers.

“It’s important that patients not feel alone when they enter a clinical trial,” she says. “With us, they have a large team of people supporting them.”

Overall, Dr. Bhave says patients at Winship embrace clinical trials as a treatment avenue that could improve their long-term health. But, for many study participants, it’s also an investment.

“Many of our patients reflect on the fact that their participation will help future generations of cancer patients,” she says.

Learn more about cancer trials at Winship Cancer Institute.

Frequently asked questions about clinical trials

• Can I leave a clinical trial at any time?
  Yes. Participation in a clinical trial is completely voluntary. Patients can choose to withdraw at any time for any reason. In some cases, a care team may also recommend withdrawal if the treatment is no longer effective or if the patient experiences harmful side effects.

• Will I receive treatment if I join a cancer trial?
  Yes. Patients in cancer clinical trials always receive treatment. If a placebo is used, it is never given alone. Instead, it is combined with the standard of care. All participants receive either the current best treatment, the new therapy being studied, or a combination of both.

• How do I know if I qualify for a clinical trial?
  Eligibility depends on several factors, including your type and stage of cancer, as well as personal health characteristics. Your oncologist may recommend a trial, or you can search for available studies and discuss them with your care team. Winship’s research coordinators will review your information and match you with appropriate trials.

• What are the risks of participating in a cancer trial?
  As with any medical treatment, there are potential risks, including side effects or the possibility that the new therapy may not be more effective than standard care. However, patients are closely monitored by both their clinical care team and the trial’s scientific team to ensure safety throughout the study.