Is a Clinical Trial Right for Me?

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A nurse discusses the pros and cons.

Once patients are aware that clinical trials are an option for cancer treatment, many have questions about whether this might be the right path for them. Blanca Ledezma of UCLA Health in Santa Monica, California, a nurse practitioner who holds a master’s degree in nursing, has some insights that can help patients decide. Here, she answers some top patient questions about the pros and cons of joining trials.

When and why should I consider joining a clinical trial?

Patients should ask about clinical trials as soon as they receive their diagnosis and are making their first treatment decisions. They should always know what trials are available to them and whether they want to take part. Perhaps there aren’t any trials that are appropriate for them, but at least they’ve asked and providers know they are interested.

Some patients find it worthwhile to participate in trials because it means they will get a novel agent that could be more effective than standard care alone (Standard, or standard-of-care, treatment is approved by the Food and Drug Administration — the FDA — and widely recommended for use for patients with a particular condition). This may especially be of interest to patients with cancers for which there are few standard treatments or none that are very effective. Patients may also feel good knowing they are contributing to research that could help others in the future.

How will I know if I’m eligible for a clinical trial?

For a trial to be appropriate for an individual, that patient must meet its eligibility requirements. These can include having a specific cancer or one that is associated with a certain genetic mutation, having cancer that is a certain stage, being within a specified age range, meeting a certain level of healthiness and ability to complete the daily activities of living, and having had or not had certain treatments in the past.

What kinds of drugs would I take in a clinical trial?

Trials can test targeted agents or immunotherapies, which are now available in numerous cancer types and are being tested in even more. They may also test chemotherapies, new classes of drugs, combinations of medications or medical devices.

These types of drugs work in different ways. For instance:

  • Chemotherapy affects cells that are rapidly growing and dividing; this includes cancer cells but also some healthy cells.
  • Targeted drugs home in on cells affected by a specific genetic mutation that drives cancer. Typically, they stop the activity of cancer-causing proteins that are made by the body due to such mutations. This means that targeted drugs mainly affect cancer cells.
  • Immunotherapy stimulates the body’s own immune system to fight cancer.

Both targeted drugs and immunotherapies have changed the landscape quite significantly for certain disease types for which there wasn’t much treatment to begin with. Now, we’ve been able to give patients more treatments, either alone or in combination with other therapies, that are personalized to their specific disease types, and they’re doing better than they have in the past.

Unfortunately, none of these drugs are free from side effects. Chemotherapy can cause fatigue, nausea, vomiting, diarrhea, hair loss, mouth sores and low blood counts that can lead to infection. Targeted drugs can cause diarrhea, liver problems and rash and interfere with blood clotting and wound healing. Immunotherapies can cause fatigue, nausea, rash and, less often, problems in organs including the lungs, liver, kidneys, intestines and glands that make hormones.

Why is molecular profiling important prior to a clinical trial?


In standard treatment or clinical trials, molecular profiling of a patient’s tumor is essential. Many targeted drugs and immunotherapies work by targeting a specific genetic mutation and counteracting its cancer-causing activity. Generally, these drugs won’t work well, or at all, unless the cancer has these mutations, so it wouldn’t make sense to give them without first testing tumor tissue or cancer cells found in blood to determine which genetic alterations are present.

In standard treatment, having this genetic information can help doctors decide which therapies to give and the order in which to administer them to maximize the options available.

When there are no FDA-approved treatments that target certain mutations, clinical trials of novel drugs can be especially helpful, although the efficacy of such medications can never be certain until these studies have been concluded.

Because knowing all a tumor’s treatable mutations is so crucial in terms of picking therapies, it’s very important at diagnosis for patients to wait until all molecular test results have come back before choosing a course of treatment.

If I join a trial, is it possible I will get a placebo instead of real medicine?


In trials for patients with metastatic cancer, participants would never get just placebo but would always get some treatment — in many cases the current standard of care. Giving these patients nothing would be unethical.

If it’s a trial of an adjuvant drug (given after the primary treatment) for earlier-stage disease, and it’s not standard practice to give any therapy after the main treatment for a particular cancer, in that case there is a possibility that a treatment arm may consist of placebo alone.

How can I know if a trial drug will work for me?

Once patients are found to be eligible to take a drug, the only way to know if it is going to work is by trying it and that’s true in standard-of-care therapy and in clinical trials. The difference is that we have data from studies of standard-of-care drugs and know, based on this data, that a treatment has been shown to have a certain efficacy for a particular disease type. In a clinical trial, we’re trying to develop (those) data so we can have more treatment options for patients.

Like standard-of-care patients, those in studies are followed with scans to evaluate their response to treatment.

How can I weigh the pros and cons of enrolling in a trial?

Trials are something many patients are open to because they gain access to a new novel agent that is not otherwise available to them. They get the added benefit of being followed very closely.

On the other hand, some patients may say, “I’m working and trying to maintain normalcy, and that doesn’t work for me,” because being in a trial generally requires a greater time commitment than get- ting standard treatment. We always have to consider the patient, their current situation, their desires and their current physical state.

We inform patients that trials are voluntary and that we don’t want to make them feel pressured to enter or stay in a trial. At the end of the day, they should do what they feel is the best decision for them.

How can I learn about clinical trials that might be right for me?

First, it’s important that patients educate and empower themselves and really understand their disease and the available treatment options. Providers will have a detailed discussion with patients regarding treatment options, including standard of care and possibly clinical trials. If patients have further questions regarding treatment options, they should contact their oncologists.

Patient advocacy groups can be of benefit in providing information about available treatments, as long as they’re reputable organizations such as the American Cancer Society, CancerCare, Cancer Support Community and disease-specific groups including the American Lung Association, the Prostate Cancer Foundation, the Multiple Myeloma Research Foundation and the Cholangiocarcinoma Foundation. Some patients may want to seek a second opinion to make sure they’re aware of all their options. Clinical treatment guidelines written by the National Comprehensive Cancer Network (nccn.org/patients/guidelines/cancers.aspx) specifically for patients can also be helpful in spelling out what standard treatment should consist of.

Those who want to learn about clinical trial options can ask their doctors, nurse navigators or other members of their health care teams for suggestions about trials that may be appropriate for them. Some practices run trials and can enroll patients; others don’t offer trials, but may refer patients to other institutions that do.

To search on their own for trials, patients can go to ClinicalTrials.gov and search by disease type and/or drug type. Some patient advocacy groups have searchable online databases of clinical trials, and some offer navigators at no cost who can help patients find trials.