Clinical Trials for Metastatic Breast Cancer

Clinical Trials for Metastatic Breast Cancer

Clinical trials are research studies in which people volunteer to try a new drug under careful supervision to help doctors find out if it’s safe and effective.

What are clinical trials?

Clinical trials are research studies in which people volunteer to try a new drug under careful supervision to help doctors find out if it’s safe and effective. By joining a clinical trial, you can potentially try a treatment that isn’t otherwise available and that might be beneficial for you. These studies help improve the overall standard of care.

According to the American Cancer Society, a shortage of people taking part in clinical trials is the biggest reason trials can’t be done. It’s estimated that fewer than 5% of adults diagnosed with cancer take part in a clinical trial. Why? One factor is that information about current trials and how to enroll in a trial are often not well understood.

In October 2014, the Metastatic Breast Cancer Alliance, of which is a member, released an extensive landscape analysis report covering the current state of research, quality of life in patients, available information and support services, metastatic breast cancer incidence and survival rates, and public awareness. According to the findings, women with metastatic breast cancer often do want to enroll in clinical trials so they can try new treatments that might extend their lives. However, these women also say they don’t often learn about clinical trials for new treatments, especially if the trials are being done outside of their hospital. Those who do participate often say they were motivated by their doctors to enroll.

Clinical trials are an important step in discovering new treatments for breast cancer and other diseases as well as new ways to detect, diagnose, and reduce the risk of disease. Clinical trials show researchers what does and doesn’t work in people. Clinical trials also help researchers and doctors decide if the side effects of a new treatment are acceptable when weighed against the benefits offered by the new treatment.

The uncertainty of not knowing how a clinical trial turns out can make it hard to decide whether you want to participate. In rare cases, clinical trial volunteers have been hurt by the treatment or procedure being tested. At the same time, hundreds of thousands of people have benefited and are alive because other people chose to participate in a trial that resulted in a new, more effective treatment. While clinical trials are important, the choice to participate in one is very personal and depends on your unique situation. As with any breast cancer treatment, it’s a good idea for you and your doctor to weigh the benefits against the risks and decide what’s best for you.’s Research News covers several clinical trials. All treatments for breast cancer — including targeted therapies, aromatase inhibitors, tamoxifen, radiation, immunotherapy, and more — were tested in several clinical trials.

Clinical trials are just one type of research that’s done before a new treatment becomes available to people. New medicines must first be discovered, purified, and tested in preclinical trials before researchers even think about testing them on participants. According to the American Cancer Society, about 1,000 potential medicines are tested before one makes it to clinical trials. On average, researchers study a new medicine to treat breast cancer for at least six years (and sometimes many more) before they can begin to test it in a clinical trial.

Read more about clinical trials, including their benefits and risks and what you should know before deciding to participate in one.


Metastatic breast cancer trial search tool

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Expanded Access and Right to Try: Alternative paths to experimental treatments for metastatic breast cancer

If you would like to find out whether there’s an experimental drug that could be beneficial for you, talk to your medical team. Make sure that you’ve thoroughly researched whether there are any clinical trials you can join. In most cases, you won’t be eligible to request an experimental drug through Expanded Access or Right to Try unless participating in a clinical trial is not possible for you.

Doctors recommend enrolling in a clinical trial if you’re eligible since your participation helps advance treatments for breast cancer and, by participating, you receive extra medical attention and monitoring of side effects during the trial.

Learn more about Expanded Access and Right to Try.


Seven Things People With Metastatic Breast Cancer Want You To Know About Joining a Clinical Trial

We asked members of the Community at who have participated in clinical trials for metastatic breast cancer to share the things they think are most useful to know before you enroll. Here are their insights.

1. In some clinical trials, you may receive a standard FDA-approved treatment for metastatic breast cancer plus a placebo

If the trial that you join is a randomized, placebo-controlled trial for metastatic breast cancer, you’ll be randomly assigned to either a test group receiving an experimental medication or a control group receiving the treatment that is the current standard of care plus a placebo. A placebo is an inactive pill or infusion that looks just like the treatment being studied. Placebos help researchers compare the benefits and side effects of treatment with and without an experimental therapy.

In a double-blinded trial, neither the researchers nor the participants know who is getting the experimental medication and who is getting the standard treatment plus a placebo. Double-blinding helps prevent the researchers and participants from having expectations about what will happen and possibly biasing the results.

You’ll always be able to find out before you enroll in a trial if there’s a possibility that you could receive a placebo (and if you won’t be able to learn whether or not you received the placebo).

For example, when Community member Gramen decided to enroll in HER2CLIMB, a randomized, double-blinded clinical trial, she knew from the beginning that she could either wind up receiving Herceptin (chemical name: trastuzumab), Xeloda (chemical name: capecitabine), and the investigational medication tucatinib (ONT-380) or Xeloda, Herceptin, and a placebo.

After three months, she had to drop out of the trial because she was having bad side effects and disease progression.

“The biggest downside for me was that I wasn’t able to find out if I was given tucatinib or a placebo. I can’t base my future treatment decisions on that knowledge,” she says. “But I knew going into it that I wouldn’t be able to find that out.”

2. You might find that you appreciate the increased medical attention and the close monitoring of side effects during a clinical trial

Typically, clinical trial participants are in more frequent contact with their medical team and can more easily get advice or other assistance.

“I liked that during the trial, I had a direct line to the protocol nurses,” says Community member Denny123. “I could call them anytime during business hours. If I had a problem — like I developed a rash — I could talk with them about it right away.”

Gramen found it helpful to have a trial coordinator as her main point of contact. The coordinator handled some tasks that patients often have to manage on their own when they’re not in a clinical trial.

“She answered my questions, set up my appointments, and coordinated the delivery of my medications,” Gramen says. “My job was just to deal with the side effects and make it to the appointments. When you’re not in a trial, a lot of the scheduling and coordinating falls on you.”

Gramen also liked that she had to fill out a diary to keep track of her treatment and side effects during the trial.

“Each day, I recorded when I took the pills and the side effects I had,” she says. “It was helpful to see the patterns. I may keep a diary like that now that I’m starting a new chemo treatment (not as part of a clinical trial).”

3. It’s best to learn as much as possible in advance about the schedule of the trial and any costs that might be involved

When you join a clinical trial, you agree to follow a specific schedule of visits to the clinic or hospital, medical tests and/or other procedures, and a schedule for taking medication.

Community member Pajim recommends that you ask questions about the schedule of a clinical trial before you enroll so you know what to expect.

“You may need to consider: am I prepared to go to the medical center for appointments every week for the first month and then every two weeks after that? If you have any trips out of town planned during the time the trial would be going on, you need to ask about how to handle that,” she says.

Additionally, she suggests that you make sure you clearly understand what costs will be covered by the trial. Don’t assume that the trial will cover all the costs of your treatment, other medical care, travel, or other expenses during the study.

“I didn’t ask the trial coordinators and my insurance company enough questions in advance about the costs,” she says. “I had a CT scan during the trial and I thought the costs for that would be covered by the trial. But it turned out that it was billed to my insurance as part of my ‘routine care’ and I was charged a co-pay. That bugged me.”

4. During the screening process or during the trial itself, speak up if you’re concerned about some aspect of your care

Some people may find that some aspect of their care is not handled the way they expect during the screening process for a trial or during the trial itself. This happened to Gramen, who recommends you share your concerns and advocate for yourself when participating in a clinical trial.

“There was a period of seven or eight weeks in which I stopped taking any cancer treatments while I completed the various things I needed to do as part of the screening process for the clinical trial, including getting a biopsy,” she says. “My doctor and I had been hoping the screening process would just take two or three weeks and that I wouldn’t have to stop treatment for such a long period. If I were to do it again, I would be more vocal and would try to push the trial coordinators to get the screening process over with faster. I can’t afford such a long waiting period with no treatment.”

5. Remember that you can drop out (or “withdraw consent”) at any time

It’s important to keep in mind that you can opt out of a clinical trial at any time because you’re having bad side effects or the trial isn’t working out for you for any other reason. “You’re not locked in,” says Denny123. “In my case, I knew when I was having some severe reactions to a trial drug that I could have stopped taking it earlier than I did.”

6. The experimental treatment you receive during a clinical trial could make a big difference in your health

The hope in joining a clinical trial is that an experimental drug will work well for you. While that isn’t always the case, many people do have success stories.

Since she was diagnosed with stage IV de novo breast cancer in 2002, Denny123 has participated in two clinical trials, both of which she says were “miracles” for her.

The first one was in 2004.

“At the time, I had liver metastases — my liver was filled with tumors and the biggest one was the size of a baseball,” she says. “I enrolled in a trial in which I received Herceptin (chemical name: trastuzumab) and the chemotherapy drug Gemzar (chemical name: gemcitabine).”

After receiving this treatment, she says her cancer went into remission and she had no evidence of disease for seven years.

“I felt like that trial saved my life.”

In 2011, she then developed metastases in the lymph nodes in her chest, and in 2018, she enrolled in a second clinical trial of an experimental drug called poziotinib.

“I wound up taking it for only eight days because the side effects were so severe,” she says. “I had blisters on my lower face and scalp, oral thrush, severe mouth sores, and hair loss. My blood pressure tanked. I had to quit the trial early. The protocol called for taking the drug for 21 days. But apparently those eight days of heck cleared my tumors. CT scans have showed that I’m in remission. Now I’m taking Xeloda and Herceptin to help keep me in remission. I would do that trial again even though it was nasty.”

7. You may not regret participating in a clinical trial even if it doesn’t work out the way you hope

Experimental drugs won’t work for everyone, but you may find something positive to take away from the experience even if the treatment doesn’t work for you.

Gramen, for example, ended up having disease progression during the clinical trial she enrolled in, but she says she learned from the experience and would consider participating in another.

“It didn’t work out for me. But I still think clinical trials are a good option to potentially get access to a treatment that you couldn’t otherwise access,” she says. “I also liked that my first trial was a learning experience. I feel that I have to consider participating in other clinical trials in the future.”

Similarly, Pajim says that she would join another clinical trial even though the two trials she enrolled in didn’t work out for her.

“Even though I had adverse events that caused me to have to quit two clinical trials early on, I think clinical trials are great and I will enroll in one again,” she says. “There’s no way to know what will happen when you start, and it’s worth taking your chances.”

Additionally, Pajim says she found it rewarding to participate in a clinical trial.

“It makes me feel good to know that I’m helping the scientific process,” she says. “When you participate in a clinical trial, you’re helping the next generation of patients coming after you, and that’s really important.”

— Last updated on July 27, 2022, 1:46 PM