Charlotte Mom, 49, Saved By Experimental Life Saving Medicine, Thriving 12-Years Later: Guide To Accessing Clinical Trials

Finding Hope In Clinical Trials

• A woman diagnosed with advanced triple-negative breast cancer in 2012 had already seen several treatment options fail, leaving her doctors to give her a year left to live. However, she found hope in a clinical trial that used an immunotherapy treatment that allowed her to live “cancer-free” 12 years after her diagnosis.
• In 2021, the FDA approved the immunotherapy drug pembrolizumab for the treatment of early-stage triple-negative breast cancer (TNBC). For patients with stage 2 or 3 TNBC, adding the immune-boosting medication pembrolizumab to combination chemotherapy before surgery increases the chances of living free of breast cancer.
• Clinical trials help doctors better understand cancer and discover more effective treatment methods. They also allow patients to try a treatment before it’s approved by the U.S. Food and Drug Administration (FDA), which can potentially be life-changing.
• Despite the great benefits of clinical trials, they also come with risks (like potential side effects that are not fully understood yet). People interested in participating in clinical trials must first talk with their doctor to see if they would be a good fit.
• For help finding a clinical trial that’s right for you, try our easy-to-use Clinical Trial Finder .

A 49-year-old mom who enjoys sailing on cruises with her son is beyond grateful for the life she has today. Just 12 years ago, Stephanie McConnell’s future was far less certain. When she was 37, she was diagnosed with advanced triple-negative breast cancer, an aggressive form of the disease. After a series of treatments failed to improve her condition, her doctors gave her roughly 12 months left to live. Then, she found hope in a clinical trial. “I used to say that I’d never do a clinical trial, that I’m nobody’s guinea pig,” McConnell said to Charlotte Magazine. However, if McConnell wanted to see her son, who was five when she was diagnosed, grow up, she knew she had few options left.

“When a doctor tells you to go home and die? I have a child. That was not going to be his story,” McConnell added. Clinical trials help doctors better understand cancer and discover more effective treatment methods. They also allow patients to try a treatment before it’s approved by the U.S. Food and Drug Administration (FDA), which can potentially be life-changing. McConnell enrolled in a Phase 1 clinical trial that involved using immunotherapy to help treat her triple-negative breast cancer. “Triple-negative” means the cancer is not fueled by any of the three main types of receptors: estrogen, progesterone, or the HER2 protein. Because of this, the cancer won’t respond to certain common therapies. Watch: Dr. Jim Allison explains how immunotherapy works Immunotherapy has emerged as a vital advancement in cancer treatment by stimulating the body’s immune system to fight cancer cells. While McConnell did not explain the specific drug she used for treatment, the results defied expectations. After three years on the clinical trial, her scans did not find any signs of cancer anymore. Today, she remains cancer-free and receives maintenance treatments every six weeks.

Expert Resources on Triple-Negative Breast Cancer

• ‘It’s A Game Changer’: FDA Approves Keytruda, Chemo Combo To Treat Aggressive Triple-Negative Breast Cancer
• Chemo Plus Immunotherapy for Metastatic Triple-Negative Breast Cancer
• Metastatic Triple-Negative Breast Cancer Treatments To Consider
• More Than 80% of Women With Early-Stage Triple-Negative Breast Cancer Experienced a Prolonged Event-Free Survival With Keytruda Plus Chemotherapy Regimen
• New Study Identifies Genes Linked to Increased Risk for Triple-Negative Breast Cancer
• Treating Early Stage Triple-Negative Breast Cancer

Understanding Triple-Negative Breast Cancer and How It’s Treated

Triple-negative breast cancer means your cancer is not fueled by any of the three main types of receptors — estrogen, progesterone, or the HER2 protein. Because of this, the cancer won’t respond to certain targeted therapies, including hormone therapy or HER2-targeted agents like Herceptin. Chemotherapy is typically the treatment, and there are several options. If the triple-negative breast cancer patient does not have many symptoms, oral chemotherapy like Xeloda (generic name capecitabine) may be prescribed. If the patient becomes resistant to the oral chemo drug, IV (intravenous) chemotherapy may be offered weekly or bi-weekly. WATCH: Understanding triple-negative breast cancer Triple-negative breast cancer is one of the most aggressive forms of the disease and accounts for about 20 percent of all breast cancers. The main treatments are chemotherapy, immunotherapy, or participating in clinical trials.

Immunotherapy Treatments for Triple-Negative Breast Cancer

The immune system works by having the immune system use its white blood cells to attack cells in the body that are abnormal or foreign. Cancer cells can stop the immune system from working like it should because they produce certain proteins that protect the tumor from white blood cells. As a result, the body does not recognize the cancerous tumor as abnormal. Immunotherapy stops this process by ensuring white blood cells recognize and attack cancer cells. Immunotherapy drugs like Keytruda (generic name Pembrolizumab) are a form of immunotherapy called a checkpoint inhibitor. Checkpoint inhibitors are a class of immunotherapy drugs specifically targeting proteins found on immune or cancer cells to prevent their binding. The advantage of checkpoint inhibitors in treating cancer is that they don’t kill cancer cells directly, but they stimulate the immune system to find the cancer cells and attack them while hopefully not affecting other surrounding healthy cells. Checkpoint inhibitors work on the background knowledge that the immune system can protect our bodies by eliminating any foreign cells that aren’t produced in the body. It sets out to do this by identifying specific proteins (also known as checkpoint proteins) found only on normal cells; when it finds them, it binds to this cell to mark it as safe and leaves it be. However, sometimes cancer cells can trick the immune system by displaying these same proteins, binding to the immune cells, switching them off, and starting to divide and reproduce, which manifests in the onset of cancer. Nevertheless, checkpoint inhibitors can help by specifically targeting proteins found on normal or cancer cells to prevent this binding and keep the immune cells alert to any foreign cells so that they can find and stop cancer cells. Known side effects for Keytruda include: The most common side effects from Keytruda include:

• Feeling tired (fatigue)
• Rash
• Cough and shortness of breath
• Decreased appetite
• Itching
• Low levels of thyroid hormone.
• Nausea and vomiting
• Diarrhea or constipation
• Abdominal pain

What to Consider When Clinical Trials Are an Option?

Within the U.S., all new drugs must go through clinical trials before the FDA approves them. Although the rewards of clinical trials can be great, they also come with risks. Talking to your doctor about this before enrolling in a trial is important. Some risks to consider include:

• The risk of harm and/or side effects due to experimental treatments
• Researchers may be unaware of some potential side effects of experimental treatments
• The treatment may not work for you, even if it has worked for others

Dr. Beth Karlan is a gynecologic oncologist at UCLA Health. She says the goal of clinical trials is to advance cancer research to the point where the disease becomes manageable, akin to diabetes. “ Clinical trials hopefully can benefit you, but it also provides very, very vital information to the whole scientific community about the effectiveness of these treatments,” Dr. Karlan said . “They can be life-saving. We’ve seen many children and adults who have participated in trials and had miraculous results in the last few years,” Dr. Karlan continued. WATCH: Clinical trials can be life-saving. Before you enroll in a trial, you must be allowed to read the consent documents thoroughly and to ask any questions you may have. The documents will likely contain the following:

• The purpose of the research
• Any risks and benefits expected from the research
• Information about procedures that may cause discomfort (like frequent blood tests)
• Any alternative procedures the patient might consider instead
• How the patient’s information will be kept private
• How long the study is expected to take
• A form confirming you are participating in research voluntarily
• Whether any compensation or additional medical care is available if some sort of injury occurs
• The patient’s rights (like the right to stop research in the middle of the trial)
• Contacts for any patient questions

Patients are allowed to walk away at any time during the trial. Understanding your rights as a voluntary patient is important before you participate in a clinical trial, and understanding that the treatment may not work is also crucial.