Immunotherapy is on the tip of everyone’s tongue when it comes to current advances in cancer treatment, but for many patients with cancer, access to immunotherapy is often out of reach unless they consider participating in a clinical trial. Many cancer immunotherapies are still in the experimental phase. According to the National Institutes of Health, more than 900 active immunotherapy clinical trials are currently being conducted in the United States.
This explosion in immunotherapy research has already resulted in new and exciting cancer immunotherapy drugs recently approved by the FDA for a growing number of cancer types—bladder cancer, head and neck cancer, Hodgkin lymphoma, kidney cancer, leukemia, lung cancer, melanoma, and multiple myeloma.
The abundance of clinical trials provides an opportunity for patients with cancer to receive immunotherapy drugs that are not yet approved or for combinations of immunotherapy drugs that are still only available through clinical trials. These novel drugs or combinations of drugs may offer patients long-term cancer remission without the toxic side effects of other therapies, especially chemotherapy.1
According to the Cancer Research Institute, only 3% to 6% of eligible patients with cancer participate in clinical trials in the United States, so the majority of patients (more than 90%) may be missing out on the opportunity to benefit from new treatments that are not yet available by prescription.
Patients with cancer are encouraged to discuss with their doctor the possibility of joining a clinical trial. But before you jump aboard the immunotherapy clinical trial train, consider the following factors.
Weighing the Risks
Although immunotherapy is generally considered to be a safer and less-toxic option than chemotherapy or radiation, there are still side effects and risks to consider. Most side effects of immunotherapies are immune-related and can be minor, such as inflammation, rash, fatigue, and fever. However, there is also a chance of more serious autoimmune disorders as a result of immunotherapy. In addition, in some cases, immunotherapy can become too active and attack the patient’s healthy immune system, which could be life-threatening and must be immediately addressed.
Can I Participate in a Clinical Trial?
Most immunotherapy clinical trials have very specific requirements for participation. Patients need to ask their physician if a particular trial is the right choice for them.
Because many immunotherapies are still experimental and have not definitively shown benefit over traditional treatments, there are ethical and legal requirements to give patients the current standards of care (usually surgery, radiation, and/or chemotherapy) first; therefore, many trials are open only to patients who have already tried standard options. A growing number of immunotherapies, however, have already shown benefit over other therapies and are gradually being approved by the FDA for different types of cancer.
The American Society of Clinical Oncology reports that fewer than half of patients selected for treatment actually benefit from immunotherapy. As a result, researchers are narrowing the criteria for clinical trial participation even further to determine why some patients respond to treatment and others do not. Results gained from very specific studies will also allow researchers to predict which treatments are best for a specific patient.
It May Take Time
The response of cancer cells to immunotherapy is often not immediate. In fact, many patients find that the cancer stays the same, or even progresses (worsens), before they see any improvement and tumor reduction.
Patience can pay off for patients who can tolerate the treatment. For those whose tumor responds to immunotherapy drugs, the benefits are worth the time and effort. Over time, in many cases, cancer immunotherapy drugs have been shown to offer long-lasting, life-extending benefits that continue even after the treatment is stopped.
What to Expect
Because immunotherapy is a relatively new way of treating cancer, clinical trials often include additional and follow-up studies, which help researchers to understand exactly how the treatment is working on the immune system. These studies are typically conducted within a lab and require many visits over a long period, as well as many procedures, such as blood tests. According to the Cancer Research Institute, these studies are critical to identify biologic signs or markers (called biomarkers) that can predict who will respond to the specific treatment and, therefore, which patients will best benefit from this type of therapy.
Because many patients are not responding to a single immunotherapy drug, many cancer-related clinical trials are now using combinations of 2 or more immunotherapy drugs, or using an immunotherapy drug plus other drugs, or adding chemotherapy or radiotherapy to get the best results.1
The Table lists just a few examples of clinical trials available for patients with cancer who may wish to have access to an immunotherapy drug. As can be seen in the Table, many new clinical trials are now testing the use of immunotherapies in combination with chemotherapy or with other immunotherapy drugs.
A recent study of immunotherapy drugs used in patients with melanoma showed that when 2 immunotherapy drugs were used in combination, patients had a longer period without the cancer progressing or getting worse (known as progression-free survival) than when one of the immunotherapy drugs was used alone.2
As more immunotherapy drugs are being tested together with older therapies, such as chemotherapy, radiation, or surgery, new opportunities to receive immunotherapy will open up for patients who are currently using only standard therapies.
Expect Trial Exclusions
Because immunotherapy drugs largely depend on a properly functioning immune system, many immune-related conditions and other medical circumstances may exclude some patients from joining a clinical trial. These may include active infections, chronic infections (including HIV, hepatitis B, and hepatitis C), serious heart disease, immunosuppression, cancer that has spread to the brain, and other conditions.
Patients should find out specific details when preparing to participate in immunotherapy clinical trials, such as what the study requires and what it will cost. It is important to know that most treatments used in clinical trials are provided with no cost, but there may be other costs involved so get the information before making a final decision.
Not all patients will have success when participating in a clinical trial, but you will not know unless you try.
Finding an Immunotherapy Clinical Trial
The National Institutes of Health offers a searchable database of clinical trials at www.clinicaltrials.gov. To find immunotherapy clinical trials, enter “immunotherapy” and a specific tumor type into the search box. You can modify the search results in several ways, by location, eligibility criteria (gender, age, specific conditions), and whether the trial is still accepting participants.
Once you have a list of viable trials, talk with your doctor to find out if any of those trials would be appropriate for you.
Many active clinical trials involve treatments already approved by the FDA. The Table lists some active clinical trials of immunotherapies, including some that have already been approved by the FDA.
Each trial receives a National Clinical Trial (NCT) identifier number, so each trial number begins with the letters NCT, as can be seen in the Table. To find more details about a specific trial, use the NCT identifier number to search that trial on www.clinicaltrials.gov.
- Ott PA, Hodi FS, Kaufman HL, et al. Combination immunotherapy: a road map. Journal for ImmunoTherapy of Cancer. 2017;5:16.
- Larkin J, Chiarion-Sileni V, Gonzales R, et al. Combined nivolumab and ipilimumab or monotherapy in untreated melanoma. New England Journal of Medicine. 2015;373:23-34.
- For many patients with cancer, access to immunotherapy can be out of reach unless they consider participating in a clinical trial
- Because many immunotherapies are still not approved by the FDA, many clinical trials are open only to patients who have already unsuccessfully utilized standard options
- The response to immunotherapy can take time; many patients find that the cancer stays the same, or even progresses (worsens), before they see any improvement
- To find immunotherapy clinical trials, visit www. clinicaltrials.gov and enter “immunotherapy” and a specific tumor type into the search box
Cancer Research Institute
National Institutes of Health