04/22/2021 By Dr. Dan Platt
Before you start researching for specific trials, it is crucial to take a moment to decide whether a clinical trial makes sense for you. Some questions to consider:
Where are you in your treatment journey?
Clinical trials are often performed to assess whether a drug works at a particular time in a patient’s treatment. Some trials test drugs as “first-line” therapy, meaning that the medication will be one of the first drugs you receive after your diagnosis. Other trials are only for advanced-stage patients who have failed all other treatments. Then there are all the trials for patients between those two poles, such as those requiring the patient to have been on a specific therapy before receiving the study drug.
Patients at different points in their journey often have very different goals and expectations for treatment, so take a moment to think about your goals and to understand where you are in your journey. Some critical benchmarks to consider are:
- The stage of your disease
- Your cancer treatment thus far (drugs received—how much, how long)
- The cancer medications you are currently taking
- The genetics of your cancer
- The results of recent tests or scans
What are your treatment options right now? What are the pros and cons of each?
Depending on where you are in your treatment journey, you may have many options for treatment or just a few. Before starting your research on clinical trials, sit down with your doctor and make a list of all the different options available to you. Make sure to ask about the pros and cons of each treatment, including the expected benefit to your health or alleviation of symptoms and the risk of side effects. Having a good sense of what is important to you when deciding on treatment will be very helpful when weighing the positives and negatives of potential trials.
Do you have the time to participate in a trial?
Clinical trials require patients to have frequent check-ups, testing, scans, and follow-up. During treatment, most clinical trials will require you to go to the testing site once a week or once every other week. After the treatment is finished, you will need to return to the medical center so that the drug’s effect on your cancer can be closely monitored over time, usually every 2-3 months. For many patients, this “follow-up period” can last several years and continue until the patient completes the trial or stops responding to the medication.
If you enroll in a trial, it will be imperative that you keep all scheduled appointments and abide by the rules of the treatment protocol. A consistent appointment schedule is the only way that the doctors can be sure the results are valid! If you have many other responsibilities and cannot keep up with the demands of a trial, it may not be the right choice for you. Before starting your research, understand how much free time per week and month you are willing to spend at the hospital or medical center receiving care. Being able to manage your schedule around the trial requirements will be essential to your health and emotional well-being, so take the time now to understand how committing to a trial will impact your life in the days, weeks, and months ahead.
Some other questions to ask yourself, answers to common concerns, and general information about trial costs can be found on the American Cancer Society website.
Gather Information About Your Cancer
Start by filling out the Cancer Details Checklist from the National Cancer Institute. Don’t be afraid to get help with the checklist from someone on your care team!
To enroll in a particular clinical trial, participants must meet specific “eligibility criteria.” These are the demographic and medical requirements that specify (1) who the trial is intended to help (which depends on things like how the drug works, when in a patient’s treatment it is expected to be used, etc.), and (2) the patients from whom unequivocal data can be gathered. The first point dictates the “inclusion criteria” for the trial or the details about your cancer that must be present for you to enter the trial. These include cancer types (e.g., breast cancer, non-small cell lung cancer, mantle cell lymphoma).
Cancer comes in many different forms, mainly determined by where cancer first occurs and what the cancer cells look like under a microscope. The type and subtype of cancer are essential to know because they help determine which drugs will be, or will not be, effective against cancer.
Stage of cancer (e.g. 0, 1, 2, 3, or 4)
The stage of cancer refers to the extent of growth of cancer in the body. Different types of cancer are staged differently, so it is vital to understand the stage and progress of your disease. A typical system for “solid” tumors (cancers of the organs, like lung, liver, breast, pancreas) is TNM, or Tumor, Node, Metastasis.
- Tumor: The size of the cancer in the primary location (the place where it first occurred)
- Node: Involvement of lymph nodes close to the tumor
- Metastasis: Spread of the cancer to another part of the body (a different organ)
Cancer results from the buildup of mutations in a cell that lead to it growing uncontrollably. There are thousands of gene changes that can turn a cell cancerous. Many of the recent advances in cancer treatment have come about by targeting specific mutations with drugs engineered to affect only that mutation (“targeted therapies”). However, scientists are also now learning that the genetic changes in cancer can strongly impact how cancer responds to “non-targeted” drugs, including chemotherapy. Therefore, for many forms of cancer, genetic analysis of a piece of tumor tissue, such as a biopsy, is the primary way doctors determine how and what drugs should be used. Many clinical trials now require that a patient’s cancer be genotyped and that only patients with a particular mutation be included in the study.
The other form that eligibility criteria take is “exclusion criteria.” These are the details about you and your cancer that must be absent to enter the trial. Most exclusion criteria focus on the patient’s medical history and current health status, especially regarding other diseases the patient may have unrelated to cancer. Since one of the primary outcomes that cancer trials measure is how long a patient survives his/or/their cancer, patients at risk of death from a different cause, such as heart disease, may not be able to join a clinical trial. Thus, an unfortunate consequence of the need for good data about the study drug is that the sickest patients (either due to their cancer or another condition) are disqualified from receiving it.
This is another reason why patients should investigate clinical trials earlier in their treatment when the chances are better to enroll in the trial and derive a benefit from the study drug.
Deskside with Dr. Dan
“One small way I am personally contributing to education is through sharing my take on academic and industry articles, using my medical background to boil down the jargon and pull out the benefits of the news for you.”
– Dan Platt, M.D.
Chief Medical Officer