Clinical Trial

Testing Your Way to Treatment Success

Clinical trials are an important part of cancer care for nearly every cancer patient. Most of us benefit from clinical trials conducted in the past. Treatment that doctors consider “standard of care”, the go-to protocol for a particular type of cancer, was previously tested with a clinical trial and proven to be “better”. That means it either is more effective in irradicating the cancer, extending life and reducing rates of recurrence, or at least, does as well with fewer side effects—than the treatment previously recommended.

Through the use of highly structured trials, peer-reviewed analysis, collaboration and debate, over time oncology professionals have built up an arsenal of treatments and arrived at an agreement about what works best. But what happens when the standard of care doesn’t work? Or when someone builds a better mouse trap?

Academic and pharmaceutical researchers are constantly trying to identify new ways of treating cancer. When they think they have something that works in the lab (usually in mice, which actually have a lot in common with humans), they develop a testing protocol, present that protocol to an internal review board, get funding and approval to proceed with the testing and then initiate a rigorous process of clinical (in the clinic, with people) trials. And through innovation and careful testing, researchers have identified hundred of new treatments in the last 10 years alone.

A Phased Approach

The first step of a clinical trial is to test the safety of the treatment in humans. These “Phase 1” trials are usually very small and carefully monitored to identify potential side effects and figure out how best to deliver the treatment. Does this treatment work for this disease (cancer) in this group of people? Do patients tolerate the treatment at dosage levels that might be effective? Can we deliver the treatment in this way? (For example, as a pill or as an infusion, alone or in combination with other drugs, etc.) If a treatment makes it through a Phase 1 trail, it moves forward to the next round of testing. 

Researchers structure Phase 2 trials to determine the effectiveness of the new treatment, while continuing to assess safety. At this point, researchers offer treatment to a slightly larger, but still limited group of people. They are hoping to determine if a specific dose of the treatment will work for the specific cancer intended. They are also watching closely to determine if there are any new side effects emerging in a larger study population.

Most trials that cancer patients may be eligible for will be Phase 3 or Phase 4 trials. In these phases, researchers are continuing to test effectiveness and side effects, but in comparison to other treatments, and will include hundreds, if not thousands, of participants. The goal is to gather sufficient information to gain approval for the new treatment from the Food and Drug Administration. Researchers will structure the test to assess effectiveness in specific patient populations (age, ethnicity, stage of disease, prior treatments, etc.) and to determine whether or not the new treatment improves outcomes by either improving quality of life (reducing side effects of treatment) or length of life (increasing time to recurrence or progression.)

Trial by Fire?

So, when should you consider a clinical trial and what should you expect?

Sometimes your doctor may recommend a trial when the standard of care options don’t seem to be working, or when your cancer has a high likelihood of recurring and new treatments appear to reduce that likelihood. For a rare cancer, there may not be a standard of care option and a clinical trial may offer hope for a magic bullet.

For some people, the thought of a clinical trials may seem scarier than regular treatment because the drug is unproven, and because, in most trials, you won’t know whether or not you are receiving the new treatment or a placebo—a harmless treatment that has no known benefits, such as a sugar pill or a plain saline infusion. Who wants to be a ginea pig in a lab experiment?

Added Benefits

But there are some important positives to keep in mind. First of all, if there is a standard of care for your disease, in a Phase 3 or Phase 4 trial, at a minimum, you will receive that treatment, along with either a placebo or the test treatment. The clinical trial treatment will be in addition to, not instead of, proven treatments. And it may work better than the standard of care.

Second, they will watch you like a hawk. If accepted into a trial, the team will provide you with more medical supervision than usual as they monitor you for side effects and treatment efficacy. That may mean extra blood test and/or scans, but it also can mean extra security that someone will notice if the cancer isn’t responding—there will be no slipping through the cracks. And the clinical trial medication and extra care will be free, paid for by the researchers, not by you or your health insurance. (Note, standard of care treatment received as part of a trial will still be your responsibility.) 

It’s also helpful to keep in mind that if you are not responding well to the treatment in the trial, you can drop out. Or, if it turns out you are receiving the placebo, and the group of patients receiving the new treatment are doing well, you may be given an option to receive that new treatment instead of dropping out.

And don’t overlook the fact that participation in a clinical trial helps to advance the science of cancer treatment, helping to ensure that better treatments are available for all. No one can force you to be in a trial—you have to opt in—so you’re never really a guinea pig, just a willing participant in advancing science. and helping to find a cure.

Talk to your Doctor

Making the decision to participate in a clinical trial should be done in careful consultation with your doctor. There are 15,000 to 20,000 trials for new cancer treatments going on at any given time, but which ones, if any, may be right for you is difficult to assess on your own. Trials often look for and enroll patients with specific characteristics related to demographics, cancer stage, prior treatments and a host of other factors. Not everyone who is interested will qualify for a particular trial. Perhaps your liver function is not strong enough or your age is outside the boundaries they are testing at this point.

If you are curious, you can go to to find out about all approved clinical trials and search by your disease to narrow the list. Or, start a dialog with, a start-up that uses artificial intelligence to help sort through available clinical trials based on your disease and help you manage the process, and Clearity Foundation will help if your cancer is ovarian. But regardless of the approach, the key ingredient will be a discussion with your doctor who will help guide you through the process of choosing and participating in a clinical trial.

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