Clinical Trials: You Don’t Know If You Don’t Try

Early in January, I learned that the cancer in my liver has grown—“substantial progression” the PET/CT scan results say. Two doctors, however, have said it’s still only a fairly small part of my liver affected; I like the oncologists’ interpretation better than the radiologist’s.

This progression was not entirely a surprise. The monthly tumor marker tests had been rising slowly over the course of several months, so we figured something was going on. Now we know and can chart a course forward.

My oncologist recommended me for a clinical trial that looks like a good fit given my type of breast cancer, my treatment history, and my current health status—that is, still symptom free, not showing any decline in liver function, and generally feeling good, minor side effects from medications notwithstanding.

So, a few days ago, I met with a new oncologist, the principal investigator of clinical trial NCT03280563—A Study of Multiple Immunotherapy-Based Treatment Combinations in Hormone Receptor (HR)-Positive, HER2-Negative Breast Cancer, also called MORPHEUS-HR+ Breast Cancer. Sounds like something out of “The Matrix.” The study is being conducted in 22 cities, including Pittsburgh. It’s being fielded out of Hillman Cancer Center and UPMC Magee Women’s Hospital. I’ll be going to Magee.

Immunotherapy hasn’t worked well in slowing or reversing progression of HR-positive, HER2-negative breast cancer (like I have), so this test is exploring the combination of an immunotherapy drug with one, two, or three other drugs that are already used regularly or have shown promise through other studies to stop cancer growth, kill cancer cells, or otherwise disrupt the disease process. The hope is the other drugs will boost the effectiveness of the immunotherapy. The study will look at how safe the drug combos are and how well they work, as well as which doses seem to provide the best outcomes (if any) for the least amount of adverse events and side effects.

In the big picture, the goal is to determine if one or more of these combos offers another weapon in the arsenal against this disease. In the small picture—that is, my personal disease—the hope is that whatever treatment arm I’m assigned to will stop the cancer from progressing further for awhile or, even better yet, knock it back and shrink it.

I still have to complete the screening process that’ll include a liver biopsy (to make sure the tumor hasn’t changed in characteristics and also to get a sample for genetic testing) and several blood tests to confirm I’m a good fit. The doctors fully expect I will be. I should know for sure in a couple weeks. I’m excited about the possibilities.

While talking with folks about this recent development, I’ve realized that not everyone understands fully what a clinical trial is all about, so I thought I’d provide some basic Q & As.

What exactly is a clinical trial?

A clinical trial is a type of research that studies a test or treatment given to people. It’s one of the ways scientists make progress in finding new disease treatments or cures. A trial could study a brand new treatment or an existing treatment that’s used in a different way or in a different combination. Clinical trials study how safe and helpful tests and treatments are—often in comparison to the type of treatment that’s usually given for a condition (called the “control). When found to be safe and helpful, the studied treatment or test may become tomorrow’s standard of care. 

The National Comprehensive Cancer Network has a really good overview of clinical trials, written in easy-to-understand language, if you want to read more.

Couldn’t you end up getting a placebo and getting no benefit?

A placebo is an inactive medicine that’s given to a control group in a trial when there isn’t already a standard of care to which a new treatment is being compared. The trial I’m on doesn’t have a placebo. It’s comparing the new combo treatments to an existing standard of care (Faslodex)—that’s the control.

Participants in the trial are assigned randomly by a computer to one of the groups. I could end up in the control group. Initially, I was concerned about that because it seemed like a lesser treatment than I had been getting (I was on two meds, and the control is only one drug). But the research oncologist assured me that control medication should be beneficial by itself. And if it’s not, we’ll know quickly and can pull me out of the trial. My regular oncologist had already mapped a new treatment plan outside of the trial, so I’d either start that or we’d re-examine options.

Although the assignment to groups is random, it is not blinded. Participants will know which group they’re assigned to. I’ll know exactly which medication(s) I’m getting.

What if the new drugs don’t work? Or they work but greatly reduce your quality of life?

That’s a legitimate possibility. It’s a test—so we don’t know whether these new drug combos will work at all and if they do, how well or for how long. And we don’t know if they’ll cause side effects that are tough to live with or if they’ll be hard on the body in ways that make it unsafe to continue taking them. But, I’ll be watched really closely. Scans will be scheduled about every 6 weeks, and I’ll have blood work every couple of weeks. If there are signs of trouble, we’ll know in a matter of weeks. And if I feel uncomfortable about anything at any time, I can pull out of the trial.

If the cancer progresses on the trial drugs, I could be offered a second treatment stage with different medications. Or I could choose treatments outside the trial. In the latter case, I’d go back to my previous oncologist and continue with plan B he and I discussed before.

Does going into a clinical trial mean you’re out of “regular” treatment options?

No. Clinical trials are not a last-ditch effort. In fact, some women with metastatic breast cancer have gone into clinical trials as their first line of treatment. In the case of this trial, to be eligible, I had to progress while on Ibrance or Verzenio but NOT yet had Faslodex (the control drug) or any chemo. In other words, I had to be pretty early in the treatment process. There are many other options still available to me outside of this trial. I’ve heard of many women going through a dozen or more lines of treatment; I’ve only gone through the first line.

Is the treatment considered “experimental”? Will your insurance pay for it?

Clinical trials have sponsors (often drug companies or organizations that receive federal grants) who pay for the trial’s medications and many other associated costs. And before I start any tests of treatments, the trial staff where I’m participating will check with my insurance company to make sure there aren’t any financial surprises.

Are the risks really worth it?

Everyone needs to decide for themselves whether participating in a clinical is worth the potential risks. You’re taking medication that hasn’t been proven. It might not work. It might cause unexpected and even serious side effects. That’s a lot to consider.

I’ve weighed the pros and cons and decided it IS worth it to participate. If the treatment I receive in the trial works, I’ve added a treatment option that wasn’t on the list for me before. If it doesn’t work, it’s a small setback but not one that should have a serious impact on the disease.

Regardless of the outcome, I’ll be contributing to progress in learning what works, or doesn’t, to fight metastatic breast cancer. It makes me feel like I’m doing everything I possibly can—both for myself and for the greater good.

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