Experimental Treatments: When Patients Want Something Outside Of Standard Of Care

I was reading my emails this morning and came across one from the Foundation for Women’s Cancers, announcing September as Gynecologic Cancer Awareness Month. Their social media campaign centered on the hashtag #sharethepurplelove, and the focus on clinical trial awareness and participation. It got me thinking again about the enterprise of clinical trials, and just how much goes in to clinical trial enrollment.

Just this morning I was on clinicaltrials.gov looking at trials related to dietary interventions and breast cancer. There were 92 trials listed, all actively enrolling, and this was in breast cancer alone. As a layperson, I might look at this list and think of it as a menu — a confusing yet all-inclusive menu — and all I would need to do is choose one. But with each link, one would discover that it is not a menu; it is a listing. The difference is that not all of the things listed are accessible to you. It might be because the trial is enrolling in Los Angeles, and you live in Boston. Or it might be a trial for a particular subtype of breast cancer, but not the one your loved one has. But just as likely, it might be enrolling in the Boston metro, but it is not open in your own institution. Suffice to say that “actively enrolling” is not synonymous with “available to you.” This in and of itself can be quite a frustrating thing — for both patients and their providers, especially when our own patients come in with these search lists, inquiring why don’t we offer such-and-so clinical trial.


But just as much as trial availability is an issue, the notion of experimental treatments continues to be a hindrance for some patients. Experimental implies unproven, and offering something unproven means that proven treatments are not available, suggesting that one is terminal. It is a discussion I have had at least once per week when discussing trial options. However, perhaps with the emergence of terms like “personalized therapy” and “immunotherapy,” the public consciousness is beginning to understand that there is promise to experimental treatments, and as many of our organizations and societies state, “for many, the best option for treatment is on a clinical trial.”

I sense that for women with gynecologic cancers, the promise of better treatments is something they have embraced as much as we have. Five years ago, when I offered a clinical trial, it was with a look of dread. Now, it seems that the option of a trial comes as frequently from my own patients as it does from me. For women who have recurred, they want to know what else is available. Perhaps it is because of the wide availability of medical information thanks to Dr. Google or the many ways social media educates and engages patients, my own patients appear keenly aware that for them, the “already available” therapies don’t offer much.

This was the case with Alex (named changed). She had been referred to me after a first recurrence of ovarian cancer — one that recurred six months after the end of carboplatin and paclitaxel. She understood her standard options and also knew that at six months, standard wasn’t very good. So, she came to see me specifically for clinical trials. I reviewed several options we had available, and we both chose one particular trial. I gave her the informed consent to read and made certain that she understood the experimental nature of the treatment and that I could not guarantee any outcome.

“Alex, when I speak of outcomes, what does that mean to you?” I asked.

She answered, “Doc, if you’re asking me if I hope I can be cured, the answer is yes. Ask anyone in my situation; they’d rather not have cancer. But, I also know that’s not realistic. So, what I hope for is quality of life and a long life — not longer, but long. I want to live and live well. I don’t want to be nauseous or bald, both of which I went through the first time, and if a non-chemotherapy clinical trial regimen can do that for me, then I am willing to try it.”

Looking back, we were both fortunate. She wanted to do something outside of standard of care, and I was in a place where we had the options. She hoped for the best, but realized that a clinical trial holds no guarantee for “success,” whichever way it is defined.

Clinical trials require so much, but in the end, they have to be available. I applaud the Foundation for Women’s Cancers for highlighting the importance of trials, and join them in their push for more trial options for not only my own patients, but for all patients with cancer. Wherever there is a patient asking for a trial opportunity, the worst response any of us can give is, “Sorry, there isn’t anything available.”

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