“Never, never, never give up.”
— Winston Churchill
In high school, I had slips of paper with inspirational quotes taped to a cabinet in my room. Every day I would go up to this cabinet and read a quote by Winton Churchill, “Never, never, never give up.”
Three decades later, Churchill’s words were still with me. Especially when someone told me ‘No’.
Winston Churchill, 1941
I shouldn’t have been surprised by the ‘No’ that I received from a leading researcher of a breast cancer study to my query asking if my husband could be included in her trial. Three years ago, my husband was diagnosed with male breast cancer, a rare disease in men. Men were more likely to have advanced breast cancer when they were diagnosed and more likely to die than women from their disease. They also didn’t respond to some of the drugs in the same way as women.
“I found your breast cancer study on NCT Trials.gov. My husband has breast cancer. Is it possible to include my husband in your trial?” I wrote. I knew that her study excluded men with breast cancer but wanted to ask the question anyway. If for no other reason than to call her attention to the fact that a man wanted to participate in her study.
If my husband could participate in clinical trials for breast cancer, he might benefit from new drugs before they were officially approved. But the clinical trial door was often closed to him as many studies excluded men. As a physician and scientist, I wanted to understand the reasons why. And where the scientific rationale for excluding men was weak, I wanted to point it out and advocate for change.
“Unfortunately, he is excluded,” wrote the researcher. “Due to the small number of cases in men, we did not have sufficient power to include men for this trial.”
The reference to power meant ‘statistical power’, a term that referred to how likely it would be for the study to find a statistically significant effect. This was an educated guess based on assumptions of how likely a treatment was to cause an effect. She meant that they were unlikely to recruit enough men with breast cancer to study the impact of the treatment on men separately from women.
Was this true? I wondered. I sat down at the computer, opened a browser, and found my way to PubMed, an internet ‘warehouse’ of scientific manuscripts. I quickly found a scientific manuscript describing the study. Within three minutes of reading, I realized that this wouldn’t be a good study where I could make a case for including men. I should have done my homework before launching the email.
Rookie mistake, I told myself and smiled with sarcasm. After nearly three decades in science and receiving grants from the U.S. National Institutes of Health, I was far from a rookie. But this was definitely the wrong breast cancer study for me to begin with.
First, this study had nearly finished recruiting patients, so the horse was metaphorically out of the barn. It was too late to recruit men with breast cancer. Next time, I would choose a breast cancer clinical trial that had not yet begun recruitment so that my efforts might change their plan to include men. Recruitment of men for this study would have needed an aggressive recruitment strategy and be more expensive. With some irony, I recalled the current NIH policy that expense could not be used as an argument to exclude women or minorities from clinical trials.
Secondly, this study was focused on how exercise changes inflammation and whether it might improve breast cancer survival. This meant that participants had to show up to an exercise facility several times a week to have their workouts supervised by an exercise trainer. Would it be feasible to find enough men with breast cancer from the Boston area to enroll in the study and come in for exercise sessions several times per week? Doubtful.
Next time, I would choose a study that was enrolling at many sites across the U.S. where there was a better chance to enroll enough men with breast cancer. The bigger the cancer trial, the harder it would be to argue that men shouldn’t be included.
It was also a mistake to choose a clinical trial focusing on exercise. My time wasn’t limitless. It would be better to focus my efforts on large breast cancer trials involving new drugs, which might have the greatest impact on survival. If I could convince the leading scientists to change their enrollment criteria to include men, this would be a precedent that I could leverage again and again.
There was one final question that was still bugging me. I didn’t want to deal with it, because I knew the answer might complicate my singular hope to increase enrollment of male breast cancer patients in clinical trials.
The question was simple. How different is breast cancer in women and men? I imagined a tiny cancer cell in the breast and how it might respond to signals from the environment that differed between men and women, like hormone levels. Could these differences in hormones change the way a little breast cancer cell made the decision to stay dormant or divide? Or stay put in the breast or travel to new places in the body?
Yes, on some level, I replied to myself. Would this difference in how male and female breast cancer behave be so different that it might confuse the entire results of a drug trial if the results were analyzed as one big group? Probably not. But to be safe, the person analyzing the results could exclude the men to see if they got the same result in the women. Or, if there were enough men, they could analyze results in men only.
Wouldn’t this be a dream! The knowledge of which drugs may or may not work in my husband might make a difference in the choices his oncologist would make for a treatment regimen down the line.
In short, I had to start over. Next time, I would be selective in choosing a breast cancer clinical trial that excluded men. And had no business excluding men. I would look for a large breast cancer trial that enrolled from many sites across the country and were studying drugs that might change men’s survival. If only they could be included.
I had no idea where this was going, nor whether I could make a difference. It just felt like the right thing to do.
And I wasn’t giving up now.