I sign up, bleed, and sweat like clockwork. I know what’s at stake and what can be achieved when people volunteer to help move medicine forward. And I ask myself, as a scientist who develops drugs that aim to help people live healthier, longer: What right do I have to expect people to test my drugs if I won’t take the plunge myself?
I took part in my first clinical trial 30 years ago. My mother broke down the concept of a clinical trial to its most basic elements to make a complex idea digestible. This was not easy; I was only 6. “You come into the hospital and take a new kind of medicine and the doctors give you money.” I remember asking two questions right away and maybe only these two: how many shots, and how much money?
The first answer gave me pause. I don’t know what the exact number was, but knowing what I do now about trials, there was at least one blood draw per visit, one or two dozen stabs in all. Like any kindergartner, I had a fear of needles. (In my case, that would eventually fade from excessive exposure.) The chance to say no and skip a dozen blood draws was tempting.
The second answer made it a tough call. This trial would pay about $200 in early 1990s dollars, more money than I’d ever had. There was a Lego set — Ice Station Odyssey — that retailed for just shy of $60. A value I still remember decades later because of how long I spent staring at that box at Toys ‘R’ Us. Now it was being offered for just a bit of blood and (because of my specific ailment) sweat. I chose to participate in my first of many clinical trials.
That choice, or informed consent, is critical in clinical trials. Because I was a minor, my mother could have enrolled me regardless of my stance, but she believed in this concept enough to present the choice to a 6-year-old. Modern clinical trials are an extremely controlled environment, with regulations designed to protect trial participants from adverse events and ensure their anonymity, and to safeguard future patients from unsafe drugs. The sponsor (the organization developing the drug) and the Food and Drug Administration come together and agree on the safest possible approach to testing in humans. Then the sponsor has to find volunteers.
Any trial is a commitment for a patient, who needs to schedule around school, work, and family. Responsibilities can range from journaling about diet and taking pills to blood draws, biopsies, and multiweek IVs. And at the end, you could have been in the placebo group the whole time — so why bother?
My motivation, at first, was purely financial. Money for Legos, then for gas, late-night delivery, beer, and eventually gifts for my fiancée. Yet another motivation developed over time.
My indication — that is, my disease being treated — is a specific type of cystic fibrosis, a genetic disorder. Traditionally, CF has been characterized by (among other things) a gradual decrease in lung function as bacterial infections cause more damage, replacing healthy tissue with scar tissue. When I was diagnosed as a toddler, the prognosis for CF was overwhelmingly negative.
The only chance for survival past a depressingly young age was one of these trial drugs working. As I grew older, my criteria for selecting which trials I participated in shifted from the best compensation to ones I thought had the best chance of success. But that assessment isn’t straightforward. A new drug must go through three trial phases. The first looks at the drug’s safety and will often depend on healthy volunteers because they are easier to find and it’s easier to distinguish between potential side effects versus something caused by the progression of a disease. The next two phases measure efficacy. If the drug shows improvement over the standard of care, it’s approved, though chances are typically slim.
Presented with a panel of choices, each one validated by a host of experts in the field, I had to choose one I thought had the best chance to hold off the grim reaper. Picking the right drug is not easy for industry professionals, let alone a young patient. But not trying had only one outcome.
Like the drug-development industry itself, I failed repeatedly, but got better over time. I learned high school biochemistry while testing new antibiotics. Then studied in college while inhaling new nebulized compounds. I started working in the field, helping to develop new drugs while other new drugs flowed through the IV in my arm. But most of these failed, and none made the difference I was hoping for.
Two decades after my first trial, I enrolled in a trial for what would turn out to be my first breakthrough medication, Orkambi. In six months, I gained 50 pounds — which, surprisingly, was a good thing. I went from being emaciated to bulky, and I stopped constantly coughing. Having watched my transformation, co-workers bemoaned that they hadn’t bought stock in the company making the drug. Since that trial, Orkambi has been replaced with Trikafta as the standard of care for CF patients, adding as much as 40 years to life expectancy for some.
It’s been 10 years since that drug company’s breakthrough led to mine — I’m healthier now than most people my age. For me, trials are no longer a matter of survival and have long since stopped being about money. But I still enroll. Because without volunteers, no medicine can move forward; there can be no more stories like mine.
I look around me and I know, the science depends on all of us, and we’re all going to depend on it, sooner or later.
Max Leabo is a senior scientist of bioanalytics at Mediar Therapeutics in Boston. Send comments to firstname.lastname@example.org.