Clinical Trials for the Rarest: Worth the Head Scratching
Clinical trials are hard to come by when one has a rare disease, doubly so when that person has a rare form of that rare disease, and triply so when deemed ineligible due to severe health complications. That was me when I hit rock bottom in my cystic fibrosis fight only to find there were no available trials to give me a fighting chance, thanks to my complex medical history and ultra-rare mutations.
It was not exactly flattering to be told — via a cold, factual email — that there were no trials for my mutations because “there’s no profit there,” nor that I was seen as an uncontrollable variable in experimentation. Yes, these matters are realities of research: Money funds most science and experiments must remove unnecessary variables. But I’m a person with a story. I’m not a number or a footnote.
Without a terminal illness of their own, a clinician or researcher will never understand the suffering involved in being a patient held at arm’s length as a stranger, rather than being seen as a person frantically opening every door in desperate attempts to survive. They won’t know what it’s like to know that once options are exhausted, the only gamble available is to turn to alternative medicines and potentially further jeopardize health in the process. They won’t know what it’s like to be up at night, debating whether to continue risking fights or surrender silently to death.
All that people like me want is a fighting chance. The end goal isn’t to be a number or case study on a research paper — it’s to survive or at least die with hope in our hearts. Researchers rightfully focus on a future with new treatments, but many patients enroll in studies because they want a treatment now, even without assurances it will work. When suffering, even a placebo tastes hopeful.
There are no easy answers, no “well, forget the money” or “you know what, variables don’t really matter.” But “no easy answers” does not translate to “no answers.” Science is the art of finding difficult answers and that art should expand beyond the machinery and chemicals. Every patient is a person with a story, worthy of infinite head scratches and dried Expo markers.
Clinical trials encompass legislature, economics, storytelling, biology, chemistry, engineering. What would it look like to get the brightest of each field to put their heads together for a solution? What would it take to uphold scientific integrity while expanding trials and experimental treatments for the rarest and severest cases? It would take a lot of thinking, to start.
Four years ago I was saved by a bilateral lung transplant rather than through an experimental treatment. Once upon a time, something as strange and intimidating as a lung transplant seemed ludicrous to scientists. But ambitious minds got together, perhaps with head scratchers, Expo markers at the ready, and they performed beautiful science. See how far we’ve come; realize how far we have to go. Let’s get to work.
