Hard to Fathom - By Robert Urban
We all have expectations. We have dreams. Each day we rise to a set of promises about “the weather,” about our schedules – perhaps to go to work, school or maybe to just take a day to relax. The ratio and intensity of these expectations shift as we move through the chapters of our lives. Thankfully an increasing fraction of the world’s population has the resources and freedom to live in such a way. But for many, on some unplanned day, these daily rituals and expectations will be eclipsed by news of a life-threatening disease. In the mere moments required to utter and comprehend a “we are so sorry to have to tell you” diagnosis we are transformed from an individual, a customer, a voter, a parent, son or daughter into – “a patient.” Not just a patient, but one with a lethal disease.
The world beneath falls away; gravity seems to exist but without a source. Time, meaning, cause-and-effort all distort. The aperture of our world swiftly contracts. We turn to our doctors, to medicines, to our healthcare system and to each other to plead for help. We need answers, or at least recommendations. This is not a time that we can “be patient,” to us, nothing is more urgent.
For many lethal diagnoses, remarkable progress and even curative treatments have been discovered. Every year fewer and fewer individuals die from infectious agents. Even patients infected with HIV – what was once a certain death sentence – are now living nearly full and vibrant lives, with the right medication. For most patients infected with the hepatitis C virus (HCV) now new treatments exist and more than 90 percent are, in fact, cured.
But our deepest fears are fully manifested by cancer. A condition in which our own bodies seemingly turn against us. Just a few cells to begin, then a rogue horde on a mission to kill us. But why? What have we done? Is this somehow our own fault? Will we require a deforming surgery, will we have to undergo a toxic concoction of chemotherapy? Will they use radiation on us? If we can survive it, will it provide a cure, remission or perhaps nothing at all? Could the treatment itself cause other lasting debilitations or even spawn other cancers? We have endless questions yet find few, if any, meaningful answers.
We are now a “cancer patient.” Our early expectations seem so distant and we long for “those days.” We quickly forget the dreams and aspiration of yesterday. We want to just survive. Our journey into the healthcare system is rarely one that we find rewarding, personalized or in many ways even “caring.” We are just one of many, and now just one of the unlucky ones.
But even in cancer, impressive progress is being made. And behind each (and all new treatments) are the true heroes of modern medicine – the patients who participate in clinical trials. The bravery required, to step across this line into the true unknown and into randomized clinical trials is dizzying. Will I be eligible? will I get the experimental drug (or the placebo)? If I do get lucky enough to be randomized to the active treatment group will it be too toxic, or just maybe it will be remarkably effective and put my cancer into remission? Or better yet result in a cure? For these reasons and many more, only about 4 percent of cancer patients enter into clinical trials. But it is from that small group, who withstand these uncertainties and endure the lack of answers, that we all owe a great debt. It is this group of heroes that make new treatments possible for all others.
This week we were privileged to be visited by one such hero. In her case, acute lymphoblastic leukemia had been the life changing diagnosis in her late 20’s. Her journey included numerous highly toxic treatments and seven separate heartbreaking relapses. A loss of vision, bone necrosis in both knees and more than 200 separate spinal taps, before being sent to hospice where she “expected to just die,” were just a few of the hard-to-fathom things she experienced prior to finding a means to enroll in a clinical trial. For her, the bet was placed on immunotherapy and a cutting-edge new autologous cell-based treatment called chimeric antigen receptor – T cell or “CAR-T.”
Inspired by the remarkable story of Emily Whitehead, and to the surprise of her treatment team, she declined a perfectly matched bone marrow transplant in order to be eligible. She boarded a plane and at the clinical site she promptly donated her T cells. All this on the hope to be randomized to the group that got their modified cells reinfused (instead of placebo).
She and the blinded clinical team awaited a sign, looking for any clue if the treatment was helping. They did not have to wait too long. Within days, her modified T cells were working their “miracle.” The leukemic cancer cells in her blood were vanishing. And perhaps most surprising to all, even her vision began to return. A combination of her disease and earlier treatments had her left only able to see in black-and-white. But beyond everyone’s wildest dreams, as the modified T cells continued their specialized work, even her vision was restored and restored to full color!
Hers was a story of pure struggle. Of hopes restored only to be stripped away again and again. Of confusion, pain, and suffering but also one of passion and belief. A story that holds so many messages on what we (the healthcare system) need to focus on. A vivid reminder of each patient’s vulnerabilities and the centrality of keeping them (and their families) in the center of all that we aspire to do. But perhaps most remarkably, hers is a story of a “hero.” Of stepping into the void, of finding a way. For now, she is cured and her story will pave the way for many, many that follow and on behalf of many, we are truly grateful, humbled and inspired.