Hard to Fathom
By Robert G. Urban
We all have expectations. We have dreams. Each day we rise to a set of promises about the weather or 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 and cause-and-effect 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 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 – once a certain death sentence – are now living full and vibrant lives, with the right medication. For most patients infected with the hepatitis C virus (HCV) new treatments now 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 aberrant cells first emerge, followed by 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 or undergo a toxic concoction of chemotherapy? Will radiation be used 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, definititive answers.
We are now a “cancer patient.” Our early expectations for our lives seem so distant and we long for those days. We quickly forget the dreams and aspirations of yesterday. We just want to survive. Our journey into the healthcare system is rarely one that we find rewarding, and for a cancer patient it can often be overwellingly complex – too seldom feeling 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. At the recent annual meeting of the American Society of Clinical Oncologists (ASCO) in Chicago, the largest conference dedicated to cancer research in the world, new treatments and exciting combination therapies were revealed that allow more and more patients to live longer. And astonishingly, in an increasing percentage of cases, some of these cancer patients may even be “cured”.
Yet 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, the standard of care or perhaps just a placeabo? If I do get lucky enough to be randomized to the experimental treatment group will it be too toxic, or 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 to 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.
We were privileged to be visited by one such hero recently. In her case, acute lymphoblastic leukemia had been a life-changing diagnosis received 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 die, were just a few of the hard-to-fathom experiences she endured 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 for the study. She boarded a plane and at the clinical site promptly donated her T cells. All this on the hope that the CAR-T procedure would work and her modified cells could be reinfused.
After infusion, she and the 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, her vision began to return. A combination of her disease and earlier treatments had left her 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 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. But also of how remarkable new cancer treatment outcomes can be. 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 and finding a way. For now, she is “cured” and her story will pave the way for many, many more that follow. On behalf of her and so many like her, we are truly grateful, humbled and inspired.