By: Susan Gubar
Ongoing breakthroughs in cancer care involve personalized medicine, we are often informed. Because every malignancy is unique in terms of its genetics and genomics, one size (or protocol) cannot fit all.
A diagnosis of multiple myeloma, cancer of the plasma cells, motivated Dr. C. Anthony Blau of the University of Washington to come up with an innovative approach to his disease. He combined his research in hematology, his physician wife’s clinical expertise and crowdsourcing to develop an individualized treatment plan.
As a cancer patient whose life has been extended by an experimental drug, I was curious about Dr. Blau’s approach. By what methods can medical practices be tailor-made? And how do scientists find the ways and means to extend analyses of individual cases to a significant aggregate of patients?
Whereas patients conventionally receive treatment options from an oncologist, Dr. Blau’s strategy is to help patients learn about treatment decisions and consequences from other patients and from a number of experts whose ideas could then be explored with an oncologist.
Let’s begin Dr. Blau’s story with a romantic encounter over a pencil. In 2000, Dr. Blau, who goes by Tony, met a woman named Sibel (who had trained in Istanbul and then in Cleveland) at Seattle’s Fred Hutchinson Cancer Research Center, specifically on the stem cell transplant ward. He was an attending physician; she a senior fellow. His pencil rolled under one patient’s closed door; a few seconds later it rolled back out: Sibel and Tony fell in love. She became an oncologist, he a cancer researcher, and they lived so happily ever after that eventually they joined forces to investigate one of the most recalcitrant types of breast cancer.
With some dismay, Dr. Blau had observed that he was better equipped to analyze the condition of his lab mice than his wife was to comprehend the state of her patients with metastatic triple negative breast cancer, an incurable condition.
In the lab, he had the time and expertise to analyze the data of every mouse’s cancer and compare it to that of all the other mice he has studied. With human subjects, most oncologists don’t have data on the evolving tumors of each individual patient and of a cohort group. Dr. Blau wanted a way to characterize the effect of various treatments on malignancies in humans at a molecular level.
To address the problem, he helped create the Center for Cancer Innovation at the University of Washington in 2013 and the next year he helped design a new type of clinical trial.
It brought together clinicians and researchers with nurses, geneticists, specialists in information technology, computational biologists and statisticians, not only at one hospital but globally. Each breast cancer patient would be closely monitored. There would be multiple biopsies of tumors from multiple sites on each patient — many enrolled as a last-ditch effort to help themselves and future patients — as well as DNA and RNA sequencing to produce huge databases. Hypotheses about treatment were put on the cloud; experts around the world could provide input and feedback. Dr. Blau said he discovered that most scientists appreciated the opportunity to offer their arcane knowledge to aid people in dire straits.
After Dr. Blau’s myeloma was diagnosed in 2015, he decided to apply this approach to himself. “I had 22 biopsies of the tumor in my left pelvis, as well as bone marrow biopsies,” he said, “and we compared the genomic profile of the myeloma cells in my pelvic tumor with that of myeloma cells in my bone marrow.” In the process, he learned about a molecular feature that kicked him out of the most favorable prognostic category.
Because he still had a tiny percentage of myeloma cells in his bone marrow after an arduous autologous stem cell transplant, which uses the patient’s own cells, he rejected the advice of specialists and underwent an allogeneic stem cell transplantation, using cells from a genetically matched person. His donor, a brother and a Methodist minister, believes that he infused Dr. Blau with the Holy Spirit.
With (possibly related) zeal, Dr. Blau soon started All4Cure, a knowledge-sharing platform for clinicians, researchers and myeloma patients who register (without cost) to allow their medical records to be accessed. Once names are replaced by numbers, these files can be tapped to create “a personalized dashboard”: a graph depicting all treatments, tests and responses as well as tumor sequencing. Beneath the graph, a discussion panel enables participants to comment on the particular patient’s situation. To date, about 150 myeloma patients, 30 clinicians and 20 researchers have enrolled.
Personalized medicine depends upon information technology and knowledge sharing. Since every cancer contains a singular collection of mutations that can vary significantly across patients or over time within a single patient, computational analysts map how mutations interact within a specific individual, Dr. Blau explains. When a physician reviews data about a faraway patient, that doctor may draw upon her experience — for example, with the synergy of combining three drugs in a similar case — to propose an alternative approach.
Such global networking has the capacity to break down geographic and institutional barriers to communication. It also enables patients to report outcomes immediately so that protocols with deleterious side effects can be quickly changed. Patients can compare their treatment with those of others; researchers can broadcast breakthroughs. The growing database should help predict which regimens might be most effective for a particular person.
Of course maintaining and extending the platform could be expensive. When I asked Dr. Blau how his for-profit company was financed, he said that the initial investment came from a house he and his wife sold three years ago. He hopes that future revenue might come from pharmaceutical companies. All4Cure could accelerate the approval of investigational drugs and provide feedback on approved drugs.
Dr. Blau’s enthusiasm sparks my realization that the sort of networking available to myeloma patients through All4Cure would transform the lives of the women in my gynecological cancer support group. Most of us feel only tenuously connected to an oncologist who often does not specialize in ovarian or uterine disease.
It is paradoxical that personalized medicine involves crowdsourcing. You need not understand all that Dr. Blau knows about cancer to revel in his survival and to thrill at the promise of the maxim underscoring the work it makes possible: “All for one and one for all.”
To read this entire article by The New York Times, please click here.