“From the outset, we recognized that outstanding care of MPN patients required not just hematologists (who would be their primary doctors) but also the participation of other specialists who had expertise in the complications of MPNs, such as cardiologists, neurologists, vascular surgeons, interventional radiologists, dermatologists, infectious disease specialists, gastroenterologists, and rheumatologists.”
– Andrew I. Schafer, MD
In honor of MPN Awareness Day 2023, MPNRF spoke with a pioneer in the research and treatment of MPNs, Andrew I. Schafer, MD, director of the Richard T. Silver, M.D. Myeloproliferative Neoplasm Center at Weill Cornell Medicine in New York City.
Dr. Schafer is renowned for his early commitment to treating MPN patients holistically, bringing multi-disciplinary specialists together in a coordinated care team that focuses on the entire patient. A leader in MPN research, he has authored more than 220 original articles; edited multiple books on hemostasis, thrombosis, and hematology; and is editor of “The Vanishing Physician-Scientist?” He is also a hematology/oncology clinician.
MPNRF is grateful for Dr. Schafer’s many contributions as a valued advisor for many years, as chair of the MPNRF Scientific Advisory Board, chair of the MPN Challenge and Thrive Initiative peer review committees, and co-chair of many past MPN Roundtable meetings. He is also a recent addition to the MPNRF Board of Directors.
MPNRF: Was there a particular reason you became interested in MPNs?
Dr. Schafer: As a 4th year medical student at Penn, I was undecided about what specialty to pursue. I signed up for an elective 4-week rotation in hematology under the tutelage of Dr. Frank Gardner, a preeminent hematologist and MPN expert. When he was in Boston, Dr. Gardner himself had worked with Dr. William Dameshek, a great hematologist who is widely credited today with developing the concept of what was then called “myeloproliferative disorders” (“MPD”), presciently recognizing the interrelatedness of polycythemia vera, essential thrombocythemia, and myelofibrosis and their clonal origin in a classic paper in BLOOD in 1951.
During that month seeing patients with blood disorders, much of which was focused on MPN patients who constituted the majority of Dr. Gardner’s practice, he introduced me to clinical research. When we came across patients with atypical presentations of their diseases, he pushed me to come up with a hypothesis of what could be the basic problem. Then, if the hypothesis involved blood cells, he would bring me into his laboratory, introduced me to his technicians who would teach me the experimental methods needed to isolate platelets or other blood cells from samples of whole blood from the patients who agreed, making sure that I wouldn’t ineptly spread radioactive isotopes around the lab. This allowed us to test certain functions of individual blood cells from MPN patients.
As a Hematology Fellow at Harvard, five years later, I eagerly picked up my interest in the MPNs and began a series of studies to understand how certain abnormalities in the platelets of MPN patients might be involved in their complications of thrombosis and bleeding.
During the first summer of my faculty appointment at Harvard, I went to Cape Cod for a lengthy vacation with my family. But it was not meant to be. Just prior to that, I had discovered a unique biochemical abnormality in the platelets of MPN patients that was not found in the platelets of healthy subjects. I now had to reproduce this defect in many more MPN patients, but the experiments demanded fresh blood samples from which I could isolate platelets. (Frozen samples to be analyzed later were not valid.) So, on many days when such MPN patients were available, I made the 3-hour drive from the Cape, consented the patients, worked late through the evening in the lab, and then drove back to the Cape often arriving after midnight, much to my wife’s chagrin. But the final results were deemed important and novel enough by the reviewers at the New England Journal of Medicine to merit their publication as a single-authored paper. I was to some extent lucky to have chosen a project that would end successfully, but that is not infrequently the case with starting physician-scientists, providing even more enthusiasm to work in the field.
From there on, I stayed active throughout my career in managing MPN patients (uninterrupted, even while carrying heavy administrative responsibilities) and doing further clinical research on the causes of their thrombotic complications.
MPNRF: Was there an individual who was a great influence in that decision?
Dr. Schafer: In addition to Dr. Gardner, in honor of whom I was able to raise funds to build a library at Penn after I became Chairman of the Department of Medicine there, I was fortunate to have some other great mentors who directly or indirectly reinforced and supported my interest in the MPNs. They included William Castle, the founder of modern Hematology and the discoverer of the cause and treatment of pernicious anemia, a previously fatal disease, with whom I had the privilege of working late in his career when his intellect was in no way dimmed. Also Aaron Marcus at Cornell (many years before I came here) who taught me how to study blood platelets. It was only after Dr. Marcus died that I found out how much he did to promote my career “behind my back.”
MPNRF: How do you view your leadership role at the Richard T. Silver MPN Center?
Dr. Schafer: I have been director of the Silver MPN Center since its inception 10 years ago. From the outset, my overarching goal for the Richard T. Silver MPN Center has been to help develop a cutting-edge comprehensive program for the diagnosis and treatment of MPN patients, first and foremost of course, seamlessly supported by both clinical and basic science research carried out mostly by physician-scientists who are themselves involved in patient care.
Also, from the outset, we recognized that outstanding care of MPN patients required not just hematologists (who would be their primary doctors) but also the participation of other specialists who had expertise in the complications of MPNs, such as cardiologists, neurologists, vascular surgeons, interventional radiologists, dermatologists, infectious disease specialists, gastroenterologists, and rheumatologists. This concept has been recently supported by the extraordinary findings of “clonal hematopoiesis of indeterminate potential” (“CHIP”), a potentially pre-neoplasticism stage of MPNs. CHIP investigators are discovering unexpected links between the MPNs with cardiovascular risk and possibly also inflammatory bowel disease and emphysema.
MPNRF: Describe your personal feelings about this role, given your connection to Dr. Silver as a colleague and presumably a mentor?
Dr. Schafer: It has been a personal privilege to serve as Director of the Center. In many ways I consider it to be a capstone for a 45-year career dedicated to the MPNs and their complications. Of course, it is Dr. Silver who is mostly responsible for the creation of this center that is most aptly named for him. I knew Dr. Silver for many years prior to my coming to Cornell in 2007, so I was delighted to link up with him at that time. He has indeed been a great mentor for me. Working with him, I now understand his “magic” as a brilliant, thoughtful and compassionate clinician as well as a pioneering clinical investigator who remains at the top of his field. For so many of us, he has been a treasure who has never lost his shine.
MPNRF: How would you describe the importance of mentoring? Is it particularly important with a group of rare diseases such as MPNs?
Dr. Schafer: In many ways, mentoring is the very essence and lifeblood of academic medicine. It is vital to all its aspects, ranging from many types of research, clinical practice, and education. Mentoring is not a function of aging. I have always said, especially to our junior faculty members and even trainees, that one is never too young to mentor nor old enough to be mentored. It is enormously gratifying, but it’s not always easy. An excellent career mentor, encompassing all aspects of academic medicine, has to play many roles vis-a-vis the mentee, not just being a personal teacher but sometimes needing to be an advisor, a role model, a coach, an advocate, or even a confidant. Regarding research mentoring specifically, especially in areas of rare diseases including the MPNs, it is more challenging only because the pool of potential mentors of this kind is still limited.
From a clinician’s perspective, how important are the contributions MPNRF is making today?
Dr. Schafer: MPNRF has had and continues to have, more than ever today, incalculable impact on the care of patients with MPNs. With a relatively modest budget for funding new research, applications for projects that have imminent clinical applicability are carefully and preferentially selected for support by a large group of our expert peer reviewers. The foundation has also been successful in identifying research project proposals that are not quite ready yet for clinical application but turn out to be studies of new pathways for drug development. As our founder, Bob Rosen, used to say, “we get a big bang for the buck.” The competition for MPNRF research funds has become enormous, with new ideas being proposed every year from leading MPN scientists and clinicians from around the world.