The Cotter Report
The MPN Patient Symposium 2017
by Mary Cotter
A dark cloud swept in, turning the Big Apple into a gloomy Gotham City, bringing with it reminders of another attack on Manhattan in September 2001. A terror attack had once again slaughtered innocent folks, out and about, just enjoying the abnormally warm Halloween Eve weather while biking, walking, chatting with friends, along a path by the river in Manhattan on a beautiful fall afternoon. 8 dead. Mowed down by a mad man in a rental truck.
That was our back drop to the 9th International Patient Symposium on Myeloproliferative Neoplasms at the Griffis Faculty Club, Weill Cornell Medical College, Manhattan. Cancer Research & Treatment Fund, Inc (CR&T), brings patients, caregivers, and clinicians together biennially to meet and learn about the latest developments in treatments for the myeloproliferative diseases of essential thrombocytosis, polycythemia vera, and primary myelofibrosis. The three big MPN diseases. 200 hundred patients and caregivers alongside 50 or so medical researchers, who are working to prolong and improve the lives of those living with chronic disease and blood cancer.
This was my fourth MPN Symposium. I have attended since my first year of diagnosis in 2011, hoping to learn something that would help me make sense of these rare diseases that none of my family or friends have heard of and few in the medical community remembered from their training.
Bringing in medical professors and hematologists from around the country and world is a huge event, one that only those who have earned a high degree of respect and professional accomplishment can achieve. Dr. Richard Silver, clinician-researcher-educator, is just the person who can do this. The Richard T. Silver MD Myeloproliferative Neoplasm Center at Weill Cornell Medical College in New York City supports talented investigators who are conducting not only basic research, but cutting edge clinical studies that may one day bring hope, treatment, or a cure to the blood cancer community. The Belfer Research Laboratories are home to the cells, mice and equipment that might one day make a discovery that eradicates our orphan neoplasm.
The MPN Symposium follows a familiar agenda. A full day of ‘patient friendly’ lectures given by a roster of big names from the international community of hematology research. Each lecture, about 30 minutes long, is designed to give patients accurate information, yet recognizing most do not have a medical or scientific background, and come from diverse educational experiences.
Dr. Jerry Spivak of Johns Hopkins University School of Medicine; Dr. Alison Moliterno of Johns Hopkins University School of Medicine; Dr. Richard Silver of Weill Cornell Medical College; Dr. Carlos Besses of Hospital del Mar, Barcelona, Spain; Dr. Jason Gotlib of Stanford Cancer Institute, California; Dr. Ronald Hoffman of Tisch Cancer Institute of Mount Sinai, NYC; Dr. Ruben Mesa of UT Health San Antonio, Texas; Dr. Koen van Besien of Weill Cornell Medical College; Dr. Andrew Schafer of Weill Cornell Medical College, all gave presentations on aspects of the basic biology and natural history of MPNs, biomarkers and molecular abnormalities of MPNs, current medical management of ET-PV-MF, drugs in current use and those in clinical trials, what patients should expect from MPN treatment, and of course the ‘ultimate cure’ – stem cell transplantation. All this biology and science is, well frankly, overwhelming for the average patient and caregiver who might have a college biology course from 40 or 50 years ago as their frame of reference.
(Recognizing this, Dr. Spivak provided us with a hematologic glossary and a copy of a paper he had written on Myeloproliferative Neoplasms.) Mid-morning and afternoon snacks and a bagged lunch break up the presentations and allow participants an opportunity to meet fellow patients and digest not just food, but academic content as well. After a long day of lectures, participants are invited to a wine and cheese reception and tours of Belfer Research Laboratories where we meet face to face with bench scientists with infectious enthusiasm for their work.
CR&T will have videos of the presentations on its website at www.crt.org in a few weeks for those who want the experience of being there.
So, I asked myself why should I or any ‘lay person’ attend a medical conference on MPN disease? Yes, I have studied science in college and have degrees in the soft sciences of education and counseling, but hard science is, well, HARD! So why tax my MPN migraine-sleep deprived-brain with the sludgy, thick blood crawling through the vasculature? Why try to take notes when my fingers tingle and my feet cramp from hours of sitting in a crowded room? Why? Can’t I find something else to do?
The answer is ‘the people.’ The patients who ask themselves the same questions. The couple who travel from Florida interested in better treatment for her ET. Elderly (Mary) Jane with MF attending her first symposium trying to estimate how long she will live. The newly diagnosed woman with ET from South Carolina who is afraid, afraid to plan for the future without know what her future holds. The young woman with PV who just wants to become pregnant and have a baby. The couple from upstate New York who swear he found a cure for MF from ‘some guy on the internet’. The woman with MF and pulmonary hypertension who has attended each symposium since 2001. The yoga teacher with MF and cognitive symptoms who has discovered ‘clean living’.
It’s the people, the MPN community who come together to share, to learn, to let go of fears. To see if there are others living well despite chronic illness. To find hope that they can too.
I never could have imagined in this world of ‘hard science’ there would be such compassion and true caring for patients from doctors. A doctor, Dr. Mesa, who would acknowledge to his community of international peers that it was indeed a patient, Zhen Senyak – one he describes as a learned man, who formulated many of the ideas and theories this doctor would devote his precious time and research money to investigate. Or that Dr. Silver would ask me, a simple patient, to participate on his symposium advisory board.
There is a sense of community in the world of MPNs. Researchers, doctors, patients, caregivers are all part of an international family with a common goal – to make life better for MPN patients.
So, what did I learn this year at the MPN patient symposium?
Recent changes in WHO diagnostic criteria allow doctors to identify and treat patients who previously suffered due to misdiagnosis and delayed, inappropriate or no treatment.
Dr. Spivak and Dr. Moliterno are doing some amazing work in the genetics and epi-genetics (the micro environment within our body where genes live) of MPNs. The older we get, the more mutations we will acquire, but that does not mean those mutations will be expressed or result in additional disease. Men and women have different ways of expressing our MPNs! Our biology is important. PV can be aggressive or more benign. All MPNs evolve and change over time. They are not static.
Dr. Silver continues to remind us that an accurate diagnosis is essential. Hematologists must know what disease is present before treatment begins. When researchers speak of ‘early disease’ their definition of ‘early’ can mean years with an MPN, because MPNs are very slow growing leukemias. Emphasis on SLOW!!! This is why watch and wait can be an important and effective strategy for newly diagnosed patients. Doctors should carefully monitor a newly diagnosed patient to see what the disease will do before prescribing any treatment. ET can actually be PV, or MF, or just ET. Treatments come with their own set of complications and side effects. Phlebotomy results in iron deficiency, which has associated cognitive, heart, and muscle function issues. Nothing in our MPN world is without risk.
Jakafi can cause weight gain, ladies.
There is a long acting interferon coming soon to a specialty pharmacy near you. This interferon will allow the patient to self-inject once or twice a month to achieve a therapeutic response. Ropeginterferon alfa-2b is expected to reach the US market in about two years. Approval in Europe will occur first.
It was suggested that combination therapies may become our best bet for controlling disease. Interferon/ruxolitinib combinations look best at this moment. Despite notable problems with some medications that have caused the FDA to halt a few clinical trials due to safety concerns, there are many drugs in the pipeline under investigation.
Stem cell transplant is presently the only ‘cure’ for MF, but it comes with big risks. SCT doctors would like to see MPN patients sooner rather than later in their disease because survival is better for early MF patients, however, hematologists would like to take a medical approach to managing early MF and often refer only the sickest MF patients for SCT. There is no real agreement on which patients are candidates and when a patient should be referred. For that matter, SCT doctors really cannot predict the outcome of a stem cell transplant with any degree of accuracy. The ‘best’ candidates for SCT are sometimes the ones who do not have a successful result, while many patients who develop complications and infections after a SCT survive against the odds.
Researchers understand how important improving quality of life is to MPN patients. Dr. Ruben Mesa and Dr. Robyn Scherber are conducting research on diet and exercise, specifically yoga, as self-help approaches to maximizing quality of life. Dr. Mesa presented a chart showing how MPNs can affect employment status of patients and suggested there are alternatives to leaving one’s job, such as work place accommodations, that can help patients remain employed and in the work force.
The 10th International Patient Symposium on Myeloproliferative Neoplasms will occur in early November 2019. I can’t predict what the weather will be like in Gotham City or if Batman will protect Manhattan from terrorists. I can tell you there will be some amazing people with stories that sound familiar. People, who, like you, want to live without MPN worry. Fortunately, we have some good guys on our side.
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