Video Presentations: San Antonio Patient/Caregiver Program 2/24/18

MPN Advocacy and Education International hosted its very first patient and caregiver program in San Antonio, Texas on February 24. Dr. Ruben Mesa, our scientific advisor and frequent speaker, initiated and assisted with this program’s agenda. Drs. Mesa, Michaelis, Verstovsek, Scherber, Mascarehnas, Gotlib, Palmer and Lyons presented to a packed audience. We will return to San Antonio in 2020.

Click the video screen below to view each presentation

Ann Brazeau, CEO, MPN Advocacy & Education International

Dr. Jason Gotlib, MD, MS, Stanford University Medical Center

Dr. Laura Michaelis, MD, Medical College of Wisconsin/Froedert Hospital


Dr. John Mascarenhas, MD, Icahn School of Medicine

Dr. Srdan Verstovsek, MD, PhD, MD Anderson Cancer Center


Dr. Jeanne Palmer, MD, Mayo Clinic


Panel Q & A


Dr. Robyn Scherber, MD, Mayo Clinic, Arizona


Dr. Ruben Mesa, MD, UT Health San Antonio Cancer Center

A Veteran’s Story: The Frustrations of Filing a Claim with the VA

By Wayne E.

MPN Patient and Vietnam Veteran Wayne E.

I served in the USAF Security Service, 6924th Security Squadron, stationed in Da Nang, Vietnam for one year (1970-1971) and was exposed to the deadly Agent Orange/Dioxin. In 2007, after a simple pre-op blood test, I was diagnosed with essential thrombocythemia (ET). Upon further study I was told I had an incurable, but manageable, blood cancer, coupled with a gene mutation (JAK2). The word cancer scared me. I had never heard of ET and I was at a loss for what to do. I didn’t know where to go next. After much reading about these potentially deadly diseases, I found out I was one of many Vietnam Veterans who had an MPN.

In 2011, I filed my first claim with the VA. Until this filing, I was unable to get any substantial information from my primary care physician (PHP) or my hematologist/oncologist, as to what may have caused or contributed to my ET. They knew virtually nothing about Agent Orange. I contacted the National Institutes of Health, The Centers for Disease Control, and as many online medical sites as possible, all ending with a bigger question mark. Nothing could be explained to satisfy my inquiry.

It was by chance that I connected with a most remarkable group, MPN Advocacy and Education International. I could never thank them enough for the compassion and the understanding they extended to me.

After my initial rejection from the VA, I filed three more times and each time I was denied because MPNs are not on the “presumptive” list of Agent Orange-related illnesses. The same message I kept getting was I needed “clinical rationale” to support my claims. My doctors have not been able to provide me with this needed information. I don’t know what to do today. I understand there are many Vietnam vets that have won their appeals and now get benefits, but there are many others who were not approved and just gave up. I don’t plan to give up.

To my fellow Vietnam Veterans who may be dealing with one of these MPNs, don’t give up. If you have been denied, file an appeal. There is hope, comfort, and assistance available. With the help of MPN Advocacy and Education International.

 Learn more about filing a claim with the VA

 Learn more about Veterans and MPNs

A Physician’s Perspective on the use of Social Media

By Dr. Naveen Pemmaraju, MD, MD Anderson Cancer Center

“The forum of social media is for information that is meant to be a general outline, not personalized therapy for an individual.”

For physicians who are balancing a heavy workload, how do you view the role of social media?

I think at least a working knowledge of social media usage is essential in today’s modern era of information. I know many hematologists and other physicians who get a good chunk of information for the day from social media such as Twitter, including myself. In five minutes, one can quickly glean important items for the day in the hematology/oncology-specific areas of interest. These posts can be “liked,” saving it for later reference when more time permits, serving as a personal bookmarking tool. Dr. Mike Thompson, my own Twitter mentor, published two really nice primers on this exact topic, and they provide a nice guide to getting yourself involved:

Twitter 101 and beyond: introduction to social media platforms available to practicing hematologist/oncologists.

Social Media and the Practicing Hematologist: Twitter 101 for the Busy Healthcare Provider.


What are your thoughts on the use of social media for patients? Pros and Cons? 

We live in an era of readily accessible information. Overall, this is a wonderful thing for our patients and providers alike. With this, it means we have information readily available, 24-7 at our fingertips. This includes online formats in the newer platforms such as social media.

For patients the pros include the ability to see up-to-the-minute information and announcements from key opinion leaders and professionals in the MPN field via twitter using #MPNSM (this is a grassroots twitter community started by me  along with other academic investigator); the access to online support groups such as on facebook for interaction, support, group discussions, information, comfort, and caregiver help; the ability to contribute one’s own valuable experiences to the public using blogs, websites, private facebook groups. (go to MPN Advocacy’s twitter page)

On the con side, one must understand that what you post comes great responsibility. Myself and others have written in the medical literature about the potential pitfalls and unknowns in this space. As with any form of media, we all need to be very careful and mindful of what we post (all is archived!), and that we treat others with the utmost respect. We must always remember to obey HIPAA and other privacy rules /laws. One must be vigilant about misinformation and be ready to identify and report spam, and any incorrect postings.

There are many different ways a person can participate on social media, from using it to gather news, information and opinions, to re-tweeting posts (twitter) or sharing (fb) items that resonate with an individual. Ultimately, one must be comfortable enough to post original content. Take your time, follow others on topics you care about, and assess and re-assess what sources are providing you with relevant, helpful information.

How do you and should you advise others on what they post on social media?

One rule I try to follow and advise others to follow is to remember to keep things accurate and keep things general. What I mean by this is always strive to give authentic, truthful accounts – whether it is about your impressions from a meeting, detailed scientific analysis, or experiences. Remember, all items on social media are archived-they are forever. Refrain from giving specific medical advice for specific situations. Instead, direct patients to make a formal appointment.

Dr. Pemmaraju has been a pioneer in the early stages of developing social media and its interface with hematology/oncology. We are pleased to have him offer his insights on the use of social media for both patients and medical professionals.
Dr. Pemmaraju will be joining us at the MPN Patient/Caregiver Program in Cleveland, learn more.

Vanderbilt University Hosts MPN Advocacy & Education International

From Left, Dr. Palmer, Ken Rosen, Drs. Stein, Savona & Gerds and Ann Brazeau

“It is always a great honor to spend time with patients. Thank you for taking the mantle MPN Advocacy & Education International.” ~Dr. Michael Savona, MD

On Friday, April 27th, MPN Advocacy & Education International presented their first educational program in Nashville, Tennessee. Dr. Michael Savona, Associate Professor of Medicine and Director of Hematology Research at the Vanderbilt University Medical Center, invited us to Vanderbilt to engage MPN patients from several cities and states in that region of the country. MPN specialists, including Dr. Savona, gave valuable updates and spent quality time with patients and caregivers answering questions. Dr. Gerds, Assistant Professor in Medicine (Hematology and Oncology) at the Cleveland Clinic Taussig Cancer Institute, shared in detail why patients may not be feeling so well and offered advice on ways to ease some of their symptoms. Dr. Brady Stein, Assistant Professor, Northwestern Feinberg School of Medicine, presented excellent findings on gender differences. Dr. Savona offered an overview of MPNs including symptom management, treatments options, and clinical trials.

Additionally, Dr. Jennifer Powers, Senior Manager of Oncology Disease at Walgreens, imparted great direction and advice from her perspective as a patient and a pharmacist. Dr. Powers has firsthand knowledge of the challenges MPN patients endure accessing and paying for treatments. Ken Rosen, a family therapist and MF patient, added the key ingredient missing in so much of the information given to patients-ways to quiet ones brain and develop peaceful coping skills. His patient story revealed a forty year commitment to meditation and a vegetarian diet that he believes diminishes his constitutional symptoms. Thank you to all the attendees who made the effort to be at this event and to the presenters for taking the time to participate and talk with the patients.

“I want to thank you for putting together such a wonderful program.  I came away with not only important information, but a sense of support from everyone there. It was a great experience!” ~Patient Attendee

NOTE: An MPN Patient Support Group is forming in Nashville that will include those in surrounding cities and states. For more information please contact Michele Riley,

A special thank you to our generous sponsors for making these programs possible:




Blood Cancers are Outsmarting the Immune System, But How?

Researchers have discovered how some of the blood cancers known as myeloproliferative neoplasms (MPNs) evade the immune system. Their findings reveal that a subset of MPNs might be susceptible to treatment with immune checkpoint inhibitors that specifically target the PD-1/PD-L1 pathway (a signaling network that normally functions as an “off switch” that blocks T cells from attacking other cell types), such as the currently marketed therapies Keytruda™ and Tecentriq™.

Certain cancers hijack the PD-1/PD-L1 pathway to prevent T cells from eradicating malignant cells, and scientists have remained uncertain if MPNs (a group of diseases where bone marrow makes too many red blood cells, white blood cells or platelets) avoided the immune system through similar mechanisms. Many patients with MPNs have abnormally activated versions of a signaling protein named JAK2, and Alessandro Prestipino et al. determined that the mutant molecule helps activate PD-L1 production – exposing a potentially promising therapeutic vulnerability. In a patient with the MPN polycythemia vera who had undergone a stem cell transplant and subsequently relapsed, treatment with an anti-PD-1 checkpoint inhibitor led to improved symptoms.

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Ruxolitinib therapy appears safe prior to allogeneic HSCT for MF

SALT LAKE CITY — Ruxolitinib treatment prior to allogeneic hematopoietic stem cell transplant appeared safe and aided in the prevention of cytokine release syndrome among patients with myelofibrosis, according to results of a phase 2 prospective study presented at the BMT Tandem Meetings.

“[The implications of the study] will let us know if patients with myelofibrosis who take ruxolitinib prior to undergoing stem cell transplant have better posttransplant outcomes,” Rachel B. Salit, MD, assistant member in the clinical research division at Fred Hutchinson Cancer Research Center and physician at Seattle Care Cancer Alliance, told HemOnc Today. “Almost all patients are starting ruxolitinib for myelofibrosis now and they are delaying transplant. There is likely an optimal time for them to move forward when they would do much better in terms of relapse and nonrelapse mortality.”

Allogeneic HSCT is the only potential cure for myelofibrosis; however, guidelines suggest the risk for transplant-related complications are acceptable only for transplant-eligible patients with intermediate-2 or high-risk disease based on the dynamic international predictive scoring system.

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View Myelofibrosis Clinical Trials

Expanding the Use of JAK Inhibitors and Relevant Clinical Trials

Novel Approaches Show Promise in Targeting JAK Pathway

Following the identification of the JAK kinase family in the late 1980s, the novel enzyme group was colloquially known as “just another kinase.”1,2 Since then, these tyrosine kinases have defied that reputation amid abundant evidence showing that they transmit a variety of signals into the cell with many biological consequences.

Dysregulation of the JAK pathway plays a role in the development of numerous tumor types; it is particularly central to the pathophysiology of myelofibrosis (MF) and has long been recognized as a potentially valuable therapeutic target in that malignancy. Ruxolitinib (Jakafi), the first JAK inhibitor to gain FDA approval, has become a centerpiece in the treatment of patients with MF. JAK inhibition has not proved effective in other tumor types, and thus far, no other JAK-targeting therapies have become available.

That picture, however, may be changing. A better understanding of the complexities of JAK signaling in normal and cancerous cells and the design of rational drug combinations, are poised to expand the use of JAK inhibitors in anticancer therapy for hematologic, and possibly solid, tumors. Ruxolitinib continues to be explored in multiple malignancies, and several promising novel agents are being evaluated in clinical trials (TABLE).

JAK Pathway

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Learn More About MPN Clinical Trials

Imago BioSciences Receives FDA Approval of IND Application for the Treatment of Myeloid Malignancies

SAN CARLOS, Calif., Feb. 1, 2018 /PRNewswire/ — Imago BioSciences, a clinical-stage pharmaceutical company developing novel therapies for hematological and inflammatory diseases, announced that the U.S. Food and Drug Administration (FDA) has accepted their Investigational New Drug (IND) application providing clearance to proceed with the clinical development of IMG-7289 in the U.S. The IND supports the company’s ongoing Phase 1/2 clinical trial of IMG-7289 for myelofibrosis (MF).

“There is a pressing need for novel approaches to the treatment of myeloproliferative disorders including myelofibrosis,” said Hugh Young Rienhoff, Jr. M.D., Imago’s Chief Executive Officer.  “We are pleased to have received FDA acceptance of our clinical trial protocol and look forward to the imminent expansion of this study into the United States.”

This Phase 1/2 open-label clinical trial is designed to assess the pharmacodynamics of IMG-7289, an oral inhibitor of the epigenetic enzyme lysine-specific demethylase 1 (LSD1) in high-risk myelofibrosis patients aged 18 or older ( Identifier NCT03136185).  Assessments include measuring changes in spleen volume, patient reported total symptom scores, mutant allele burden, inflammatory cytokines and bone marrow fibrosis over the course of the treatment period.  The trial commenced in Australia in 2017 and will add multiple sites in the United States in 2018.

This is the second clinical trial of IMG-7289 sponsored by Imago BioSciences, Inc.  The first, evaluating IMG-7289 for the treatment of high-risk acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), was initiated in 2016 ( Identifier NCT02842827) and continues to enroll patients.

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 Go to MPN Clinical Trials

Novel Agent Active in Refractory Polycythemia Vera

Dr. John Mascarenhas, MD is associate professor of medicine at Icahn School of Medicine at Mount Sinai

Idasanutlin demonstrated clinical activity among patients with refractory polycythemia vera, according to study results presented at ASH Annual Meeting and Exposition.

The agent also appeared well tolerated. Polycythemia vera and essential thrombocythemia are associated with considerable symptom burden. These chronic myeloproliferative neoplasms also are associated with a heightened risk for thrombosis and progression to myelofibrosis.

JAK2 inhibitors, hydroxyurea and interferon are commonly used to treat these conditions; however, hematopoietic stem cell-depleting therapies may offer an alternative option to improve outcomes.

MDM2, a negative regulator of P53, often is overexpressed in CD34-positive, p53 wild-type myeloproliferative neoplasm cells.

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Qiagen JAK2 Test Gets FDA Clearance for Additional Blood Cancer Types

NEW YORK (GenomeWeb) – Qiagen said after the close of the market on Tuesday that it received US Food and Drug Administration clearance for its Ipsogen JAK2 RGQ PCR Kit for additional use in the diagnosis of all myeloproliferative neoplasms (MPNs).

In March 2017 the FDA cleared the assay as a qualitative in vitro diagnostics test for the detection of the JAK2 V617F/G1849T allele in genomic DNA extracted from EDTA whole blood to aid in the diagnosis of the blood cancer polycythemia vera.

The new FDA clearance now covers two additional types of MPNs: essential thrombocythemia and primary myelofibrosis.

The assay runs on Qiagen’s Rotor-Gene Q MDx instrument, a component of the modular QiaSymphony family of laboratory automation products.

Qiagen said that it is the exclusive worldwide licensee of intellectual property covering the detection of the JAK2 V617F mutation for diagnostic purposes, and that it recently reached a settlement with an unspecified molecular diagnostics industry supplier in Europe, which agreed to stop selling its own JAK2 V617F test kit.

“We are eager to expand the use of our Ipsogen JAK2 assay, which is already available in Europe and other markets, for use in a wider range of patients in the US,” Thierry Bernard, senior vice president and head of Qiagen’s molecular diagnostics business area, said in a statement. The assay “makes it easier for hematologists and oncologists to follow recommended diagnostic testing algorithms and international guidelines for their patients suspected of having MPNs.”

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Learn more about MPN Clinical Trials