New Data Proves Role of Thrombosis in Patients with MPNs

New research published in Annals of Internal Medicine shows that the rate of arterial and venous thrombosis was significant in patients with myeloproliferative neoplasms (MPNs).

MPNs include a variety of blood disorders, like myelofibrosis, polycythemia vera, and essential thrombocytosis, and patients with them have been reported to be at increased risk for thrombotic events. Pharmacologic treatment can stabilize the blood counts, but they generally provide only partial symptom improvement. Until recently, no population-based study has estimated the excess risk with matched control participants.

Malin Hultcrantz, M.D., PhD and colleagues, in an effort to address the gap in knowledge, evaluated data from 9,429 patients – 46% of whom were male – diagnosed with MPNs who reported to the Swedish Cancer Register between 1987 and 2009, and matched them to 35,820 control participants and compared their rates of arterial and venous thrombosis. Median age of study participants was 72 years, and a mong them, 3,001 had polycythemia vera, 3,462 had essential thrombocythemia, 1,488 had PMF, and 1,478 had MPN unclassifiable.

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Incyte and Syros to collaborate in cancer R&D

  • Gene expression-focused biotech Syros Pharmaceuticals, Inc. has snagged a collaboration deal with biopharma Incyte Corp. to develop potential therapeutics to treat rare bone marrow disorders known as myeloproliferative neoplasms (MPNs).
  • In return for $20 million upfront in cash and R&D funding, and $10 million in stock, Syros will use its platform technology to find targets in MPNs. Incyte gets options to exclusive rights for therapies modulating up to seven validated targets discovered under the collaboration.
  • If Incyte exercises these options, Syros could see up to $54 million in option fees, and up to $115 million for products against each of the targets, as well as low-single digit royalties.

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Remembering Robert “Bob” Rosen

By Ann Brazeau, CEO, MPN Advocacy and Education International

In 2005, I met with the MPN Research Foundation for a potential job opportunity. Chairman Bob Rosen, Barbara Van Husen, President, and another Board member conducted the invigorating interview. The JAK2 Gene mutation had just been discovered and the energy and enthusiasm in the room was palpable. I was intrigued to say the least. A few months later I began my career there as Associate Director.

As Bob and I learned each other’s work habits and styles, we eventually hit a stride that took the Foundation to new and rewarding places. On any given day, conversations centered on MPN science, fundraising strategies, children, and of course, how we cooked our chicken the night before.

I remember the early days at the office on the river. The logo on the front door said Bridge Development, and I always thought my job was a bridge to another calling. I loved the absolute freedom to be creative, launch a project, or find new ways to increase visibility. Bob never refused to hear me out, look at the facts and inevitably say, “Go for it.” Our travels over the years to meet with donors and other potential partners always ended with a story to add to a growing list for a laugh or two later. I still laugh when I think about meetings he would request with me and Barbara, and we’d sit baffled that no one could quite remember why we were meeting.

We always used the expression, “The earth was moving under our feet.” That has certainly continued to be the case at the Foundation and in the greater MPN Community. Every day offered new hopes and new challenges and none were too mighty for a small group of dedicated people eager to get the job done.

Bob’s legacy will forever live on in the MPN Community. From the innovative, cutting edge, funded science he blessed, to the individual lives he touched, like mine. I would not be where I am without the years of experience at the Foundation. Bob could be fatherly when you needed it, and a big brother. Mostly, he was courageous, hard working, and unwavering in his pursuit for answers to MPN mysteries. Despite his own health issues, Bob always maintained his sense of humor. He will be greatly missed.

Click here to make a donation in his name

MPN Research Foundation Announces the Passing of its Chairman and Founder Robert Rosen

MPN Research Foundation announced today with great sadness the passing of its Chairman and Founder, Robert Rosen, age 74. Diagnosed with polycythemia vera in 1997, Rosen was shocked to discover that little research was being conducted on his condition and that there were no advocacy groups working to assist people with these rare blood cancers. Polycythemia vera (PV) is one of a cluster of blood cancers known as myeloproliferative neoplasms (MPNs), which includes PV, essential thrombocythemia (ET) and myelofibrosis (MF).

In 1999 Rosen, an established and highly successful Chicago businessman, formed the MPN Research Foundation to catalyze research and advance treatments for MPN patients. To date, Rosen’s MPN Research Foundation has awarded in excess of $12 million in MPN funding that has led to over 60 MPN-focused research projects, the advancement and development of new drug therapies, the publication of cutting edge research in numerous peer-reviewed scientific journals and the development of much needed patient advocacy initiatives, including effective outreach to the U.S. Food and Drug Administration, and highly beneficial education tools for the MPN community.

Andrew I Schafer, M.D., Director of the Richard T. Silver Center for Myeloproliferative Neoplasms (MPNs) at Weill Cornell Medical College, stated, “Because of Bob Rosen’s relentless tenacity, intense work ethic and limitless optimism, the MPNRF has been involved in every major scientific and medical advancement in the field of MPN research. Bob and the MPNRF have made immeasurable differences in the lives of people living with MPNs, the physicians who care for these patients, and the researchers who continue to search diligently for a cure.”

“There are no words to describe our sense of loss over Bob’s passing,” added Barbara Van Husen, MPNRF’s President and long-time friend of Rosen. “Our best and most significant tribute to Bob will be our continued efforts to accelerate and fund innovative research, create greater connectivity and resources for improving the lives of patients, and ultimately discover a cure for families living with an MPN. This was Bob’s mission and I’m confident that the MPNRF is in a strong position both professionally and financially to succeed with his goal.”

Bob’s daughter Molly Rosen Guy, also a director on the MPN Research Foundation board, said “Dad sought the only available curative treatment – a stem cell transplant – which is fraught with complications and often unsuccessful. His death is further evidence of the unmet need to find better ways to improve and extend the lives of patients with polycythemia vera and other blood cancers. Our work will continue.” A video produced by Molly about Bob’s diagnosis and her entry to the board can be seen here.

About the MPN Research Foundation
Founded by patients for patients, the MPN Research Foundation is a catalyst for research funding in pursuit of new treatments – and eventually a cure – for myeloproliferative neoplasms (MPNs). To date, the MPNRF has funded more than $12 million in MPN research.

Research funding is only part of what we do. The MPNRF is also dedicated to helping people living with MPNs change their prognosis by serving as a valuable source of education and resources in the MPN community.

Click here to make a donation in Bob Rosen’s name

Celgene to Acquire Impact Biomedicines, Adding Fedratinib to Its Pipeline of Novel Therapies for Hematologic Malignancies

  • Fedratinib is a highly selective JAK2 kinase inhibitor that is being evaluated for myelofibrosis and polycythemia vera
  • Fedratinib demonstrated clinical improvement in a phase III trial with treatment-naïve myelofibrosis patients and in a phase II trial with myelofibrosis patients resistant or intolerant to ruxolitinib
  • A New Drug Application (NDA) submission for fedratinib in myelofibrosis is planned for mid-2018

SUMMIT, N.J. & SAN DIEGO–(BUSINESS WIRE)–Celgene Corporation (NASDAQ:CELG) and Impact Biomedicines today announced the signing of a definitive agreement in which Celgene will acquire Impact Biomedicines, which is developing fedratinib for myelofibrosis and polycythemia vera. Under the terms of the agreement, Celgene will pay approximately $1.1 billion upfront and up to $1.25 billion in contingent payments based on regulatory approval milestones for myelofibrosis. Additional future payments for regulatory approvals in additional indications and sales-based milestones are also possible.

“Myelofibrosis is a disease with high unmet medical need as the number of patients who are ineligible for or become resistant to existing therapy continues to increase”

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Fedratinib, a highly selective JAK2 kinase inhibitor, was evaluated in 877 patients across 18 clinical trials. In a randomized, placebo-controlled, phase III pivotal trial (JAKARTA-1) for patients with treatment-naïve myelofibrosis, fedratinib demonstrated statistically significant improvements in the primary and secondary endpoints of splenic response and total symptom score, respectively. In an exploratory subgroup analysis, these improvements were observed regardless of a patient’s baseline platelet count.

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From Diagnosis to Transplant in Four Months

Cheryl Bonder is the New Jersey Support Group Coordinator

I was diagnosed with a rare unclassified MPN in May of 2013. Prior to my diagnosis I was being treated for what was believed to be Urticarial Vasculitis by the head of Dermatology at NYU. This is just a big name for inflamed hives. I had extensive blood work evaluated every three months and 8 biopsies over the course of 12 years. I was diligent with opinions from several leading academic medical centers in New York City and Baltimore. My reports were always within normal range. Aside from the discomfort of these hives that were itchy and painful I was feeling well. I was put on many anti-inflammatory medications and allergy medications to no avail in arresting the hives. It was unanimously concluded that I developed an auto-immune response from the antibiotic Levaquin.

Although I was asymptomatic in May of 2013, my blood tests revealed something more was going on and I was referred to a local Hematologist who told me that I had Leukemia. The world fell out from under me and I feverishly began a search for a Hematologist at a New York Academic Medical Center. I met with several specialists from different hospitals and decided on New York Presbyterian/Weill Cornell Medical Center.

A Hematology Pathologist report stated that the Levaquin had most probably caused the migration of the cells that led to my mutation. The report also stated that I didn’t have Urticarial Vasculitis, but Sweets Syndrome which is a rare auto immune skin disorder also stemming from the Levaquin. In June I met with the Director of Hematology and Leukemia Department and the Director of the Stem Cell Transplant Department. My case was certainly unusual, but nevertheless, my only chance for a cure was to undergo an Allogenic Stem Cell Transplant. Very quickly, donor matches were sought after. I did not have a familial match but fortunately several perfect matches were found from unrelated donors. On August 9th 2013 I was checked in to the hospital for my transplant.

I was fortunate that I engrafted well and moved along this journey with few complications. I was in the hospital for 2.5 weeks and then moved in a nearby housing facility so that I could be monitored daily at the hospital. I was able to return home after just one week of being in this facility. Aside from the emotional toll of all that was going on for my family and me, the physical impact of the low intensity chemo drugs and additional drugs that followed to protect my lungs, my liver, and to ward off any possible GVHD, my body was very weak. I had experienced a physical fatigue like I had never known. Taking a few steps caused major exhaustion. I felt like there were 50 pound weights on each of my legs. I came home with needing 31 pills a day. I needed to visit my medical team every week for one year. With the amazing support from my daughters and my friends and family, I pushed through every day. Many weeks passed until I began to feel a little stronger. I can’t stress enough that this is probably the most difficult journey you will endure. Be aware of all the ramifications of a transplant and the possible outcomes before you decide if this is right for you. Initially I was 100% my donor. Unfortunately, as months passed less and less of my donor’s cell remained in my periphery blood. I had relapsed.

I see my medical team every month now and feel grateful that my disease, although present is being managed. I live a full life able to do most of what I choose.

My background has been in medical education and Program Development. I decided during my recovery that I must use this experience to assist other families and patients that will need to face this process and a cancer journey.   I became multi- certified as a cancer/life coach working with families and transplant patients. I am also an MPN Support Group Facilitator in Northern/ Central New Jersey.

One piece of advice I would offer is to become as educated as possible about your challenges just enough to seek out the right medical team at an academic medical center that can assist in proper care. I would also suggest to try to resist the temptation to constantly google your disease. You can’t believe all that you read. You are seeking the advice from a specialist and letting go of your fears to trust is very hard but it will serve you well to do so. Seeking many opinions after treatment or ongoing treatment can be very frustrating when you receive many different thoughts on treatment options. My advice is to trust your judgement as much as possible and in the hope that your medical team you have chosen will do all that they can for you. If you feel that your doctor is against second opinions or discussing your case with other doctors, well then it’s time to seek out another medical team. You will be with these doctors for a very long time. You must feel comfortable and trusting in their thoughts. The ability to have healthy, good communication with your team is very important.

There is no denying the emotional toll this will take on your ability to move forward with your life. With the right assistance you can reclaim the joys you have known. I am living proof that life goes on. It may be different, but life goes on. Learning how to navigate the ever-changing landscapes of this disease will be challenging if you attempt to do it on your own. The right coach will assist in the design of the new road map.

Cheryl Corin-Bonder is a Certified Health & Wellness Coach specializing coaching cancer survivors, survivors of chronic disease, stem cell transplant recovery & survivorship, cancer care specialists, weight management, and life transition challenges. Learn more

ASH: Stemline Presents on Recent Clinical Trial Results

Stemline Therapeutics Presents Detailed SL-401 Pivotal Data in BPDCN at ASH and Kicks Off its BPDCN Awareness Campaign; Updated Results From Ongoing Trials in Additional Malignancies Also Presented

NEW YORK, Dec. 13, 2017 (GLOBE NEWSWIRE) — Stemline Therapeutics, Inc. (Nasdaq:STML), a clinical-stage biopharmaceutical company developing novel therapeutics for difficult to treat cancers, presented detailed data from its SL-401 pivotal trial in BPDCN, as well as results from other ongoing trials in additional indications, at the 2017 American Society of Hematology (ASH) Annual Meeting and Exposition, held in Atlanta, GA. Presentations are available on the Stemline website, www.stemline.com, under the Scientific Presentations tab…

SL-401: Phase 1/2 Trial in myeloproliferative neoplasms (MPN): chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF)

  • Key outcomes (ASH ’17 data)
    – SL-401 Phase 1/2 trial consists of a Stage 1 (lead-in, dose escalation) and Stage 2 (expansion); has enrolled 24 patients
    – No dose limiting toxicities (DLT) were identified and a maximum tolerated dose (MTD) was not reached. Most common TRAEs include hypoalbuminemia (33%), thrombocytopenia (33%), and fatigue (29%). Most common TRAEs (grade 3 or higher), include thrombocytopenia (24%) and anemia (19%)
    – Durable CR (14+ months) in CMML patient
    – 65% (11/17 evaluable) of CMML and MF patients had spleen reductions >25% (range 29% to 100%)
    – Durable SD in 4 patients (2 CMML, 2 MF) for 5+ to 8+ months. Three ongoing SD patients enrolled with baseline platelet counts <100,000, including 1 patient platelet count <50,000 are on treatment
  • Next Steps in MPN
    – Continue enrollment and patient follow-up
    – Favorable tolerability and preliminary signs of activity support both single agent and combination development strategies, including JAK-inhibitors and hypomethylating agents

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ASH: Sotatercept Boosts Hemoglobin in MPN-Associated Myelofibrosis

The investigational activin receptor IIA ligand trap sotatercept safely increases hemoglobin levels in patients with myeloproliferative neoplasm (MPN)-associated myelofibrosis, both when used alone and in combination with ruxolitinib (Jakafi).

A phase II investigator-initiated trial of sotatercept in this population showed responses in 10 of 28 evaluable patients, said Prithviraj Bose, MD, of updated findings presented at the 2017 ASH Annual Meeting.

Anemia is present in about one-third of patients with myelofibrosis at diagnosis and eventually develops in all patients. Only 20% to 30% of patients respond to current therapy, such as danazol and erythroid-stimulating agents.

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ASH: The Reveal Study Results for PV Patients Treated with Hydroxyurea

Examining the Treatment Patterns and Blood Counts Among Patients with Polycythemia Vera Treated with Hydroxyurea in the United States: An Analysis from the Reveal Study

Abstract

Introduction: Polycythemia vera (PV) is associated with erythrocytosis (with or without thrombocytosis/leukocytosis), increased risk of thrombosis, and symptoms including fatigue, early satiety, and abdominal discomfort. Hydroxyurea (HU) is a common cytoreductive strategy used to control blood counts and splenomegaly in patients with PV. Although blood counts in many patients with PV are effectively controlled with HU, a proportion of patients fail to achieve controlled blood counts. The European LeukemiaNet (ELN) has defined response criteria for blood count control as well as HU resistance and intolerance in patients with PV; 1 criterion for HU resistance includes a lack of blood count control at a minimum dose of ≥ 2 g/day HU for ≥ 3 months.

The REVEAL observational study (ClinicalTrials.gov, NCT02252159) is being conducted to describe contemporary demographics, burden of disease, clinical management, patient-reported outcomes, and healthcare resource utilization among patients with PV in the United States. The objective of this analysis is to describe HU treatment patterns, blood count control, and HU intolerance among patients enrolled in REVEAL.

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ASH: Pegylated Interferon Alfa-2a Active in Hydroxyurea-Resistant PV and ET

Treatment with pegylated interferon alfa-2a led to objective responses in about two-thirds of patients with hydroxyurea (Hydrea)-resistant/intolerant polycythemia vera (PV) or high-risk essential thrombocytopenia (ET), a phase II study of salvage therapy showed.

At 12 months, 69% of patients with ET and 60% of those with PV had achieved objective responses. The overall response rates (ORR) included complete responses (CRs) in 43% of the ET group and in 22% of the PV group.

No new safety or toxicity signals emerged from the MPD-RC 111 study, Abdulraheem Yacoub, MD, an associate professor of medicine at the University of Kansas Medical Center, reported at the 2017 ASH Annual Meeting.

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