News

​Constellation Pharmaceuticals Provides Update on MANIFEST Clinical Trial of CPI-0610 in Myelofibrosis, Reviews 2018 Accomplishments, and Announces 2019 Data Disclosure Plans for Lead Programs

Allogeneic hematopoietic stem cell transplant remains the only curative treatment for myelofibrosis. Most post-transplantation events occur during the first 2 years and hence we aimed to analyze the outcome of 2-year disease-free survivors.

Although allogeneic hematopoietic cell transplantation (allo-HCT) is the only curative treatment for myelofibrosis (MF), data are limited on how molecular markers predict transplantation outcomes. We retrospectively evaluated transplantation outcomes of 110 consecutive MF patients who underwent allo-HCT with a fludarabine/melphalan (Flu/Mel) conditioning regimen at our center and assessed the impact of molecular markers on outcomes based on a 72-gene next-generation sequencing panel and Mutation-Enhanced International Prognostic Scoring System 70+ v2.0 (MIPSS70+ v2.0).

"Primary myelofibrosis (PMF) can be treated with allogenic stem cell transplant (which can cure the disease or prolong survival) or with drug therapy, splenectomy, and radiation. Determining the appropriate time for a transplant is an important objective in tailoring treatment to the individual patient, and currently, the mutation- and karyotype-enhanced prognostic scoring system (MIPSS70 + version 2.0), a risk model for overall survival, is one tool used to do so. A recent study sought to develop a complementary, robust predictive model for patients whose disease will transform into acute myeloid leukemia, a complication that is associated with a poor prognosis."

Myelofibrosis in myeloproliferative neoplasms (MPNs) with mutations such as JAK2V617F is an unfavorable sign for uncontrollable disease progression in the clinic and is complicated with osteosclerosis whose pathogenesis is largely unknown. Because several studies have revealed that macrophages are indispensable supporter for bone forming osteoblasts, we speculated that macrophages might play a significant role in the proliferation of collagen-producing myofibroblasts in marrow fibrotic tissues. Here, we show that myelofibrosis critically depends on macrophages whose differentiation is skewed by vitamin D receptor (VDR) signaling.

Background: Janus Kinase 2 (JAK2) plays pivotal roles in signaling by several cytokine receptors. The mutant JAK2 V617F is the most common molecular event associated with myeloproliferative neoplasms. Selective targeting of the mutant would be ideal for treating these pathologies by sparing essential JAK2 functions.

Constitutive JAK2 signaling is central to myeloproliferative neoplasm (MPN) pathogenesis and results in activation of STAT, PI3K/AKT, and MEK/ERK signaling. However, the therapeutic efficacy of current JAK2 inhibitors is limited.

Malignant bone marrow disease: New hope for MPN patients

"Myeloproliferative neoplasms (MPNs) are still difficult to treat. A team from Vetmeduni Vienna and the CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences/Medical University of Vienna has discovered a new therapeutic approach that could fundamentally change this situation, as evidenced by a study that was published recently in the academic journal Blood."

Are you ready to roll up your sleeves?

The MPN Research Foundation is doing our part to advocate for the MPN patient population. The needs of patients are first and foremost in our minds when we choose what researchers to support, have conversations with the FDA, and raise awareness about these rare blood cancers.

Rare Disease Day 2019 Kickoff

#BecomeAnAdvocate #ShowYourStripes #RareDiseaseDay #MPNRF

Promising Results Released from Clinical Trial for MF Patients

According to a press release published by Constellation Pharmaceuticals, the latest findings in the Manifest Clinical Trial of CPI-0610 in Myelofibrosis Patients was found to have a profound effect on trial participants.

MPNRF Releases 2019 Request for Proposals

The MPN Research Foundation is proud to announce the opening of our 2019 call for proposals for the MPN Challenge Grant Program.

Breaking news for ropeginterferon

The CHMP has given a positive opinion on the drug and recommended granting market authorization.

MPN Highlights from ASH 2018

Each year the American Society of Hematology (ASH) hosts their annual convention, where researchers, doctors, and organizations such as the MPN Research Foundation gather to discuss the ever-changing world of blood disorders and learn about the progress of research.

MPNRF Founder Bob Rosen Honored as MPN Hero

The MPN Heroes Celebration Event held annually at ASH is an opportunity to recognize the individuals and organizations who work hard on behalf of the MPN community and make a difference in the lives of patients living with PV, ET, and MF. Each year at this event, recipients of the MPN Heroes Recognition Program are honored based on those actions and traits that define them as heroes. This year, on November 30, 2018, the founder of the MPN Research Foundation, Bob Rosen, was honored as one of those heroes.