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Changing a name to fit the science

The MPD Foundation has changed its name to MPN Research Foundation.  But don't worry; you'll still be able to reach us at our current web address and email addresses, at least for the foreseeable future.   

Why the change? Because in 2008 the World Health Organization (WHO) developed a new classification system for the Myeloproliferative Disorders (MPDs), and in the process changed the official designation to Myeloproliferative Neoplasms (MPNs).   They also added a handful of new blood diseases to the category, such as chronic neutrophilic leukemia, chronic eosinophilic leukemia, hypereosinophilic syndrome and the not-very helpful "MPNs, unclassifiable."

However, the three diseases that have always been our concern - polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF) - are still considered the "classic" Philadelphia chromosome-negative [Ph(1)] myeloproliferative neoplasms.  Our focus will remain on funding research to find a cure for these three diseases.

The word "neoplasm" actually defines our diseases far more precisely than "disorder" did. Neoplasia (Greek for "new growth") means the abnormal proliferation of cells - in our case, one or more lines of blood cells.  Many kinds of neoplasia result in lumps or tumors, either benign or malignant.  They are called neoplasms, too, and are a good deal more common than our blood cell neoplasms; so friends may think you're using the term "neoplasm" incorrectly.  You won't be.

Though we've changed our name we're still pursuing the same research goals as always.  With your continued support we are able to be sure that the understanding of MPNs is constantly improving, leading to steps forward in science, therapies, and of course, nomenclature.  This name change puts us in line with the latest thinking of authorities like WHO and we think it's a change that will be beneficial to the MPN community's understanding of their condition.  Please take a moment to read about our new 2011 research grants, and consider giving the fact that this research is funded primarily by MPN patients around the world.  They will be responsible for the next advancement in treatments. 

Spotlight on New Investigators

MPD Foundation's staff and board are constantly revisiting what it means to fund research that produces results.  We look at the various ways the work we fund affects patients - from available treatments to increasing their understanding of the scientific underpinnings of their disorder.

We also happen to be impacting the lives of those in the research community, including a group we've focused on through our last two grant cycles: new investigators.  

This is a group we define as being new to the field of MPN or simply new to independent research and desiring to work in MPN.  Although we've funded up and coming researchers in the past, since 2008 it has been a goal of ours to target this group for funding along with our grants to more experienced investigators.  Both types of investigators are working towards the goal of finding the causes of and potential cures for the myeloproliferative neoplasms.  

On that note we'd like to introduce some of our newest grant recipients:

Wei Tong, PhD, Children's Hospital of Pennsylvania

Dr. Tong will be working on a project titled "K63 Ubiquitination in JAK2 Signaling and Myeloproliferative Neoplasms".  The bottom line is that Dr. Tong is trying to determine how LNK affects JAK2 signaling.  JAK2 is basically an on-off switch whose malfunction is present in many MPNs.  LNK normally regulates the JAK2 switch to prevent myeloproliferation; mutated versions fail to turn off the signaling.  

Toshiaki Kawakami, MD PhD, La Jolla Institute for Allergy and Immunology.

Dr. Kawakami's project is titled "SPS Complex in MPD".  In this project he will be studying a series of genes whose absence in mice is known to cause tumors and myeloproliferative neoplasms.  His hypothesis is that the same thing happens in humans, and if correct, the discovery could lead directly to new therapeutic targets for MPN drug development.

Saghi Ghaffari, MD PhD, Mt. Sinai School of Medicine

Dr. Ghaffari's project is titled "Understanding Molecular Mechanisms of Regulation of Myeloproliferative Disorders in Mouse and Human".  With this project Dr. Ghaffari will be investigating a different signaling mechanism altogether whose failure may be responsible for myeloproliferation.  This is important because the JAK2 mutation is not present in all MPN patients; there must be at least one other mutation to account for those cases.


We will monitor their progress and report back as their work develops.  As one of the only organizations serving the needs of patients with myeloproliferative neoplasms we are committed to not only funding such projects but also to providing information and support for MPN patients.    

Vote to help NORD's rare disease registry

NORD is the National Organization for Rare Disorders.  They bring individuals and organizations together who have one important thing in common: having or working in rare disorders.  We've mentioned our participation in their medical meetings before.  Today we discovered a new opportunity to help them create a better registry of rare diseases that is accessible to patients.  

Pepsi's Challenge Refresh Program has selected NORD to be in the running to receive $50,000 to improve and expand their registry of diseases. NORD would like to make their existing registry more user friendly, including adopting language that is more comprehensible to patients and the lay public.  

We are pleased to say that helping NORD (and, by extension, people living with a rare disorder such as MPD / MPN) is free and easy.  Just register here and vote for NORD's project.  The 10 organizations who receive the most votes will each win $50,000. For the 1 in 10 people in the United States who have a rare disease (and more internationally), having access to information on their disease is vital in managing their treatment and symptoms. 

We at MPD Foundation believe it is important to partner with organizations who are working in the area of rare diseases.  There is potential for our struggles and strengths to overlap and help each of us understand our respective struggle.  Our relationship with NORD is just one of many we have established in the orphan and rare disease community. 

San Diego Symposium Brings the Experts to the Patients

Guest post by Ann Brazeau

Keynote speaker Dr. Catriona Jamieson addresses the crowd

The MPD Foundation hosted a patient educational symposium in San Diego on October 25^th at the UCSD Moores Cancer Center in La Jolla.  Each speaker commended the attendees for taking an active role in learning about their rare blood cancer and for supporting research and awareness efforts to ensure better treatments and care.  They agreed that patients are key advocates for changing the course of their future. 

There was a brief discussion about why the World Health Organization changed MPD (Myeloproliferative disorders) to MPN (Myeloproleferative neoplasms) and why it was important.  This change clearly specifies and classifies the disease as a blood cancer.   Data is collected and gathered appropriately when a patient is diagnosed.  This change will also ensure payment from insurers who were skeptical about the classification of the disease as a cancer.

Our keynote speaker, Dr. Catriona Jamieson, MD, PhD, is an Assistant Professor in the Medicine Hematologic Malignancies Program and Director of Stem Cell Research at UCSD. Dr. Jamieson found that a specific mutation in the JAK2 signaling molecule occurs at the stem cell level in polycythemia and changes cell fate decisions in primitive hematopoietic cells. She discussed the possibility of using combination therapies for MPN patients since one drug may not manage all the symptoms.  Dr. Jamieson has earned great respect in the field and is a committed clinician who sees numerous MPN patients at the center.

Drs. John Crispino and Ross Levine speak to patients

After the presentations patients formed groups specific to their MPN.  A clinician/researcher joined each group and answered questions from individuals.  Patients were able to share their stories and get feedback from the experts and fellow patients.

The MPD Foundation will continue to advance research and bring updates on MPD research to patients.  These sessions are held in order to empower patients and give them the tools to successfully manage their disease.  By assisting primary care physicians, hematologists/oncologists and pathologists in hearing cutting edge information, we better equip them in diagnosing and treating patients, advancing treatment options in their own way.

We extend a special thanks to our sponsors, Incyte Corporation, Sanofi-Aventis and Cytopia/YM Biosciences.

Click here to view a specific speaker or the entire presentation via web cast. 

Whirlwind of activity

Wow - is it mid-November already?!  It must be, since we finished hosting our final MPD patient symposium of the year and are starting to think about wrapping up 2010.  We've also finished our review of the grant applications we received for the RFP issued this May.  I say "we", but what I really mean is the panel of experts we bring in to assist us in evaluating proposals on their scientific merit.  

Grant reviewers reviewing proposals in Chicago

Last week we asked 14 experts who specialize in the myeloproliferative disorders to join with our existing Scientific Advisory Board to evaluate the proposals we received in the New Investigator and Established Investigator grant categories.  Though our funding decisions won't be announced until February, we can say that the proposals we received covered new ground in MPD research and we are looking forward excitedly to determining what we can fund in the 2011 grant cycle.       


In addition to this we've also just received our first printing of the Spanish translated MPD brochure.  This is the only informational, free brochure on the myeloproliferative disorders to be translated into Spanish.  After repeated requests from the international and domestic physician community we are so pleased to now have something for both English and Spanish speaking patients! 

Can Environmental Causes Be Behind Some Cases of Polycythemia Vera?

By Ann Brazeau

In August 2008, ATSDR and PADOH organized a meeting with a panel of experts in Philadelphia.  Medical researchers, environmental scientists, and public health professionals met to review the findings and recommend future studies. Four major research areas were identified: epidemiology, genetics/biomarkers, toxicology, and environmental analysis.
 
The PV cluster in Carbon, Luzerne and Schuylkill counties will require a great deal of  assessment of a host of possible environmental influences including hazardous waste sites, industrial emissions and waste, and naturally occurring radiation sources as well as possible genetic risk factors. Because the area was allocated a substantial amount of federal funding to study the specific causes, comprehensive research can be conducted that will not only benefit the local community but the entire MPD / MPN (myeloproliferative neoplasms) community.  

For this rural community in Pennsylvania, the increased benefits are evident with local physician awareness, the formation of a patient support group, access to PV patient clinical trials, a community JAK2 screening and follow-up study, testing of residential properties and nation-wide attention to an orphan disease that otherwise can go unnoticed.  The MPD Foundation has paid very close attention to this evolving story in Pennsylvania and  has supported and assisted the Centers for Disease Control, researchers, and the patients in those counties.


* This article appeared in the Fall 2010 edition of MPDUpdate.  To begin receiving this free newsletter click here.