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.

New digs and new grants

In June the MPD Foundation moved from a shared sublet at Willis (nee Sears) Tower to a new office on Michigan Avenue .  In addition to cheaper rent, this new space offers a better layout for Foundation staff and volunteers.  And with this new space comes a new opportunity to show it off.  As such, those in the Chicagoland area are invited to attend our House Warming on Thursday, September 30, 2010 at 5:30.  For more details or to RVSP contact Juliana at jgordon@mpdfoundation.org or 312-683-7249.

In addition to new space we are also looking forward to making decisions regarding the new grant proposals we received with 2010's request for proposals.  Between our New Investigator and Established Investigator programs we have received over 40 proposals.  A cursory review suggests some exciting opportunities to accelerate research into the myeloproliferative disorders: polycythemia vera, essential thrombocythemia and myelofibrosis.  In November the MPD Foundation's Scientific Advisory Board will work with an additional team of reviewers to score these proposals on the basis of scientific merit, relevance to MPDs, and potential for collaboration.  We make our funding decisions and announcements in February 2011. 

The MPD Foundation has always been focused on fostering collaboration among researchers as well as between the research community, industry and clinicians.  We made a specific plea in the 2010 Challenge Grant request for proposals (RFP) for proposals that highlighted collaboration.  It is our belief that research occurring in a community instead of a silo will facilitate faster breakthroughs that will help patients.  We now know that we are not alone in our belief as more patient-driven and research focused non-profits benefit from this model of operation.  It is our goal to make this new way of doing business work for MPD patients now. 

Not your mother's PV: A young patient's battle with Polycythemia Vera

Special guest post by PV patient Donna B.  

The polycythemia vera part of my story began in August 2006, when I was diagnosed.  I went to the hospital with abdominal pain and an enlarged spleen, and several tests (and weeks) later I started phlebotomy treatments.  I was 29 years old.  That’s not the whole story.  

In January 2002, I was diagnosed with a portal vein thrombosis – or as I tell most people “a blood clot in my liver.”  That diagnosis came after several weeks of abdominal pain and several wrong diagnoses ranging from gastritis to a pulled muscle.  Back then, the clot was attributed to Factor V Leiden, a genetic clotting disorder that, while still uncommon, is more common that PV.  The Factor V Leiden diagnosis effectively masked the fact that I also had PV until 2006, when I tested positive for JAK2.

Really, the story begins as early as summer 2000, when I began experiencing chronic headaches and fatigue.  Checked against the headache symptoms I dug up online, I found that my headaches combined features of tension headaches (pressure on the back and sides of my head), migraines (visual aura) and cluster headaches (stabbing pain over one eye).  I figured that all of these symptoms must be stress-induced, and kept them in check with over-the-counter pain relievers.

I’ve been on Coumadin since that clot was discovered in 2002, and while it was enough to keep my alive, it clearly wasn’t enough to keep me healthy.  I spent a lot of time in the hospital between 2003 and 2005, and have the scars to prove it.  

Phelobotomy ended up not being enough to keep me healthy, either.  Week after week, pint after pint of my “extra thick and chunky style” blood was drained, and yet my spleen was still causing me pain. As long as I sat perfectly still I was reasonably comfortable.  A slow shuffle was bearable for short distances.  The acts of sitting down and standing up were agony.  It was like being kicked in the stomach and kidney at the same time.  Lying down to sleep was not an option.

So, I began taking Hydroxyurea.  My doctor had some reservations about me taking it, because I am young for a PV patient, and long-term risks of taking the drug haven’t been completely ruled out.  I took it for a year with no side effects.  The pain in my spleen finally resolved, and my phlebotomy treatments were reduced to every couple of months.

Weekly Pegasys injections had been mentioned to me as a treatment back in 2006. Because it was seen as very promising and doesn’t carry the long-term risks, I wanted to try it right away. Unfortunately, my insurance wouldn’t pay for it.  It would, however, pay for thrice-weekly Interferon injections, which I began in October 2007.

Headaches.  Fevers.  Achy joints.  It was like I had the flu three nights a week.  I sometimes felt very tired the next day. My hair thinned.  I never got depressed, but I think the darker, more introspective side of my personality became more dominant.  My tastes changed, too: I stopped putting sugar in my coffee and switched from eating cereal for breakfast to eating yogurt.  

Why did I stay on it?  I didn’t mind the changes that much. What mattered was how I felt overall, and overall I felt pretty good. I adjusted to the symptoms, taking the shot just before bed and chasing it with a couple of Tylenol.  I started playing softball with some friends and found that when I was outside on the field I felt better than I had in years. 

I took Interferon for two years.  The symptoms eased up after the first year.  Now, I only felt flu-ish one night a week.  I got used to jabbing myself with a needle.  Then, last December, I stopped.  There had been a recall of the form of Interferon that I was taking, and I had to switch to a different form.  For whatever reason, my blood counts dropped.  My doctor thought I’d be better off without the Interferon for a few weeks. When I went in for my next blood test, my counts had all risen again.  It seemed clear that they would keep rising if left unchecked.  Coincidentally, a new study had just been released that extolled the benefits of Pegasys for PV patients.  I walked out of the clinic that day with a one-month supply.

I’ve been on it since then.  If there are side effects, I don’t notice them.  I no longer shed more hair than my dog.  I can take my weekly shot and leave the house without feeling sick.  The most inconvenient aspect is that I have to visit the doctor every couple of weeks for a blood test.  Pegasys has been very effective for me, as well.  I started on 90 miu a week, and now I’m down to 45 miu every other week.  Even at this level, my counts are at the low end of normal.  I think that, if I had never had PV then this is where my blood counts would naturally gravitate, but there’s no way to know for sure.  

I feel really good.  I play softball and go rollerblading and hiking.  I still eat yogurt for breakfast and never put sugar in my coffee.  I know that the test results are important, but the way I feel matters more to me than the numbers, especially since it was the way I felt that told me I was sick in the first place.

This is the first of many personal stories from patients.  Feel free to share your story in the comments section or contact me at mwoehrle@mpdfoundation.org to tell your own MPD story on this blog. 

MPD patients connect through social media

With increased access to and understanding of technology, people are finding their way online, searching out interests like cooking, basketball and even myelofibrosis.  Once assumed to be the domain of the young and frivolous, a new group has turned to the internet as a means of learning about and connecting to what is important to them.  This is especially true for patients with orphan diseases like polycythemia vera, essential thrombocythemia and myelofibrosis where the next closest patient may be hundreds of miles away.

The use of new technology like Twitter, Facebook and listservs has created a network previously unheard of in the health community.  Patients (and their families) now have access to a wealth of information and anecdotal first hand accounts of patient experiences with doctors, treatments and tips for living day to day with a chronic illness.  If there is a gap between the clinical advice of a physician and the need of a patient to hear directly from someone who has experienced an MPD, social media and online networks can bridge the gap for some patients. 

We've long linked to MPD-focused forums at www.mpdfoundation.org, including MPDChat, MPDNet and MPDSupport.  All offer a means to discuss and learn from other patients and occasionally doctors.  In addition we've branched out to other social media outlets such as Facebook where you can keep up with MPD news and interface with other patients.  It seems strange to say, but if you are someone with access to technology, it's never been a better time to have an MPD.

Meeting of the minds for MPD researchers

MPD Foundation grantees meet at ASH

The annual meeting of the American Society of Hematology takes place each year in early December.  For the past 2 years the MPD Foundation has exhibited, distributing our free patient brochure as well as speaking with hematologists who specialize in MPDs about treatments and patient numbers.  During these events we also take time to meet face to face with MPD researchers who have been awarded or applied for our grants.  This past December we had the opportunity to meet with our current Research Alliance and New Investigator grantees in New Orleans.  

The MPD Foundation believes collaboration is an essential ingredient for accelerating the rate of progress for medical research.  The academic research environment often presents barriers to sharing insights and open communication among researchers who may be in competition with potential collaborators.  However, at ASH we found fewer barriers to communication among our New Investigators as Drs. Delhommeau, Kralovics and Braun (Dr. Sipkins could not attend) communicated freely among one another, discussing research progress and findings.  

In 2011 the MPD Foundation will be awarding new grants and once again emphasizing collaboration along with science that will drive treatments forward.  With each series of grantees we learn more about what it takes to emphasize what MPD patients need from medical research and science.  Right now it appears that organic collaborations are most effective in creating real communication between scientists.  It is this sharing of information that we think will accelerate the understanding of MPDs and bring about better and more treatments. 

MPD Foundation distributes brochures through NORD's Medical Meetings initiative

The mission of the National Organization for Rare Disorders (NORD) is to assist individuals with rare disorders through advocacy, funding research and promoting networking among individuals and organizations interested in orphan disorders.  We at the MPD Foundation are always looking for ways to tell people the story of myeloproliferative disorders and reach more patients.  As such, in 2010 MPD Foundation became a member of NORD.  We feel being a part of this group will help us learn about the issues specific to orphan diseases and learn how other organizations operate and spread awareness. 

A benefit of membership in NORD is participation in their Medical Meetings initiative.  As part of this program NORD collects and distributes brochures of member organizations to pertinent meetings.  The MPD Foundation utilized this service by forwarding the MPD patient brochure to various meetings, including the American Society of Clinical Oncology (ASCO) meeting that recently took place in Chicago.  

It can be difficult to connect with doctors - general practitioners and hematologists - who may see patients with a MPD.  We consider this an opportunity to educate the physician community about MPDs in the hopes that they in turn pass this information on to patients who need it. 

Challenge Grants

Requests for proposals (RFP) for MPD Foundation's 2010 Challenge Grant program left the office in May, making their way into the hands of researchers actively involved with or thinking about getting into the world of myeloproliferative disorders. MPD Foundation grant programs have gone through several incarnations that illustrate how our priorities have changed as we learn more.

The MPD Research Alliance was our collaborative program aimed at accelerating development of new treatments following the 2005 discovery of the JAK2 mutation associated with MPDs. In 2008 a category of grant was added that recognized the need for funding of researchers new to MPDs. We are proud that the New Investigator grant program has been enormously successful.

In 2011 the MPD Foundation will award a new set of grants through our 2010 Challenge Grant program. The goal is to focus on both basic and translational research. This grant program is following up on the new and intriguing discoveries upon which we hope new treatments can be discovered.

This new grant program is consistent with our role as a patient-driven organization.  Our intention is to explore all options that could lead to discoveries that benefit patients with myelofibrosis, polycythemia vera and essential thrombocythemia.  In that sense we are ourselves constantly challenging the idea that a motivated group of individuals can't make a difference. We can't wait to see how the new group of MPD researchers will challenge themselves with these new grant proposals. 

MPD Patient Symposia Educate and Empower

By Ann Brazeau

The MPD Foundation believes that an educated patient is an empowered patient who can share what they know with their health care professionals to optimize treatment protocols and be able to fully participate in important decisions for their future care. To that end, in May 2010, the MPD Foundation hosted a patient education symposium in San Mateo, CA. Guest speakers included Drs. Ayalew Tefferi and Ruben Mesa from the Mayo Clinic, Dr. Jason Gotlib from Stanford, Dr. Ann Mullally from Harvard, Dr. David Leibowitz from the Palo Alto Cancer Center and author Joy Selak. For those who couldn't make it, a video of the event is available here.

Patients, family members and physicians are encouraged to attend these programs where they will hear from leading experts in the field of myeloproliferative disorders / neoplasms. Research updates, information on current clinical trials and day to day maintenance of these rare blood cancers are discussed. Open forums are provided for questions and one on one opportunities to speak with the researchers and clinicians. Attendees enjoy meeting other patients in a safe and warm environment.

This fall, the MPD Foundation will host a symposium in San Diego, California. Dr. Catriona Jamieson will be our keynote speaker and will be joined by other leading MPD researchers and clinicians. Dr. Jamieson, MD, PhD, is Assistant Professor of Medicine/Division of Hematology-Oncology and Director for Stem Cell Research at Moores Universtiy of California San Diego Cancer Center. Dr. Jamieson studies the mutant stem cells and progenitor cells in myeloproliferative disorders / neoplasms. Her studies have run the gamut from identifying a promising treatment in the laboratory, to opening and completing the first clinical trial, to target cancer stem cells in humans. Please continue to check our Events page for updates on the next MPD symposium.

www.mpdfoundation.org

Pricing Orphan Drugs

On a daily basis at least one of us at the MPD Foundation office is actively thinking about MPD treatments - mostly how to find more through our various projects. Whether planning a fundraiser, assisting a patient support group coordinator or looking at grant proposals, everything points back to creating an opportunity for MPD patients to live better and live longer. But recently the issue of access has taken up more space in our thoughts.

For many MPD patients, getting their diagnosis is a first step in a complicated path. There are many layers to get through in pursuit of good care, including diagnosis, finding the right physician, choosing among treatment options and dealing with insurance. It is in this last step that many MPD patients face frustration. There is still much to learn about Myeloproliferative Disorders; insurance companies and Medicare are dealing with a learning curve in how to classify PV, ET and MF and how to categorize treatments not to mention how to price them appropriately.

This scenario isn't specific to MPDs, as an article in American Health & Drug Benefits points out. Orphan disease treatments in general pose an issue for health coverage providers and consequently, patients. Insurers vary on how they interpret FDA rulings about the use of a drug. For a hypothetical example, if a drug is developed for sickle cell anemia specifically but research has found it is also useful for polycythemia vera, insurers don't necessarily provide coverage for that treatment and will not agree with each other on standards.

Our hope is that as research advances and more treatments are approved insurers and Medicare develop the flexibility to address orphan diseases like myeloproliferative disorders in a manner that works for patients. 

www.mpdfoundation.org

Introducing the MPD Foundation's official blog

The MPD Foundation is a decade old this year. We are currently waiting for proposals to arrive for the 2011 grant cycle and are looking forward to continuing to hold patient-education symposia, the first of which was held in May 2010. We continue to distribute MPD brochures to doctors and patients around the world and keep up to date with scientific discoveries pertinent to MPDs.

We look forward to sharing what we’re working on and thinking about here – the MPD Foundation’s official blog. We’re also interested in hearing about what is on your mind. Please feel free to use the comment section to let us know what you’d like to hear more about. 

www.mpdfoundation.org