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. 

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.

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.