Thankfullness

by Guest Blogger Lina http://linampn.com/

I am sure many of us have seen the daily Facebook posts during the month of November about all of the things our friends are thankful for. Don’t get me wrong. I’m happy that people are recognizing how blessed they are; that is a wonderful thing. My argument is that you should be thankful every day of the year, not just one month out of the year.

Now, I realize that sometimes it’s hard to be thankful. It is much easier to think of the negatives: the bad day at work, the argument with your spouse, the bills that need to be paid, or any of the other things that may be happening. The thing is, though, all of those negatives are what make the positives that much better. Without them, we would have no idea how good our lives really are. 

Often, someone who has just learned about my health situation  will say something like “Oh, I’m so sorry!” Well, I’m not. I’m not sorry about it at all. My ET has changed my life. Not all for the good, I’ll admit, but it has helped to make me who I am. It has made me a much bolder person than I was before I was diagnosed. It has helped me to see the brighter side of things more often, which has made me more able to see the things for which I am thankful. 

I am thankful for my family, the people who have known me my entire life, and have supported me through all of my choices (good, bad or otherwise). They were there for my diagnosis, and treatment, and all of the other excitement that came along with it. Without them I don’t know what I would have done. 

Lina and her husband

I am thankful for my husband. I was diagnosed just after we met in college, and even after 
seeing what I mess I was, he still stuck with me. I love you, M. 

I am thankful for my friends. Even though I’m not much fun sometimes, and can’t go out as often as I’d like, they’re still there for me.

I am thankful for my dog and writing-buddy, Tesla. Whenever I’m having a bad day, not feeling well, he’s there. No matter how long I’ve been gone he is always happy to see me, and ready to greet me with a wagging tail and a slobbery kiss. 
This is just the short list, but I am thankful for so many things in my life, and I am thankful for these things every single day.

Even if you can’t see it right now, there are things to be thankful for. My suggestion is to find the things in your every day life for which you are thankful. The girl at the coffee shop remembered your order. The lights were all green on your drive to work. The cop let you off with a warning. Whatever it may be, there are things in your life that are wonderful.

I would like to add that I am thankful for each and every one of you who may be reading this right now. I hope in some small way I have helped you to look at your life with a different perspective, and see the great things in it!

As always, be assertive. You are your own best advocate.

Until next time,
Lina

Fears: Disease Progression

We've started a new guest blogger feature. Please welcome Lina of linampn.com

 

When I was first diagnosed, I knew absolutely nothing about MPNs. And for a while…I was totally fine with that. But after a few months of not knowing what was going on with me, or my treatment, I decided to do some research. Some of what I learned was reassuring and comforting. Some of it much less so. (I should add that the majority of my research was done on the internet because, honestly…where else does a freshman in college do research…) The internet is a wonderful place to find information; but I advise using large amounts of caution – the internet can also be a scary place.

One of the first things I saw when I started researching was that the disease could change and become something more serious. What did they mean by serious: Myelofibrosis. Acute Myeloid Leukemia (AML). This terrified me. I didn’t really have hard evidence to tell me that I would progress to MF, or AML…but it was in the back of my mind that it was a definite thing. While the likelihood of it actually happening is pretty low, it is difficult not to focus on. Toward the beginning of my diagnosis I was always paranoid or thinking the worst, and I had to force myself to stop jumping to those conclusions.

While at the Chicagoland support group, one of the questions I asked Dr. Brady Stein was about disease progression. How likely is it really? He said that the risk of progression to MF from ET goes up to about 10% after 15 years, and for PV patients it was about 15% after 10 years. I felt a lot better after hearing him say that.

Even though 10% is pretty low, I was still curious about symptoms of disease progression, so I asked Dr. Ruben Mesa, of the Mayo Clinic in Scottsdale for some information. He very kindly agreed to help me. The following information is paraphrased from an e-mail I received from Dr. Mesa.

For patients with advanced MPNs, there tend to be many difficulties and symptoms which indicate progression. For example, a drop in blood counts; either bringing blood counts to relatively normal levels where before they required intervention thru phlebotomy or drugs, or bringing them so low they require transfusion. An enlargement of the spleen is often seen, which causes pain and discomfort. The bone marrow also becomes scarred.

There is still no definitive reason behind progression in MPNs. It is thought that JAK2 inhibitors might help to delay disease progression, and also that drugs like Interferon has been shown to help to delay it. Aside from that though, there is not a clear reason/form of prevention.

Another way patients progress is typically from MF to Acute Myeloid Leukemia. (it is less likely, but it is also theoretically possible to see progression from ET or PV to AML).

As a patient I still fear that progression from ET to MF one day, or even possibly AML…but I try really hard not to let it nag at me. When something strange happens (A new or different symptom, for example) I do wonder…but I don’t automatically assume the worst anymore the way I used to.

I know I’m repeating myself when I say this; but I strongly recommend being an informed patient, and a partner in your own health care. Things are much less frightening when you know what they mean, and it is much easier to take care of yourself when you know what is going on.

You are your own best advocate. If you do not take care of yourself, who will?

Until next time,
Lina

Clinical trials news for PV and ET patients

Last week two companies reported news on their clinical trials for myeloproliferative neoplasms:

• AOP Orphan Pharmaceuticals announced that Phase III trials were going well for their study on Pegasys in PV patients. This study is being conducted in Europe and is currently recruiting.
  
• Promedior has initiated a new Phase II clinical trial for their compound, PRM-151, in people with myelofibrosis.

Ongoing clinical trials for PV, ET, and MF can be found at clinicaltrials.gov as well as the MPN Research Foundation's site. Participating in a clinical trial should be considered carefully and discussed with your physician and family. 

This September we're putting patients in the driver's seat

Last September, the U.S. House of representatives declared that September is Blood Cancer Awareness Month. In 2012 our focus was to help educate patients about the diseases and the resources available.  

 

This year is all about YOU as patients and patient-supporters, and the role you can take in combatting PV, ET, and MF.

 

This month we'll be starting a conversation on ways you can educate yourself and Take Action, and consequently help raise awareness about MPNs.

 

Starting next week, look for weekly emails from us on different components of the MPN world. Hopefully you'll pass them on to your friends and family. MPNs affect almost 300,000 people across the U.S.

 

Follow along on Twitter and Facebook with the hashtag #mpnbcam throughout the month of September.

 

You as patients know what it is to live with an MPN, and you know what a cure would mean. Play a part in informing your community that though rare, MPNs are worthy of attention and action.

Get Involved: To participate in one of September's events and for resources click here. 

 

Join us September 17th for a Twitter Chat!

On Tuesday, September 17^th we'll be hosting an #mpnbcam Tweet-Chat. Follow us at @MPN_RF then follow along with the #mpnbcam hashtag. The conversation starts at 7pm EST. Foundation chairman and PV patient Bob Rosen will be participating as will MPN blogger Lina. We hope to "tweet" you there!

Polycythemia Vera Clinical Trials for 2013 - an update by Dr. Mesa

Past MPN Research Foundation grantee Dr. Ruben Mesa (Mayo Clinic) has a new video out on polycythemia vera clinical trials. In it he discusses the clinical trials for PV that are current, including some recruiting patients. Watch the video then check out our clinical trials page to find out more about specific trials. 

Picture update from the Trek for a Cure 2013

Celebrating their journey during the Trek for a Cure 2013

Annette DeBow and her walking crew were joined by Raquel Nunez of the MPN Research Foundation for a leg of their Trek over the weekend. We'll have an update from Raquel and Annette soon. For now, Raquel snapped this picture of the trekkers with an MPN Research Foundation banner. Thanks to all the trekkers for their hard, long journey to raise funds for MPN research!

The power of patients (and an increase in the number of people with PV, ET, and MF)

Recently we shared the new prevalence data which put the number of people in the U.S. with PV, ET, and MF at nearly 300,000. That's a lot more people with MPN than we thought we were dealing with. 

This got us thinking about what people with PV, MF, and ET have done to drive MPN research to a better place, if not quite where we need to be. We also wanted a graphic way to display the new numbers. To illustrate what we think of when we think of "the power of patients" we put together this infographic:

New MPN quarterly journal launched by MPN Forum

Recently the MPN Forum launched a new quarterly journal focused on scientific medical advances. The idea behind the MPN Forum Quarterly Journal (MQJ) is to provide hematologists and scientists a professional forum to talk with other physicians and interested patients in plain English about diagnosis and treatment of myeloproliferative neoplasms.

The first issue features a roundtable discussion by MPNclinic doctors on "How I diagnose MPNs". Participants include Drs. Richard Silver, Ruben Mesa, Hans Hasselbach, Jason Gotlib, Ross Levine and Srdan Verstovsek as well as a retrospective on PV and Damanshek by Dr. J.J. Michaelis. 

The MPN Forum Quarterly Journal was created to serve family practice physicians, internists, clinicians, healthcare providers and the MPN patients and caregivers who want to explore myeloproliferative neoplasm etiology, diagnosis and therapeutic intervention. 

MQJ is produced and distributed by MPN Forum Magazine, www.mpnforum.com

MF Challenge grants for 2013 announced

Today I got to do one of my favorite tasks: call grantees to let them know they have been awarded funds for their research. For the second consecutive year, the MPN Research Foundation and the Leukemia & Lymphoma Society have collaborated to review and award research projects proposing new ideas of how to stop or reverse fibrosis. Winners of these one year concept grants focusing on fibrosis and myelofibrosis are:

• John Varga and Jonathan Licht (Northwestern University)  
• Golam Mohi (State University of New York)  
• Emmanuele Passegue (University of California at San Francisco )  
• Xiaoli Wang (Mt. Sinai School of Medicine)

As in the first round of MF Challenge grants, each researcher will receive $100,000 over one year to test his/her hypothesis for these grants, the goal of which is to provide a proof of concept for ideas on how to stop or reverse fibrosis. The grant review was conducted by a multi-disciplinary group overseen by the MPN Research Foundation's Scientific Advisory Board Chair Dr. Andrew Schafer from Weil Cornell. 

The first round of MF Challenge concept grants were funded in July 2012. Progress on these four projects will be reviewed in late 2013. Projects with potential to go further will be candidates for longer term funding. A "concept grant" is used to identify innovative and novel approaches to an issue.

Fibrosis is the formation of excessive fibrous connective tissue in place of healthy tissue. The disease myelofibrosis (one of the myeloproliferative neoplasms) is characterized by fibrosis in the bone marrow.  Prognosis for sufferers of myelofibrosis varies. A small proportion of MF patients can transform to acute myeloid leukemia (AML), a type of blood and bone marrow cancer that progresses rapidly.

The potential of the MF Challenge is its ability to stimulate research in areas that are currently untested in fibrosis and myelofibrosis, offering hope to patients struggling with a relative lack of treatment options and understanding about their disease.  More information about the MF Challenge can be found at www.mfchallenge.org.

I am so proud of the work we've done and the progress we've made in MPN science. But there is still a long way to go. After the grants are completed it's up to us and the MPN community to decide how to turn these projects into life saving and improving medicine for patients. I look forward to sharing the progress of these and our other projects with the patient community very soon. 

Sincerely,

  

Barbara Van Husen President, MPN Research Foundation 

   

MPN news from ASCO

The American Society of Clinical Oncologist just wrapped up its annual meeting in Chicago last week. The meeting brought together over 25,000 oncology professionals from all disciplines. Added to the fertile mix were drug companies, advocacy folks and journalists striving to understand what’s new in cancer research and treatment.

The MPN Research Foundation met with about 9 different pharmaceutical companies working on drugs in our space. The most significant major news relating to us is the announcement by Incyte that Jakafi is showing a clear positive impact on fibrosis in many MF patients. Bone marrow biopsies from patients who have been on this drug for up to 48 months have in many cases shown a reversal of progression, and in other cases a stabilization of fibrosis. Read more about this data here.

Our field remains crowded with pharmaceutical firms vying to provide improved versions of JAK inhibitors. Other more novel mechanisms of action are either in early stage clinical trials or headed towards trials later this year. Sanofi hopes to apply for FDA approval later this year for its JAK 2 inhibiting drug, and Promedior hopes to start trials this year for a novel drug that has shown efficacy in halting and perhaps reversing the fibrosis process in Pulmonary fibrosis. There is reason to believe, but it has not been proven, that it will be effective in bone marrow fibrosis.

The next conference where major new MPN news might be announced will be at ASH, in December 2013.

Benefit concert for Trek for a Cure to take place June 15th

The Trek for a Cure is right around the corner, and to celebrate the effort and raise funds, there will be a benefit concert on June 15th in Woodland, CA featuring violinist Emily Wells. The proceeds will benefit research to find a cure for Myeloproliferative neoplasms (MPNs). Money raised will go to the MPN Research Foundation. Annette De Bow is living with the disease and has dedicated herself to raising money for this foundation. As part of her fundraising efforts, she will be hiking the Tahoe to Yosemite Trail for 3 weeks this summer! For more information see http://trekforacure.com and http://www.mpnresearchfoundation.org.

Gearing up for new round of MF Challenge grants

The applications for the second found of MF Challenge funding will be reviewed on Friday, May 31st in Chicago. In addition to members of the Foundation's Scientific Advisory Board, thought leaders from fibrosis will be joining the review panel as the proposals are judged. In addition to scientific merit, the applications will be evaluated based on innovation and likelihood of clinical translation. The proposals will be scored on an NIH scale. Following the meeting, the Leukemia & Lymphoma Society and MPN Research Foundaiton will jointly decide which projects can be funded during this cycle.

The first round of MF Challenge grants will be evaluated by November 2013.

Chicago MPN Roundtable publishes first article

We've mentioned before about our low-cost, high-quality project titled the MPN Chicago Roundtable. Basically, it is a group of clinicians and researchers representing some of the great Chicagoland hospitals who gather around the MPN Research Foundation conference table to discuss what they're working on individually and what they can do jointly to accelerate and improve treatment options for people living with PV, ET, and MF. 

We are pleased to announce that this group has published it's first joint piece of research titled "Age-related differences in disease characteristics and clinical outcomes in polycythemia vera." The colaborators from the MPN Chicago Roundtable include Brady Stein (Northwestern), Jamile Shammo (Rush), Damiano Rondelli (University of Illinois at Chicago), Toyosi Odenike (University of Chicago) and Laura Michaelis (Loyola). We are so proud that the Foundation has a role to play in helping to pull this group together, all for the cost of a few pizzas, and look forward to what else comes from their meeting of the minds. 

MPN on the web

Lina, a young woman living with Essential Thrombocythemia, blogs about changing her medication and dealing with migraines: http://linampn.wordpress.com/2013/05/11/an-overdue-update/#more-202

MPN Forum is talking about the "silent gene" http://mpnforum.com/the-silent-gene/

Starting back up that trail

Annette DeBow will be contributing a series of gues blog posts leading up to her Trek for a Cure

Annette speaking at a Trail Mixer event

This disease has changed my life in so many ways, and most of the changes have been unexpected.

When I was diagnosed in 2008, I had a 6 month old daughter and I was already dealing with so many changes around being a new mother, I couldn’t even wrap my head around what it meant to be diagnosed with a rare disease. The first doctor who suspected that something was wrong was my family doctor. I had taken my daughter to her 6 month check up and the doctor instantly knew that something was wrong, she said, “You look thin and tired- you need a CBC.” Later that week I received a call from her, and while she has had a patient with Polycythemia Vera in the past, could not remember the full name of the MPN and told me I had, “poly something and I’ll recheck your CBC.” Well, it was confirmed and with a HCT of 65, I was off to get phlebotomized –weekly and then every other week and then once a month until my HCT was back in the low 40s.

I shared the news of my diagnosis with family members and a few friends but that was it. I did not want others to know I was ill because I have always been an incredibly active and fit person. I was the woman who mountain biked the black diamond trails at ski resorts every summer. Learning how to interface with the medical community was something completely new and at times frustrating. I also learned that there was not a lot of research going on for rare diseases, like MNPs. It wasn’t until I decided to raise money for the MPN Research Foundation by hiking the John Muir Trail in 2010, that I really experienced another change. I learned about the power of advocacy. When I asked my community to help raise money for research, folks responded from all over the world to give. Wow, what a great feeling of support! That support, which led to more energy and enthusiasm, has me heading to the hills again to trek the Sierras. I hope I can share more of my experiences as I prepare for Trek for a Cure 2013.

Dr. Mesa on Late-Stage Trials in Myelofibrosis

Dr. Mesa recently filmed a video for OncLive in which he describes some Phase 3 clinical trials ongoing for Myelofibrosis. 

Bealers' Ride for a Cure

By guest blogger Jen Bealer

In 2001, we lost our Grandma to a Myeloproliferative Neoplasm (MPN), a rare form of blood cancer that had no effective form of treatment at the time. Nearly five years ago, our mom had a blood draw showing her platelet count was over 4 times higher than normal, and she has since been diagnosed with a similar form of blood cancer.

Between 2001 and 2013, a lot has been learned about MPN's, largely in part due to the MPN Research Foundation, which is the only independent organization focused exclusively on MPN research.

Our grandma only had one option for treatment, which had the side effect of giving her Leukemia. Now, ten years later, my mom is being successfully treated. We owe a great deal to this foundation, and try to give back to them every way we can.

My brother and I, along with our awesome friends, will ride 40 miles for a cure. We will be riding in the Five Boro Bike Tour in New York City on May 5-just in time for Mother's Day.  We will also have a fun cheering section along the route, so please feel free to join! All proceeds benefit MPN Research Foundation, and will go directly to the organization when you make a donation. 

Click here to support Jen and MPN Research

Spread the word. We made over $11,000 at our bowling event last year and hope to beat our record by race day!  

Hope to see you there!

MF Challenge proposals due today!

Today our small staff is buzzing about the MF Challenge proposals coming through our email. This is the second wave of our Myelofibrosis Challenge grant program, a joint effort with the Leukemia & Lymphoma Society. 

In 2012 we awarded $100,000 each for four different projects, the goal of which is to stop or reverse fibrosis. Progress reports on the first wave are due later this year, and we're hoping to find 4 more projects worthy of the funds to continue in 2013-14. We set up a microsite to keep patients, their friends & families up to date, where you can also sign up for alerts. 

News from Mt. Sinai on their Panobinostat trial for MF

Drs. Ron Hoffman and John Mascerenhas of Mt. Sinai School of Medicine in New York have reported positive outcomes from their Phase I trial of panobinostat in myelofibrosis patients. Their article published in the British Journal of Haematology states that significant improvement occurred for patients on low dosages of Panobinostat.

Click here for the full article from Mt. Sinai's Medicine Matters blog.

Click here for the study abstract.

April patient events

Our partner on the MF Challenge, the Leukemia & Lymphoma Society, is hosting a few events in April aimed at educating the people living with blood cancer: 

Patient in and around Sioux Falls, SD are invited to attend a seminar on Myelofibrosis. Dr. Ahmed Galal will be speaking about managing and living with Myelofibrosis. More information can be found on their flyer located here. 

For Illinois, patients are invited to attend the Illinois Blood Cancer Conference taking place on April 20th in Oakbrook. There will be presentations on latest treatments and research as well as breakout sessions covering long term survival and nutrition. This free conference covers blood cancers in general, but could be of interest to MPN patients and their caregivers. 

Finally, several patient support groups are meeting in April including Houston, TX,  Pittsburgh and Philadelphia, PA, and Los Angeles, CA. 

Playing catch-up

2012 MF Challenge grant recipient
Ann Mullally of Harvard in her lab

It's the beginning of March 2013 and judging from our last post date this space has been silent for too long. It has nothing to do with the level of activity going on in the world of research for PV, ET and MF, though. On the contrary, 2012 was a very busy year.  

In July we wrote the first checks for the MF Challenge, a joint grant program funded by MPN Research Foundation and the Leukemia and Lymphoma Society, whose aim is to help discover novel ideas of how to stop or reverse fibrosis. At the sime time we continued to fund our 6 New Investigator and Established Investigator grants. 

September brought us Blood Cancer Awareness Month, and a series of posts including how the Foundation is forging partnerships with the new MPN groups popping up. 

Northwestern's Dr. Brady Stein at the
Chicago MPN Patient Symposium

September was also our Chicago Patient Symposium. We videotaped the proceedings, which allowed patients and doctors to get together and learn. November had us checking for new MPN videos. In December our Scientific Advisory distilled the highlights from ASH's annual meeting as they pertained to the MPNs.

Judging by the first quarter of 2013, it will be another banner year in which we put MPN research at the forefront of our mission and direct funds in the manner that will best serve patients. For us this means the funding of a second installment of the MF Challenge grant program, continuing to publish our free patient newsletter, and finding a way to work with industry  to direct funds and attention to the MPNs. 

Soon we'll have progress to report on our MF Challenge, New Investigator and Established Investigator grants. We're excited to learn how their insights could impact MPN patients everywhere and to see what else this year brings. We can't wait to share with you all, a bit more frequently going forward.