Sunday, December 27, 2015

MPNRF honored with Cure MPN Hero award!

Each year for the past three years, Cure Magazine and Incyte Corporation have recognized efforts within the MPN community with their MPN Hero award. The two categories are commitment to the community or individual. Past winners have included Ruben Mesa, Zhenya Senyak, Joyce Niblack, Richard Silver, and many more. We were honored to receive recognition this year for our dedication to serving the MPN community. 

We share this award with all board members and patients who work hard to make a difference for themselves and others living with PV, ET and MF. Click here to read the press release from Cure Magazine.

Tuesday, December 1, 2015

Nature Medicine: Drug stops reproduction of cancer cells in Myelofibrosis

According to a paper published in Nature Medicine, the drug MLN8237 (or Alisertib) has reduced tumor cells, blood counts and fibrosis in mouse models of myelofibrosis. Reports John Crispino, PhD, "This new paper shows that Alisertib has an anti-tumor effect in samples of myelofibrosis, similar to what we saw in megakaryocytic leukemia." 
John Crispino is also the Scientific Advisor for the MPN Research Foundation.

Click here to read the original article from Nature, or the press release from Northwestern quoting both Dr. Crispino and Brady Stein.

There is a clinical trial for Alisertib in people with myelofibrosis currently enrolling at Northwestern. Please visit the listing for this study for further information including how to enroll.

Tuesday, November 24, 2015


On October 29, the Centers for Medicare and Medicaid Services (CMS) issued a proposedrule allowing Medicare coverage for stem cell transplants in approved clinical trials for Myelofibrosis (MF) patients. Unfortunately, some of the requirements in the proposed rule would actually decrease access to stem cell transplants.  
Here's How You Can Help  
The CMS is currently taking comments on the proposed rule. Use the link to send a brief message to CMS to ask that they revise their proposed study parameters to ensure that the clinical studies make stem cell transplant an option for all MPN patients. 
We need the transplant, biomedical and lay community to share their comments with CMS. CMS is accepting comments on their proposal for 30 days only; the comment period closes on November 28, 2015.  Comments can be very brief and still be impactful.  An example might look as follows:
  • I appreciate that CMS is trying to provide coverage for Medicare beneficiaries with Myelofibrosis.
  • I encourage CMS to consider removing the requirement for concurrent controls in these clinical studies because it will limit a Medicare beneficiary's ability to access this important therapy. 
Use your own words as much as possible, and
  • Be brief
  • Be personal
  • Tell them exactly what you want:  to ensure that stem cell transplants are an option for ALL MPN patients.
The Deadline for comments is November 28, 2015  

Thursday, November 19, 2015

Do you want to double your dollars? A $25,000 Matching Gift Fund has been setup by a group of generous donors. That means that a gift of $100 made on Giving Tuesday, December 1st will become $200 to help find better treatments and a cure for MPNs.   Give to the MPN Research Foundation on Giving Tuesday, December1st.
To help spread the word, we are asking people to change their profile picture to this image and explain to friends that you will be helping to support the MPN Research Foundation on Giving Tuesday.               
Want to make a more personal statement? Jump on the Unselfie bandwagon for GivingTuesday this year. Upload YOUR selfie and why you are planning on giving this year on December 1st -  Giving Tuesday.                                                                                         
 How do I Unselfie?  1. Take a selfie with a caption or have it on a card in the picture explaining how or why you are giving this year. (Example - I'm giving because I want to find a cure for MPN Blood Cancers) 2. Post it to your Facebook, Twitter or Instagram account and share it with us and your friends. Every Unselfie Counts!

Thursday, November 12, 2015

Is this the Beginning of gene-editing medicine?


Layla Richards

Does the smiling face of Layla Richards mark a new era in genetic medicine that could change all our lives?

The story of Layla Richards may be just the beginning of a new chapter in medicine.

Layla Richards developed CLL as an infant and the day before her first birthday, Layla's parents were told she was going to die. All treatments for her leukemia had failed

An experimental treatment that had only been tried on mice was her only hope. Her family, her doctors and a biotechnology company decided they had to try it.

The biotechnology company genetically engineered some cells that were designed to kill Layla’s cancer.

Today, because of this experimental treatment, Layla has no trace of leukemia.

We could be seeing the start of a revolution in medicine. The same procedure that worked in Layla, could be used to treat, not only other cancers, but inherited diseases.

Genetic engineering is a whole new field in which missing DNA be inserted and defective DNA can be corrected. This is what doctor’s did for Layla.

This revolution may have started around the turn of the millennium when scientists were saying that gene therapy was going to transform the world. While it has taken 15 years for gene therapy technology to develop, the experimental treatment that saved Layla’s life may be just the first miracle of this new technology.

As with all advances in science, caution is the word for the day. Gene-based therapy involves changing our DNA. These are the instructions for building and running every part of our body.

Early attempts to use this technology ultimately resulted in patients developing leukemia and further attempts were abandoned.

DNA was being inserted, in such a way, that it disrupted the natural functioning of some cells and they became cancerous.

Since then the process has become much more precise. The viruses being used can precisely place DNA into safer sites in the genome. This new precision has been described as the difference between using an ax and a laser. The viruses used act as a guide that finds its way to specific sites in our DNA and a pair of molecular scissors that can edit the DNA.


DNA is our blue print of life

Will doctors be able to harness our DNA for new treatments?
A similar technique is already being tested in HIV-positive patients. The aim is to take the patient's cells out of the body, give them HIV protection, and then put them back in.

The doctor involved in treating Layla's leukemia, told the BBC: "The technology is moving very fast, the ability to target very specific regions of the genome has suddenly become much more efficient.

"The technology itself has got enormous potential to correct other conditions where cells are engineered and given back to patients or to provide new properties to cells that allow them to be used in a way we can only imagine at the moment."

Re-arming the immune system to target cancer and a wide range of inherited disorders is in the sights of doctors.

It will be easiest for them to use cells that can be taken out of the body, modified and reinserted rather than trying to edit them still in the body.

So diseases of the blood or immune system - such as MPNs or sickle cell anemia - will be easier targets than kidney or heart defects.

Doctors are predicting an "explosion" in the use of such genetic engineering in the next 10 years.

After the overhyped false dawn fifteen years ago, gene-editing is now, it seems, about to arrive.

Wednesday, November 4, 2015

Notes from CR&T MPN Symposium

There were many interesting sections from today's meeting. Bill Crowley from the MPN Research Foundation was on the ground at the event and shared some highlights:

  • Jason Gotlib presented "What is the Clinical Trial Process", touching on ethics in the history of clinical trials, how the review boards function, FDA audits of trials, industry monitoring of studies, trial design, and barriers to participation in clinical trials.
  • Srdan Verstovsek gave a rundown of "Current developments in MF treatments". He spoke of changing guidelines for stem cell transplant and mentioned that MD Anderson will do transplants up to 75, depending on overall health and donor match. He mentioned that sometimes a JAK inhibitor will lower blood counts, and use of a JAK inhibitor has to be individualized. Combinations are encouraged. Two new JAK inhibitors coming up are Pacritinib and Momelotinib. New targets are being investigated with the drugs PRM-151 and Imetelstat.

  • Jerry Spivak gave a talk on"Essential Thrombocythemia, Pathogenesis and Management" that included info on differences between male and female ET patients, with more women affected, but the men with ET tend to have more issues. He also mentioned that migraines are a big problem because of the increase in platelets. If something is not done with the platelet count, that migraine will not improve.
We'll report more after the event. If you were at the event or have a question, comment below. 

Friday, October 30, 2015

Update on Medicare coverage decision for Stem Cell Transplant in Myelofibrosis

The Centers for Medicare & Medicaid Services’ Coverage and Analysis Group released a proposed decision memo Thursday on modifications to the National Coverage Determination for allogeneic Stem Cell Transplant. CMS has proposed to expand coverage through Coverage with Evidence Development (CED) for allogeneic HCT to include myelofibrosis, multiple myeloma and sickle cell disease.

Unfortunately, this does NOT mean that coverage for SCT for those with myelofibrosis is immediately available. There are three more stages to this process: 1) completion of the 30-day comment period, 2) completion of the 60-day final analysis period that CMS has after the close of the public comment period, 3) development, submission and approval of a study that meets guidelines set forth in the final decision. Coverage through Medicare will likely not be available until sometime in the first half 2016.

The National Marrow Donor Program is working on coordinating the study to the specifications needed by the CMS. 

Public comments by people affected by the lack of Medicare coverage for Stem Cell Transplants for Myelofibrosis. We will let you know how to go about submitting a public comment ASAP. 

Tuesday, October 13, 2015

Sivana-ET "I am a fighter and don’t give up easily"

Silvana ET

I am Silvana, and I live in Rotterdam, The Netherlands. I'm 52 years old and was diagnosed with E.T December 19th, 2011, about 3 weeks after I had a bone marrow biopsy. I have 2 children, a daughter and son. My other experience with a chronic condition was with asthma, which I was diagnosed with 1993/1994.

In 2003 I was hospitalized at the ICU for 3 weeks with Acute respiratory distress syndrome (ARDS) which included 2 weeks in a drug injured coma. After this I asked my complete medical file where I read that my doctors thought at that time i had MDS. However, I didn't receive my diagnosis of ET until 2011. That’s why I believe I have been living with this long before I was diagnosed. 
In december 2013 I was also diagnosed with papillairy thyroid cancer, stage 3. I had 2 surgery's to remove the whole thyroid and I had also twice the highest dose radio active iodine treatment. In May I heard the good news that I am thyroid cancer free! My next check up is November. About 2 months ago I had a minor stroke which caused my doctors to modify my regimen from aspirin to clopidogrel. In the past 6 weeks I have also been on Hydrea.

I am a fighter and don’t give up easily! On November 13th I am going to London ((UK) to attend the ‘Living with MPN’ day!

It’s been a long time since being on a plan, and I am excited and a bit insecure to go all alone. I look forward to meeting others out there with MPN. I am also going for the 4th year in a row to the Dutch MPN day which is October 31st!

Tuesday, October 6, 2015

How to Help a Sick Friend

How to Be There for a Sick Friend

A guide for being there when it matters most

What can I do to help?" is probably the most common thing we say to a friend who is ill or in need. But despite our best intentions, this may not be the best way to lend a helping hand. When a person is diagnosed with an MPN blood cancer, patients often feel too overwhelmed or too proud to know how to respond. Now, here's some ways for you to support a sick friend.

1. Don't ask, just do. Offer something specific (big or small) that you can do to help out and suggest a time when you can follow through, such as "How about I come over on Friday at six and make you dinner?" This may help ease any embarrassment or guilt your friend may feel about asking for help.

2. Be honest. It can be difficult to find the right words—or any words at all—when a friend is in crisis. Sometimes, simply saying "I don't know what to say" is the best way to start a conversation.

3. Add a personal touch. While an iTunes gift card or a good book is a great gift, personalized presents can be the most meaningful. A baked batch of favorite cookies could be a great comfort. For someone living abroad, make me a short video to tell them that you are thinking of them. You can send dozens of hand-folded origami cranes for good luck.

4. Listen. When news of a cancer diagnosis spreads, patients can be inundated by opinions, advice, and information on everything from nutrition to spiritual outlook. Help out a friend by being a sounding board rather than an information dispenser.

Wednesday, September 23, 2015

CTI Biopharma to submit New Drug Aplication for Pacritinib

CTI Biopharma to submit NDA for Pacritinib

September 23, 2015
CTI BioPharma Corp. today announced their plan to submit a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for Pacritinib, an oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R. They are requesting approval for this drug as a treatment for patients with intermediate and high-risk myelofibrosis with low platelet counts of less than 50,000 per microliter (<50,000/uL).

Submission of an NDA after a single Phase 3 trial under accelerated approval, instead of waiting to complete two Phase 3 trials, could potentially reduce time to market by up to 14 months.

Read the full press release from CTI Biopharma here.

For more information about the FDA Accelerated Approval program click here.

The PERSIST-1 trials, which is a randomized trial of Pacritinib against best available therapy in patients with  myelofibrosis regardless of platelet counts is ongoing. CTI Biopharma is working in partneship with Baxalta on the development of Pacrinitib.
- See more at:

Thursday, September 17, 2015

What's next for MPN Research?

Before there was knowledge of the JAK2 mutation, there was the MPN Research Foundation.


Both the Foundation and MPN patients had the nerve to demand that medical science deal with us now


We were audacious enough to be impatient, to push for better and more treatment options now


On a personal level, being diagnosed with an MPN may have been one of the biggest shocks of your life. Globally, the fight against cancer is one of the most significant challenges in history, and we must not rest until we have the answers we deserve, answers that lurk in our bodies and in as-yet untried interventions.  


Sixty years after the MPNs were first identified we have finally begun to make progress. One approved medication. There are more treatments in clinical trials now than ever before, all representing a promise of new and better options. These are good things, but it is not enough, and it is not time to sit back and wait. 


Now is the time to act. So we are asking you today: what's next? If you could focus MPN research on one problem or area of interest, what would it be? Take a moment to tell us now. We're listening.

Tuesday, September 8, 2015

Our support means you are never alone.

In the fight against MPN Blood Cancer, Research & Support can make all the difference. The MPN Research Foundation is your partner. The innovative research we fund moves science forward. Support from family, friends and us means you are never alone. Together we can make a difference today, while finding better treatments and a cure for tomorrow. Patients and caregivers have options for support, both online and in person.

The MPN Research Foundation will be rolling out a list of activities for MPN patients to participate in during the month of September and will be highlighting some of the important work currently being conducted in research, advocacy and education. These activities have been spearheaded not just by MPN Research Foundation, but by a wider group of organizations working together to help patients.

Blood Cancer Awareness Month Events

Patient Support Groups
September 10, 2015

Contact: Mabel at 303-764-8523

September 10, 2015-Gilda's Club Chicago.@6-8pm
Contact: Raquel at

September 17, 2015
Contact: Jean at 724.831.0368 or Email:

Los Angeles
September 26th 11am to 3pm.

Contact: Ron Anderson at

SF Bay Area
October 3rd at 9:30am-4:30pm in San Francisco
Registration required and space is limited.

For patient support group meetings beyond September click here

- See more at:

Patient Symposiums

Cancer Support Community

September 1-30 – CSC local sites will be hosting  awareness events: Shining the Light on MPN during the month of September.

Leukemia and Lymphoma Society

Tuesday, September 8, – Blood Cancer Conference in Hershey, Pennsylvania

MPN Advocacy and Education International

September 11-12 - Women and MPN , San Diego, CA

 Patient Power

A Town Meeting for Patients, Their Families & Care Partners
Saturday, September 12, - Online and In-Person in Stanford, CA

Cancer Support  Community of Greater Philadelphia

Saturday, September 12, at 4100 Chamounix Drive, Philadelphia, PA 19131.
Breakfast will be served at 9 a.m. with the lecture starting at 9:45 a.m. Speaker is Dr. Gerald L. Messerschmidt, the director of clinical research at Lankenau Institute for Medical Research.

Leukemia and Lymphoma Society

Saturday, September 19, Blood Cancer Conference in Worcester, Massachusetts

The Canadian MPN Network

Saturday, September 26, Patient advocacy Conference

Leukemia and Lymphoma Society

September 26, Blood Cancer Conference in Chicago, Illinois

MPN Voice

September 30, Patients' Forum, Edinburgh Register Here:

·The Latest News on Research and Treatment for Myeloproliferative Neoplasms (MPNs)

A Town Meeting for Patients, Their Families & Care Partners

Being diagnosed with, a rare medical condition like a myeloproliferative neoplasm (MPN) can be overwhelming and sometimes confusing. The good news is that innovations in the field are increasing the understanding of MPNs. Research advances are making a difference and improving the lives of those living with polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF).

Saturday, September 12, 2015 - Online and In-Person in Stanford, Calif.

·The Latest News on Research and Treatment for Myeloproliferative Neoplasms (MPNs)

A Town Meeting for Patients, Their Families & Care Partners

Being diagnosed with, a rare medical condition like a myeloproliferative neoplasm (MPN) can be overwhelming and sometimes confusing. The good news is that innovations in the field are increasing the understanding of MPNs. Research advances are making a difference and improving the lives of those living with polycythemia vera (PV), essential thrombocythemia (ET) and myelofibrosis (MF).

Saturday, September 12, 2015 - Online and In-Person in Stanford, Calif.


Fundraising Events


Tim’s Run

Yarmouth, ME

Sunday, September 27, 2015


Chicago Half Marathon

Sunday, September 27, 2015