Monday, February 1, 2016

It’s World Cancer Day


There has been, from time to time, a question about whether myeloproliferative neoplasms are cancers. Let’s be clear, they are. But don’t take our word for it.


Cancer Support Community defines Myeloproliferative Neoplasms (MPNs) as types of blood cancers that overproduce blood cells in the bone marrow, due to genetic mutations that originate in the stem cells. This genetic mutation leads to the overproduction of white blood cells, red blood cells, and platelets in the bone marrow.

The World Health Organization (WHO) reclassified MPDs to “Myeloproliferative Neoplasms” to reflect the consensus that these diseases are blood cancers (neoplasms) in 2008. MPNs were previously identified as “Myeloproliferative Disorder” or “Myeloproliferative Disease” (MPDs).  

Though myeloproliferative neoplasms are serious, and may pose certain health risks, people with these conditions often live for many years after diagnosis. The prognosis largely depends on the type of MPN. –


The Leukaemia Foundation in Australia offers this definition. Myeloproliferative neoplasms (MPN) are a group of diseases that affect blood-cell formation. In all forms of MPN, a bone marrow problem leads to increased levels of blood cells circulating in the bloodstream.

  • “Myelo” refers to bone marrow, which is the body’s blood-cell factory.
  • “Proliferative” refers to the rapid growth and production of cells.
  • “Myeloproliferative” means increased growth and production of bone marrow and blood cells.
  • “Neoplasm” means an abnormal growth of cells. A neoplasm can be either benign (noncancerous) or malignant (cancerous). In MPN, the neoplasm starts out benign; over time it may turn into malignant disease (cancerous).
    From our website
    The myeloproliferative neoplasms are progressive blood cancers that can strike anyone at any age, and for which there is no known cure. A gene marker, JAK2, was discovered in 2005. It is present in most PV patients and 50% of ET and PMF patients. A second gene marker, Calreticulin or CALR, was discovered in 2013. It is present in 23.5% of people with MF and ET.

Tuesday, January 12, 2016

MPN Community joined by legislators in speaking up on Medicare's plan for Myelofibrosis

For the past several months we've kept you informed and taking action on Medicare's pending decision of whether to provide coverage for people with myelofibrosis seeking a stem cell transplant. Currently there is no official ruling from Medicare on whether this treatment is covered, leaving many patients to risk paying out of pocket (running potentially several hundred thousand dollars) or scrambling to find alternative ways to cover it. 

While we are awaiting decisions, we are pleased that The Honorable Scott Garrett, Congressman from the 5th District of New Jersey, has written a letter to the Centers for Medicare & Medicaid Services in support of providing full coverage to patients. Please join us in thanking him for his support for the myeloproliferative neoplasms community! 

Expanding upon their success with myelodysplastic syndromes, Be The Match and the National Marrow Donor Program have been leading the charge to increase coverage for people with myelofibrosis. We expect word about Medicare's decision in the coming weeks and will let you know ASAP. 

Read our previous posts about this issue:

Help Fix Medicare Coverage for Stem Cell Transplant - May 2015
Update on Medicare coverage decision for Stem Cell Transplant in Myelofibrosis - Oct. 2015

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.