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!

Tuesday, November 17, 2015

Giving Tuesday: When America Gives as Good as it Gets. 

Giving Tuesday was founded in 2012 by New York’s 92nd Street Y, in partnership with the United Nations Foundation. The goal of Giving Tuesday is to create a global day of giving back that celebrates generosity and kindness. The day itself falls on the Tuesday following Thanksgiving each year, meaning Giving Tuesday for 2015 falls on December 1.

The same way that Black Friday and Cyber Monday kicks off the buying season, Giving Tuesday kicks off the giving season. It’s a day to step back from the commercialization and consumerism that comes with the holiday season, and promote doing good for others.

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!