Friday, July 17, 2015

MPN Clinical Trial Highlights for Summer 2015

If you have MF, PV or ET but don’t need medication now, you may think that clinical trials don’t matter to you. But that could change, at which point you may want to review your options. Thankfully, over the past years the interest in developing treatments for people living with these rare, chronic blood cancers has exploded. Here are some clinical trials we’re keeping our eye on:


Baxalta / CTI BioPharma had good press at ASCO with details on their Pacritinib trial and are recruiting more patients at this time for their trial “Persist-2: A randomized, controlled Phase 3 study of oral Pacritinib vs. best available therapy in patients with thrombocytopenia and primary myelofibrosis, post-polycythemia vera myelofibrosis, or post-essential thrombocythemia myelofibrosis” For trial locations and more information: www.persistprogram.com


Gilead is conducting a Phase 3 trial of momolotinib, and is recruiting patients with Primary Myelofibrosis (MF), Post-polycythemia Vera MF, or Post-essential Thrombocythemia MF. For trial locations and more information: https://clinicaltrials.gov/ct2/show/NCT02101268?term=momelotinib&rank=4


Janssen acquired Imetelstat from Geron and has an open study to Evaluate Activity of 2 Dose Levels of Imetelstat in Participants With Intermediate-2 or High-Risk Myelofibrosis (MF) Previously Treated With Janus Kinase (JAK) Inhibitor. For more information Contact Laurie Sherman lsherma4@its.jnj.com

KaloBios Pharmaceuticals, Inc. is now recruiting the Phase 2 portion of their trial of KB004 in MF and MDS patients. The study consists of IV infusion once weekly for a 21 day dosing cycle with KB004 250 mg (the recommended Phase 2 dose).   This portion of the study will be to further study the activity of KB004 and explore the safety of KB004  For more information contact Kate Doherty at Kdoherty@kalobios.com / 1-650-243-3126. They’ve also made further information on this study available here: http://www.mpnresearchfoundation.org/Kalabios-Details

The Myeloproliferative Disorders Research Consortium (MPD-RC) is an international, multi-institutional non profit consortium funded by the National Cancer Institute set up to coordinate, facilitate, and perform basic and clinical research investigating with the ultimate goal of developing new treatment options for people with PV, ET and MF. They are doing several investigator-initiated clinical trials now, including trials with Pegasys, RG7388, decitabine + ruxolitinib and more.  Patients interested in learning more can reach Dr. John Mascerenhas directly at john.mascarenhas@mssm.edu / 212 241 3417 or reach Ruben Mesa at mesa.ruben@mayo.edu / 480-301-8335. Information on these trials is also available at http://mpdrc.org/readarticle.php?article_id=14

Novartis is testing LCL-161 an antagonist of inhibitor of apoptosis protein (IAP), which protect cancer cells from cell death by inhibiting caspases—the enzymes that mediate apoptosis. Their trial” Phase 2 LCL-161 in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF) or Post-Essential Thrombocytosis Myelofibrosis (Post-ET MF)” is recruiting right now at University of Texas, MD Anderson Cancer Center. For more information Contact: Naveen Pemmaraju, MD 713-792-4956

NS-Pharma is actively recruiting for their trial “Safety and Tolerability Study of Oral NS-018 in Patients with Primary Myelofibrosis (MF), Post-PV MF or Post-ET MF” with various locations across the United States. For more information contact Tomonori Uno of NS Pharma at: uno@nspharma.com


Pfizer is testing their Hedgehog Inhibitor PF-04449913 in people who have been previously treated with a JAK Inhibitor. Please check this link as sites are continuously added: https://clinicaltrials.gov/ct2/show/NCT02226172 or call the Pfizer Clinical Trial Call center: 1-800-718-1021  https://www.pfizer.com/contact/email_contact?inquiry=Clinical%20Research

This is meant to be informative rather than to suggest a course of action. It is our policy to share any relevant trial in the MPN space that is Phase II, III or IV. Remember to consult your doctor if you're considering participation in any clinical trial!

Tuesday, June 30, 2015

MF HIKE

"With research there is hope"



"WITH RESEARCH THERE IS HOPE"



On August 19, 2014, Julie Libon was diagnosed with Myelofibrosis. She learned that MF is a rare blood cancer that affects approximately 16,500 to 18,000 people in the United States with no cure and no treatments to slow down the progression of this disease.

After her diagnosis, she and her family felt compelled to act. They formed HikeMF, an organization that sponsors an annual hike with the sole purpose fund research on Myelofibrosis. The funds raised from the hike support the work of the MPN Research Foundation.

On Saturday, May 30th they had their first annual hike at beautiful Moose Hill Wildlife Sanctuary. Over 125 people laced up their boots and joined the fun. After hiking for about 90 minutes on the Bluff Overlook Trail, the hikers had refreshments, a silent auction, and raffles in the main building. They raised over $19,000 and they expect to hit $20,000 soon. Not bad when their original goal was $10,000 and they expected about 25 people hiking.
If you're inspired by Julie's actions email Bill @ wcrowley@mpnresearchfoundation.org with your idea, big or small.
On August 19, 2014, Julie Libon was diagnosed with Myelofibrosis. She learned that MF is a rare blood cancer that affects approximately 16,500 to 18,000 people in the United States with no cure and no treatments to slow down the progression of this disease.

After her diagnosis, she and her family felt compelled to act. They formed HikeMF, an organization that sponsors an annual hike with the sole purpose fund research on Myelofibrosis. The funds raised from the hike support the work of the MPN Research Foundation.

On Saturday, May 30th they had their first annual hike at beautiful Moose Hill Wildlife Sanctuary. Over 125 people laced up their boots and joined the fun. After hiking for about 90 minutes on the Bluff Overlook Trail, the hikers had refreshments, a silent auction, and raffles in the main building. They raised over $19,000 and they expect to hit $20,000 soon. Not bad when their original goal was $10,000 and they expected about 25 people hiking.
If you're inspired by Julie's actions get in touch with your idea, big or small.
- See more at: http://www.mpnresearchfoundation.org/2015-Hike-MF#sthash.41Fitl0k.dpuf
On August 19, 2014, Julie Libon was diagnosed with Myelofibrosis. She learned that MF is a rare blood cancer that affects approximately 16,500 to 18,000 people in the United States with no cure and no treatments to slow down the progression of this disease.

After her diagnosis, she and her family felt compelled to act. They formed HikeMF, an organization that sponsors an annual hike with the sole purpose fund research on Myelofibrosis. The funds raised from the hike support the work of the MPN Research Foundation.

On Saturday, May 30th they had their first annual hike at beautiful Moose Hill Wildlife Sanctuary. Over 125 people laced up their boots and joined the fun. After hiking for about 90 minutes on the Bluff Overlook Trail, the hikers had refreshments, a silent auction, and raffles in the main building. They raised over $19,000 and they expect to hit $20,000 soon. Not bad when their original goal was $10,000 and they expected about 25 people hiking.
If you're inspired by Julie's actions get in touch with your idea, big or small.
- See more at: http://www.mpnresearchfoundation.org/2015-Hike-MF#sthash.41Fitl0k.dpuf

Friday, June 5, 2015

MPNs made news at ASCO this year

The American Society of Clinical Oncology has its big meeting each year in Chicago in early June. In the past solid tumor cancers have received the most attention, saving blood cancer announcements for the American Society of Hematology meeting where the focus is more narrow. This year, however, was different, with big news coming from Baxter/CTIBiopharma on their Pacritinib data. Dr. Ruben Mesa presented at a press conference on progress for this drug, which is intended for people with myelofibrosis who have low platelet counts.

Patient Power was also on the scene and filmed a roundtable discussion with Dr. Mesa, Dr. Allison Moliterno, Dr. Naveen Pemmaraju and PV to MF patient Heidi. They discussed the trends in therapy for MPNs, especially in the wake of the 10 year anniversary of the discovery of the JAK2 mutation upon which much work developing drugs has been focused. Also mentioned was a presentation on a 5 year follow-up of people who had underwent allogenic stem cell transplants for myelofibrosis. Heidi also discussed how she as a patient has sought out information, choosing to go both to her doctor as well as the online forums. According to Heidi, "patients should do their own research from multiple reputable sources". 

Click here for the "All About Myelofibrosis" infographic.
     
                                   
                                   


Thursday, May 21, 2015


You may have heard that earlier this week the Federal Trade Commission filed a lawsuit against four cancer groups for defrauding donors. The four organizations collected $187 million between 2008 and 2012. Founder James Reynolds, Sr. and his friends and family members operated all the “sham” organizations. Less than 2 cents of every dollar donated went to aid for cancer patients. 

Let's be clear: this is not ALL cancer charities. Many organizations are working hard to find a cure for cancer, provide services to patients and families and fund research. With the government contributing less to fund medical research and provide services to patients, cancer charities are more important than ever.

How do you know if your donation supports the cause that is important to you?  Donors have to do some due diligence before making a gift. Several groups, including Wise Giving Alliance, Charity Navigator and Charity Watch evaluate millions of charities on a regular basis and are easily searchable online.

If you get a late-night phone call or see a fund box in the supermarket, ask basic questions: How long has the charity been in business? How much goes toward programs, staff, and board members? The more questions you ask, at some point, either they will get tired of answering or they will give enough information to make you comfortable.

You should also check the 990 of organizations you support (we list ours here http://www.mpnresearchfoundation.org/Annual-Reports) and understand how they are spending your money.

To see what the Better Business Bureau’s Wise Giving Alliance, Charity Review Council or Guidestar says about the MPN Research Foundation, go to our website and click on the link “Our Accreditations” at the bottom of the page.

 

Give from the heart, but give smart.

Monday, May 4, 2015

Help fix Medicare's gap of coverage for Myelofibrosis patients seeking a stem cell transplant

I just submitted my first public comment ever to the Centers for Medicare and Medicaid Services (CMS), the body that governs what is covered by Medicare and Medicaid. By the time you finish reading this I hope you'll decide to follow suit. 

In my position at MPN Research Foundation I have had the opportunity to interact with many myelofibrosis patients, as well as people with polycythemia vera and essential thrombocythemia. Patients face anxiety about what the future holds for them as they struggle with MF, for which there is only one cure that works for a portion of the population: a hematopoetic cell transplant.

There is data to back this up, and also there's stories like Ron's

Right now, there is no standing rule that Medicaid will cover the costs of a Stem Cell Transplant for people living with Myelofibrosis. Without guarantee that this is covered, patients and hospitals face severe financial risk that has already kept people from getting a SCT even though their physicians recommend it. 

Medicare should be encouraged to explicitly cover transplant for myelofibrosis. Be the Match and the American Society for Blood and Marrow Transplantation (ASBMT) have lobbied CMS to open a National Coverage Analysis on coverage for Myelofibrosis. 

This is where you come in. We are joining with Be the Match and ASBMT to encourage the public - YOU - to submit a comment to CMS about why it is important to MF patients seeking care they and their doctors decide they need. This is a chance to tell your story. Put in your own words why this is unjust. Click here to submit your comment before the deadline - May 30th. 


If you have any questions or comments get in touch at 312-683-7243 or mwoehrle@mpnresearchfoundation.org


Wednesday, April 22, 2015


PA Support Group Leader, Jean Diesch, Rally's "MPN Warriors" to raise Awareness at the  "Light the Night Walk" during BCAM.

We took a few minutes to talk with Jean about her experiences as a Support Group Leader. Prepare to be inspired!


Why did you become a Support Group Leader?
I became a Support Group Leader for a variety of reasons. The core reason is that I love to help people. When I was diagnosed, it was difficult finding information, whether it be medical professionals treating the disease with the latest treatments, learn what the latest research is or talking to someone that simply understood my experiences. When presented with the opportunity to start a Support Group, I had no other choice. It gave me a purpose. I retooled my skills that I developed from my work experience to focus on helping others.

Where did you begin your journey as a SGL?
My journey to start a Support Group began at the time I attended the MPNRF Educational Conference, in Chicago, IL, 2012. I was so amazed. The presenters and the quality of research and the wealth information presented were outstanding! There were approximately 200 people in the room; they too were thirsting to learn about our disease. I asked one of the conference planners if they could bring this program to my home town. They informed me that it was not possible at that time. They told me the best way to get the information out was to become a Support Group Leader. So I set out to do just that!

What were some of the challenges you came across? How did you overcome them?

My challenges in the beginning were that there was no real direction as to how to become a Support Group Leader. I was not a social worker and I had no medical experience, nor did I ever attend a support group meeting, I just was living with this disease! I contact the MPN Research Foundation to find out who the other Support Group Leaders were. I spoke to them over the phone. They told me what they were doing at their meetings. I contact other cancer groups, but didn’t get a lot of support due to the fact that I was not a social worker nor did I have any form of medical experience. I eventually received information from and individual who gave me some additional direction. I then contact several places to have a meeting however, they wanted to charge me. Eventually I was given a room at the local community hospital. I requested the room for 2 month in advance not having any patients in the group. My next step was to find patients! I requested the MPN Research Foundation notify their patient base to inform people of the meeting time and place. At the first meeting I had 4 patients, 2 widows to the disease, and 3 caregivers. Getting this group together was like making a cake backwards; had the end product a good cake but didn’t know what the ingredients were, didn’t know what it tasted like or how long to bake it!

I have a major challenge with the 2 major hospital groups in our region. They are more interested in their concerns of their “MODEL HOSPITALS” instead of caring for the needs of the sick patient! Other challenges were that I did not get a lot of support from the physicians’ offices. Some physician offices simply have told me “They Don’t support, Support Groups!” I have persevered and reach patients and patients have found me whether it be Facebook, flyers that I post, emailing’s to physician’s office or simply work of mouth.

Currently, we meet at the former “Gilda’s Club of Western PA” now known as “Our Club House.” They have been very supportive of our needs. If we have a “Meet the Doctor” program they provide us with a great meeting room and all the amenities; conference phones, projection equipment, large screens and etc. Another reason I really enjoy their facility is that if there comes a time that I physically cannot continue with the group, “Our Club House” is well equipped, with qualified leaders, that could take over the group and everything I have done can continue.

One person in particular has been very helpful, her name is Francine Morrison. She was the Patient Care Coordinator at LLS of Western PA at that time. She became the mixer to forming our cake! She helped me broaden my resources from pharmaceutical companies to healthcare providers and other resources that I would never have known, if it wasn’t for Francine!


Please run us through SG Meeting from beginning to end.
Generally we start out with my welcoming everyone. I reiterate at every meeting that I have no CLINICAL EXPERIENCE. The purpose of this meeting is to listen, speak of what has worked for us in similar circumstances, and to help member explore their options. We are to respect each member’s rights and personal decisions. The meeting is for patients, their families, and care givers. There is no cost to the meeting. At this point I have the participants introduce themselves to the group. Some people want to tell us which disease they have and what they are taking to control the condition and share other experiences. After the introductions I present one of the following
• give an update of information that I have learned from a conference
• If I have a CD from one of the conferences I will show a segment of it
• have a conference call or meeting from a medical professional
• I ask if I can help with anything until the next meeting

Generally the 1 ½ hours is up quickly. We meet bimonthly from March to November. Many of the patients travel an hour to get to the meeting.

What would you say to an aspiring Support Group Leader?
Many times this is the only option a patient has, when diagnosed with a rare disease, to learn and connect to other patients. A rare disease is not RARE when you have it! Sometimes it a lonely feeling, when you have a condition that is not common. Support Groups can help fill that void!

I have found the role of a Support Group Leader has been very rewarding. I have had patients have the disease for over 20 years and never spoken to another individual with their condition. They are so appreciative. I would say reach out to others that have formed groups, learn everything you can then run with it! Don’t look back!