MPNforum Magazine

February, 2014. Pub: January 15 2014, Second Edition

 Coming Thursday — February 27…

MPN likes FACEBOOK – A Survey

                The Imetelstat Road Show – Tefferi Speaks

                Fatigue Project launches–  Now it’s up to us.

                MPN Research Foundation banks on 2014 science… Harvey hits wall on long winding road … PLUS  TSR and List of Hematologists and more… See you at The Forum.   

Patient Safety in Clinical Trial, the Sanofi Story
News release here.    Full story here.

Stem Cell Transplant and the Clinical Trial– Our last lines of defense

In this second edition of January’s MPNforum we look at both. Here’s SCT seen through a window opened by one of own, Harvey Gould, as he proceeds through the induction phase of an anticipated stem cell transplant. Harvey’s cheerful courage rallies our spirit even as his body engages in the first rounds of a heavyweight battle for a full cure… And the Zebra Coalition is moving ahead with a petition to secure MPN Patient Clinical Trial rights while minimizing risks and improving outcomes. It’s a simple idea that’s already embedded in the FDA advice to trial sponsors. Give patients a voice. “Nothing about us without us.”…  

– Zhenya Senyak

The MPNclinic has re-opened. Symptoms, therapies, new approaches? …The most reliable place on Earth to get your answers: (Three easy steps start here.)

(Our investigative report on the Sanofi Fedratinib trial publishes Monday.)

The long and winding slide to the edge of the cliff…

Harvey Gould’s popular series, “The Long and Winding Road,” has appeared here for months. He traces his MPN journey in vivid, sometimes hilarious, stories. He also helped introduce us to the 23andMe MPN project, spearheaded by his daughter Ashley, that produced a genome based MPN database researchers can use to explore possibly significant correlations. When his blasts spiked to AML proportions, Harvey and his family settled in for action Now Harvey takes us with him into the hospital, alongside his bed as he begins his preparation for stem cell transplant. So far his white cells have fallen sharply but not, as you’ll see, his wicked sense of humor. (Story here.)

“Happy to be a Zebra…but not for lunch”

That was the very first comment we got on the Draft Patient Rights in Clinical Trial petition. It neatly sums up the deadly serious business of advancing our right to know what’s going in the high stakes game of Clinical Trial. Give patients a voice. The petition will be deployed in two weeks. This draft – including the petition and background – are published to give all of us a chance to weigh in on changes, comments or additions. (Click here for the story and the petition.)

Jeremy’s New Year’s Resolutions for the MF patient MPNforum’s resident fitness, diet, exercise and inspirational guru, Jeremy Smith sets out his four step program for a rebounding healthy 2014. And, no, you don’t have to have myelofibrosis to benefit. (You can start your program right here.)

There is a tribute to Ian Sweet here, a small addition to the flood of grief and acclaim published on his beloved MPN-NET and across the internet as news of his departure spread. This one is from his homeland, from Australia, as if we needed to be reminded that from a place so distant and remote from centers of World population, a living human voice can reach out and sustain us all across oceans and continents.

We feel the loss this week of Patsy Bushee.  She told her SCT story openly and fully to her friends in the MPN community and shared her SCT procedures, photos, hopes and setbacks on Facebook. She spoke her mind and was a good strong friend. There is a page in honor of Patsy Bushee here .  And Ann Haehn shares her memories of Patsy in Honey, a tribute..

The MPNclinic’s doors are open now...

There is no equivalent in the cancer world, perhaps not in all of Medicine. The leading MPN specialists from the world’s major medical centers — Weill-Cornell, Stanford, Guy’s and St Thomas, Sloan Kettering, Roskilde, Mayo Clinic, MD Anderson — assemble to answer patient and healthcare provider questions in an open, free and public international forum.  (Click HERE for 3 steps to get your MPN questions answered in the opening session.)  

What Sanofi doesn’t want us to know…

It has become clear since Sanofi’s abrupt termination of the SAR302503 trial November 18 that the French drug giant does not want to reveal publicly what actually happened. The conclusion of our preliminary investigation — as we await documents from our FDA Freedom of Information Act request — reveals part of that story. (Rescheduled: This report will be available Tuesday, January 28.)

TSR – The Senyak Report

… The new, much better-looking, face of MPNforum. A new year, fresh start, fresh faces taking a hand in MPNforum…with new roles.

Meet the new Associate Editors. They’ll be working on articles, projects, back office production and management, volunteering their time and energy to help MPNforum serve the many needs of our MPN community. Those of us who follow the magazine have already seen their work as each has written for MPNforum. Jamie, a healthcare professional, has been active in social media MPN support. Kathy, fresh from her second stem cell transplant, has taken us along with her during her trials and recovery. Ann, the founding director of Genny’s Hope, is a marrow donor activist. Her series of articles on her daughter Genny’s decision process and stem cell transplant opened the SCT discussion for many of us. Mary, a serious agility dog trainer in her spare time, has been Patient Coordinator for the MPNforum Fatigue Project from the very beginning. As a Rehabilitation Counselor working with patients suffering from chronic illness and disability, she was well equipped to take on that role…. Beyond writing and advocating, the new Associate Editors have something else in common. They’re all moms… something MPNforum has needed from the beginning.

… Promedior heads for Round Two. Fresh from successful toxicity trials of its PRM-151, Promedior announced completion of recruitment for the first part of its two-part Phase II myelofibrosis combo trial to be test for efficacy both alone and with ruxolitinib (Jakafi). PRM-151, the fibrosis busting drug, using a recombinant version of the body’s own Pentraxin-2, has already demonstrated its effectiveness in pulmonary fibrosis applications… Pentraxin-2 is a protein active in the body’s healing resolution process to help stop and reverse scarring. “We’re not sure yet how durable this response will be, “said Dr. Trehu, “but right now this is the simplest approach. PRM-151 acts directly on monocytes and while it doesn’t affect the genetics it might act directly on the malignant cell .”

A portion of this current myelofibrosis trial pairs PRM-151 with Jakafi to see if the known ability of ruxolitinib to relieve symptoms, particularly splenomegaly, can in combination with PRM-151 affect the underlying MF disease state. Part A is a small scale trial of 25 patient-participants divided into two cohorts to test the efficacy of PRM-151 both with and without ruxolitinib in varying dosages. “Based on what we see in terms of IWG response,” Beth Trehu told MPNforum “we can enroll more patients in each of those groups for Part B or change the group composition.” The plan is to ultimately enroll 80 additional patients in the study, perhaps as early as this summer…A complete description of the trial and locations can be found at clinicaltrials.gov.

KDF reports on MD Anderson and Patient Power event …

Take action: Remember Sanofi. Be a Zebra.

Join the taskforce to petition government and private sponsors of research to guarantee MPN patients on clinical trial the right to be informed of developing risks. Just type “Zebra” in the subject of an E-mail to ourMPNforum@gmail.com

You can read the current issue of the Q … right here!

How I treat MPNs? …Silver, Mesa, Hasselbalch, Harrison

Aspirin, Platelets and Thrombosis… by J.J. Michiels…

Interferon instructions for my patients… H.C. Hasselbalch

The List — 100+ Patient-Recommended Hematologists

The Catalog of Articles. ..

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