MPNforum Magazine

One small step for man. One giant leap for MPN therapy.

A tiny study, three patients only. A Phase One genetic engineering safety study, one that doesn’t even look at efficacy. 

But this first in-human clinical trial  NCT03399448 is an historic milestone in the search for an MPN cure.  Jenifer Doudna the co-discoverer of CRISPR and its leading ambassador, reviews the findings and UPENN’s Carl June, the key scientist behind CAR-T, is principal author of the paper reporting on this study in SCIENCE.

Bottom line:  Yes, there are many remaining questions, but CRISPR editing of T cells taken from, modified, and reinfused into blood cancer patients, is both safe and durable. 

It works. It lasts.

AND TALKING OF CRISPR:    MPN and Covid-19, the Corona Virus: It’s a war and we’re in the primary fire zone.  On the one hand we’re particularly vulnerable. Compromised immune system, many of us older, some with co-morbidities. On the other hand we have a few distinct advantages and some powerful allies.. (More)

After MPNforum

Some of you have been with us, have been part of MPNforum, for a decade. Some of you are new to our band of blood brothers and sisters. As we start closing down our game-changing cooperative patient-led movement I ask you all to read as much of this document as possible.

It’s part history, part urgent concern, part hope …and it involves us all. Because the subtraction of MPNforum from the MPN media environment leaves a dangerously large and critical beat uncovered.  Between our friends, allies and each other, we can get it covered. We have to.

So have a look.   And then weigh in, with comments, with small donations. With ideas going forward.

   Thanks,   
                Zhenya Senyak    

I am truly worried.

Beyond myelofibrosis, secondary to essential thrombocythemia. I have multiple co-morbidities, progressive and incurable.  Generally, I feel OK, but I have aged out of all curative options. .
I’m not worried about me. I have had a full, good life and it still looks like a few more turns around the dance floor ahead. I look forward to the next chapter, the mystery of apoptosis, free of body, home again.  And in the time I have left, there are other passions calling to me. A book to write. Some people to see.  Designing the Clinical Trials Project.  Securing the MPNforum archives.

I am worried about you and our MPN community that has banded together in support this past decade.

MPNforum was always a loose federation of MPN patients, caregivers, physicians, and scientists. We’ve been a band of regular reporters, contributors, and columnists, editors, proofreaders, production and graphics folks. And the core cadre was always bolstered by opinion, stories and profiles from friends dropping in now and then. With the proliferation of social media we’ve grown out of mutual coordinated publishing efforts and embraced individual projects, web pages, Facebook groups, podcasts, etc. Our problem is not a lack of opinion and support.

You’ve met — and maybe been among —  excellent regular writers in our pages like Jeremy Smith, Dr. Arch McCallister, Harvey Gould, Charlie Nielsen, Chris Harper, Julie Libon (and familly), Kathy Dubin Flynn, Dr. Michael Goldstein, Ann Haehn, Bonnie Evans,  Cyndy, Sam, Joe, Nathalie Cook and memorable contributors like Marina Peed, Barbara Kurtz, Jane Frantz,  Ian Sweet, Anthony Tiano, Barbara Van Husen,  Manuela Manuel, Karen Duffy, Robert Rosen, Kelly Lanier, Robert Tollen, Patsy Bushee, Ann Brazeau, Michelle Woerhle, Elizabeth Goldstein, Barbara Beckman, Janice Creed,  Mary Morochnick,  Ellen Jacquardt, Zeta Charania, Ashley Gould, Mary Cotter, plus hundreds of others through comments, contributions  and  Small Donations.

In addition, physicians,  the cream of MPN medical specialists selflessly contributed articles and opinion in the  MPNforum and  counseled us through the MPNclinic,  deliberated in the  Stem Cell Taskforce and published in our  sister Journal, the MPN Quarterly — Doctors Ruben Mesa,  Richard T. Silver, Claire Harrison,  Ayalew Tefferi,  Jason Gotlib, Srdan Verstovsek, Robyn Scherber, Nicolai Kroeger, Hans Hasselbalch, Jean-Jacques Kiladjian, Jan-Jacques Michels, Attilio Orazi, Ross Levine, Ann Mullally and many others.

After nine years and 800,000  visits to our pages it’s time to wind down. But we plan to continue some features of MPNForum and need your financial support  (1) To preserve the invaluable MPNforum historic and scientific archives; (2) To make our Lists and resources available to MPN patients today and tomorrow…and (3) To leave the door open for future Special Reports and on-going “Best of…” issues.

Before you reach into your pocket to make a Small Donation (as ever $5 to $100).. consider what we’ve done together through the Forum…and how you might participate in the work that lies ahead. 

As the first on-line MPN magazine, MPNforum introduced us to one another in its graphic  pictorial and personal  presentation of MPN patients, caregivers  and doctors .  MPNforum sponsored the Fatigue Project, contributing to multiple peer-reviewed papers and helping to advance our understanding of this most debilitating aspect of MPNs… MPNforum spearheaded the Zebra Coalition that secured FDA approval of a patient representative on every clinical trial…MPNforum Introduced the MPN community to Jennifer Doudna,  Rachel Hurwitz, Zhaohui Yi, George Church, Shaoguang Li  and the first CRISPR gene editing breakthroughs…covered meetings at ASH, CR&T and MPN A&E… took us behind lab doors, into the MPNRF conference room…  led the CREATE seminar with MPNRF to investigate the use of gene editing in stem cell transplant procedures…Organized the Stem Cell Transplant Taskforce to create an on-line free tool to help assess therapeutic timing options for myelofibrosis patients…Published MAGIC  (www.mpn-magic.com), a prognostic tool accessed a quarter million times by patients and physicians in the past year. And an overview of the MPN Mutational landscape. 

Together, dozens of us – sometimes thousands —  did all that this past decade and more, publishing over a thousand articles and hundreds of on-line issues in the process, hosting meetings, presenting posters and papers at ASH. 

Along with so many of us, I am proud of the work we did together in the past… and truly worried about the future without us.

Without MPNforum who is there to stand against the rampant commercialization of our healthcare?

There are  harsh truths we need to confront:  MPNforum reported on breaking scientific and medical news but we were also there to probe the darker side of MPN care. Where are the healthcare providers who raised their voices against Sanofi’s radical betrayal of patient and healthcare provider confidence?  In all our on-line and print media why did MPNforum alone express the need for the FDA to step up and demand Incyte grant clear and prominent space to the serious side effects of Jakafi?  And who else reported on the sometimes astonishing depth and breadth of physician and medical institution financial engagement with drug companies  and potential conflicts of interest revealed by the Sunshine Act?  As an independent patient-supported cooperative media project MPNforum has been there for us for most of the past decade.

In the brutal blowup of the Sanofi Fedratinib trial —  perhaps the coldest, clearest example of callous drug company behavior — among all MPN media only MPNforum deeply investigated, interviewed patients, Sanofi officers, investors, physicians to ferret out the awful truth that thousands of us were abandoned at the very end of a Phase III trial. Tossed out like the day’s rubbish to seek what solace we could from our own private doctors.  There were  deaths and unreported severe adverse effects in that trial that raised no advance warning signals to investigators or patients.  And there was no ultimate accounting, no corporate taking of responsibility, no transparency.

Who will try to wean MPN patients away from our naïve belief that clinical trials are staged for our benefit, that it makes sense to place hope in profiteering corporate testing of chemical molecules in human experiments.  The NIH labs, the university scientists are focused on research and science.  And while there are good and brilliant people within Big Pharma, the drug companies are laser focused on profit and shareholder value.

In the rush to promote promising new drugs,  why are there so few of us to point out the cruel waste in failed JAK inhibitor drug trials, the merry go round of changing corporate hands – YMI, Gilead, TargeGen,  Celgene, Sierra, Sanofi, Cytopia, Fedratinib– after failures to reach  clinical endpoints, squandering the time, energy, immune resistance and hopes of thousands of MPN patients?

And without MPNforum who will underscore how Incyte has papered the MPN landscape with millions of dollars accumulated through sale and aggressive extension of its temporarily palliative billion dollar drug?   When physicians, medical institutions, non-profits and commercial organizations depend on injections of Big Pharma capital,  where are the voices of protest going to come from?

I look around and regret our lost early innocent days when we came together in solidarity as an MPN patient cooperative to petition the FDA, to investigate gene therapy, to challenge CEOs on drug pricing,  to enjoy monthly MPNforum columns by Dr. Arch, Harvey, Jeremy and Mike…and dare to hope for an ultimate cure.

But we do have allies today.  Among us are caring, engaged patients and caregivers, some like David Denny and Diane Blackstock at Myelofibrosis Facebook and the hardworking admins  and patient/caregiver contributors at several of the MPN Facebook sites offering support on social media. Myelofibrosis Private Support, Jeremy Smith’s MPN Life,, Polycythemia Support Group, MPN Voice, Essential Thrombocythemia Support Group,  MPN Interferon Forum and others.   For years, the amazing Ellen Jacquart patiently collects, sorts, and posts vetted entries to the List of Hematologists. Others like Kevin Walsh (MPN Investigation and Discovery) and Michelle Woerhle and Brandon Goetzman  (MPNRF) carefully follow and alert us to the science. Dr. Margaret Warner, D.O. stepped up as our Patient Advocate.   And we have a handful of physicians on our side.  A few institutions, too. The MPN Research Foundation, CR&T and MPN Advocacy and Education, may accept Big Pharma grants, but they are driven by long-standing commitment to the MPN patient and run by scrupulous people.

It could be enough if we work together.  After MPNforum, who will speak for us? There’s only you and our friends among patients, physicians and caregivers. Together, we can make ourselves heard.

Thanks for listening and thanks for all the good times and shared joys and sorrows. Let’s keep it rolling…

“I’ve made my point and before I get a frozen shoulder from patting our backs, I better sit down in my corner and resume being the company curmudgeon. Hopefully more often the loyal opposition. Am I egocentric?  Of course. Aren’t we all?  Some show it with reversed humility, others with poorly disguised self-promotion, most of you with simplicity and honest restraint.” — Arch’s  Corner, by Dr. Arch McC., MPNforum, March 2012, 

While reducing regular reporting, MPNforum will continue to maintain free community access to all files and report on significant breaking news. We need your financial support  (1) To preserve the invaluable MPNforum historic and scientific archives; (2) To make our Lists and resources available to MPN patients today and tomorrow…and (3) To cover production and system expenses for Special Reports and publication of on-going issues.Why Donate? Why Small?     Our policies, from the beginning:  MPNforum is produced by and for MPN patients. The publication is freely available to the Internet community without cost or paywall.  Gifts of small donations to MPNforum assure our ability to function independently, report objectively. No one is compensated for creative or production work contributed to the Forum.. We pay for hosting, web services and technical software consultants.  There are volunteers but no paid staff. No funds are accepted from institutions, drug companies, or corporate entities.  All operating expenses are covered by small donations or our own funds.  To maintain a level playing field, all donations over $100. are returned to the provider.Your donation is not tax deductible.  

Opening the Archives:  The Best of MPNforum

If  you have a personal favoritie you’d like see in the next issue of “Best of…” email us: ourmpnfourma@gmail.com.

Arch’s Corner….(Dr. Arch McCallister):  Ascites and edema come up for discussion from time to time with us and I thought to write something about it that might help some of you... (more) 

Long and Winding Road... (Harvey Gould): The night before we left for Ireland, I got sick and was up most of the night. Karen and I both wrote it off to too much sushi at dinner.…(more)  

Betting the farm on Imetelstat….The reports from two women on the imetelstat clinical trial lay on my desk. They could have been reporting on different drugs...(more)

ET Pajama Party…(Terri Libenson): Living an MPN life out loud…with humor, comic panels and talk balloons (more)

An Open Letter to the MPN Community in the United States….(Dr, Hans Carl Hasselbalch):  About 25 years ago recombinant interferon (IFN) was used successfully for the first time in the treatment of essential thrombocythemia and a few years later in the treatment of polycythemia vera and hyperproliferative myelofibrosis  (more)

On the pathway to beat polycythemia vera...(Shaoguang Li): This story starts in 沈阳 Shenyang, China, moves to its Sister City, Chicago, and is working its way to a conclusion in the Boston area. (more) 

 List of 300+ Patient-Recommended Hematologists from 27 Nations & 47 States

The MPNclinics – 302 answers to 141 Patient questions… with Index

Stem cell transplant — Six front-line stories and some vital links.

THE ARCHIVES:  A Visual Catalog of MPNforum Articles.

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