Science & Medicine

Posts tagged ‘clinical trial’

Advance: MPNforum Summer Issue –


Summertime popsicle


Imetelstat is back in the headlines.  Results from its myelofibrosis trial reports at ASCO next week…(Nobel Prize winners and patients weigh in as the drug continues its tortured path to approval)…

Cachexia, A look at the deadly, most ignored, sign of progressing cancer and what you can do about it. …

Clinical trial vs. stem cell transplant  with eyes wide open…

News: Will medicare pay for myelofibrosis SCT?…  What’s the 3rd leading cause of death in the US?…. Close up, MPN A&E,…Welcome to MPN Actjion Network and …congratulations to HikeMF.

It’s a thick package to take along on your Memorial Day Weekend.  Enjoy


Happy Summer, 2016.


  •  As ever, this is an advance subscriber copy. Please send along your comments, edits, typos, misspellings, broken links, etc. so we can clean things up before publication tomorrow and thank you!

March issue available/Clinical trial


This is a difficult MPNforum issue, emotional and challenging.

I have to finally get it out of my computer and into the world but I know it still needs proof-reading and editng,  If you can help this afternoon, please send any comments/corrections to me:

The melanoma story that kicks off the issue was researched and written before news broke about pacritinib.  I left it in as the lead because of the brilliant scientific work Leonard Zon did uncovering the stem cell origins of  of cancer formation. It stands in sharp contrast to the rest of the issue.

The pacritinib affair – FDA suddenly putting a stop to the clinical trial and prohibiting further distribution of the CTI myelofibrosis drug – has had a devastating effect on Patients.

That makes it twice in two years that a Phase III MPN drug collapsed at the finish line. This time however, it was the Feds that did it…and by every indication through brutal over-reach.

Either way we the patients are left powerless, unconsulted about matters that directly affect our lives, and lost in a maze of clinical trial mumbo jumbo

This March Issue of MPNforum is given over to the Clinical Trial Lottery, the pacritinib affair, a view of MF patients abandoned without alternatives, and an overview of how messy the clinical trial system is and how we can avoid getting ensnared.

MPN Lives Matter,




Summer 2015 — Advance Issue Available Now



The advance Summer, 2015 issue of MPNforum is now available to Subscribers and Friends of the Forum.

The Clinical Trial option is a card we know– or at least hope — we can play if and when we need it. Given the inherent risk and recent history of clinical trials the arrival of Margaret Warner, the first MPN Clinical Trial Patient Advocate, is cause for celebration.   Her story and a picture of the new clinical trial landscape is in this issue.

One focus this time around is Points of Light, a celebration of those who illuminate, who lighten our MPN burdens. This weekend the Most Important MPN Event of the Year takes place in a room filled with some of those folks.  It’s a secret meeting with an inside report from someone who knows what happens behind closed doors.

There’s the story of another Point of Light, a young woman who refused to take her MF diagnosis sitting down.  The results roll out this Saturday.

There’s a lot more in this issue – a chance to comment on Medicare covering SCT medical expenses…The full report in the journal Blood on the Fatigue Project and Mayo Clinic (Scottsdale) work..exploring the impact our meds and other health issues have on MPN fatigue… and lots  more in TSR and our regular features.,

Enjoy. celebrate the Summer…and be well,


*As ever, the “TAKE ME BACK TO THE CONTENTS” link won’t work until publication date, tomorrow, and forwarding your discovery of errors and typos in advance of that pub date to me at  would be greatly appreciated

Patient Safety Report



Report reveals under-reported problems, patient safety concerns

Sanofi’s clinical trial continues months after reported brain damage. Toll: 1 dead, 7 injured.

Contact: “media”,

Tuesday, January 28, 2014Sudden collapse of the big multinational Sanofi study of its myelofibrosis drug fedratinib exposed serious problems in clinical trial patient safety monitoring. That’s the conclusion of a Special Patient Safety Report appearing today in MPNforum.

The abrupt termination of the Sanofi Phase III clinical trial in November stunned patients and site investigators. Reportedly, one death and seven cases of Wernicke’s encephalopathy (WE), a potentially deadly neurological disorder, occurred in the course of the clinical trial.

A Mayo Clinic (Rochester) neurologist confirmed one WE case this spring, a 71 year old woman with myelofibrosis on clinical trial. That case study was published in the medical literature in August but the Sanofi drug trial continued until mid-November. News of the severe adverse event did not reach supervisors, the FDA, or field investigators. Despite this confirmation of Wernicke’s encephalopathy, no change was made in the clinical trial protocol. Physicians at the clinical trial sites remained ignorant of this unanticipated serious and potentially fatal development. Patients were not informed of the expanded trial risk.

The FDA confirmed to MPNforum:Sponsors investigating a drug under an IND (Investigative New Drug) are required to notify the FDA and all participating investigators, in a written IND safety report, of any adverse experience associated with the use of the drug that is both serious and unexpected.”

According to the MPNforum report: “Revealing the deep and deadly ignorance that permeated the trial, just a few weeks before Sanofi pulled the plug, the company made a milestone payment confirming fedratinib achieved acceptable safety and efficacy results. Sanofi was only days away from filing its FDA approval application when it reversed course and unceremoniously backed out. Trial closed. Drug development terminated. Every myelofibrosis patient on the trial out in the cold. Doctors, patients and overseers all taken by surprise as the seemingly successful study of a good drug spectacularly blew up right at the finish line….”

On November 18, 2013 Sanofi terminated its trial and advised patients to seek medical testing and advice from their physicians. The giant French drug company released scant details and refused to answer questions. The FDA claimed it was unable to respond to Freedom of Information Act requests for additional information due to mandated restrictions.

According to the MPNforum report:More than two full months past trial termination, no official public accounting has been provided, no data on death or injury was made available to patients or their physicians. At least some of the patients are believed to have suffered irreversible neurological and cognitive damage. No one has taken responsibility for failure to diagnose, failure to report, or failure to terminate the trial earlier.” The full story can be accessed at:

  • Wernicke’s encephalopathy (WE) is a serious neurological disorder that can result in disability and death, The disease, caused by depletion of thiamine (vitamin B1) resources, is characterized by confused mental state, unsteady gait, and vision issues. It can be difficult to diagnose but is easily treated in early stages and irreversible in later stages.
  • MPNforum Magazine ( is an international on-line publication serving the myeloproliferative neoplasm patient, caregiver,and healthcare provider communities. A not for profit collective effort of the MPN community, MPNforum, founded in 2011, also publishes the MPN Quarterly
  • Myelofibrosis is a myeloproliferative neoplasm, a rare blood cancer in which fibrosis of the bone marrow capsule can lead to blood production in distant organs, reduced hematopoiesis, severely restricted quality of life and reduced longevity.


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