Science & Medicine

Posts tagged ‘myelofibrosis’

Gilead and momelotinib. An update.



Momelotinib…  An alternative to Jakafi?

Maybe…but don’t hold your breath waiting for momelotnib

Overall, I’m a little disappointed, having heard momelotinib was finally going to report exciting results. A news release published by Gilead doesn’t have much to write home about.

In SIMPLIFY-1, the trial of momelotinib v. rux or BAT, the MF momelotinib pts  who were never on rux  apparently reached the ≥ 35 spleen reduction outcome. Jakafi reported the same six years ago. They also hit the three anemic endpoints  so maybe there’s something there, not specified in the release but worth looking at in the final paper.

In that weird PR-speak favored by some pharmas, Gildead reported:

“Non-inferiority was not achieved for the key secondary endpoint of response rate in total symptom score (TSS)”  

Unpacking the double negative gobbledygook of that sentence, it simply says momelotinib was inferior to ruxolitinib in improving symptoms.

Adverse events seem a bit worse than those Jakafi reports– peripheral neuropathy twice as common —  except for the anemia benefit.

The SIMPLIFY-2 study — MF pts who had rec’d rux in the past on momelotinib or BAT arms —  appears to be a bust. Most pts on BAT continued to receive rux (88%)  There was some claimed benefit but   “SIMPLIFY-2 did not achieve its primary endpoint of superiority of momelotinib compared to BAT in patients previously treated with ruxolitinib ” Still, having an alternative to Jakafi for patients for whom it fails, might be a good thing, particularly for those of us who are transfusion dependent.

The Full Release is here:

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!

The ASH Report


forumsphere stretched


The theme of MPNforum’s  Orlando ASH Report is “Behind the Scenes.”

Most of us never get to ASH… so this time we show you what it’s really like. Come on inside the CREATE MPN gene editing luncheon… and then head on out to see a surprising about-face at the MPN Heroes’ shindig. There’s all  that plus a quick overview of drugs and a couple of clinical trials (and a partridge in a pear tree.)

Enjoy… As always, this is an Advance Subscriber edition so if you find errors worth correcting, please let me know.

You can find it all at:

                              Happy Holidays,


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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