Another drug bites the dust: how to stay hopeful when frustration is all around

Dr. Srdan Verstovsek spoke with Patient Power during ASCO early this month. During his interview he gave a summary of the state of JAK inhibitor trials and drug trials in MPN generally. 

The MPN community is approaching 10 years from approval of Jakafi, with no new drug breakthroughs since. After the reports made in November 2016 regarding the Momelotinib trial, we were skeptical that Gilead would take Momelotinib to the finish line, and during ASCO we heard definitively that is so. Given that there are by our count at least 20 different compounds or combinations being tested in PV, ET or MF, we are left wondering not only why, but what can we as a patient advocacy organization focused on research do about it? 

We bring our perspective about the need for more drug options beyond the usual suspects to each meeting with drug developers and academic scientists. We have also shared this POV with the FDA multiple times, and will also be speaking to them about this September 2017 as part of their series of rare cancer meetings. The goal is to not only share the thoughts and struggles of the patient community, but to also learn about what ways we can be more effective in seeing more safe and effective therapies brought to patients, sooner. 

We'd love to hear from you: what message do you want the FDA and drug makers to hear about your need for more drug options? What role do you think the Foundation should play? Send us an email at with your thoughts and we'll include your answers (without your name) in a follow up post. 


Michelle Woehrle
Executive Director, MPN Research Foundaiton
MPN Community News and Events

Upcoming Events for Patients and Caregivers
June 17, 2017 - MPN Canadian Network is having a meeting in Montreal (Free) Click here for details
June 24, 2017 - Patient Power MPN event at Northwestern in Chicago, IL (Free)  Click here for details

UT San Antonio makes Dr. Ruben Mesa head of their Cancer Center
Congratulations to Dr. Mesa, who is accepting the role of the head of the University of Texas at San Antonio's Cancer Center, where he will remain committed to MPNs. Click here to read more
Give strength to hope: support ground breaking MPN research into PV, ET and MF today!
Make MPN Research part of your legacy.  
Find out more about including the MPN Research Foundation in your estate. Click here for information or reach out to Bill Crowley at 312-683-7226 or 

Phase 2 Study of Ruxolitinib Versus Anagrelide in Subjects With Essential Thrombocythemia Who Are Resistant to or Intolerant of Hydroxyurea (RESET-272)

The purpose of this study is to evaluate the efficacy and safety of ruxolitinib versus anagrelide in subjects with essential thrombocythemia who are resistant to or intolerant of hydroxyurea.  Primary Outcome Measures are efficacy of ruxolitinib versus anagrelide as measured by proportion of subjects who achieve platelet and white blood cell (WBC) control. Secondary Outcome Measures are safety and tolerability of ruxolitinib measured by adverse events and proportion of subjects who achieve complete remission or partial remission. Click here for more information .

Polycythemia Vera, Myelofibrosis and Essential Thrombocythemia: Identification of PV, MF & ET Genes

The purpose of this project is to find genes whose mutations cause Polycythemia Vera, Essential Thrombocythemia and Primary Myelofibrosis. The Primary Outcome Measures are to identify genes whose mutations cause Polycythemia Vera, Essential Thrombocythemia and Primary Myelofibrosis. Secondary Outcome Measures are to determine if there are proteins expressed by cells from patients that might be targets for the immune response. Click here for more information.