ASH 2017 Research Updates

Publishers Intro (by David Wallace):

ASH 2017 AtlantaThis year I covered 5 abstracts from the 2017 American Society of Hematology (ASH) meeting in snow covered Atlanta with 3 renowned MPN experts.  The videos and more details will be forth coming soon.  The first one with Dr. Michael Grunwald covers Patient Reported Symptom Burden and Peripheral Blood Counts Among patients with Polycythemia Vera – An Analysis from the REVEAL Study with “Surprising Findings.”

Two noteworthy research studies that I attended presentations on were:

A two-part study of givinostat in patients with PV:  The Maximum Tolerated Dose selection and the Proof of Concept final results – by Dr. Alessandro Rambaldi, Univerisity of Milan.  This HDAC (histone deacetylases) inhibitor produced an overall response rate of more than 80% according to the ELN Response Criteria.

Open Label Phase I Study of Single Agent Oral RG7388 (idasanutlin) in Patients with Polycythemia Vera and Essential Thrombocythemia – by Dr. John Mascarenhas, Icahn School of Medicine at Mount Sinai, NY.  IDA is well tolerated and demonstrates a clear signal activity in patients with therapy refractory PV.  A multi-center phase II trial of IDA is underway.  More “patient friendly” details can be found here.

MPN Research Foundation shared the following highlights:

What’s old is new again
Claire Harrison presented an analysis of Wernicke’s Encephalopathy (WE) in MPN patients to see if the incidences of WE reported among those on the defunct JAKARTA trial testing Fedratinib areASH 2017 inside higher than the norm for MPN patients.  Fedratinib was discontinued by the drug company Sanofi in 2013 after the FDA put the drug on clinical hold because of instances of WE in the study population.  However, some patients had received benefit and there were doubts about whether WE was related to the drug or not.  Dr. Harrison presented that WE seen in the JAKARTA trial participants was on par with what is to be expected on MPN patients generally. With the recent news about the investment in Fedratinib, this could suggest renewed hope for those patients who did well on the drug.

In this category is also Ropeginterferon (Ropeg), a new formulation of pegylated interferon (commonly known as Pegasys and used off-label by MPN patients).  Hans Gisslinger presented data from a randomized trial comparing Hydroxyurea to Ropeg, stating that patients on Ropeg experienced “high and durable hematologic response and symptom improvement”, with the drug.  They further describe the excellent safety and tolerability profile of Ropeg and mention the drug’s ability to reduce the JAK2 allele burden, which makes it truly novel among other drugs that have been tried in MPNs.

Novel Therapies
Representing an entirely new angle to new therapies for MPN is LCL-161, an oral smac mimetic which is being tested now in high risk MF patients. Naveen Pemmaraju was interviewed by Patient Power at the beginning of their ASH wrap up, describing this drug and how smac mimetics work.

Diagnostic and Prognostic Update
Drs. Tefferi and Vannuchi presented an updated DIPPS  (Dynamic International Prognostic Scoring System) for Myelofibrosis.  This new “Revised” DIPPS-plus” incorporates clinical, cytogenetic and molecular information, in order to provide a comprehensive prognostic tool for facilitating treatment decision-making in PMF.  RDIPSS-plus was most effective in extracting patients with better than expected outcome, from specific DIPSS-plus risk categories.  This is important, especially for doctors who don’t see as many MF patients as the specialists in major cancer centers who nevertheless must make decisions about how best to manage their patients treatment plan.

Click here for the link back to MPNRF summary.

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