ASH Conference Features Exciting MPN Updates Including MPNRF Funded Research

American Society of Hematology Conference Features Exciting MPN Updates Including MPNRF Funded Research

Normally the annual meeting of the American Society of Hematology (commonly referred to simply as “ASH”) takes place in a conference center in a major city. True to 2020 style, this year’s conference was all virtual but, fortunately, no less productive!

Of all the takeaways from ASH, perhaps the most exciting was on the final day, Tuesday, which featured a talk selected for its significance by the ASH committee to be included first among a shortlist of “Late Breaking Abstracts”.  In 2019 MPNRF decided to fund a two-year project proposed by Jyoti Nangalia from the University of Cambridge Wellcome Sanger Institute titled “Understanding the timing of acquisition of driver mutations and dynamics of clonal expansion.” The preliminary results of her study were so compelling that this research was featured little over a year later at ASH! In her presentation, Dr. Nangalia detailed how she and her team have been able to track the acquisition and growth of mutations from as early as conception. This research could potentially lead to a better ability to predict when some individuals may present with signs and symptoms of the disease and influence the choice to treat preventatively. Her research continues with the support of MPNRF and donors like you!

The meeting officially opened on Saturday, December 5th with a fireside chat between ASH’s President, Dr. Stephanie Lee, and Dr. Anthony Fauci who provided his thoughts on the Covid19 pandemic, vaccines under development, and how they will likely be distributed. Future studies will focus on elucidating why some individuals with Covid are more symptomatic than others and individuals who are on chemotherapy or other immunosuppressive drugs should receive the vaccine as a matter of priority. “Some degree of immunity is better than no degree of immunity,” Fauci said.

The fireside chat was followed by an oral presentation session chock full of updates for drugs in clinical trials for MPNs. Among those presented were Imetelstat (Geron), Bomedemstat (Imago), Navitoclax (Abbvie), Momelotinib (Sierra Oncology), and Constellation’s CPI-0610. Interestingly, early results in the Imago and Imetelstat trials show early efficacy in patient cohorts that were believed to be difficult to treat – those with the ASXL1 mutation (Imago) and triple negative driver mutations (Imetelstat). The session closed with a great discussion of the positive news that had been shared and Srdan Verstovsek from MD Anderson in Houston noted that it is reflective of a great team effort by MPN doctors, researchers, advocates, and patients. We should also mention that, while the trials above were for MF patients, a later session featuring additional trial updates reported that early results looked promising for a new drug, PTG-300. This is a drug that is in Phase 2 clinical trial for PV patients requiring therapeutic phlebotomies. 

On Sunday Christian Marinaccio from Northwestern University shared his work showing that a loss of LKB1 STK11 in hematopoietic stem cells facilitates leukemic progression of MPNs and may be exacerbated by additional mutations. We also heard additional presentations by John Mascarenhas about the appropriate dosing of Imetelstat, the design of a phase 3 trial as well as Imetelstat’s role as a telomerase inhibitor which may partly explain why the drug is effective in triple-negative patients. Several other researchers presented their latest work in sessions that focused on earlier stage “translational” research for MPNs including some specific genetic markers and splicing factor mutations that may impact disease progression.

ASH 2020 also featured a healthy dose of good news on the Interferon front. Hans Gisslinger from Vienna reported the latest results from the PROUD PV and CONTINUATION PV studies of Ropeg Interferon alfa 2b vs. control arm (predominantly Hydroxyurea). After 5 years of continued monitoring, Interferon continues to show benefit with 82% of patients in the IFN arm of trial are phlebotomy-free vs. 63% in the control arm and 53% on IFN have a Jak2 allele burden of less than or equal to 10% vs. 12% of patients in the control arm. Further, hematocrit was well controlled and there were very few thromboembolic events in the IFN group.  

One of the most interesting discoveries on the topic of Interferon came from Paris where Rafael Daltro De Oliveira showed data on a retrospective cohort study of patients who discontinued Interferon after achieving complete hematological response (CHR).  Not only was there no significant difference in event free and overall survival between the cohort who discontinued Interferon alpha treatment and those who remained on treatment following CHR, the study also found that restarting IFN treatment was successful and 80% of those who did achieved CHR, again.  Stay tuned for the results of MPNRF’s Interferon Initiative which will be completed this spring.

There were many posters at ASH this year addressing various aspects of MPNs including one submitted by Johns Hopkins’ Alison Moliterno sharing data from a cohort of underrepresented minority patients in MPNRF’s myMPN, the patient registry. The size of the cohort highlighted that minority patients are not participating at the same rate as their Caucasian counterparts in the registry but their data also showed that they have a higher burden of disease. The poster was also included in a themed “Poster Walk” in which hematologists from ASH’s Committee on Promoting Diversity led a discussion of various posters addressing issues of healthcare disparities and inclusion chosen from among all those shared at ASH.

In addition to Dr. Nangalia, many of our current and previously funded researchers were featured at ASH including, Drs. Yelena Ginzburg, (Mt. Sinai), Vijay Sankaran, (Boston Children’s Hospital), Vikas Gupta, (University Health Network, Toronto), Stephen Oh (Washington Univ., St. Louis), Ann Mullally, (Dana-Farber), Alison Moliterno, (Johns Hopkins), Catriona Jamieson (UC, San Diego), and Isabelle Plo (INSERM, Paris). Overall ASH 2020 delivered exciting news for MPN patients and promises an exciting year ahead for patients, clinicians and the community at large. Keep an eye out in the weeks ahead for more exciting news about the many drugs in trial and don’t hesitate to reach out to us if you have questions or would like to learn more!

Author: Lindsey Whyte

Lindsey has assisted several rare and ultra orphan disease groups to create research assets to improve treatment prospects for patients. She came to the MPNRF in 2016 to help launch myMPN, a patient-driven registry.

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