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IACH Focus on Leukemia 2020 | Major breakthroughs in BPDCN treatment

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Naveen Pemmaraju

Naveen Pemmaraju, MD, University of Texas MD Anderson Cancer Center, Houston, TX, discusses the major breakthroughs in blastic plasmacytoid dendritic cell neoplasm (BPDCN) research. The first breakthrough finding was that CD123 is overexpressed in all cases of BPDCN. Secondly, due to the discovery of CD123 overexpression, the first therapy for BPDCN, tagraxofusp, has been approved by the FDA. Finally, there have been developments in other novel therapies such as other targeted CD123 agents, BCL-2 inhibitors and combinations with chemotherapy. This interview took place during the 2020 Annual Meeting of the International Academy for Clinical Hematology (IACH).

Transcript (edited for clarity)

It’s always an honor to present at the IACH meeting. I really think this is one of the great meetings in the world each year put on by Professor Mohamad Mohty, and it was my honor to be able to join this year virtually for the very first time due to the coronavirus pandemic. I was able to present the invited talk on management and state-of-the-art of plasmacytoid dendritic cell neoplasm or BPDCN...

It’s always an honor to present at the IACH meeting. I really think this is one of the great meetings in the world each year put on by Professor Mohamad Mohty, and it was my honor to be able to join this year virtually for the very first time due to the coronavirus pandemic. I was able to present the invited talk on management and state-of-the-art of plasmacytoid dendritic cell neoplasm or BPDCN. The full name is blastic plasmacytoid dendritic cell neoplasm leukemia.

And my talk was centered around this emerging field that I have dedicated my career to, which really boils down to three major items. So number one is the breakthrough finding that CD123 or IL-3 receptor alpha is over-expressed in 100% of cases. So that’s important for everybody to know. Number two, this important finding has led to therapeutic breakthroughs, including the first and only drug that’s FDA approved, which I was able to lead a multi-center group towards, which is known as SL-401 or tagraxofusp, CD123 targeted therapy approved for BPDCN patients ages two and older. This was in the New England Journal of Medicine by me and my co-authors, April of 2019. FDA approved here in the United States almost two years ago. And then the third finding is, we are now building in two separate areas for a novel therapy. So other CD123 targeted agents, which are now actively in clinical trials for BPDCN and AML, and then a second route, which is novel therapies altogether. So BCL2, so with venetoclax and other therapies, and then in that same bucket combination, so CD123 combined with BCL2 combined with chemotherapy. So I think these are the three major items and breakthroughs that I was able to present here in 2020 in the field of BPDCN.

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