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MPN Workshop of the Carolinas 2024 | Major breakthroughs in the field of BPDCN: combination therapies, managing CNS involvement & more
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and highly emerging disease. In this video, Naveen Pemmaraju, MD, The University of Texas MD Anderson Cancer Center, Houston, TX, outlines three crucial breakthroughs in the field that could provide hope for patients. Dr Pemmaraju discusses the potential of trials investigating tagraxofusp combination therapies and comments on the discovery of central nervous system (CNS) involvement, which has led to changes in clinical practice. To conclude, Dr Pemmaraju highlights the development of other CD123-targeting agents, such as pivekimab sunirine, which have shown significant potential in BPDCN treatment. This interview took place at the 1st Annual MPN Workshop of the Carolinas in Asheville, NC.
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Transcript
So BPDCN, blastic plasmacytoid dendritic cell neoplasm, is a rare but highly emerging disease that many of my colleagues are starting to find out about. My group and I have been working on this disease, this field, for over 15 years now, and we have three exciting and important breakthroughs that I think can provide hope for our patients in the field.
One is that we and others have shown that BPDCN is highly CD123 overexpressing, that’s a surface marker on all of these cancer cells, and so we have already led the first and only approved drug tagraxofusp or tag exactly six years ago to FDA approval...
So BPDCN, blastic plasmacytoid dendritic cell neoplasm, is a rare but highly emerging disease that many of my colleagues are starting to find out about. My group and I have been working on this disease, this field, for over 15 years now, and we have three exciting and important breakthroughs that I think can provide hope for our patients in the field.
One is that we and others have shown that BPDCN is highly CD123 overexpressing, that’s a surface marker on all of these cancer cells, and so we have already led the first and only approved drug tagraxofusp or tag exactly six years ago to FDA approval.
So that drug has been on the market, we’ve been using it as a single agent with high response rates, particularly in the frontline setting. But what’s very exciting is we’re working on combination studies, doublets and now triplets, combining the tag with aza/ven in an ongoing clinical trial, or with hyper-CVAD ALL-based chemotherapy, also an enrolling clinical trial. So both of those are open and available at our center and we’re looking for patients actively.
The second exciting breakthrough is that we found out that a lot of these patients have central nervous system involvement, way more than we thought before, and more than in ALL and AML patients, 20%-30% plus of these patients. So we’ve made the practice changing finding after my publication in Blood a few years ago to incorporate lumbar puncture intrathecal chemos, the number is still being debated, but multiple intrathecal chemos for CNS prophylaxis. So that’s just to prevent CNS because it’s so common, and then more if it’s needed to treat. So I think that’s a big one to help patient outcomes, particularly lower CNS relapses.
The third exciting development has been the development of other drugs in the BPDCN field, building on the CD123 story, there was an exciting drug program that I led called the IMGN632 of pivekimab sunirine where we published or presented those results in the EHA meeting published in the Abstract Guide a year ago, showing that as a monotherapy that had high activity rates in BPDCN, and that trial and data is ongoing.
So I think these are some of the exciting updates in the field for BPDCN.
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Disclosures
Consultancy: ClearView Healthcare Partners, Imedex, Protagonist Therapeutics Inc., EUSA Pharma, Curio Science, CareDx, Harborside Press, Dava Oncology, CancerNet, Aplastic Anemia & MDS International Foundation, Pacylex, Patient Power, Menarini Group, Astellas, BMS, PeerView Institute for Medical Education, Stemline, Medscape, OncLive, Physician Education Resource (PER), Celgene, PharmaEssentia, Novartis Pharmaceuticals:, Magdalen Medical Publishing, Neopharm, Intellisphere, Blueprint, Cimeio Therapeutics AG, ImmunoGen, CTI BioPharma, Incyte, Aptitude Health, AbbVie, Bristol Myers Squibb Pharmaceuticals; Membership on Board of Directors/Advisory Committees: ClearView Healthcare Partners, Imedex, Protagonist Therapeutics Inc., EUSA Pharma, Curio Science, Dan’s House of Hope, CareDx, Harborside Press, Dava Oncology, CancerNet, Aplastic Anemia & MDS International Foundation, Pacylex, Patient Power, Menarini Group, Astellas, BMS, PeerView Institute for Medical Education, Stemline, Medscape, OncLive, Physician Education Resource (PER), Celgene, PharmaEssentia, Novartis Pharmaceuticals:, Magdalen Medical Publishing, Neopharm, Intellisphere, Blueprint, Cimeio Therapeutics AG, ImmunoGen, CTI BioPharma, Incyte, Aptitude Health, AbbVie, Bristol Myers Squibb Pharmaceuticals; Speakers Bureau: ClearView Healthcare Partners, Imedex, EUSA Pharma, Curio Science, CareDx, Harborside Press, Dava Oncology, CancerNet, Aplastic Anemia & MDS International Foundation, Pacylex, Patient Power, Menarini Group, Astellas, BMS, PeerView Institute for Medical Education, Medscape, OncLive, Physician Education Resource (PER), Celgene, PharmaEssentia, Novartis Pharmaceuticals, Magdalen Medical Publishing, Neopharm, Intellisphere, Blueprint, Cimeio Therapeutics AG, ImmunoGen, CTI BioPharma, Incyte, Aptitude Health, AbbVie, Bristol Myers Squibb Pharmaceuticals; Research Funding: United States Department of Defense (DOD), National Institute of Health/National Cancer Institute (NIH/NCI); Licenses: Karger Publishers; Leadership role on ASH Committee on Communications and ASCO Cancer.Net Editorial Board.
