Bomedemstat as an investigative treatment for myeloproliferative neoplasms

Expert Opin Investig Drugs. 2023 Oct 7. doi: 10.1080/13543784.2023.2267980. Online ahead of print.ABSTRACTINTRODUCTION: Myeloproliferative neoplasm (MPN) are heterogeneous group of hematopoietic stem cell disorders characterized by clonal proliferation of one of more of the hematopoietic stem cell lineages. Clinical manifestations result from uncontrolled myeloproliferation, extramedullary hematopoiesis with splenomegaly and excessive inflammatory cytokine production. Currently available therapy improves hematologic parameters and symptoms but don't adequately address the underlying neoplastic biology. Bomedemstat has thus far demonstrated clinical efficacy and tolerability in the treatment of MPNs with recent evidence of impacting the malignant stem cell population.AREAS COVERED: This reviews summarizes the mechanisms of action, pharmacokinetics and pharmacodynamics, safety and efficacy of bomedemstat in MPN with specific emphasis on essential thrombocythemia (ET) and myelofibrosis (MF).EXPERT OPINION: In patients with MPNs bomedemstat appears effective and well tolerated. The signs and symptoms of these disease are managed as A reduction in the frequency of mutant cells was demonstrated in patients with ET and MF. Ongoing and planned studies of bomedemstat in MPN will establish the position of bomedemstat in MPNs and may help to redefine treatment endpoints of MPNs in the future.PMID:37804041 | DOI:10.1080/13543784.2023.2267980
Source: Expert Opinion on Investigational Drugs - Category: Drugs & Pharmacology Authors: Source Type: research