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http://dx.doi.org/10.1016/j.htct.2024.09.2484 | DOI Listing |
Expert Opin Pharmacother
February 2025
Hospices Civils de Lyon, Department of Clinical Hematology, Centre Hospitalier Lyon-Sud, Pierre Bénite, France.
Introduction: The aim of treatment in very low-, low- and intermediate-1-risk myelodysplastic syndrome (MDS) is mainly to relieve symptoms due to cytopenias. Only a few therapeutic drugs are currently available, but novel drugs are under clinical investigations. In this setting, imetelstat, a telomerase inhibitor, is a promising new agent.
View Article and Find Full Text PDFClin Transl Sci
February 2025
Morcos Pharmaceutical Consulting, LLC, Marlboro, New Jersey, USA.
Evaluation of the proarrhythmic potential of imetelstat, a novel oligonucleotide telomerase inhibitor, in nonclinical and clinical studies is presented. In vitro, imetelstat sodium ≤ 750 μg/mL and negative (vehicle) and positive (cisapride) controls were evaluated for hERG channel current inhibition. In vivo, cynomolgus monkeys received a single vehicle control or imetelstat sodium (5 mg/kg [2-h infusion], 10 mg/kg [6-h infusion], or 15 mg/kg [6- or 24-h infusion]); cardiovascular parameters were collected before and after drug administration.
View Article and Find Full Text PDFBr J Pharmacol
April 2025
Department of Pharmacology and Pharmacotherapy, Semmelweis University, Budapest, Hungary.
In the past year, the European Medicines Agency (EMA), the Food and Drug Administration (FDA) and the Medicines and Healthcare Products Regulatory Agency (MHRA) authorised 53 novel drugs. While the 2024 harvest is not as rich as in 2023, when 70 new chemical entities were approved, the number of 'orphan' drug authorisations in 2024 (21) is similar to that of 2023 (24), illustrating the dynamic development of therapeutics in areas of unmet need. The 2024 approvals of novel protein therapeutics (15) and advanced therapy medicinal products (ATMPs, 6) indicate a sustained trend also noticeable in the 2023 new drugs reviewed in this journal last year (16 and 11, respectively).
View Article and Find Full Text PDFHematol Transfus Cell Ther
February 2025
Liaquat National Hospital, Karachi, Pakistan.
Haematologica
February 2025
Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf.
Myelodysplastic syndrome (MDS) is considered to be a heterogeneous myeloid malignancy with a common origin in the hematopoietic stem cell compartment and is generally divided into lower- and higher-risk forms. While the treatment goals for lower-risk MDS are to decrease transfusion requirements and transformation into acute leukemia, the major aims for higher-risk MDS are to prolong survival and ultimately cure the patient. Although novel agents such as luspatercept and imetelstat have recently been approved as new treatment options for lower-risk MDS, hypomethylating agents currently remain the only approved non-transplant option for higher-risk MDS and are the standard of care for patients not eligible for allogeneic hematopoietic stem cell transplantation (HSCT).
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