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Nanoparticles from Marine Biomaterials for Cancer Treatment
Published in Se-Kwon Kim, Marine Biochemistry, 2023
The U.S. Food and Drug Administration (FDA) has approved a number of important anticancer drugs derived from marine organisms, such as cytarabine (Cytosar U®), a chemotherapeutic drug used to treat leukemia. Trabectedin (Yondelis®) is another antitumor agent that is used to treat advanced soft tissue sarcoma. Breast, prostate, and pediatric sarcomas are all in clinical studies. Another anticancer medication, eribulin mesylate (Halaven®), is used to treat breast cancer and liposarcoma. Brentuximab vedotin (Adcetris®) is an antibody-drug conjugate (ADC) medication used to treat HL and anaplastic large cell lymphoma that has relapsed or become resistant. Midostaurin (Rydapt®) is a protein kinase inhibitor used to treat acute myeloid leukemia and myelodysplastic syndrome (van Andel et al. 2018). Furthermore, 19 anticancer substances obtained from the sea are at various stages of clinical testing (Zuo and Kwok 2021). Numerous studies have also demonstrated the anticancer properties of marine-derived chemicals in vitro or in vivo.
Leukemias
Published in Pat Price, Karol Sikora, Treatment of Cancer, 2020
For patients with MPNs, treatment emphasis is on the reduction of risks for thrombosis and bleeding, and symptom control. For patients with higher-risk MF, there are two licensed JAK inhibitors that accord rapid and substantial clinical benefits but few molecular remissions and no significant impact on transformation to secondary AML. Hydroxycarbamide (hydroxyurea) remains the standard treatment in high-risk patients with PV and ET. IFNα and ruxolitinib are useful as second-line therapies for patients with PV. Midostaurin is now licensed for the treatment of patients with SM.
Pemigatinib for the treatment of myeloid/lymphoid neoplasms with FGFR1 rearrangement
Published in Expert Review of Anticancer Therapy, 2023
Craig W. Freyer, Mitchell E. Hughes, Alison Carulli, Adam Bagg, Elizabeth Hexner
Pre-clinical data have also demonstrated the anti-leukemic activity of midostaurin (PKC412) in MLNFGFR1 cell lines with ZNF198–FGFR1 gene fusion. Midostaurin is a FLT3, KIT, PDGFR, and protein kinase C inhibitor that is now FDA approved for AML and systemic mastocytosis. In this early report, a single patient with MLNFGFR1 with ZNF198-FGFR1 was treated with single agent midostaurin 100 mg by mouth twice daily. The patient experienced reduction in peripheral blood leukocytosis, lymphadenopathy, and splenomegaly but with a persistently hypercellular bone marrow with all metaphases positive for t(8;13). The patient remained clinically stable through 6 months of midostaurin monotherapy and then proceeded to alloHSCT [42]. To our knowledge no additional reports of midostaurin in the management of MLNFGFR1 have been published.
Avapritinib for the treatment of KIT mutation–negative systemic mastocytosis
Published in Baylor University Medical Center Proceedings, 2023
Farhan Azad, Jiahua Zhang, Eunice Wang
Therapies traditionally used for SM have largely been ineffective. Imatinib and nilotinib are tyrosine kinase inhibitors that have shown limited response against D816V.5 D816V mutation shows resistance to imatinib via a change in receptor conformation that blocks the drug from binding.6 Similarly, nilotinib has shown only a partial response in MC cytoreduction in patients with a D816V mutation.7 Cladribine, a purine analog, has been shown to destroy MC independent of D816V mutation. However, increased susceptibility to infections secondary to immunosuppression has been reported.8 Hydroxyurea is a DNA synthesis inhibitor that has been shown to provide symptomatic relief.9 Both cladribine and hydroxyurea do not reduce the neoplastic MC burden drastically, and evidence of efficacy is weak since it is from observational studies.10 The emergence of midostaurin and avapritinib have revolutionized the therapy for SM. Midostaurin, a multikinase inhibitor, has shown a promising response. However, adverse effects including nausea, vomiting, and diarrhea have been reported.11
Current and emerging pharmacotherapy for the treatment of childhood acute myeloid leukemia
Published in Expert Opinion on Pharmacotherapy, 2022
Branko Cuglievan, David McCall, Lindsay Robusto, M. Estela Mireles, Suzanne C. Gettys
Midostaurin is an oral multikinase inhibitor that is FDA approved for treating adult patients with newly diagnosed FLT3-positive AML in combination with standard chemotherapy based on the results of the CALGB 10603/RATIFY study. This prospective, randomized trial demonstrated a significantly longer median OS (74.7 months vs 25.6 months, p = 0.009, HR = 0.78) and EFS (8.2 months vs 3 months, p = 0.002) in the midostaurin plus chemotherapy group compared to chemotherapy alone [39]. A phase I/II open-label study was developed for pediatric patients with relapsed/refractory FLT3-positive AML or ALL with rearrangement in the mixed lineage leukemia gene family (MLL). It enrolled 22 patients, 9 of whom had FLT3-positive AML, but was closed early due to poor accrual [40]. A phase II study is currently recruiting newly diagnosed FLT3-positive pediatric AML patients to assess the dose-limiting toxicity of midostaurin and assess safety, tolerability, and efficacy in combination with chemotherapy [41].