Agent exhibits activity in leukemias, MDS

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AML cells
The experimental agent prexigebersen (formerly BP1001) was considered well-tolerated and demonstrated early evidence of activity against relapsed/refractory hematologic disorders in a phase 1/1b trial. The drug reduced blasts in the bone marrow and peripheral blood for patients with acute myeloid leukemia (AML), chronic myeloid leukemia (CML), and myelodysplastic syndrome (MDS). When given... [Read Article]
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FDA approves nilotinib for kids with CML

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Nilotinib (Tasigna)
Photo from Novartis
The US Food and Drug Administration (FDA) has expanded the approved indication for nilotinib (Tasigna®) to include the treatment of children. The drug is now approved to treat patients age 1 and older who have newly diagnosed Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in the chronic phase. Nilotinib... [Read Article]
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Drug could improve treatment of CML, team says

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Lab mouse
A microRNA-targeting drug could improve the effectiveness of tyrosine kinase inhibitors (TKIs) against chronic myelogenous leukemia (CML), according to preclinical research published in Nature Medicine. The drug, miristen, targets miR-126, a microRNA expressed in leukemia stem cells (LSCs). Researchers found that miristen “enhanced the anti-leukemic effects of TKI treatment” in mouse... [Read Article]
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CHMP backs bosutinib for newly diagnosed CML

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CML cells
Image by Difu Wu
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended expanding the approved use of bosutinib (BOSULIF) to include treatment of patients with newly diagnosed, chronic phase, Philadelphia chromosome-positive (Ph+) chronic myelogenous leukemia (CML). Bosutinib is currently approved in Europe to treat patients with... [Read Article]
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Expanded UCB product can stand alone

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Mitchell Horwitz, MD
The expanded umbilical cord blood (UCB) product NiCord can be used as a stand-alone graft, according to research presented at the 2018 BMT Tandem Meetings. Researchers found that a single NiCord unit provided “robust” engraftment in a phase 1/2 study of patients with high-risk hematologic malignancies. NiCord recipients had quicker... [Read Article]
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FDA expands approved use of bosutinib in CML

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CML cells
Image by Difu Wu
The US Food and Drug Administration (FDA) has expanded the approved indication for bosutinib (BOSULIF®). The tyrosine kinase inhibitor (TKI) is now approved to treat adults with newly diagnosed, chronic phase, Philadelphia chromosome-positive (Ph+) chronic myelogenous leukemia (CML). Bosutinib has accelerated approval for this indication. The approval was... [Read Article]
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