Drug under priority review for BPDCN

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Vials of drug
Photo by Bill Branson
The US Food and Drug Administration(FDA) has accepted for priority review the biologics license application seeking approval for tagraxofusp (Elzonris, SL-401) to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN). The FDA expects to make a decision on this application by February 21, 2019. The FDA grants priority review... [Read Article]
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Familial risk of myeloid malignancies

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Three generations of
women in a family
A large study has revealed “the strongest evidence yet” supporting genetic susceptibility to myeloid malignancies, according to a researcher. The study showed that first-degree relatives of patients with myeloid malignancies had double the risk of developing a myeloid malignancy themselves, when compared to the general population. The researchers... [Read Article]
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Ixazomib could improve treatment of AML

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Lab mouse
New research suggests the FOXM1 protein plays an important role in acute myeloid leukemia (AML) progression, and targeting FOXM1 could improve AML treatment. With a retrospective study, researchers showed that overexpression of FOXM1 was associated with increased resistance to chemotherapy and inferior overall survival. Subsequent preclinical research showed that ixazomib inhibits FOXM1, exhibits... [Read Article]
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Orphan designation recommended for PCM-075

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AML cells
Image by Lance Liotta
The European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) has recommended that PCM-075 receive orphan drug designation as a treatment for acute myeloid leukemia (AML). PCM-075 is an oral adenosine triphosphate competitive inhibitor of the serine/threonine Polo-like kinase 1 (PLK1) enzyme, which is overexpressed in hematologic and... [Read Article]
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Method may enable eradication of LSCs in AML

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Micrograph showing LSCs
Image by Robert Paulson
Disrupting mitophagy may be a “promising strategy” for eliminating leukemia stem cells (LSCs) in acute myeloid leukemia (AML), according to researchers. The team found that AML LSCs depend on mitophagy to maintain their “stemness,” but targeting the central metabolic stress regulator AMPK or the mitochondrial dynamics regulator FIS1... [Read Article]
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Inhibitor receives breakthrough designation for AML

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AML cells
The US Food and Drug Administration (FDA) has granted breakthrough therapy designation to quizartinib, an investigational FLT3 inhibitor, for the treatment of adults with relapsed/refractory FLT3-ITD acute myeloid leukemia (AML). The FDA granted quizartinib breakthrough designation based on results from the phase 3 QuANTUM-R study, which were presented at the 23rd Congress of... [Read Article]
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Study could change treatment of MLSM7

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Study authors Tamara
Lamprecht, Jason Schwartz,
Jing Ma, and Jeffrey Klco
Photo from St. Jude
Children’s Research
Hospital/Justin Veneman
New findings could help improve treatment of an inherited bone marrow disorder known as myelodysplasia and leukemia syndrome with monosomy 7 (MLSM7), according to researchers. While studying families affected by MLSM7, researchers identified germline mutations... [Read Article]
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Treatments, disease affect spermatogonia in boys

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Male germinal epithelium
showing spermatogonia,
spermatocytes, spermatids,
and spermatozoa
Image from Dreamstime
Alkylating agents, hydroxyurea (HU), and certain non-malignant diseases can significantly deplete spermatogonial cell counts in young boys, according to research published in Human Reproduction. Boys who received alkylating agents to treat cancer had significantly lower spermatogonial cell counts than control subjects or boys... [Read Article]
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FDA approves drug for IDH1-mutated AML

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Ivosidenib (Tibsovo®)
Photo courtesy of
Agios Pharmaceuticals
The US Food and Drug Administration (FDA) has granted full approval for the isocitrate dehydrogenase-1 (IDH1) inhibitor ivosidenib (Tibsovo®). The drug is approved to treat adults with relapsed or refractory acute myeloid leukemia (AML) who have an IDH1 mutation, as detected by an FDA-approved test. Ivosidenib was approved... [Read Article]
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FDA approves biosimilar filgrastim

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Vials of drug
Photo by Bill Branson
The US Food and Drug Administration (FDA) has approved the leukocyte growth factor Nivestym™ (filgrastim-aafi), a biosimilar to Neupogen (filgrastim). Nivestym is approved to treat patients with nonmyeloid malignancies who are receiving myelosuppressive chemotherapy or undergoing bone marrow transplant, acute myeloid leukemia patients receiving induction or consolidation chemotherapy,... [Read Article]
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Study suggests dasatinib could treat AML, JMML

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Micrograph showing JMML
© Universitätsklinikum Freiburg
New research suggests dasatinib could treat certain patients with juvenile myelomonocytic leukemia (JMML) or acute myeloid leukemia (AML). The study showed that TNK2 inhibition has a negative effect on PTPN11-mutant leukemias. PTPN11-mutant JMML and AML cells were sensitive to treatment with dasatinib, which inhibits TNK2. Dasatinib also induced hematologic... [Read Article]
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Explaining enasidenib resistance in AML

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Enasidenib (IDHIFA®)
Photo from Business Wire
New research helps explain enasidenib resistance among patients with IDH2-mutant acute myeloid leukemia (AML). Researchers found that leukemic cells stop responding to enasidenib when IDH2 clones develop additional mutations. This may mean that enasidenib will have to be combined with other drugs to prevent AML relapse, the researchers said.... [Read Article]
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