Small molecules, big impact: 20 years of targeted therapy in oncology

PL Bedard, DM Hyman, MS Davids, LL Siu - The Lancet, 2020 - thelancet.com
The identification of molecular targets and the growing knowledge of their cellular functions
have led to the development of small molecule inhibitors as a major therapeutic class for …

[HTML][HTML] Integrating next-generation sequencing into clinical oncology: strategies, promises and pitfalls

P Horak, S Fröhling, H Glimm - ESMO open, 2016 - Elsevier
We live in an era of genomic medicine. The past five years brought about many significant
achievements in the field of cancer genetics, driven by rapidly evolving technologies and …

Prediction of risk for myeloid malignancy in clonal hematopoiesis

LD Weeks, A Niroula, D Neuberg, W Wong… - NEJM …, 2023 - evidence.nejm.org
Background Clonal hematopoiesis of indeterminate potential (CHIP) and clonal cytopenia of
undetermined significance (CCUS) are defined by somatic mutations in genes associated …

Single-cell RNA-seq reveals AML hierarchies relevant to disease progression and immunity

P van Galen, V Hovestadt, MH Wadsworth II… - Cell, 2019 - cell.com
Acute myeloid leukemia (AML) is a heterogeneous disease that resides within a complex
microenvironment, complicating efforts to understand how different cell types contribute to …

Ivosidenib induces deep durable remissions in patients with newly diagnosed IDH1-mutant acute myeloid leukemia

GJ Roboz, CD DiNardo, EM Stein… - Blood, The Journal …, 2020 - ashpublications.org
Abstract Ivosidenib (AG-120) is an oral, targeted agent that suppresses production of the
oncometabolite 2-hydroxyglutarate via inhibition of the mutant isocitrate dehydrogenase 1 …

Highly efficient therapeutic gene editing of human hematopoietic stem cells

Y Wu, J Zeng, BP Roscoe, P Liu, Q Yao… - Nature medicine, 2019 - nature.com
Re-expression of the paralogous γ-globin genes (HBG1/2) could be a universal strategy to
ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by …

Ivosidenib or enasidenib combined with intensive chemotherapy in patients with newly diagnosed AML: a phase 1 study

EM Stein, CD DiNardo, AT Fathi… - Blood, The Journal …, 2021 - ashpublications.org
Abstract Ivosidenib (AG-120) and enasidenib (AG-221) are targeted oral inhibitors of the
mutant isocitrate dehydrogenase (mIDH) 1 and 2 enzymes, respectively. Given their …

[HTML][HTML] Reconstructing the lineage histories and differentiation trajectories of individual cancer cells in myeloproliferative neoplasms

D Van Egeren, J Escabi, M Nguyen, S Liu, CR Reilly… - Cell stem cell, 2021 - cell.com
Some cancers originate from a single mutation event in a single cell. Blood cancers known
as myeloproliferative neoplasms (MPNs) are thought to originate when a driver mutation is …

Ultraviolet radiation shapes dendritic cell leukaemia transformation in the skin

GK Griffin, CAG Booth, K Togami, SS Chung, D Ssozi… - Nature, 2023 - nature.com
Tumours most often arise from progression of precursor clones within a single anatomical
niche. In the bone marrow, clonal progenitors can undergo malignant transformation to …

Pediatric acute lymphoblastic leukemia, version 2.2020, NCCN clinical practice guidelines in oncology

P Brown, H Inaba, C Annesley, J Beck… - Journal of the National …, 2020 - jnccn.org
Acute lymphoblastic leukemia (ALL) is the most common pediatric malignancy.
Advancements in technology that enhance our understanding of the biology of the disease …