Hematopoiesis News 8.40 October 10, 2017 | |
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TOP STORYGene Therapy Halts Progression of Cerebral Adrenoleukodystrophy in Clinical Trial In a clinical trial, a gene therapy to treat cerebral adrenoleukodystrophy — a neurodegenerative disease that typically claims young boys’ lives within ten years of diagnosis — effectively stabilized the disease’s progression in 88 percent of patients. [Press release from the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center discussing online prepublication in The New England Journal of Medicine] Press Release 1 | Press Release 2 | Press Release 3 | Full Article | Editorial | |
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PUBLICATIONS(Ranked by impact factor of the journal)Scientists used CRISPR/Cas9 genome engineering of primary adult and umbilical cord blood CD34+ human hematopoietic stem and progenitor cells, the cells of origin for myeloid pre-malignant and malignant diseases, followed by transplantation into immunodeficient mice to generate genetic models of clonal hematopoiesis and neoplasia. [Cell Stem Cell] Full Article | Press Release | Graphical Abstract The Telomere Binding Protein Pot1 Maintains Hematopoietic Stem Cell Activity with Age Investigators showed that protection of telomeres 1A (Pot1a), a component of the Shelterin complex that protects telomeres, improves HSC activity during aging. Pot1a was highly expressed in young HSCs, but declined with age. In mouse HSCs, Pot1a knockdown increased DNA damage response and inhibited self-renewal. [Nat Commun] Full Article A small molecule screen identified prostaglandin E2 (PGE2) as a positive mediator of lentiviral transduction of CD34+ cells. Supplementation with PGE2 increased lentiviral vector transduction of CD34+ cells approximately two-fold compared to control transduction methods with no effect on cell viability. [Mol Ther] Abstract | Full Article Using a mouse bone marrow retroviral transduction/transplantation approach combined with tamoxifen-inducible, CreERT2-mediated stem cell factor spalt-like transcription factor 4 (Sall4) gene deletion, scientists studied SALL4 functions in leukemic transformation that was induced by mixed lineage leukemia (MLL)-AF9 — one of the most common MLL-rearranged oncoproteins found in patients. [J Hematol Oncol] Full Article The authors showed that deletion of Ca2+/calmodulin-dependent protein kinase kinase 2 (CaMKK2) promotes hematopoietic stem and progenitor cell (HSPC) regeneration and hematopoietic recovery following radiation injury. Using Camkk2-enhanced green fluorescent protein reporter mice, they found that Camkk2 expression is developmentally regulated in HSPC. [Cell Death Dis] Full Article Researchers elucidated benzene’s effect on the lineages-committed progenitors and genes-regulating self-renewal and differentiation of hematopoietic stem and progenitor cells. 1,4-BQ exposure significantly lowered the clonogenicity of the myeloid progenitor at 1.25 and 2.5 μM, but affected neither the erythroid nor Pre-B lymphoid progenitors. [Toxicol In Vitro] Abstract CLINICAL RESEARCHThe authors present a cohort of 14 patients from six countries who received hematopoietic stem cell transplantation (HSCT) for deficiency of adenosine deaminase 2 (DADA2). In 14 patients, HSCT was an effective and definitive treatment for DADA2. [Blood] Abstract Investigators compared the incidence of secondary solid cancer in a monocentric cohort of thalassemia major (TM) patients who received hematopoietic cell transplant versus a hematopoietic cell donor monocentric cohort and versus a large multicenter cohort of age- and sex-matched TM patients who received conventional therapy. [Bone Marrow Transplant] Abstract | |
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REVIEWSImpact of DNA Methylation Programming on Normal and Pre-Leukemic Hematopoiesis The authors outline the current view of the hematopoietic system and the epigenetic mechanisms regulating hematopoiesis under homeostatic conditions, with a particular focus on the role of DNA methylation in this process. [Semin Cancer Biol] Abstract Visit our reviews page to see a complete list of reviews in the hematopoiesis research field. | |
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SCIENCE NEWSSyros Pharmaceuticals announced that the biomarker status of patients screened for the ongoing Phase II clinical trial of SY-1425, its first-in-class selective retinoic acid receptor alpha agonist, in genomically defined subsets of patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), was predictive of myeloid cell differentiation in the patients’ blood samples treated ex vivo with SY-1425. [Press release from Syros Pharmaceuticals, Inc. discussing research presented at the European School of Haematology (ESH)’s 4th International Conference on Acute Myeloid Leukemia ‘Molecular and Translational’ Advances in Biology and Treatment, Estoril] Press Release | |
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INDUSTRY NEWSFDA Awards 15 Grants for Clinical Trials to Stimulate Product Development for Rare Diseases The FDA announced that it has awarded 15 new clinical trial research grants totaling more than $22 million over the next four years to boost the development of products for patients with rare diseases. [U.S. Food and Drug Administration] Press Release FDA Awards Six Grants for Natural History Studies in Rare Diseases The FDA announced it has awarded six new research grants for natural history studies in rare diseases. The aim of the research is to inform medical product development by better understanding how specific rare diseases progress over time. [U.S. Food and Drug Administration] Press Release Nohla Therapeutics Inc. announced that it has received a $6.9 million grant from the California Institute of Regenerative Medicine. The grant will support the ongoing clinical development of Nohla’s lead regenerative cell therapy program, NLA101. NLA101 is an off-the-shelf ex vivo expanded hematopoietic stem and progenitor cell therapy that has the potential to provide functional bridging hematopoiesis in patients receiving myeloablative or high-dose chemotherapy. [Nohla Therapeutics Inc.] Press Release Porteus Awarded Grant for Work on Possible Treatment for Sickle Cell Anemia The California Institute for Regenerative Medicine has awarded a researcher a grant of $5.2 million to lay the groundwork for a clinical trial of a possible treatment for sickle cell disease. [Stanford Medicine] Press Release 1 | Press Release 2 Actinium Pharmaceuticals, Inc. provided an update on its Actimab-A drug candidate in acute myeloid leukemia (AML). The Phase II trial is currently enrolling patients with AML age 60 and older who are unfit for standard induction chemotherapy. [Actinium Pharmaceuticals, Inc.] Press Release Trovagene, Inc. announced that the FDA granted an orphan drug designation to PCM-075 for the treatment of patients with AML. PCM-075 is an oral, highly-selective adenosine triphosphate competitive inhibitor of the serine/threonine polo-like kinase 1 (PLK1) enzyme, which appears to be over expressed in several different hematologic malignancies and solid tumor cancers. [Trovagene, Inc.] Press Release | |
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POLICY NEWSPublishers Threaten to Remove Millions of Papers from ResearchGate Millions of articles might soon disappear from ResearchGate, the world’s largest scholarly social network. Five publishers said they had formed a coalition that would start ordering ResearchGate to remove research articles from its site because they breach publishers’ copyright. [Nature News] Editorial Navajo Nation Reconsiders Ban on Genetic Research When the Navajo Nation opens its first oncology center next year in Tuba City, Arizona, clinicians there may be able to offer a service that has been banned on tribal lands for 15 years: analyzing the DNA of Navajo tribe members to guide treatments and study the genetic roots of disease. [Nature News] Editorial Scientists Plead with Brazilian Government to Restore Funding Anxiety is growing in Brazil over the country’s collapsing research budgets. President Michel Temer had slashed funding for science by 44% and has proposed additional decreases for 2018 — even as some science institutes run out of money for basic needs, such as paying electricity bills. The 2017 science budget, at 3.2 billion reais (US$1 billion), is the lowest the country has seen in at least 12 years. [Nature News] Editorial
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EVENTSNEW Keystone Symposia: Precision Medicine in Cancer Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESPostdoctoral Fellow – Hematopoietic Stem Cells (Memorial Sloan Kettering Cancer Center) Staff Scientist – Translational Hematology (University of Copenhagen) Postdoctoral Positions – Stem Cell and Cancer Biology (Johns Hopkins University) Scientist – Hematopoietic Precursors and Immune Cells (Università di Genova) Postdoctoral Opportunities – Hematological Malignancies (Albert Einstein College of Medicine) Postdoctoral Scholar – Hematopoiesis and Leukemia (Penn State College of Medicine) Director – Allogeneic Hematopoietic Stem Cell Transplantation (Princess Margaret Cancer Centre) Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.
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