SciBX: Science-Business eXchange Contents: June 5 2014, Volume 7 / Issue 22

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TABLE OF CONTENTS June 5 2014, Volume 7 / Issue 22 Analysis Cover Story Translational Notes Targets and Mechanisms The Distillery: Therapeutics Cancer Cardiovascular disease Endocrine/metabolic disease Musculoskeletal disease Neurology Ophthalmic disease Other The Distillery: Techniques Assays and screens Disease models Drug delivery Drug platforms Advertisement Biopharma Dealmakers A supplement to Nature Biotechnology and Nature Reviews Drug Discovery The November 2013 issue of Biopharma Dealmakers showcases companies with partnering opportunities. This week, find out about how you can collaborate with QRxPharma.    Analysis Cover Story Top Alas, porphyria Amy Donner doi:10.1038/scibx.2014.631 Treatment of acute porphyrias has long relied on hemin infusions, but the therapy has many drawbacks. A team led by Alnylam has created an siRNA therapeutic that reduces disease symptoms in mice. The company still needs a strategy to reduce misdiagnosis of the rare disorder. Full Text | PDF Translational Notes Top Go west, FDA C. Simone Fishburn doi:10.1038/scibx.2014.632 The FDA's first two centers for regulatory sciences were focused on modernizing methods and building bridges to academia. The agency now is at earlier drug development and has partnered with Stanford and UCSF to create a center focused on quantitative pharmacology that will give the FDA a foothold on the West Coast. Full Text | PDF Sanford-Burnham goes fourth Michael J. Haas doi:10.1038/scibx.2014.633 The Sanford-Burnham Medical Research Institute has been seeking broader industrial partnerships that tap both its basic research and drug discovery expertise. Its new deal with Daiichi is the most expansive of its four recent industry partnerships. Full Text | PDF Targets and Mechanisms Top De-stressing glaucoma Benjamin Boettner doi:10.1038/scibx.2014.634 Glucocorticoids are standard care for a host of allergic and inflammatory eye conditions, but they can elevate intraocular pressure and result in secondary open-angle glaucoma. A chemical chaperone called sodium phenylbutyrate could eliminate that side effect, according to new research from the University of Iowa. Full Text | PDF Distillery: Therapeutics Cancer Top BET bromodomain proteins; myeloid-lymphoid or mixed-lineage leukemia 3 (MLL3) doi:10.1038/scibx.2014.635 In vitro and mouse studies suggest BET inhibition could help treat AML with chromosome 7q deletions. Full Text | PDF Human cytomegalovirus (CMV) doi:10.1038/scibx.2014.636 Studies in patients suggest autologous, CMV-specific T cell infusions could help treat glioblastoma multiforme (GBM). Full Text | PDF Fas ligand (TNF superfamily, member 6; FASL); VEGF-A; cyclooxygenase (COX) doi:10.1038/scibx.2014.637 Mouse studies suggest inhibiting FASL could improve the efficacy of T cell–based cancer therapies. Full Text | PDF NDC80 homolog kinetochore complex component (NDC80); NIMA-related kinase 2 (NEK2) doi:10.1038/scibx.2014.638 SAR studies suggest compounds that block the interaction between NDC80 and NEK2 could help treat cancer. Full Text | PDF Proteasome subunit-β type 4 (PSMB4); serine hydroxymethyltransferase 2 mitochondrial (SHMT2) doi:10.1038/scibx.2014.639 In vitro and mouse studies suggest inhibiting PSMB4 or SHMT2 could help treat cancer. Full Text | PDF Receptor activator of NF-κB ligand (RANKL; TNFSF11) doi:10.1038/scibx.2014.640 In vitro and mouse studies suggest inhibiting RANKL could help promote antitumor immunity. Full Text | PDF SET and MYND domain containing 3 (SMYD3); K-Ras (KRAS); MAP kinase kinase 1 (MAP2K1; MEK1); MAP2K2 (MEK2) doi:10.1038/scibx.2014.641 Mouse studies suggest antagonizing SMYD3 could be useful for treating cancers driven by KRAS mutations. Full Text | PDF Actin α2 smooth aorta muscle (ACTA2; α-SMA); smoothened (SMO); sonic hedgehog homolog (SHH) doi:10.1038/scibx.2014.642 Studies in mice and patients suggest depleting tumor stroma and fibrosis could be deleterious as opposed to helpful in treating pancreatic cancer. Full Text | PDF Cardiovascular disease Top S100 calcium binding protein A9 (S100A9; calgranulin B; MRP14) doi:10.1038/scibx.2014.643 Studies in mice and patients suggest S100A9 inhibitors could help prevent arterial thrombosis. Full Text | PDF Histone deacetylase (HDAC) doi:10.1038/scibx.2014.644 Mouse studies suggest enhancing the revascularization of primary endothelial colony-forming cells (ECFCs) ex vivo with HDAC inhibitors could help treat ischemia. Full Text | PDF Endocrine/metabolic disease Top Bacterial bile salt hydrolase (bsh) doi:10.1038/scibx.2014.645 Mouse studies suggest bsh-expressing probiotics could help treat obesity. Full Text | PDF Aminolevulinate synthase-1 (ALAS-1) doi:10.1038/scibx.2014.646 Mouse studies suggest an siRNA targeting ALAS-1 could help treat porphyria. Full Text | PDF Musculoskeletal disease Top Transforming growth factor-β (TGFB; TGFβ) doi:10.1038/scibx.2014.647 Mouse studies suggest inhibiting TGFB signaling could help treat osteogenesis imperfecta (OI). Full Text | PDF Neurology Top Adenosine A2A receptor (ADORA2A) doi:10.1038/scibx.2014.648 Nonhuman primate studies suggest antagonists of presynaptic ADORA2A could help treat cannabis addiction. Full Text | PDF GABAB receptor doi:10.1038/scibx.2014.649 Mouse studies suggest specific GABAB receptor agonists could help treat narcolepsy. Full Text | PDF Not applicable doi:10.1038/scibx.2014.650 Mouse studies suggest transplantation of hippocampal γ-aminobutyric acid (GABA)-containing interneurons could help treat schizophrenia. Full Text | PDF Ophthalmic disease Top Not applicable doi:10.1038/scibx.2014.651 Mouse studies suggest decreasing the chemical chaperone sodium 4-phenylbutyrate (PBA) could help prevent glucocorticoid-induced glaucoma. Full Text | PDF Other Top Plasminogen activator inhibitor 1 (SERPINE1; PAI1) doi:10.1038/scibx.2014.652 Mouse studies suggest inhibiting PAI1 could prevent age-related tissue dysfunction. Full Text | PDF N-acetyltransferase 10 (NAT10) doi:10.1038/scibx.2014.653 In vitro studies suggest inhibiting NAT10 could help treat Hutchinson-Gilford progeria syndrome (HGPS) and other laminopathies. Full Text | PDF Distillery: Techniques Assays and screens Top Semiconductor sequencing platform (SSP) for noninvasive diagnosis of incorrect chromosome numbers doi:10.1038/scibx.2014.654 A benchtop SSP could help carry out noninvasive prenatal diagnosis of incorrect chromosome numbers using cell-free fetal DNA from maternal plasma. Full Text | PDF Disease models Top Mouse model of inducible narcolepsy doi:10.1038/scibx.2014.655 Mice with inducible ablation of hypothalamic orexin (hypocretin; Hcrt) neurons could be useful models for evaluating narcolepsy therapeutics. Full Text | PDF Recombination activating gene 2 (RAG2) knockout pigs to model immunodeficiency and evaluate transplant-based therapies doi:10.1038/scibx.2014.656 Pigs with RAG2 knocked out could be useful as models of human immunodeficiencies and for testing transplant-based therapies. Full Text | PDF Drug delivery Top Fibrin hydrogel for controlled and sustained delivery of recombinant VEGF doi:10.1038/scibx.2014.657 In vitro and mouse studies suggest a fibrin hydrogel that provides controlled and sustained release of VEGF could help treat ischemia. Full Text | PDF Drug platforms Top Adoptive cell therapy using mutation-specific, CD4+ T cells in epithelial cancer doi:10.1038/scibx.2014.658 A single-patient study suggests adoptive cell therapy using tumor mutation–specific, CD4+ T cells could help treat epithelial cancer. Full Text | PDF Expansion of CD19-specific chimeric antigen receptor (CAR) T memory stem cells to improve adoptive T cell therapy doi:10.1038/scibx.2014.659 Studies in patient samples and mice suggest increasing the subpopulation of T memory stem cells could help improve the efficacy of CAR-based T cell therapy. Full Text | PDF Expansion of primitive, CD34+ cord blood cells with histone deacetylase (HDAC) inhibitors doi:10.1038/scibx.2014.660 Mouse and ex vivo studies suggest HDAC inhibitors could be used to expand functional cord blood stem cells for transplant. Full Text | PDF In vitro condensation of human mesenchymal stem cells (MSCs) to aid in vivo formation of functional cartilage doi:10.1038/scibx.2014.661 In vitro studies suggest using human MSCs to form structures called condensed mesenchymal cell bodies (CMBs) can generate cartilage that could help promote joint regeneration and bone repair. Full Text | PDF Top

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