Preeclampsia – Will Orphan Drug Status Facilitate Innovative Biological Therapies?

Brosens, 2011, The “great obstetrical syndromes” are associated with disorders of deep placentation, Am J Obstet Gynecol, 204, 193, 10.1016/j.ajog.2010.08.009

Romero, 2014, Preterm labor: one syndrome, many causes, Science, 345, 760, 10.1126/science.1251816

Ghulmiyyah, 2012, Maternal mortality from preeclampsia/eclampsia, Semin Perinatol, 36, 56, 10.1053/j.semperi.2011.09.011

Lawn, 2014, Preterm birth: now the leading cause of child death worldwide, Sci Transl Med, 6, 263, 10.1126/scitranslmed.aaa2563

Liu, 2014, Global, regional, and national causes of child mortality in 2000-13, with projections to inform post-2015 priorities: an updated systematic analysis, Lancet, 10.1016/S0140-6736(14)61698-6

Sibai, 2013, What to expect from expectant management in severe preeclampsia at <34 weeks gestation: pregnancy outcomes in developed vs developing countries, Am J Obstet Gynecol, 209, 400, 10.1016/j.ajog.2013.08.024

2014, WHO Fact Sheet No. 348: Maternal Mortality

Fisk, 2008, Market failure and the poverty of new drugs in maternal health, PLoS Med, 5, e22, 10.1371/journal.pmed.0050022

2015, WHO Fact Sheet No. 297: Cancer

Ridings, 2013, The thalidomide disaster, lessons from the past, Methods Mol Biol, 947, 575, 10.1007/978-1-62703-131-8_36

Fischer, 2014, Structure of the DDB1-CRBN E3 ubiquitin ligase in complex with thalidomide, Nature, 512, 49, 10.1038/nature13527

Palosse-Cantaloube, 2014, Analysis of chats on French internet forums about drugs and pregnancy, Pharmacoepidemiol Drug Saf, 23, 1330, 10.1002/pds.3709

Koren, 2014, Bias against the null hypothesis: scaring pregnant women about drugs in pregnancy, Can Fam Physician, 60, 441

Fisk, 2009, Systematic analysis of research underfunding in maternal and perinatal health, BJOG, 116, 347, 10.1111/j.1471-0528.2008.02027.x

Gross, 1999, The relation between funding by the National Institutes of Health and the burden of disease, N Engl J Med, 340, 1881, 10.1056/NEJM199906173402406

2014, Orphanet: Preeclampsia (ORPHA275555)

Fisk, 2008, Public-private partnership in cord blood banking, BMJ, 336, 642, 10.1136/bmj.39489.454699.AD

Fisk, 2011, Relative and absolute addressability of global disease burden in maternal and perinatal health by investment in R&D, Trop Med Int Health, 16, 662, 10.1111/j.1365-3156.2011.02778.x

Rayburn, 1995, Off-label drug prescribing on a state university obstetric service, J Reprod Med, 40, 186

Clarke, 2014, Toward a functional definition of a “rare disease” for regulatory authorities and funding agencies, Value Health, 17, 757, 10.1016/j.jval.2014.08.2672

Hall, 2014, The current status of orphan drug development in Europe and the US, Intractable Rare Dis Res, 3, 1, 10.5582/irdr.3.1

Moliner, 2010, Creating a European Union framework for actions in the field of rare diseases, Adv Exp Med Biol, 686, 457, 10.1007/978-90-481-9485-8_25

Dunkle, 2010, Advocacy groups and their role in rare diseases research, Adv Exp Med Biol, 686, 515, 10.1007/978-90-481-9485-8_28

Byrnes, 2014, To crowdfund research, scientists must build an audience for their work, PLoS One, 9, e110329, 10.1371/journal.pone.0110329

Brenan, 2014, Science by the masses: is crowdfunding the future for biotech start-ups?, IEEE Pulse, 5, 59, 10.1109/MPUL.2013.2289465

Dragojlovic, 2014, Crowdfunding drug development: the state of play in oncology and rare diseases, Drug Discov Today, 19, 1775, 10.1016/j.drudis.2014.06.019

Perlstein, 2013, Anatomy of the Crowd4Discovery crowdfunding campaign, Springerplus, 2, 560, 10.1186/2193-1801-2-560

Siva, 2014, Crowdfunding for medical research picks up pace, Lancet, 384, 1085, 10.1016/S0140-6736(14)61661-5

Paidas, 2013, PRESERVE-1 Study Group. Exploring the role of antithrombin replacement for the treatment of preeclampsia: a prospective randomized evaluation of the safety and efficacy of recombinant antithrombin in very preterm preeclampsia (PRESERVE-1), Am J Reprod Immunol, 69, 539, 10.1111/aji.12091

Jacobsen, 2008, Incidence and risk patterns of venous thromboembolism in pregnancy and puerperium – a register-based case-control study, Am J Obstet Gynecol, 198, e231

Paternoster, 2004, Efficacy of AT in pre-eclampsia: a case-control prospective trial, Thromb Haemost, 91, 283, 10.1160/TH03-06-0388

Maki, 2000, Antithrombin therapy for severe preeclampsia: results of a double-blind, randomized, placebo-controlled trial. BI51.017 Study Group, Thromb Haemost, 84, 583, 10.1055/s-0037-1614071

Kobayashi, 2003, Treatment of severe preeclampsia with antithrombin concentrate: results of a prospective feasibility study, Semin Thromb Hemost, 29, 645, 10.1055/s-2004-815632

Akerstrom, 2014, A1M, an extravascular tissue cleaning and housekeeping protein, Free Radic Biol Med, 74, 274, 10.1016/j.freeradbiomed.2014.06.025

Wester-Rosenlöf, 2014, A1M/alpha1-microglobulin protects from heme-induced placental and renal damage in a pregnant sheep model of preeclampsia, PLoS One, 9, e86353, 10.1371/journal.pone.0086353

Prather, 2008, Pluristem Therapeutics, Inc, Regen Med, 3, 117, 10.2217/17460751.3.1.117

Prather, 2008, Placental-derived and expanded mesenchymal stromal cells (PLX-I) to enhance the engraftment of hematopoietic stem cells derived from umbilical cord blood, Expert Opin Biol Ther, 8, 1241, 10.1517/14712598.8.8.1241

Prather, 2009, The role of placental-derived adherent stromal cell (PLX-PAD) in the treatment of critical limb ischemia, Cytotherapy, 11, 427, 10.1080/14653240902849762

Lahiani, 2015, Human placental expanded (PLX) mesenchymal-like adherent stromal cells confer neuroprotection to nerve growth factor (NGF)-differentiated PC12 cells exposed to ischemia by secretion of IL-6 and VEGF, Biochim Biophys Acta, 1853, 422, 10.1016/j.bbamcr.2014.11.009

Pluristem Therapeutics Submits FDA Orphan Drug Application for Preeclampsia

Chaiworapongsa, 2014, Pre-eclampsia part 1: current understanding of its pathophysiology, Nat Rev Nephrol, 10, 466, 10.1038/nrneph.2014.102

Naljayan, 2013, New developments in the pathogenesis of preeclampsia, Adv Chronic Kidney Dis, 20, 265, 10.1053/j.ackd.2013.02.003

Lynch, 2010, Dysregulated complement activation as a common pathway of injury in preeclampsia and other pregnancy complications, Placenta, 31, 561, 10.1016/j.placenta.2010.03.010

Than, 2014, Placental protein 13 (PP13) – a placental immunoregulatory galectin protecting pregnancy, Front Immunol, 5, 348, 10.3389/fimmu.2014.00348

Hahn, 2012, Neutrophil NETs in reproduction: from infertility to preeclampsia and the possibility of fetal loss, Front Immunol, 3, 362, 10.3389/fimmu.2012.00362

Thadhani, 2011, Pilot study of extracorporeal removal of soluble fms-like tyrosine kinase 1 in preeclampsia, Circulation, 124, 940, 10.1161/CIRCULATIONAHA.111.034793

Burwick, 2013, Eculizumab for the treatment of preeclampsia/HELLP syndrome, Placenta, 34, 201, 10.1016/j.placenta.2012.11.014

Khandpur, 2013, NETs are a source of citrullinated autoantigens and stimulate inflammatory responses in rheumatoid arthritis, Sci Transl Med, 5, 178, 10.1126/scitranslmed.3005580

Sur Chowdhury, 2014, Enhanced neutrophil extracellular trap generation in rheumatoid arthritis: analysis of underlying signal transduction pathways and potential diagnostic utility, Arthritis Res Ther, 16, R122, 10.1186/ar4579

Garcia-Romo, 2011, Netting neutrophils are major inducers of type I IFN production in pediatric systemic lupus erythematosus, Sci Transl Med, 3, 73, 10.1126/scitranslmed.3001201

Lande, 2011, Neutrophils activate plasmacytoid dendritic cells by releasing self-DNA-peptide complexes in systemic lupus erythematosus, Sci Transl Med, 3, 73, 10.1126/scitranslmed.3001180

Gupta, 2014, Efficient neutrophil extracellular trap induction requires mobilization of both intracellular and extracellular calcium pools and is modulated by cyclosporine A, Plos One, 9, e97088, 10.1371/journal.pone.0097088