Objective "Glioma is a rare brain cancer with one of the highest mortality rates. It is considered an orphan disease due to its low prevalence (less than 0.5 cases per 10,000 inhabitants in the EU) and the lack of plausible therapies. Based on the discovery of the lipid proliferation switch (high membrane PE-to-SM molar ratio that enables recruitment of cell growth transducers to the membrane), the SME Lipopharma (leading this application) defined a novel anticancer drug target, the tumor repressor protein sphingomyelin synthase 1 (SMS1). An innovative SMS1 activator, 2OHOA, was designed and showed safety and efficacy in preclinical GLP and non-GLP studies. A first-in-man clinical trial I/IIa (ClinicalTrials.gov identifier #NCT01792310) further demonstrated its safety and efficacy in humans. The European Medicines Agency (EMA) designated 2OHOA orphan drug for the treatment of glioma and approximately half of the patients with glioma submitted to >2 months of treatment showed positive response. The present project aims to perform a clinical phase IIB study to demonstrate 2OHOA’s efficacy against glioblastoma multiforme, the most aggressive form of glioma. In this context, a written formal report from the EMA after scientific advice and protocol assistance (SA/PA-EMA/CHMP/SAWP773534/2014) indicates that 2OHOA would obtain Conditional Marketing Authorisation if this phase-IIB study further demonstrates statistically significant efficacy. In addition this project will further investigate 2OHOA’s safety, mechanism of action and biomarkers for glioblastoma diagnosis, prognosis and response to 2OHOA treatment. These studies will let us (i) know the molecular basis underlying the response to 2OHOA treatment, (ii) define new biomarkers, (iii) design more efficacious personalized treatments and (iv) investigate therapeutic alternatives in patients who do not respond to treatment." Fields of science medical and health sciencesbasic medicinepharmacology and pharmacydrug discoverymedical and health scienceshealth sciencespublic healthsocial sciencessociologydemographymortalitymedical and health sciencesbasic medicinepharmacology and pharmacypharmaceutical drugsmedical and health sciencesclinical medicineoncology Programme(s) H2020-EU.3.1. - SOCIETAL CHALLENGES - Health, demographic change and well-being Main Programme H2020-EU.3.1.3. - Treating and managing disease Topic(s) SC1-PM-08-2017 - New therapies for rare diseases Call for proposal H2020-SC1-2016-2017 See other projects for this call Sub call H2020-SC1-2017-Two-Stage-RTD Funding Scheme RIA - Research and Innovation action Coordinator LAMINAR PHARMACEUTICALS SA Net EU contribution € 2 985 285,09 Address Ctra valledemossa km.7,4 c/ issac newton, s/n block a, planta 1, puerta 3, parc bit edificio norte 07121 Palma de mallorca Spain See on map Region Este Illes Balears Mallorca Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 Participants (12) Sort alphabetically Sort by Net EU contribution Expand all Collapse all PRAXIS PHARMACEUTICAL SA Participation ended Spain Net EU contribution € 618 655,78 Address Hermanos lumiere 5 01510 Minano mayor See on map Region Noreste País Vasco Araba/Álava Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 Third-party Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure. BIOKERALTY RESEARCH INSTITUTE AIE Participation ended Spain Net EU contribution € 82 059,13 Address Entidad arcaute 005 arcaute 01192 Arcaute alava See on map Region Noreste País Vasco Araba/Álava Activity type Other Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 SPECIALIZED MEDICAL SERVICES-ONCOLOGY BV Participation ended Netherlands Net EU contribution € 1 212 500,00 Address Walaardt sacrestraat 401 1117 BM Schiphol See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region West-Nederland Noord-Holland Groot-Amsterdam Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 UNIVERSITY OF NEWCASTLE UPON TYNE United Kingdom Net EU contribution € 238 750,00 Address Kings gate NE1 7RU Newcastle upon tyne See on map Region North East (England) Northumberland and Tyne and Wear Tyneside Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA Italy Net EU contribution € 117 000,00 Address Via giovanni celoria 11 20133 Milano See on map Region Nord-Ovest Lombardia Milano Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 RESEARCH FUND OF THE HADASSAH MEDICAL ORGANIZATION (R.A) Israel Net EU contribution € 222 500,00 Address Kiryat hadassah ein karem 91120 Jerusalem See on map Activity type Research Organisations Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 UNIVERSITAT DE LES ILLES BALEARS Spain Net EU contribution € 195 500,00 Address Carretera de valldemossa km 7.5 07122 Palma de mallorca See on map Region Este Illes Balears Mallorca Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 UNIVERSITA DEGLI STUDI DI SALERNO Italy Net EU contribution € 101 875,00 Address Via giovanni paolo ii 132 84084 Fisciano sa See on map Region Sud Campania Salerno Activity type Higher or Secondary Education Establishments Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 LANA MANAGEMENT AND BUSINESS RESEARCH INTERNATIONAL LLC United States Net EU contribution € 50 000,00 Address 360 massachusetts avenue suite 203 01720 Acton, massachusetts See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 THE ROYAL MARSDEN NATIONAL HEALTH SERVICE TRUST United Kingdom Net EU contribution € 117 000,00 Address Fulham road 203-6 SW3 6JJ London See on map Region London Inner London — West Kensington & Chelsea and Hammersmith & Fulham Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 INSTITUT GUSTAVE ROUSSY France Net EU contribution € 117 000,00 Address Rue camille desmoulins 39 94805 Villejuif See on map Region Ile-de-France Ile-de-France Val-de-Marne Activity type Research Organisations Links Contact the organisation Opens in new window Website Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00 LIPODOM KUTATO FEJLESZTO ES TANACSADO KORLATOLT FELELOSSEGU TARSASAG Hungary Net EU contribution € 97 000,00 Address 35-37 dorottya utca 6726 Szeged See on map SME The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed. Yes Region Alföld és Észak Dél-Alföld Csongrád Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Links Contact the organisation Opens in new window Participation in EU R&I programmes Opens in new window HORIZON collaboration network Opens in new window Other funding € 0,00
