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A Clinical Phase IIB trial with 2OHOA in patients with newly-diagnosed malignant glioma.

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."

Field of science

  • /social sciences/sociology/demography/mortality
  • /medical and health sciences/clinical medicine/oncology/cancer

Call for proposal

H2020-SC1-2017-Two-Stage-RTD
See other projects for this call

Funding Scheme

RIA - Research and Innovation action
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Coordinator

LAMINAR PHARMACEUTICALS SL
Address
Ctra Valldemossa Km 7,4 Parcbit, Edificio 17, Incu Empresas, 2A Planta, Modulo C - 8
07121 Palma De Mallorca
Spain
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
EU contribution
€ 2 582 500

Participants (11)

PRAXIS PHARMACEUTICAL SA
Spain
EU contribution
€ 1 103 500
Address
Hermanos Lumiere 5
01510 Minano Mayor
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
SPECIALIZED MEDICAL SERVICES-ONCOLOGY BV
Netherlands
EU contribution
€ 1 212 500
Address
Walaardt Sacrestraat 401
1117 BM Schiphol
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
UNIVERSITY OF NEWCASTLE UPON TYNE
United Kingdom
EU contribution
€ 238 750
Address
Kings Gate
NE1 7RU Newcastle Upon Tyne
Activity type
Higher or Secondary Education Establishments
FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA
Italy
EU contribution
€ 117 000
Address
Via Giovanni Celoria 11
20133 Milano
Activity type
Research Organisations
RESEARCH FUND OF THE HADASSAH MEDICAL ORGANIZATION (R.A)
Israel
EU contribution
€ 222 500
Address
Kiryat Hadassah Ein Karem
91120 Jerusalem
Activity type
Research Organisations
UNIVERSITAT DE LES ILLES BALEARS
Spain
EU contribution
€ 195 500
Address
Carretera De Valldemossa Km 7.5
07122 Palma De Mallorca
Activity type
Higher or Secondary Education Establishments
UNIVERSITA DEGLI STUDI DI SALERNO
Italy
EU contribution
€ 101 875
Address
Via Giovanni Paolo Ii 132
84084 Fisciano Sa
Activity type
Higher or Secondary Education Establishments
LANA MANAGEMENT AND BUSINESS RESEARCH INTERNATIONAL LLC
United States
EU contribution
€ 50 000
Address
360 Massachusetts Avenue Suite 203
01720 Acton, Massachusetts
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
THE ROYAL MARSDEN NATIONAL HEALTH SERVICE TRUST
United Kingdom
EU contribution
€ 117 000
Address
Fulham Road 203-6
SW3 6JJ London
Activity type
Research Organisations
INSTITUT GUSTAVE ROUSSY
France
EU contribution
€ 117 000
Address
Rue Camille Desmoulins 39
94805 Villejuif
Activity type
Research Organisations
LIPODOM KUTATO FEJLESZTO ES TANACSADO KORLATOLT FELELOSSEGU TARSASAG
Hungary
EU contribution
€ 97 000
Address
35-37 Dorottya Utca
6726 Szeged
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)