Methods and pharmaceutical compositions for the treatment of myelodysplastic syndromes

US9675590B2 · kind B2 · utility

Assignees

• Institut National de la Sante et de la Recherche Medicale (INSERM) · FR
• Moraine Corporation · FR
• ASSISTANCE PUBLIQUE—HÔPITAUX DE PARIS (APHP) · FR
• UNIVERSITE DE PARIS DESCARTES · FR
• CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE · FR

Inventors

• Olivier Hermine · Palaiseau, FR
• Flavia GUILLEM · Paris, FR
• Jean-Benoit Arlet · Paris, FR
• Genevève Courtois · Paris, FR
• Michaela Fontenay · Paris, FR

Key dates

• Filing date: Jun 9, 2015
• Grant date: Jun 13, 2017
• Expiry date: Jun 9, 2035

Classification

• Technology area (CPC A): Human Necessities
• CPC primary: A61P35/02
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present invention provides methods and pharmaceutical compositions designed to intervene in this defective process and to promote or restore erythrocyte maturation in individuals suffering from a myelodysplastic syndrome. The methods involve maintaining the activity of GATA-1 by preventing sequestration of Hsp70 in the cytoplasm. Accordingly, it is an object of this invention to provide methods of restoring or increasing erythrocyte maturation in a subject suffering from a myelodysplastic syndrome by preventing proteolytic inactivation of GATA-1. In some embodiments, preventing is achieved by administering to the subject a compound that inhibits the XPO1 nuclear transporter.