Abstract: The present invention provides a chemical class of active agents to be used as efficacious drugs in preventing erythrocyte dehydration, thus helping ameliorate the symptoms of certain hemoglobinopathies. The active agents include a class of compounds that can deliver to an erythrocyte in vivo a divalent cation. Increased intracellular concentrations of the divalent cation in the erythrocyte block K—Cl cotransport preventing dehydration of the erythrocyte, thus improving erythrocyte survival. These agents are to be administered by any preferred route of administration including oral, intravenous and parenteral routes. These agents may be co-administered with any other anti-hemoglobinopathic compounds. The methodology is effective for both long term and short term therapy; may be employed prophylactically and/or therapeutically; and may be used in acute crisis clinical situations.
Type:
Grant
Filed:
November 9, 2000
Date of Patent:
December 18, 2001
Assignees:
Children's Medical Center Corporation, Insitut National de la Sante et de la Reicherche Medicale
(Inserm), Establissement Public Assistance Publique Hopitaux de
Paris
Inventors:
Carlo Brugnara, Yves Beuzard, Frederick Galacteros, Lucia De Francheschi