Methods and compositions for delivery of pharmaceutical agents

C - Chemistry – Metallurgy – 12 – N

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Details

C12N 15/88 (2006.01) A61K 9/127 (2006.01) A61K 47/24 (2006.01) A61K 47/28 (2006.01) A61K 47/40 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2384425

Methods and compositions for delivering pharmaceutical agents into cells, in particular urothelial cells of the bladder, are provided. In the methods and compositions of the invention, a solubilized cholesterol composition is used to facilitate delivery of pharmaceutical agents. Preferably, the cholesterol is solubilized by a cyclodextrin (e.g., methyl-.beta.-cyclodextrin) and the pharmaceutical agent comprises a polynucleotide and either a cationic lipid, a cationic polymer or a dendrimer. Improved methods for transfecting polynucleotides into cells thus are also provided, using cationic lipids, cationic polymers or dendrimers and solubilized cholesterol, wherein the transfection efficiency is enhanced compared to use of cationic lipids, cationic polymers or dendrimers alone. Preferred methods of the invention involve transfecting polynucleotides into urothelial cells, preferably for therapeutic treatment of bladder cancer using, for example, a polynucleotide(s) encoding an interleukin(s), an interferon(s), a colony stimulating factor(s) and/or a tumor suppressor(s).

L'invention concerne des procédés et des compositions destinés à l'administration d'agents pharmaceutiques dans des cellules notamment les cellules urothéliales de la vessie. Dans les procédés et les compositions de l'invention, on utilise une composition de cholestérol solubilisé en vue de simplifier l'administration desdits agents pharmaceutiques. Le cholestérol est solubilisé de préférence au moyen d'une cyclodextrine, telle que méthyl-.beta.-cyclodextrine, et les agents pharmaceutiques contiennent un polynucléotide et un lipide cationique ou un polymère cationique ou un dendrimère. L'invention concerne également des procédés améliorés permettant de transfecter des polynucléotides dans des cellules, qui utilisent des lipides cationiques, des polymères cationiques ou des dendrimères ainsi que du cholestérol solubilisé, dans lesquels l'efficacité de la transfection est accrue par rapport à l'utilisation de lipides cationiques, polymères cationiques ou dendrimères seuls. Des procédés préférés de l'invention consistent à transfecter des polynucléotides dans des cellules urothéliales, de préférence pour un traitement thérapeutique du cancer de la vessie utilisant notamment des polynucléotides codant pour des interleukines, des interférons, des facteurs de stimulation de colonies et/ou des suppresseurs de tumeurs.

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