US2010273894A1PendingUtilityA1

Treatment of leber's hereditary optic neuropathy and dominant optic atrophy with tocotrienol quinones

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Assignee: MILLER GUY MPriority: Apr 28, 2009Filed: Apr 27, 2010Published: Oct 28, 2010
Est. expiryApr 28, 2029(~2.8 yrs left)· nominal 20-yr term from priority
Inventors:Guy M. Miller
A61P 25/00A61P 27/00A61K 9/0053A61P 25/02A61P 27/02A61K 9/0048A61K 31/122A61P 25/28A61P 25/16A61P 27/06
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Claims

Abstract

The present invention relates to methods of treating Leber's hereditary optic neuropathy and dominant optic atrophy with tocotrienol quinones, including alpha-tocotrienol quinone, in order to alleviate symptoms of the disease.

Claims

exact text as granted — not AI-modified
1 . A method of treating Leber's hereditary optic neuropathy (LHON) or dominant optic atrophy (DOA) in an individual, comprising administering a therapeutically effective amount of a compound selected from the group consisting of tocotrienol quinones and tocotrienol hydroquinones to an individual suffering from Leber's hereditary optic neuropathy (LHON) or dominant optic atrophy (DOA). 
     
     
         2 . The method of  claim 1 , wherein the individual is suffering from Leber's hereditary optic neuropathy (LHON). 
     
     
         3 . The method of  claim 1 , wherein the compound is selected from the group consisting of alpha-tocotrienol quinone, beta-tocotrienol quinone, gamma-tocotrienol quinone, and delta-tocotrienol quinone. 
     
     
         4 . The method of  claim 1 , wherein the compound is selected from the group consisting of alpha-tocotrienol hydroquinone, beta-tocotrienol hydroquinone, gamma-tocotrienol hydroquinone, and delta-tocotrienol hydroquinone. 
     
     
         5 . The method of  claim 3 , wherein the compound is alpha-tocotrienol quinone. 
     
     
         6 . The method of  claim 1 , wherein the individual suffering from LHON has at least one mutation selected from the group consisting of 11778G>A, 3460 G>A or 14484T>C. 
     
     
         7 . The method of  claim 1 , wherein the individual is suffering from LHON and has a 11778G>A point mutation. 
     
     
         8 . The method of  claim 1 , wherein the individual is suffering from LHON and has at least one mutation in at least one gene, said mutation affecting Complex I of the mitochondrial electron transport chain. 
     
     
         9 . The method of  claim 1 , wherein the individual suffering from DOA has at least one mutation of at least one OPA gene selected from the group OPA1, OPA2, OPA3, OPA4, OPA5, OPA6 and OPA7. 
     
     
         10 . The method of  claim 1 , wherein the individual suffering from DOA has at least one mutation in at least the OPA1 gene. 
     
     
         11 . The method of  claim 1 , wherein the individual has one or more symptoms selected from the group consisting of: loss of visual acuity, loss of central vision, impairment of color vision, centrocecal scotomas, temporal pallor of the optic disc, circumpapillary telangiectatic microangiopathy, sparing of pupillary light responses, swelling of the retinal nerve fiber layer around the disc (pseudoedema), or optic atrophy. 
     
     
         12 . A pharmaceutical preparation containing from 50 mg to 500 mg of alpha-tocotrienol quinone suitable for use in treatment of Leber's hereditary optic neuropathy (LHON) or dominant optic atrophy (DOA). 
     
     
         13 . A pharmaceutical preparation containing sufficient alpha-tocotrienol quinone to provide a therapeutic level of compound in at least the retina or the optic nerve system when administered to a patient. 
     
     
         14 . The preparation of  claim 12 , wherein the alpha-tocotrienol quinone comprises at least 50% by weight of the tocotrienols and tocotrienol quinones present in the preparation. 
     
     
         15 . The preparation of  claim 14 , wherein the alpha-tocotrienol quinone comprises at least 80% by weight of the material present in the preparation, excluding the weight of any added pharmaceutical carriers or excipients. 
     
     
         16 . A unit dosage formulation of alpha-tocotrienol quinone. 
     
     
         17 . The unit dosage formulation of  claim 16 , wherein the alpha-tocotrienol quinone comprises at least 95% by weight of the tocotrienols and tocotrienol quinones present in the preparation. 
     
     
         18 . The unit dosage formulation of  claim 17 , wherein the alpha-tocotrienol quinone comprises at least 95% by weight of the material present in the preparation, excluding the weight of any pharmaceutical carriers or excipients. 
     
     
         19 . The unit dosage formulation of  claim 16 , wherein the formulation contains from 50 mg to 500 mg of alpha-tocotrienol quinone. 
     
     
         20 . The unit dosage formulation of  claim 18 , wherein the formulation contains from 50 mg to 500 mg of alpha-tocotrienol quinone. 
     
     
         21 . The pharmaceutical preparation of  claim 13 , suitable for use in treating Leber's hereditary optic neuropathy or dominant optic atrophy. 
     
     
         22 . The pharmaceutical preparation of  claim 13 , for use in treating an individual with Leber's hereditary optic neuropathy, said individual having at least one mutation in Complex I of the mitochondrial electron transport system. 
     
     
         23 . The pharmaceutical preparation of  claim 13 , for use in treating an individual with dominant optic atrophy, said individual having at least one mutation in the OPA1 gene. 
     
     
         24 . The unit dosage formulation of  claim 16 , for use in treating LHON or DOA. 
     
     
         25 . The unit dosage formulation of  claim 16 , for use in treating an individual with LHON, said individual having at least one mutation in Complex I of the mitochondrial electron transport system. 
     
     
         26 . The unit dosage formulation of  claim 16 , for use in treating an individual with DOA, said individual having at least one mutation in the OPA1 gene. 
     
     
         27 . A method of treating Leber's hereditary optic neuropathy (LHON) or dominant optic atrophy (DOA) in an individual, comprising administering the preparation of  claim 12  to an individual suffering from Leber's hereditary optic neuropathy (LHON) or dominant optic atrophy (DOA). 
     
     
         28 . The method of  claim 27 , wherein the individual has one or more symptoms selected from the group consisting of: loss of visual acuity, loss of central vision, impairment of color vision, centrocecal scotomas, temporal pallor of the optic disc, circumpapillary telangiectatic microangiopathy, sparing of pupillary light responses, swelling of the retinal nerve fiber layer around the disc (pseudoedema), or optic atrophy. 
     
     
         29 . The method of  claim 27 , wherein the preparation is administered via topical, periocular, or intraocular administration. 
     
     
         30 . The method of  claim 27 , wherein the preparation is administered via oral administration.

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