Local treatment of neurofibromas

Abstract

A method for treating a neurofibroma, e.g. dermal neurofibroma, a subdermal neurofibroma, or a superficial plexiform neurofibroma, in a subject in need of such treatment is disclosed. The method comprises locally applying a composition to a neurofibroma either topically or intralesionally. This method does not encompass systemic administration of the composition to the subject to have an effect on the neurofibromas. Compositions useful for such treatments and methods of preparing the compositions are disclosed.

1 . A method of treating a dermal neurofibroma, a subdermal neurofibroma, or a superficial plexiform neurofibroma in a subject in need of such treatment, which method comprises locally administering a composition to the region of the tumor, wherein the composition comprises (a) at least one non-carcinogenic agent for treating the neurofibroma on the subject, and optionally (b) a pharmaceutically acceptable excipient that aids in transporting the non-carcinogenic agent into the tumor where it is preferably maintained for a sufficient period of time to treat the neurofibroma;
 wherein the non-carcinogenic agent is selected from the group consisting of an anti-metabolite or a nucleoside analogue; a topoisomerase inhibitor; a microtubule inhibitor; an HDAC inhibitor; an immunomodulators an angiogenesis inhibitor; a chemopreventative agent; an alkylphospholipid; a heat shock protein (HSP) inhibitor; and an agent restores the function of a mutated gene.   
     
     
         2 . The method of  claim 1 , wherein said local administration is topically applying said composition to the surface of the skin in the region of the tumor, and wherein said excipient aids in transporting the non-carcinogenic agent across the skin and to the tumor and preferably maintains the non-carcinogenic agent on the skin of the subject. 
     
     
         3 . The method of  claim 1 , wherein said local administration is intralesionally injecting said composition into the tumor. 
     
     
         4 - 5 . (canceled) 
     
     
         6 . The method of  claim 1 , wherein the immunomodulator is an immunoregulator, an immunosuppressant, or an immunostimulant. 
     
     
         7 - 17 . (canceled) 
     
     
         18 . The method of  claim 1 , wherein the non-carcinogenic agent is 5 fluorouracil, triciribine, sangivamycin, or tubercidin; podophyllotoxin; mebendazole,
 tricostatin A or valproic acid; a retinoid, fenretinide; miltefosine; geldanamycin derivatives, 17-AAG, radicicol or analogues thereof; halofuginone, gentamicin, rapamycin, imiquimod, or a combination thereof.   
     
     
         19 . The method of  claim 1 , wherein the pharmaceutically acceptable excipient includes a skin penetrant. 
     
     
         20 - 38 . (canceled) 
     
     
         39 . The method of  claim 1 , wherein the non-carcinogenic agent is an anti-metabolite or a nucleoside analogue. 
     
     
         40 . The method of  claim 39 , wherein the anti-metabolite or a nucleoside analogue is 5-fluorouracil. 
     
     
         41 . The method of  claim 6 , wherein the immunomodulator is imiquimod.