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 agent for abating the negative impact of the neurofibroma on the subject, and optionally (b) a pharmaceutically acceptable excipient that aids in transporting the agent into the tumor where it is preferably maintained for a sufficient period of time to negatively impact the neurofibroma. 
     
     
         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 agent across the skin and to the tumor and preferably maintains the 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 . The method of  claim 1 , wherein the agent comprises a chemotherapeutic agent, wherein (1) the agent interferes with DNA replication by topoisomerase inhibition; (2) the agent disrupts the microtubule and/or mitotic spindle; (3) the agent acts as an alkylating or damaging agent to DNA; (4) the agent interferes with nucleotide synthesis or a combination thereof. 
     
     
         5 . The method of  claim 1 , wherein the agent comprises a sclerosing agent. 
     
     
         6 . The method of  claim 1 , wherein the agent is an immunomodulator, an immunoregulator, an immunosuppressant, an immunostimulant, or an non-steroidal anti-inflammatory agent. 
     
     
         7 . The method of  claim 1 , wherein the agent modulates gene transcription. 
     
     
         8 . The method of  claim 1 , wherein the agent is an angiogenesis inhibitor. 
     
     
         9 . The method of  claim 1 , wherein the agent alters the structure, function, localization, or post-translational modification of small GTPases. 
     
     
         10 . The method of  claim 1 , wherein the agent is a chemopreventative agent. 
     
     
         11 - 19 . (canceled) 
     
     
         20 . A composition useful for treating a dermal neurofribroma, a subdermal neurofibroma, or a superficial plexiform neurofibroma in a subject, which composition comprises (a) at least one agent for abating the negative impact of the neurofribroma on the subject, and optionally (b) a pharmaceutically acceptable topical excipient that aids in transporting the agent across the skin into the tumor and preferably maintains the agent on the subject's skin for a period of time. 
     
     
         21 . The composition of  claim 20 , wherein the agent is a chemotherapeutic agent, wherein (1) the agent interferes with DNA replication by topoisomerase inhibition; (2) the agent disrupts the microtubule and/or mitotic spindle; (3) the agent acts as an alkylating or damaging agent to DNA; (4) the agent interferes with nucleotide synthesis or a combination thereof. 
     
     
         22 . The composition of  claim 20 , wherein the agent comprises a sclerosing agent. 
     
     
         23 . The composition of  claim 20 , wherein the agent is an immunomodulator, an immunoregulator, an immunosuppressant, an immunostimulant, or an non-steroidal anti-inflammatory agent. 
     
     
         24 . The composition of  claim 20 , wherein the agent modulates gene transcription. 
     
     
         25 . The composition of  claim 20 , wherein the agent is an angiogenesis inhibitor. 
     
     
         26 . The composition of  claim 20 , wherein the agent alters the structure, function, localization, or post-translational modification of small GTPases. 
     
     
         27 . The composition of  claim 20 , wherein the agent is a chemopreventative agent. 
     
     
         28 . The composition of  claim 20 , wherein the agent is an inhibitor selected from the group consisting of a protein kinase inhibitor, a lipid kinase inhibitor, a heat shock protein inhibitor, a protein chaperone inhibitor, a phosphatase inhibitor or a protease inhibitor. 
     
     
         29 - 37 . (canceled) 
     
     
         38 . A method of preparing a medicament for topically treating a dermal neurofibroma, a subdermal neurofibroma, or a superficial plexiform neurofibroma, which method comprises combining at least one agent for abating the negative impact of a neurofibroma on a subject with a pharmaceutically acceptable topical excipient that aids in transporting the agent(s) across the skin into the tumor and maintains the agent(s) on the skin of the subject for a period of time.