Method for the treatment or prophylaxis of lymphangioleiomyomatosis (lam) and animal model for use in lam research

Abstract

Treatment of lymphangioleiomyomatosis with the MEK1/2 inhibitor CI-1040 delayed the development of primary tumors and blocked the estrogen-induced lung metastases in treated animals. Such treatment also reduced the number of circulating ELT3 cells and decreased their lung colonization after intravenous injection.

What is claimed is: 
     
         1 . A method for identifying agents having efficacy for the treatment or prevention of LAM, comprising:
 a) generating a xenograft mouse model of LAM via introduction of a sufficient number of ELT3 cells into a scid mouse thereby forming tumors having metastatic capability; and   b) administering a test compound to said mice and determining whether said compound alters tumor size or inhibits metastatic capability thereof.   
     
     
         2 . The method of  claim 1  further comprising the step of determining whether said agent induces anoikis in said ELT3 cells. 
     
     
         3 . A method for the treatment or prophylaxis of LAM comprising administering to a patient in need thereof a therapeutically effective amount of RAD001. 
     
     
         4 . A xenograft animal model of LAM obtained by introducing into a scid mouse a sufficient number of ELT3 cells to form tumors having metastatic capability. 
     
     
         5 . The animal model of  claim 4 , wherein said ELT3 cells are subcutaneously injected into the flanks of said mouse. 
     
     
         6 . The animal model of  claim 4 , wherein said ELT3 cells are injected into a tail vein of said mouse.