Therapeutic agent for neuropathy in organic acidemia of which mechanism relies on increase in camp

Abstract

[Object] To clarify the mechanism associated with neuropathy in methylmalonic acidemia and to develop a new therapeutic drug or the like for neuropathy in organic acidemia on the basis of this finding. [Solving Means] The inventors established technologies for the establishment of iPS cells derived from a methylmalonic acidemia patient and establishment of a stable maintenance and culturing method using peripheral blood lymphocytes of a methylmalonic acidemia patient, and for the differentiation of methylmalonic acidemia patient-derived iPS cells into nerve cells. The inventors made clear that neuropathy in organic acidemia can be treated and prevented by replenishing cAMP using a series of these experiment technologies. The drug of the invention treats or prevents neuropathy by increasing cAMP in organic acidemia.

1 . A therapeutic drug for neuropathy, treating or preventing neuropathy by increasing cAMP in organic acidemia. 
     
     
         2 . The therapeutic drug for neuropathy according to  claim 1 , further activating CREB. 
     
     
         3 . The therapeutic drug for neuropathy according to  claim 1 , wherein the organic acidemia lowers the activity of Adenylate cyclase. 
     
     
         4 . The therapeutic drug for neuropathy according to  claim 1 , wherein the organic acidemia is selected from any one or a plurality of methylmalonic acidemia and propionic acidemia. 
     
     
         5 . The therapeutic drug for neuropathy according to  claim 1 , wherein the increase in cAMP is achieved by any one of Forskolin, GW9508, NECA, SKF77434, nicotinamide, dobutamine, and db-cAMP. 
     
     
         6 . The therapeutic drug for neuropathy according to  claim 3 , wherein the increase in cAMP is achieved by suppressing or preventing a decrease in the activity of Adenylate cyclase. 
     
     
         7 . The therapeutic drug for neuropathy according to  claim 1 , wherein a life prolongation effect is increased by treating or preventing neuropathy. 
     
     
         8 . A method for treating or preventing neuropathy in organic acidemia by using the therapeutic drug for neuropathy according to  claim 1 .