Ex Parte AVERBACK - Page 3


              Appeal No. 1999-0494                                                                                       
              Application 08/482,768                                                                                     

              intracerebral injection of DMS.  DMS is an acronym for "dense microspheres."                               
              Specification, page 4.  As explained:                                                                      
                            A microscopic structure referred to as the dense microsphere is known to                     
                     exist in normal brain and in brain affected by Alzheimer's disease.  See                            
                     Averback, Acta Neuropathol. 61: 148-52 (1983); results confirmed by Hara, J.                        
                     Neuropath. Exp. Neurol. (1986).  Evidence for the existence of dense                                
                     microspheres (DMS) comes from microscopic histological section studies of fixed                     
                     whole brain tissue, where the dense microspheres are seen to have a                                 
                     proteinaceous central region ("DMS protein") surrounded by continuous                               
                     membrane ("DMS membrane").  The dense microspheres are observed as                                  
                     randomly dispersed, very infrequent structures which occupy an estimated 10-9 or                    
                     less of total brain volume, at a unit frequency roughly estimated at                                
                     10-16 or less, relative to other definable brain structures such as mitochondria.                   

              Id.  Appellant believes that development of amyloid fibrils associated with a number of                    
              conditions including Alzheimer's disease is tied to the unchecked disruption of DMS                        
              in vivo.  Specification, page 8.  Appellant describes a screening procedure for identifying                
              compounds which inhibit the disruption of DMS in vivo.  As explained:                                      
                            It has also been discovered that compounds which are effective, at a in-                     
                     tissue concentration of about 10-5 M or less, in impeding the formation of amyloid                  
                     fibrils in test animals which receive DMS via intracerebral injection can be used                   
                     to treat cerebral amyloidosis, including Alzheimer's disease.  Particularly effective               
                     in this regard are compounds that act on DMS protein or DMS membrane, for                           
                     example, via intracellular or extracellular binding, so as to prevent a structural                  
                     transition of DMS protein in the brain to a β-pleated sheet conformation.                           

              Specification, page 8, lines 15-26.                                                                        
                     Example 2 of the specification describes use of this assay to identify compounds                    
              meeting the requirements of claim 13.  Of the seven compounds tested in Example 2,                         
              appellant states that only three compounds, pyrimethamine, cromolyn sodium, and                            
              erythromycin were found to inhibit amyloid formation in vivo at in-tissue levels in the                    
              range of 10-5 to 10-6 M.  On the basis of those results, appellant states that those three                 

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