Compound and method for the treatment and diagnosis of neurodegenerative conditions

Abstract

A reagent selected from cholestenoic acid or an inhibitor of an enzyme in the cholestenoic acid biosynthetic or metabolic pathway for use in the treatment of neurodegenerative conditions. in particular, the reagent is a cholestenoic acid of a particular form, such as 3β,7α-dihydroxycholest-5-en-26-oic (3β,7α-diHCA), not previously associated with neural tissue or CSF. Pharmaceutical compositions, methods of treatment or prevention of neurodegenerative conditions as well as diagnostic methods and novel biomarkers form further aspects of the invention.

1 - 7 . (canceled) 
     
     
         8 . A pharmaceutical or veterinary composition comprising a cholestenoic acid, a cholestenoic acid precursor or inhibitor of an enzyme in the cholestenoic acid biosynthetic or metabolic pathway, or a pharmaceutically acceptable salt for use in the treatment of neurodegenerative conditions. 
     
     
         9 - 17 . (canceled) 
     
     
         18 . A method for diagnosing or predicting a predisposition towards a neurological disorder, which method comprises determining the relative amounts of specific forms of cholestenoic acid and/or a cholestenoic acid precursor present in a biological sample, and relating the result to the presence or absence of or a predisposition towards development of a neurological disorder. 
     
     
         19 . The method of  claim 18  wherein the cholestenoic acid is selected from 3β,7α-diHCA, 3β,7β-diHCA, 7α, 26-diHCO, 7α, 26-diHC, 3β-HCA and 26-HC. 
     
     
         20 . A method for diagnosing neurodegenerative conditions or a predisposition to neurodegenerative conditions which comprises using a cholestenoic acid and/or a cholestenoic acid precursor as a diagnostic biomarker for said conditions. 
     
     
         21 . The method of  claim 20  wherein the cholestenoic acid or cholestenoic acid precursor is selected from 3β,7α-diHCA, 3β,7β-diHCA, 7α,26-diHCO, 7α,26-diHC, 3β-HCA and 26-HC. 
     
     
         22 . The method of  claim 20  wherein the neurodegenerative condition is motor neuron disease. 
     
     
         23 . A method for screening a candidate compound for efficacy in treating a neurodegenerative condition, said method comprising identifying compounds that inhibit an enzyme in the cholestenoic acid biosynthetic or metabolic pathway that leads to harmful or undesirable cholestenoic acids in the context of neurodegenerative conditions.