Matrix Metalloproteinase-2/-9 Inhibitor For The Treatment Of ALS

Project Summary AbstractAquilus PharmaceuticalsIncproposes to evaluate the effects of a selectivedual activematrix metalloproteinaseMMPand MMPinhibitorAQUin the superoxide dismutaseSODmouse model of amyotrophic lateral sclerosisALSThe goals for Phase I are to chemically synthesize AQUdetermine its oral pharmacokineticsPKin SODmice and then determine its efficacy in the SODmouse model of ALS with dose responseLastlya tissue morphology andampimmunohistochemical study will then be performed to determine whether or not oral dosing with AQUresults in any biochemical changes within spinal tissues as compared to controlsThe ultimate goal of the proposed work is to evaluate whether a dual active MMPinhibitor can be used to treat ALS Project NarrativeOverpeople are diagnosed in the US with amyotrophic lateral sclerosisALSor Lou Gehrig s Disease each yearThe life expectancy for those diagnosed with ALS on average is two to five yearsThe primary FDA approved drugRiluzolehas been found to extend the life of people with ALSPALSby only a few monthsAquilus PharmaceuticalsAquiluswill test a novel inhibitor of a group of proteins that have recently been found to be upregulated in both PALS and in a key mouse model of the diseaseAquilus will test this inhibitor in this ALS mouse model to determine if it can not only slow or arrest the disease but more importantly prolong overall survivalIf the project is successfulAquilus will be able to rapidly bring to the clinic this first in class experimental drug to treat this devastating disease