Targeted Riluzole Delivery by Antioxidant Nanovectors for Treating Amyotrophic Lateral Sclerosis

: The goal of this proposal has been to determine whether a novel nanovector consisting of hydrophilic carbon clusters (HCCs) (pegylated) can serve as a therapeutic in a murine model of amyotrophic lateral sclerosis. HCCs were produced by Dr. James Tour of Rice University and provided to Dr. Grill to assess in his colony of G93A hSOD1 mutant mice. Aims were to determine whether PEG-HCCs functionalized with antibodies against the transferrin receptor could enhance lifespan, protect motoneurons and enhance motor function when delivered via sustained intravenous route at the first sign of disease. Second aim was to assess whether riluzole, in combination with functionalized PEG-HCCs could enhance the outcomes used in Aim 1.Progress in this grant was significantly reduced through two incidences with the Jackson Laboratory who had improperly sent us the wrong animals for the study. This resulted in a significant loss of time on two occasions as the colony needed to be refurbished. We report now, however that PEG-HCCs produce a significant enhancement in several indices of motor function, but did not rescue lumbar motoneurons nor enhance lifespan, when applied to the G93A mouse.