Drug delivery systems in Krabbe disease—present and prospective approaches

Lysosomal enzymes help in the degradation of various complex biomolecules. Mutations arising from genes encoding them result in deficiency of these enzymes causing serious disturbances in specific metabolic pathways. Krabbe Disease (KD), is one such condition, where galactocerebrosidase enzyme deficiency occurs which is critical for galactosylceramide (GalCer) degradation, which if present abundantly results in a toxic secondary metabolite psychosine accumulation in myelin. It also incites globoid cell infiltration, ultimately resulting in myelination cessation affecting both central nervous system (CNS) and peripheral nervous system. Current life-supportive treatments are not curative since accompanied limitations impede attaining better therapeutic efficacy. Also, the complex pathogenic cascade makes addressing the target difficult. Therapeutic efficacy can be improved by designing ideal drug delivery systems with potential therapeutic agents. This chapter discusses current treatment modalities followed in KD and challenges associated along with a few novel therapeutic approaches which might provide leads for treatment improvements and advancements.