Research into Parkinson’s Disease

At the level of basic research, new experimental models are being developed to overcome the limitations of animal models: fibroblast cultures, reprogramming to induced pluripotent stem cells (iPSCs), and differentiation into dopaminergic neurons, for example, from patient-derived fibroblasts, allowing the study of genetic, epigenetic, and metabolomic profiles.

At the level of clinical science with diagnostic application, research is being carried out on molecular biomarkers in cerebrospinal fluid, and currently, on peripheral blood analysis techniques. Several molecules of interest include microRNAs, cytokines, and especially proteins involved in neurodegeneration such as alpha-synuclein, beta-amyloid, and tau protein. These molecules are measured using various techniques, such as single ELISA or multiplex platforms.

Another diagnostic method is RT-QuIC (real-time quaking induced conversion), which is the focus of significant research efforts and already has clinical applicability.

Currently, the main focus of interest is on clinical trials of molecules potentially modifying the course of the disease, such as passive and active immunization against alpha-synuclein, aimed at counteracting the excess of synuclein that promotes intraneuronal aggregation. Also under evaluation are drugs with a different primary indication (such as exenatide, a GLP-1 agonist indicated for diabetes), following promising results from pilot clinical trials.

These approaches are awaiting results from international phase 3 trials.