Disease Modification

The ‘holy grail’ of PD therapy is to provide a treatment that can alter the underlying course of the disease. By impacting the actual disease process, disease modification therapy could delay or even prevent progression of symptoms. Though progress in developing disease-modifying therapies in research models continues, clinical development is complicated by the absence of a clear regulatory path and well-tested strategies for measuring disease modification in clinical trials.

Neuroprotective approaches to disease modification attempt to promote neuronal health and prevent neuronal death and dysfunction by targeting PD pathogenic processes. Other efforts involve neurorestorative or neuroreplacement approaches utilizing trophic factors, or transplantation using stem cell-based treatments.  It has also been hypothesized that compensatory mechanisms that operate to counter the effects of neurodegeneration in PD may be potentiated as a means of slowing or arresting disease.