Disease modification in neurodegenerative diseases

In this issue of Neurology®, a meta-analysis by Feustel et al.¹ reminds us that the holy grail of identifying disease-modifying treatments for neurodegenerative diseases remains elusive. Most important, they remind us that our patients, who endure the relentless, debilitating progression of neurodegenerative diseases, nevertheless cling to the hope that a disease-modifying treatment might be just around the corner. The extent of the desperation is exemplified in patients becoming stem cell tourists or succumbing to promises by unscrupulous providers of treatments that lack sound evidence.^2,3 This same desperation leads to inappropriate expectations by many patients who enter clinical trials for disease-modifying treatments. To allay the fear of being assigned to a placebo group and perhaps to attract more participants, trial designers increase the ratio of people in treatment arms to those in the placebo groups. Maintaining clinical equipoise is of little concern for those with a debilitating, relentless disease. Understandably, the fear of not receiving the study drug is emotionally devastating for some trial participants.^4,5