Encouraging Signs in Alzheimer Drug Development Despite Failures: Jeffrey Cummings, MD, ScD

WATCH TIME: 2 minutes

"We have biomarkers now, which are so informative. You can see the clearance of plaque in 6, 12, or 18 months, depending on when they do the scans. That’s been the remarkable thing. Although it’s not as much as we want, we’ve finally cracked the disease modification ceiling.”

The FDA’s approval of Biogen’s investigational antiamyloid therapy aducanumab (Aduhelm; Biogen) represented a landmark decision, marking it as the first novel approval for a treatment for patients with Alzheimer disease (AD) since 2003. While historical in context, the decision comes after years of failed attempts. To quantify the amount of private expenditures that have gone into research and development of AD trials in the past quarter century, Jeffrey Cummings, MD, ScD, and colleagues conducted a retrospective exploration.

A total of 1099 clinical trials in all 4 phases were included, with cumulative private expenditures estimated at $42.5 billion. Of the 235 agents analyzed in the study, 112 remain in active clinical development, 6 have reached commercialization, and 117 have had negative outcomes in various stages of clinical development—equating to a 95% failure rate. Cummings, professor of brain science, and director, Chambers-Grundy Center for Transformative Neuroscience, University of Nevada–Las Vegas, noted better means of reducing and distributing costs, sharing risks, and improving development success are needed.

In a conversation with NeurologyLive®, Cummings discussed how clinicians should interpret the data and which take-home points should carry the most weight. On the flip side, he provided insight on the encouraging signs and trends observed in recent years, including the success of monoclonal antibodies.

REFERENCE

Cummings JL, Goldman DP, Simmons-Stern NR, Ponton E. The costs of developing treatments for Alzheimer disease: a retrospective exploration. Alzheimer Dement. Published online September 28, 2021. doi:10.1002/alz.12450