Pursuit of Alzheimer Disease Treatments Relying Heavily on Publicly and Academically Funded Research as Pharma Steps Back
The journal Alzheimer's & Dementia: Translational Research & Clinical Interventions and Studies, published by the Alzheimer's Association, has published 2 studies showing a shift in how research for potential Alzheimer disease (AD) treatments is funded. Analysis of the US National Library of Medicine database clinicaltrials.gov and annual reviews of the pipeline of potential AD drugs document a retreat from this therapeutic area by pharmaceutical companies. In particular, early phase clinical trials in humans funded by pharmaceutical companies have decreased substantially.
Nonprofit organizations and public agencies have picked up the slack and are funding increased numbers of clinical trials to drive growth and innovation in an area where there is pressing unmet medical need. Clinical trials by academic medical centers have increased by 78% over the last 5 years, funded primarily by the Alzheimer Association's Part the Cloud initiative, the Alzheimer's Drug Discovery Foundation (ADFF) and the National Institutes of Health (NIH).
Although the number of potential treatments in clinical trials for AD has been relatively steady, there is an increasingly broad range of targets and potential mechanisms of action for the drugs in development. The authors suggest that after many negative phase 3 trials, the increase in early trials is driven by innovation and pursuit of previously underfunded novel approaches. Early stage clinical trials are more common, and biomarkers are being used more often. It was also found that the pharmaceutical companies who are continuing work on AD are funding mostly phase 3 trials.
Dr. Jeffrey Cummings, research professor of the University of Las Vegas (UNLV) School of Integrated Health Sciences and lead author of the studies said, “Our study of funding reveals that developing new treatments for AD depends on a complex ecosystem of collaboration among the NIH, biopharmaceutical companies, the Alzheimer’s Association and other advocacy groups, philanthropists, and academic medical centers. Together this system is providing more opportunities to advance therapies needed for patients and eagerly anticipated by their families.” Dr. Cummings is also a member of the Practical Neurology Editorial Board.
“Alzheimer’s Association funding, partnerships--including the NIA and ADDF--and advocacy for federal AD research funding are now the primary drivers of growth in AD clinical trials, filling the gap left by pharma’s retreat, and growing and diversifying the front end of the drug pipeline,” said Maria C. Carrillo, Alzheimer’s Association chief science officer.
These studies also highlight the importance of the upcoming Food and Drug Administration decision of whether to approve aducanumab (Biogen; Cambridge, MA) for treatment of AD. If approved, aducanumab would be the first new AD treatment in nearly 20 years bringing hope to patients and potentially increasing commercial interest in funding more studies. In clinical trials, aducanumab provided a significant improvement in AD progression; however, it did not meet primary endpoints in a second trial, although post hoc analysis showed benefit for a subgroup of participants. These mixed results have created controversy over the potential approval of aducanumab.