25 New Drug Candidates Now in Human Trials: Hope on the Horizon (5/25/25)

NIH reports an unprecedented surge in Alzheimer's treatments advancing to clinical trials, including one targeting the neuropsychiatric symptoms that devastate families.

The National Institutes of Health recently released its 2025 progress report on Alzheimer's research, and the numbers are staggering: at least 25 new drug candidates developed with NIH funding have now advanced to human trials. For a disease that saw a 99.6% clinical trial failure rate between 2002 and 2012, this represents a sea change in our approach to treatment development.

Of these 25 candidates, 18 are in early Phase 1 trials and 7 have progressed to mid-to-late stage Phase II and III trials. But what caught my attention was a specific mention: one of these new candidates targets the neuropsychiatric symptoms of Alzheimer's—the agitation, anxiety, depression, and sleep disturbances that often devastate families even more than memory loss itself.

Beyond Memory: Treating the Whole Person

Anyone who has cared for someone with dementia knows that the behavioral symptoms can be the most challenging aspect. Sundowning, agitation, wandering—these behaviors exhaust caregivers, disrupt households, and often precipitate placement in long-term care facilities. Yet our treatment options for these symptoms have been limited, often relying on sedating medications with significant side effects.

This is exactly where music therapy has shown its greatest value. Research consistently demonstrates that personalized music interventions can reduce agitation and anxiety, often as effectively as medications but without the side effects. Imagine a future where pharmaceutical treatments for neuropsychiatric symptoms could work alongside music-based interventions, giving families and care teams a comprehensive toolkit for managing the most distressing aspects of dementia.

Gene Therapy on the Horizon

The NIH report also highlighted exciting developments in gene therapy. Researchers are exploring ways to increase APOE ε2—a gene variant associated with reduced dementia risk—in people who carry the high-risk APOE ε4 variant. Early animal studies show this approach can reduce amyloid deposits and improve markers of brain health.

While gene therapy may sound like science fiction, human trials are already underway. We're entering an era where treating Alzheimer's may mean not just managing symptoms, but fundamentally altering the biological factors that drive the disease. For the 7 million Americans currently living with Alzheimer's and their caregivers, this research represents real hope.

References:

1) https://www.nia.nih.gov/about/2025-nih-dementia-research-progress-report