Today we present LITHIUM: Newsworthy but not yet ready to use to treat Alzheimer’s Disease and Related Dementias
ATRI is always exploring new potential therapies for care and cure. Some of these have
sufficient safety and efficacy data to show they are safe for trials in humans (for example,
the medications we are currently testing that reduce amyloid and tau proteins and their
brain impacts).
Other treatments may emerge as scientists collect more information. You may hear about
these in the news. These include agents like lithium and insulin. Though exciting
scientifically, we do not yet have sufficient information to use these for testing in people.
We continue to monitor the data emerging from the basic science community and are in
frequent communication with them.
Lithium and Alzheimer’s disease (by Michael Rafii, MD PhD)
Recent research has sparked renewed interest in lithium as a potential therapy for
Alzheimer’s disease (AD). Long recognized for its role in mood stabilization, lithium is now
being studied for its neuroprotective properties. Emerging evidence suggests that lithium
naturally occurs in the brain and contributes to neuronal health. Intriguingly, studies
indicate that lithium levels may decline early in Alzheimer’s progression, possibly due to
sequestration into amyloid plaques—the protein aggregates that characterize the disease.
This depletion could accelerate plaque formation and tau pathology, worsening cognitive
decline.
Preclinical studies in animal models have shown that restoring lithium levels can reduce
pathological protein buildup and improve memory performance. These findings point to
lithium’s potential to influence underlying disease mechanisms rather than simply
alleviating symptoms. Researchers hypothesize that lithium may help stabilize neuronal
signaling and reduce neuroinflammation, both critical factors in Alzheimer’s pathology.
However, promising laboratory results do not automatically translate into safe and effective
treatments for humans. Lithium has a narrow therapeutic window, meaning the difference
between a beneficial dose and a harmful one is small. High doses can lead to serious side
effects, including kidney and thyroid dysfunction. This underscores why randomized,
controlled clinical trials are essential. Such trials are designed to rigorously evaluate
whether lithium-based interventions can deliver cognitive benefits while maintaining safety
over time.
Clinical trials provide the framework to answer key questions: What is the optimal dose?
How should treatment be monitored? What are the long-term effects? Without this
evidence, any use of lithium for Alzheimer’s remains speculative and potentially
dangerous. Experts strongly caution against self-medication or off-label use, as risks could
outweigh any theoretical benefits.
The renewed focus on lithium highlights a broader truth about Alzheimer’s research:
breakthroughs require careful validation. While early findings are encouraging, only well designed trials can confirm efficacy, establish dosing protocols, and ensure patient safety.
Until those results are available, lithium should be viewed as a promising but unproven
approach.
In short, lithium offers an intriguing avenue for addressing Alzheimer’s disease at its roots.
Yet the path from discovery to therapy runs through clinical trials—the cornerstone of
evidence-based medicine.