Researchers from Washington University in St. Louis and Brigham and Women's Hospital published findings in Science Translational Medicine showing that inhaling xenon gas improved cognition in mouse models of Alzheimer's disease. According to STAT, the study demonstrated that xenon gas inhalation not only reduced the accumulation of pathological tau and amyloid proteins but also improved behaviors related to cognitive function, such as nest building.
Xenon gas, currently used in medicine as an anesthetic and neuroprotective agent for treating brain injuries, showed potential in protecting the brain in studies. Because xenon can easily penetrate the blood-brain barrier—which protects the brain from infection but also prevents most drugs from reaching it—scientists were curious whether it could also protect the brains of people with Alzheimer's disease.
"One of the main limitations in the field of Alzheimer's disease research and treatment is that it is extremely difficult to design medications that can pass the blood-brain barrier—but xenon gas does," said Oleg Butovsky, a neuroscientist at Brigham and Women's Hospital and senior and co-corresponding study author. "We look forward to seeing this novel approach tested in humans."
The research team plans to begin a Phase 1 clinical trial of the xenon gas treatment in healthy volunteers in early 2025 at Brigham and Women's Hospital to evaluate safety and dosage, as reported by La Razón. Howard Weiner, co-director of the Ann Romney Center for Neurological Diseases and leader of the clinical trial, expressed optimism about the potential of xenon, stating, "If the clinical trial goes well, the opportunities for the use of xenon gas are great," according to Gizmodo.
Alzheimer's disease, a currently incurable condition affecting millions worldwide, is characterized by the accumulation of amyloid plaques and tau tangles, which disrupt communication between neurons, leading to progressive brain degeneration.
Dysregulation of microglia, the brain's main immune cells, is a key component of Alzheimer's disease, and xenon inhalation may help restore their protective functions. STAT notes that unlike most current treatments that focus on targeting beta-amyloid plaques and tau tangles, xenon acts by modulating microglial activity to foster a healthier brain environment.
During Alzheimer's disease, microglia lose their ability to break down beta-amyloid proteins that accumulate between neurons, contributing to progressive brain abnormalities and neuronal damage. Defective microglia also contribute to brain inflammation and the accumulation of amyloid beta plaques characteristic of the disease.
In mice with Alzheimer's-like disease, inhaling xenon gas was shown to reduce inflammation and brain shrinkage, and it helped microglia regain their ability to break down beta-amyloid proteins. "In both animal models that model different aspects of this disease, amyloid pathology in one model and tau pathology in another model, xenon has had protective effects in both situations," said Dr. David M. Holtzman.
The team is designing technologies to help with the more efficient use of xenon gas, including innovations in delivery devices and gas recycling systems aimed at making this therapy more accessible and sustainable. As the initial phases of the trial are launched to establish safety and dosage, the research team plans to continue studying the mechanisms by which xenon gas achieves its effects.
The researchers also plan to study the potential of xenon gas to treat other diseases such as multiple sclerosis, amyotrophic lateral sclerosis (ALS), and eye diseases involving neuron loss. "It is a very novel discovery that demonstrates that simple inhalation of an inert gas can have such a profound neuroprotective effect," said Butovsky, according to STAT.
This article was written in collaboration with generative AI company Alchemiq