Why Alzheimer's Disease?
Approximately 6 million Americans and more than 30 million people worldwide have either clinical AD or mild cognitive impairment due to AD.
As the population ages, the U.S. numbers are expected to grow to 7.7 million by 2030 and approximately 16 million by 2050. Despite advances in understanding of Alzheimer’s disease (AD) biology, there are no disease-modifying treatments or preventions. The cost to the U.S. government is more than $200 billion a year (AD is the nation’s third most expensive disease). The financial costs, of course, do not include the enormous personal tragedy of AD for patients and their families, for which no cost can be assigned. Despite decades of research, the development of disease-modifying therapies has been difficult to achieve. Treatment of AD will require therapy that targets multiple pathways, and a combination of drugs will ultimately be required to control the disease.
Our Approach
We restore microglial phenotype and function to alleviate neuroinflammation.
Emerging evidence shows that homeostatic dysregulation of the brain’s immune system, which is orchestrated by microglia, plays a significant role in the onset and progression of AD. We recently identified homeostatic microglia and neurodegenerative microglia, also called disease-associated microglia (DAM), that are regulated by the reciprocal suppression of transforming growth factor beta (TGFβ) and induction of apolipoprotein E (APOE) signaling in different neurodegenerative models including AD. We showed that these MGnD are cytotoxic in multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), and AD. However, the major question is how to restore microglial phenotype and function to treat AD. Restoring functional M0microglia in the brain of AD patients is an unmet need.
Our Technology
Xenon gas inhalation modulates microglial phenotype.
General Biophysics discovered that xenon gas treatment via inhalation modulates microglial phenotype associated with reduction in Aβ plaque pathology and neuroinflammation. Xenon penetrates the blood-brain barrier; suppresses expression of MGnD molecules, including APOE; and restores microglial functions in neurodegenerative diseases. Importantly, xenon gas treatment directly modulates the MGnD phenotype. Thus, xenon gas inhalation can serve as a novel treatment for AD and has clinical applicability for other neurodegenerative diseases.
General Biophysics demonstrated that xenon inhalation treatment modulates microglia and reduces Aβ in animal AD models, and it established an optimal xenon dose. Xenon is a biologically active noble gas used as an anesthetic in some countries. Xenon treatment has been shown to have an excellent clinical safety profile. Xenon crosses the blood-brain barrier and can act on CNS microglia. In the cellular membranes, xenon influences N-methyl-D-aspartate (NMDA) signaling and oxidative phosphorylation in microglia.
Our Product
The company’s product is a device-drug combination of xenon gas mixture and inhalation system.
It is a safe treatment that modulates microglia and inhibits neuroinflammation in a known and controllable fashion. This treatment is an intervention perhaps ultimately being given in an outpatient facility or at home by caregivers, that will reduce the burden to the health care system and being embraced by multiple stakeholders, including patients, clinicians, and payers. The expected benefit is that MGnD modulation will lead to slowing disease progression and cognitive decline.