The treatment was found to reverse blood-brain barrier degradation, DNA damage, oxidative stress, and neuroinflammation.
Scientists have successfully reversed Alzheimer's disease in mice, potentially revealing a path toward treating the condition in humans, according to a peer-reviewed study published in the journal Cell Reports Medicine. While Alzheimer's has traditionally been considered irreversible, researchers treated two groups of mice with the pharmacological agent P7C3-A20. One group carried human mutations related to amyloid processing, while the other carried a tau protein mutation. Both amyloid and tau pathologies are primary early events in the progression of the disease. Researchers noted that because these mice develop brain pathologies closely resembling the human condition, they serve as ideal subjects for testing how P7C3-A20 might affect patients.
The treatment enhanced synaptic plasticity and neurogenesis in the hippocampus, a process where new functional neurons are created
These systemic changes led to "complete cognitive recovery and a reduction in plasma levels" of the p-tau217 protein among the amyloid-model mice, the researchers stated. The p-tau217 protein is a key clinical biomarker for Alzheimer's that helps predict cognitive decline. Remarkably, when administration of P7C3-A20 began in mice at 6 months of age—a stage where they already exhibited advanced pathology and cognitive deficits—the treatment fully restored brain health and function by the 12-month mark. P7C3-A20 also reversed advanced disease in mice with the tau mutation and was found to protect human brain microvascular endothelial cells (BMECs) from oxidative stress. BMECs are the primary component of the blood-brain barrier. "Our results challenge the long-standing view that Alzheimer's disease is inherently irreversible," the researchers wrote, suggesting that restoring NAD+ levels could be a "potentially transformative therapeutic approach."
Although many study authors disclosed conflicting interests due to holding patents related to the research, University Hospitals in Ohio, whose researchers participated in the study, stated on December 22 that the findings should spark new research into supplementary approaches and clinical trials for patients. Andrew A. Pieper, the study's lead author, expressed that they are "very excited and encouraged" by the results. "The key takeaway is a message of hope—the effects of Alzheimer's may not be inevitably permanent. The damaged brain can, under certain conditions, repair itself and regain its function," he said. Alzheimer's is a progressive disease that typically begins with mild memory loss and eventually leads to an inability to hold conversations or perform daily activities.
Alzheimer's usually affects individuals aged 60 and older
While the exact causes remains unknown, the disease is likely the result of various factors such as genes, the environment, family history, and lifestyle behaviors, according to an August 2024 report from the Centers for Disease Control and Prevention (CDC). An October 2024 study further discovered that a "silent phase" of Alzheimer's begins up to 20 years before symptoms appear. This phase is characterized by subtle cellular changes, such as inhibitory neurons becoming vulnerable and disrupting brain cell communication. During this period, there is a gradual buildup of beta-amyloid plaques and tangles, which are the hallmarks of the condition.
According to data from the non-profit Alzheimer’s Association, more than 7 million Americans are currently living with the disease, a figure expected to soar to nearly 13 million by 2050. Approximately one in nine people aged 65 and older suffers from Alzheimer's. Furthermore, nearly 12 million Americans are estimated to provide unpaid care to individuals with Alzheimer's or other types of dementia, a service valued at over 413 billion dollars last year.
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