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FDA-Approved Cancer Drugs May Reverse Alzheimer’s, Groundbreaking UCSF Study Reveals
In a landmark breakthrough, scientists at UC San Francisco (UCSF) and Gladstone Institutes have discovered that two widely used, FDA-approved cancer medications—letrozole and irinotecan—may hold the key to not only slowing but possibly reversing the effects of Alzheimer’s disease. These findings, recently published in the journal Cell, signal a pivotal moment in neurodegenerative disease research and could pave the way for swift clinical trials.
New Hope from Old Drugs
Alzheimer’s disease affects over 7million Americans and remains a formidable challenge, with few effective treatments that truly address the underlying causes. Current FDA-approved medications such as Leqembi and Kisunla only modestly slow disease progression, leaving families waiting desperately for better solutions.
Researchers turned to the vast database of already-approved drugs in search of therapies that could counteract the gene expression changes seen in Alzheimer’s brains. By analyzing single-cell gene activity and millions of patient records, the team pinpointed letrozole—traditionally used for breast cancer—and irinotecan, a mainstay for colon and lung cancers, as the most promising candidates.
How Do Letrozole and Irinotecan Work Against Alzheimer’s?
The two drugs target different types of brain cells: letrozole appears to rebalance gene expression in neurons, while irinotecan aids glial cells, crucial for supporting neurons and mitigating brain inflammation. Strikingly, patients with cancer who received these medications had a lower incidence of Alzheimer’s disease compared to others.
When combined and tested in mouse models of aggressive Alzheimer’s, letrozole and irinotecan not only reduced toxic protein clumps—hallmarks of the disease—but also halted neurodegeneration and, most importantly, restored memory and learning abilities in the affected mice.
Rapid Path to Clinical Trials
This breakthrough represents a major leap because both drugs are already widely used and considered safe in humans for cancer treatment. Repurposing them for Alzheimer’s could vastly accelerate the journey from lab to patient, with clinical trials expected to begin soon.
Dr. Marina Sirota, co-senior author and interim director at UCSF Bakar Computational Health Sciences Institute, remarked, “We’re excited that our computational approach led us to a potential combination therapy for Alzheimer’s based on existing FDA-approved medications.”
The Road Ahead
While the results in animal models are promising, the ultimate proof will come in human trials. Success could transform the treatment landscape for Alzheimer’s disease, offering hope where little has existed before.
UCSF’s Dr. Yadong Huang emphasized, “If completely independent data sources guide us to the same pathways and the same drugs, and then resolve Alzheimer’s in a genetic model, then maybe we’re onto something—we’re hopeful this can be swiftly translated into a real solution for millions of patients with Alzheimer’s.”
Clinical trials are expected to be announced soon. This development could mark the beginning of a new era in treating one of the world’s most devastating brain diseases.