Researchers at the University of California San Francisco (UCSF) have discovered that two FDA-approved cancer drugs — letrozole and irinotecan — may offer a potential breakthrough in the treatment of Alzheimer’s disease. Their study, published in the journal Cell, found that when these two drugs were combined and tested in mice, they significantly reduced harmful tau protein clumps and improved memory and learning abilities, suggesting a possible reversal of the disease’s effects.
The researchers approached the problem of Alzheimer’s by examining how the disease affects gene expression in brain cells. They then analyzed a vast database of medical records from adults over the age of 65, looking for medications that might counteract the disease’s impact. The study identified letrozole and irinotecan as two of the most promising candidates, despite their primary use for cancer treatment. These findings represent an innovative use of existing FDA-approved drugs to address a condition with no clear cure.
While the results are encouraging, the researchers emphasized that the study had limitations. The database used to identify potential drugs was built on cancer cell data rather than brain tissue, and the findings were based on mouse models. These factors raise questions about the real-world applicability of the results in human patients. Additionally, the study found that male mice responded better to the treatment than female mice, prompting further investigation into how hormonal factors might influence the drugs’ effectiveness.
The study’s authors, including co-senior authors Marina Sirota and Yadong Huang, highlighted the complexity of Alzheimer’s and the challenges it poses for drug development. Traditional Alzheimer’s treatments target specific genes or proteins, but this approach may not account for the multifaceted nature of the disease.