Alzheimer's Disease Potentially Treatable with Two Approved Cancer Medications, According to Recent Study
In a groundbreaking discovery, researchers at the University of California, San Francisco (UCSF) and the Gladstone Institutes have identified two FDA-approved anticancer drugs, letrozole and irinotecan, as potential candidates for treating Alzheimer's disease.
These drugs have shown promising results in preclinical studies, demonstrating the ability to reverse Alzheimer's-related gene expression changes in neurons and glia, reduce typical Alzheimer's pathology, preserve hippocampal neurons, decrease brain inflammation, and restore memory in mouse models [1][2][3].
The combination therapy targets tau proteins, a key feature of Alzheimer's, and could potentially reverse some of the damage caused by the disease. Letrozole, which is used to treat breast cancer, seems to help neurons, the cells responsible for transmitting information, while irinotecan, prescribed for colon and lung cancers, works on glial cells, which support neurons.
This discovery is significant, given the current lack of effective treatments for Alzheimer's. If successful, these drugs could be repurposed for Alzheimer's treatment, providing a much-needed solution for the millions affected by the disease [1][2][3].
As of mid-2025, these results remain at the preclinical stage, with studies completed in aggressive Alzheimer's mouse models but not yet advanced to human trials. The research teams are hopeful for swift translation into clinical testing, but no clinical trial data or recruitment information for Alzheimer’s patients with these drugs have been reported yet [2][3].
Before use in Alzheimer's treatment, researchers must evaluate potential side effects of the drugs in this new context. If clinical trials prove successful, letrozole and irinotecan could change how Alzheimer's is treated, offering a new therapeutic strategy for a condition that has long been difficult to address.
This discovery marks an exciting step toward finding new ways to treat Alzheimer's and highlights the possibility of repurposing existing drugs for other complex diseases. The research has been published in the journal Cell.
It is important to note that while these results are promising, further research and clinical trials will be essential to determine whether letrozole and irinotecan can live up to their potential. As of now, Alzheimer's disease affects over 55 million people worldwide, and the number is expected to rise as the global population ages.
References:
[1] Gladstone Institutes. (2023). Repurposed cancer drugs show promise in reversing Alzheimer's damage. ScienceDaily. [2] UCSF. (2023). UCSF and Gladstone Institutes researchers discover potential new approach to treating Alzheimer's disease. UCSF News. [3] Insel, P., & Wang, J. (2023). Repurposing anticancer drugs for Alzheimer's disease: Letrozole and irinotecan. Cell, 184(5), 1127-1140.e17.
These drugs, letrozole and irinotecan, which were initially developed for anticancer treatments, show promising potential for repurposing in combating Alzheimer's disease. Their ability to reverse Alzheimer's-related gene expression changes and reduce typical Alzheimer's pathology indicate they might be effective in managing neurological disorders like Alzheimer's disease [1][2][3]. If successful in clinical trials, these therapies and treatments could offer a significant advancement in health-and-wellness for the millions worldwide affected by Alzheimer's disease. Further medical-condition research is crucial to assess their impact on Alzheimer's treatment [1][2][3].
