Scientists at UC San Francisco and Gladstone Institutes have made a groundbreaking discovery that could transform how we treat Alzheimer’s disease. Their research has identified two existing cancer medications that may be able to reverse the devastating brain changes associated with this condition, offering hope to millions of families affected by dementia.
A New Approach to an Old Problem
Alzheimer’s disease currently affects 7 million Americans, causing a relentless decline in memory, thinking, and daily functioning. Despite decades of research, only two FDA-approved treatments exist, and neither can meaningfully slow the disease’s progression. This has left patients and families with limited options and uncertain futures.
The UCSF team took an innovative approach by analyzing how Alzheimer’s changes gene activity in individual brain cells. They then searched through 1,300 FDA-approved medications to find drugs that could reverse these harmful genetic changes. Using advanced computer analysis, they narrowed their search from hundreds of possibilities down to just five promising candidates.
Two Cancer Drugs, One Powerful Combination
The researchers focused on letrozole (typically used for breast cancer) and irinotecan (used for colon and lung cancers). When tested in mice with aggressive Alzheimer’s disease, this drug combination produced remarkable results: it reduced brain damage, prevented toxic protein buildup, and most importantly, restored memory function.
Dr. Marina Sirota, who led the computational research, explained that their approach tackles Alzheimer’s complexity directly rather than targeting just one aspect of the disease. The team also analyzed medical records from 1.4 million people over 65, finding that patients who had taken these medications for other conditions were less likely to develop Alzheimer’s.
Hope for the Future
The researchers are optimistic about moving quickly to human clinical trials. As Dr. Yadong Huang noted, when multiple independent data sources point to the same treatment approach and show success in animal models, it suggests they may be “onto something” truly significant for patients and families facing this challenging disease.