FDA-Approved Drugs Show Promise in Reversing Alzheimer’s Symptoms

In a significant advancement in Alzheimer’s research, scientists have discovered that two FDA-approved drugs can not only slow the progression of the disease but may also reverse its effects, including restoring memory in mice. The drugs, which are currently approved for cancer treatment, have shown potential in tackling the complexities of Alzheimer’s disease.

Researchers at the University of California, San Francisco (UCSF) focused on understanding how Alzheimer’s affects gene activity in brain cells. By utilizing the Connectivity Map database, which includes over 1,300 FDA-approved drugs, they identified five medications that could reverse gene expression associated with Alzheimer’s. Among these, they concentrated on two specific cancer drugs: letrozole, commonly used to treat breast cancer, and irinotecan, utilized in treating colon cancer and small cell lung cancer.

In what was described as a “mock clinical trial,” the researchers examined the medical records of 1.4 million patients. Their findings indicated that individuals who had received either letrozole or irinotecan for cancer had a significantly lower likelihood of developing Alzheimer’s. When administered together in a mouse model exhibiting severe Alzheimer’s symptoms, the combination of these drugs reversed disease-related gene expression, dissolved toxic tau protein clumps, and prevented further brain degeneration.

Most impressively, the treatment restored memory and learning abilities in the affected mice, marking a promising breakthrough in a field that has long struggled to find effective therapies. Marina Sirota, the interim director of the UCSF Bakar Computational Health Sciences Institute, expressed excitement about these findings, stating, “Our computational tools opened up the possibility of tackling the complexity directly.”

The complexity of Alzheimer’s disease presents significant challenges for drug development. According to Yadong Huang, a professor of neurology and pathology at UCSF, “Alzheimer’s is likely the result of numerous alterations in many genes and proteins that, together, disrupt brain health.” This multifaceted nature complicates traditional approaches to drug development, which typically focus on single genes or proteins.

The research was published in the journal Cell, highlighting the potential for these existing cancer drugs to be repurposed for Alzheimer’s treatment. Because both medications are already FDA-approved, this could accelerate the timeline for human clinical trials. Nonetheless, repurposing cancer drugs poses its own set of complexities and risks.

The discovery adds to an expanding array of potential treatments for Alzheimer’s disease. For instance, carnosic acid, a compound found in rosemary and sage, has demonstrated the ability to reverse memory loss and reduce brain inflammation in mice with Alzheimer’s. Additionally, a study from Stanford Medicine revealed that seniors who received the shingles vaccine were 20% less likely to develop dementia over a seven-year period. Moreover, researchers at Penn State and Stanford University have also identified a cancer drug that may restore memory and brain function in early-stage Alzheimer’s models.

As research progresses, the hope is that these promising findings will lead to effective treatments that can significantly improve the quality of life for those living with Alzheimer’s disease. The ongoing exploration of existing medications highlights the potential for breakthroughs in understanding and managing this complex condition.