A groundbreaking study from UC San Francisco and the Gladstone Institute has unveiled exciting news: two FDA-approved cancer drugs might turn back the clock on the biological changes that lead to Alzheimer’s disease.
This could represent a game-changing opportunity for the over 7 million people in the U.S. currently battling this progressive ailment.
Even though scientists have studied Alzheimer’s for decades, only two medications have made it through the FDA approval process, neither effectively halting the harmful trajectory of cognitive decline. To tackle this challenge, the researchers took a unique path by employing data-focused methods to explore the potential of existing drugs already on the market.
Marina Sirota, a UCSF professor and head of the Baker Computational Health Sciences Institute, explained, “We began with 1,300 medications, many of which were cancer treatments, although we didn’t start with a specific theory in mind. It was all driven by data, and the results were quite unexpected.”
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Dr. Yaqiao Li, a postdoctoral researcher who formerly studied at UCSF, led this ambitious research team. Their goal? To identify ways to help Alzheimer’s patients regain cognitive function. Utilizing the Connectivity Map—a comprehensive drug database—they examined how different compounds affected the brain on a molecular scale.
Among the discoveries, two cancer drugs stood out: letrozole, used for breast cancer, and irinotecan, usually administered for colon and lung cancers. Remarkably, when test rats were given a mix of both drugs, their brain regeneration accelerated, and some memory functions returned.
“Both of these drugs are already approved by the FDA. Plus, we have evidence from cancer patients showing that their risk of developing Alzheimer’s is lower when taking these medications,” stated Dr. Li, expressing optimism about this research avenue.
Developing a new drug specifically for Alzheimer’s can be an incredibly lengthy and expensive process, often costing millions and taking over ten years. However, because the medications in question already have FDA approvals for different uses, moving toward clinical trials specifically for Alzheimer’s treatment could be expedited. Researchers are currently fundraising for the initial human trials, during which they plan to monitor any potential side effects closely.
Recent developments include the FDA’s approval of donanemab, an Alzheimer’s treatment from Eli Lilly, but resistance from pharmaceutical companies to pursue parallel paths, especially after failed trials, has made the prospect of reusing existing medications with known safety records particularly enticing.
Sirota believes these findings could open doors for treating other difficult diseases, such as endometriosis. She’s grateful for the progress so far and encourages fellow researchers to keep pursuing innovative drug discovery methods.
“There’s a lot of promise here,” Dr. Li concluded, urging academic peers to invest in this potentially groundbreaking approach.
