AI Tool for Drug Repurposing May Offer New Hope for Treating Rare and Neglected Diseases

Researchers at Harvard Medical School report they have developed a groundbreaking artificial intelligence tool, TxGNN, that can identify potential drug candidates for 17 080 diseases, many of which currently lack treatments, offering new hope for patients with rare and neglected conditions.

Developed by scientists at Harvard Medical School, TxGNN is the first AI model specifically designed to identify drug candidates for diseases with no current treatments. The tool outperformed existing AI models for drug repurposing. By predicting potential side effects and contraindications, TxGNN could offer a faster and more cost-effective approach to developing therapies compared to traditional methods.

Researchers share that using repurposed drugs for new treatments provides the advantage of utilizing medications with established safety profiles, regulatory approval, and extensive research data. It is common for medications to have multiple effects beyond their intended use, with many undiscovered effects only emerging after years of use by large populations. While the approach to drug repurposing may seem random, it offers opportunities to find new uses for existing drugs and potentially improve treatment options for various conditions.

The AI tool, trained on vast amounts of data including DNA information and clinical notes, has the capacity for reasoning like a human clinician. It was tested and validated on 1.2 million patient records to identify drug candidates for various diseases. The tool was also asked to predict patient characteristics that would contraindicate certain drug candidates. Additionally, it identified existing small molecules that could block the activity of disease-causing proteins.

“The researchers caution that any therapies identified by the model would require additional evaluation for dosing and timing of delivery,” shared the Harvard research release. “But, they add, with this unprecedented capacity, the new AI model would expedite drug repurposing in a manner not possible until now. The team is already collaborating with several rare disease foundations to help identify possible treatments.” 

Reference
Pesheva E. Researchers harness AI to repurpose existing drugs for treatment of rare diseases. Harvard. Published September 25, 2024. Accessed September 27, 2024. https://hms.harvard.edu/news/researchers-harness-ai-repurpose-existing-drugs-treatment-rare-diseases