| AI for Good: Drug repurposing |
Source: Unsplash |
| We’ve talked before about the clinical plight of rare diseases, that collection of at least 7,000 diseases that don’t get too much attention because they don’t individually impact more than 200,000 people in the U.S. But, in aggregate, rare diseases impact a minimum of 30 million Americans, and hundreds of millions of people around the world. |
| The combination of intense impact and lack of focus makes the study of these diseases ripe for the application of machine learning tools and technologies. But it goes beyond simple diagnostics and into clinical drug explorations. |
| What happened: Specifically, I’m talking about drug repurposing, the discovery of new drugs from existing (and already FDA-approved) medicines. Last year, researchers at Harvard Medicine launched an AI model called TxGNN that’s specifically designed to identify candidates for rare diseases among existing drugs. |
| Trained on a vast quantity of biological and medical data — including DNA information and gene activity — the model was validated across more than a million patient records to identify subtle commonalities between rare illnesses. It is made up of two components; one that identifies potential candidates and their side effects, and another that provides a rationale for its identification, injecting a needed boost of transparency and explainabilty into related medical decision-making. |
| In a test, the TxGNN identified potential candidates among a pool of 8,000 existing medicines for more than 17,000 diseases. |
| Why it matters: Solely identifying candidates doesn’t make them accessible; the model’s predictions then require evaluation and experimentation. Still, the model — which the researchers freely released — promises to drastically speed up the process. |
| “We’ve tended to rely on luck and serendipity rather than on strategy, which limits drug discovery to diseases for which drugs already exist,” Marinka Zitnik, the paper’s lead researcher, said in a statement. “Even for more common diseases with approved treatments, new drugs could offer alternatives with fewer side effects or replace drugs that are ineffective for certain patients.” |
| The team of researchers has already begun work with rare disease foundations to identify potential treatments. |
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Source: Unsplash