AI-Powered Drug Development Led to This Recent Breakthrough

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For all that artificial intelligence has accomplished over the last few years, one thing it has not yet achieved is the creation of a new blockbuster drug from start to finish…

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Essentially, going from drawing board to pharmacy.

But AI has made major contributions to the drug development process.

A recently approved therapy from one of the world’s largest pharmaceutical companies benefited from such a contribution.

Back in the early 1990s, biopharma firms Eli Lilly and Co. (LLY) and a partner discovered a new drug they called xanomeline. Early tests found this drug was helpful in slowing cognitive decline related to Alzheimer’s. It also reduced the delusions and hallucinations that come with the disease.

There was, however, a major problem. The side effects associated with xanomeline were so severe that more than 50% of Phase 2 study participants dropped out. Xanomeline was eventually shelved.

But that wasn’t the end of the story…

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Almost a decade later, the Boston-based biotech company PureTech Health PLC (PRTC) became interested. They realized that xanomeline’s efficacy came from its ability to bind to certain receptors inside the brain, while its side effects were caused by activating receptors outside he brain.

That’s a complex issue, but suffice to say, it meant that the problems with xanomeline were solvable. So, PureTech bought the rights to the drug from Lilly.

The PureTech team went on to identify at least 65 binders and 114 suppressors that could be combined to offset xanomeline’s side effects. However, they knew that testing all 7,410 of those combinations would have been impossible in real life.

To produce a solution, PureTech’s team turned to predictive algorithms, a form of AI.

The details of their process remain a trade secret. But we do know that PureTech’s AI eventually stumbled onto a perfect combination. Even better, it was a molecule named trospium chloride, a generic drug for bladder control that the U.S. Food and Drug Administration (FDA) had already approved in 2004.

Trospium chloride, PureTech’s researchers discovered, could counteract xanomeline’s severe side in patients’ bodies, while leaving xanomeline to perform its work in their brains.

While we still don’t know if this newly created drug can be used to treat Alzheimer’s, PureTech was able to demonstrate its ability to reduce delusions and hallucinations. And that proved useful in treating a separate disease: schizophrenia.