The use cases for AI have spread far and wide across numerous different fields including life saving ones such as pharmaceuticals where AI is helping speed up discovery of new drugs. However, a less celebrated use case has been in ‘drug repurposing’, a field where healthcare practitioners look among existing drugs for treatment of diseases beyond what they were originally meant for.
“Repurposing is fairly common in pharmaceuticals: Minoxidil, developed as a blood pressure medication, has been repurposed to treat hair loss. Viagra, originally marketed to treat a cardiac condition, is now used as an erectile dysfunction drug. Semaglutide, a diabetes drug, has become best known for its ability to help people lose weight.”
The alternative use case of a drug like in the above – hair treatment, erectile dysfunction or weight loss, itself can be a huge market and hence an incentive for drug repurposing. But when it comes to treatment of rare diseases, its utility is even greater as the pharmaceutical industry doesn’t find economic viability in discovering a new drug with limited use case. Except, without AI, there was no systematic way of doing this.
“In labs around the world, scientists are using A.I. to search among existing medicines for treatments that work for rare diseases. Drug repurposing, as it’s called, is not new, but the use of machine learning is speeding up the process — and could expand the treatment possibilities for people with rare diseases and few options.
Thanks to versions of the technology developed by Dr. Fajgenbaum’s team at the University of Pennsylvania and elsewhere, drugs are being quickly repurposed for conditions including rare and aggressive cancers, fatal inflammatory disorders and complex neurological conditions. And often, they’re working.
There is a “treasure trove of medicine that could be used for so many other diseases. We just didn’t have a systematic way of looking at it,” said Donald C. Lo, the former head of therapeutic development at the National Center for Advancing Translational Sciences and a scientific lead at Remedi4All, a group focused on drug repurposing. “It’s essentially almost silly not to try this, because these drugs are already approved. You can already buy them at the pharmacy.”
The National Institutes of Health defines rare diseases as those which affect fewer than 200,000 people in the United States. But there are thousands of rare diseases, which altogether affect tens of millions of Americans and hundreds of millions of people around the world.
And yet, more than 90 percent of rare diseases have no approved treatments, and pharmaceutical giants don’t commit many resources to try to find them. There isn’t typically much money to be made developing a new drug for a small number of patients, said Christine Colvis, who heads drug development partnership programs at NCATS.
That’s what makes drug repurposing such “an enticing alternative” route to finding treatments for rare diseases, said Dr. Marinka Zitnik, an associate professor at Harvard Medical School who studies computer science applications in medical research. Dr. Zitnik’s Harvard lab has built another A.I. model for drug repurposing.”
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