The most famous (or perhaps infamous) example is sildenafil — aka: Viagra. Originally developed to treat high blood pressure, the little blue pill received US Food and Drug Administration approval in 1998 to treat erectile dysfunction, and very quickly became a blockbuster drug.
Another notable example is thalidomide. Given to women in the late 1950s to prevent morning sickness — and soon found to cause severe birth defects — it got a second life in 1998 as treatment for leprosy (now called Hansen’s disease), and then a third life in 2006, when it was approved to treat multiple myeloma, cancer of the bone marrow.
When Covid-19 became a pandemic last year, the race was on to find any treatment that could help against the novel virus that gravely sickened and even killed some, while leaving others completely unscathed.
One option was developing new medications that specifically targeted SARS-CoV-2, the virus that causes Covid-19, from scratch. New treatments, such as monoclonal antibodies, are the result of those efforts. But developing, testing and then getting approval from the FDA, not to mention creating the infrastructure for the production and distribution of a new medication, all takes time — time the world didn’t, and still doesn’t, have.
Other researchers quickly began to look around at what was already on hand, in hospitals and pharmacies, and on drug store shelves.
“The advantage of drug repurposing is that that drug is already approved. It’s already gone through the regulatory process to show that it’s safe and effective for something. So if you can find additional uses for that drug, you already know there’s a good safety profile,” explained Dr. David Fajgenbaum, an immunologist at the University of Pennsylvania, and the director of the Center for Cytokine Storm Treatment and Laboratory.
Not to mention: Cheap, generic versions of many older medications exist because their patents have expired.
“It’s just a matter of matching the right drug to the right disease,” Fajgenbaum said. “Thankfully, there are over 2,000 drugs that are already approved by the FDA for at least one disease, and we’ve learned that there are many other diseases that those drugs can also be repurposed for.”
In fact, Fajgenbaum said he has “dedicated [his] life towards advancing drug repurposing” for diseases without specific treatments. His passion is driven in part by his own experience. In 2010, while in medical school, he first became ill with Castleman disease, a rare autoimmune disorder that, like Covid-19, can trigger the immune system to suddenly flood the body with inflammatory chemicals in a so-called cytokine storm; the result can be tissue and organ damage, and sometimes death.
“I’m actually alive today because of a drug that was developed 30 years ago for another condition that we identified through a very systematic process and we thought it could help to save my life,” he said. “Here I am, over seven years later.”
A blueprint to save lives
When Covid-19 emerged, Fajgenbaum realized that he was perfectly positioned to look for repurposed drugs to fight it. On March 13, 2020, the day when much of the country began to shut down, “I found myself that night sitting next to my wife, hoping and praying that some researchers somewhere would follow the blueprint that we went through to identify this drug that saved my life,” he said.
About a minute later, he said, he realized “that researcher somewhere” would be him. He tapped his team at the Center for Cytokine Storm Treatment and Laboratory.
“I turned to my team and said, ‘This is a disease that has many…
Read More: Repurposing drugs to treat Covid-19: Everything old is new again