Early in the pandemic, researchers began exploring various avenues to repurposing drugs for COVID-19.
There are many paths to repurposing, including high-throughput drug screening, which uses automated methods to screen thousands or even millions of molecules for targeted biological activity in a disease of interest. Other approaches include focusing on a certain biological mechanism of action (like testing other antivirals in COVID); combing through clinical trial data and public records; or, in the case of sildenafil citrate (Viagra), incidental clinical observation. (See
MedPage Today s full story on how drug repurposing is gaining momentum as demand for a COVID pill grows.)
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Everything old is new again: Repurposing drugs to treat Covid-19
The practice of finding new uses for old medications called repurposing or repositioning drugs is not new.
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.
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A new focus
In the EU, a disease is defined as rare if it affects no more than 1 in 2,000 people and similarly in the US it is defined as a condition that affects less than 200,000 people.
1 Despite being referred to as ‘rare’, collectively, there are 6,000 to 8,000 known rare diseases
2 and more than 300 million people around the world are living with one.
3 For these people, living with a rare disease can mean a long wait to receive an accurate diagnosis, less options in terms of care, and many unanswered questions. In order to address this current unmet need, we must bring a new focus to the challenges faced by those with rare diseases.