rare disease

A team of researchers is finding different ways of using existing drugs to treat the rarest of diseases, saving huge research costs.

One in 10 people in America is fighting a rare disease, or a disorder that affects fewer than 200,000 Americans. Although there are more than 7,000 rare diseases that collectively affect more than 350 million people worldwide, it is not profitable for the pharmaceutical industry to develop new therapies to treat the small number of people suffering from each rare condition.

Those with financial resources can at least pay for the expensive medication, but those who can’t must simply try to survive. 

But now, a research team from Louisiana State University has managed to develop a sophisticated and systematic way to identify existing drugs that can be repositioned to treat a rare disease or condition. The research is described in the npj Systems Biology and Applications journal. 

To do this, researchers have fine-tuned a computer-assisted drug repositioning process that can potentially save time and money for the various pharma companies producing the drugs, and, more importantly, allow patients receive affordable and effective treatment.

To systematise drug repurposing, the team combined eMatchSite, a software developed by the same group, with virtual screening to match FDA-approved drugs and proteins that are involved in rare diseases.

These supercomputers allow them to test millions of possibilities that will cost billions of dollars to test in the lab.

“In the past, most repurposed drugs were discovered serendipitously,” said Dr Michal Brylinski, head of the computational systems biology group at LSU.

“For example, the drug amantadine was first introduced to treat respiratory infections. However, a few years later, a patient with Parkinson’s disease experienced a dramatic improvement of her disease symptoms while taking the drug to treat the flu.”

Now, amantadine is approved by the Food Drug Administration as both an antiviral and an antiparkinsonian drug. But, we can not only rely on chance to find a treatment for an orphan disease.”