Major breakthrough lets us find new drugs for the rarest of diseases

13 Mar 2018

The antibody drug protein Pembrolizumab monoclonal. Image: molekuul_be/Shutterstock

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

Not all medication is treated equally and, for the 350m people globally with more than 7,000 very rare diseases, they are forced to miss out on life-saving drugs simply because of the number of people who suffer from said diseases, as the drugs cannot be produced in large enough quantities to be profitable.

In the best-case scenario, those with the financial resources can at least pay for the very expensive medication; at worst, those who can’t must simply try to survive.

But now, a research team from Louisiana State University (LSU) has managed to develop a sophisticated and systematic way to identify existing drugs that can be repositioned to treat a rare disease or condition.

To do this, the team fine-tuned a supercomputer-assisted drug repositioning process that could potentially create significant savings for the pharma companies producing the drugs, and, more importantly, allow the patients to receive affordable and effective treatment.

This new system computationally finds matches between rare disease protein structures and functions, and existing drug interactions to help treat patients with some of these so-called ‘orphan diseases’.

‘We cannot rely on chance’

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

In doing so, the supercomputer can churn and digitally test millions of different possibilities that would otherwise cost billions of dollars in a lab.

“In the past, most repurposed drugs were discovered serendipitously,” said Dr Michal Brylinski, the 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.”

He continued: “Now, amantadine is approved by the [US] Food Drug Administration as both an antiviral and an antiparkinsonian drug. But, we cannot only rely on chance to find a treatment for an orphan disease.”

The team’s research is set to be published in the NPJ Systems Biology and Applications journal.

Colm Gorey was a senior journalist with Silicon Republic