Motor neurone disease, also called amyotrophic lateral sclerosis (ALS), is a rare condition that slowly kills off nerve cells in the brain and spine, leading to paralysis and eventually death.

Though currently incurable, a new study may have brought us closer to finding one.

Scientists have taken lab samples of one form of ALS and been able to reverse one of the hallmark biological abnormalities the disease introduces in cells.

Although this is yet to be applied in other forms of the disease or in human beings, the finding represents a large step forward in understanding how ALS could be combatted, providing some hope that the disease could one day be beaten.

What the study found

The researchers found that three RNA binding proteins, which help regulate RNA, get stuck in the wrong place for most people with ALS.

Instead of staying in the motor neuron nucleus, they end up in the surrounding cytoplasm.

The team then found that blocking a particular enzyme, called VCP, was able to reverse this in their human cell samples and return the distribution of RNA binding proteins in the nucleus and cytoplasm back to normal.

According to the scientists, this suggests that this enzyme becomes mutated and overactive in some cases of ALS.

“Demonstrating proof-of-concept for how a chemical can reverse one of the key hallmarks of ALS is incredibly exciting,” said Jasmine Harley, a neuroscientist from the Francis Crick Institute in the UK.

“We showed this worked on three key RNA binding proteins, which is important as it suggests it could work on other disease phenotypes too.”

The drug they used to inhibit the enzyme is also being tested in cancer trials, which could speed up its development and availability if it is found to help cancer patients and ALS patients.

In a second study, published in the journal Brain, the same researchers found over 100 types of RNA fragments, called intron-retaining transcripts, which can also move from the nucleus of cells into the cytoplasm in ALS cases.

The researchers found that these fragments have sequences that bind to the RNA binding proteins, and they suspect these sequences are drawing the proteins out of the nucleus and into the cytoplasm.

Illustrations depict nervous system or neurological disease in ALS patient.

Image: Getty Images

“To imagine what’s going on here we can consider watching a movie at the cinema,” neuroscientist Jacob Neeves explained.

“Typically, we don’t expect to see adverts throughout the film, but, if something goes wrong these ads might start cropping up at odd and unexpected points. These retained introns are a little bit like these abnormal ad breaks.”

Why this matters

Though only 1-2 percent of ALS cases have the mutated enzyme the scientists found in the first study, both pieces of research add to our understanding of motor neurone disease.

This additional information offers new hope that scientists could eventually figure out how to undo some of the damage that the disease causes to the brain and nervous system.

“More research is needed to investigate this further,” Harley said. “We need to see if this might reverse other pathological hallmarks of ALS and also, in other ALS disease models.”

This article originally appeared on Over60.