An already existing drug may help treat some cases of hereditary motor neuron disease (MND), a study has found.
MND is an incurable, progressive disease that attacks the nerves controlling movement so muscles no longer work, said researchers from the University of Liverpool in the UK.
Inherited MND is a rare form of the disease (5-10 per cent of total cases) that runs in families.
Around 20 per cent of hereditary MND cases are caused by mutations in a gene which codes for a protein called SOD1.
When the SOD1 gene is mutated, the protein assembly process malfunctions and steps are missed out.
This makes the SOD1 protein structurally unstable leading to the formation of protein clumps in the motor neurones, causing them to die.
The study, published in the journal Nature Communications, used state-of-the-art crystallography, mass-spectrometry and in-cell NMR technologies to search for a drug molecule which could correct the SOD1 assembly line.
They found that ebselen, a drug which was discovered in the 1980s and has been investigated as a potential treatment for a variety of nervous system disorders, can effectively restore several important steps in the SOD1 assembly process including folding, dimerization and zinc binding.
“This discovery has the potential to prevent the accumulation of SOD1 into the large aggregates we see within the motor neurons of effected individuals. If we can stop that, we might be able to stop the neurons dying,” said Gareth Wright from the University of Liverpool.
“The next step is to test ebselen in settings more accurately resembling human neuronal cells and optimising it so that it can become useful as a drug for motor neuron disease,” said Samar Hasnain from the University of Liverpool.
May 18, 2018