‘ Old drug may help treat motor neurone disease’
London, May 18: An already existing drug may help treat some cases of hereditary motor neurone 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.