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Scientists identify drug that could block multiple sclerosis

Prof Luke O’Neill with flasks of inflamed white blood cells from the immune system in a laboratory in Trinity. 

Tests reveal remedy could also halt diseases such as rheumatoid arthritis and alzheimer’s

Researchers in Dublin have led an international study identifying a remarkable drug that may be able to block major diseases such as multiple sclerosis (MS), alzheimer’s, rheumatoid arthritis and most other inflammatory conditions.

Initial tests show it could instantly block MS and the effects of blood poisoning in mouse models. It also halted a rare inflammatory disease called Muckle-Wells syndrome using human blood samples as a test.

Muckle-Wells syndrome is a disorder characterised by episodes of skin rash, fever and joint pain. Progressive hearing loss and kidney damage also occur with this illness.

The drug, called MCC950, stops a very early trigger that sets off the inflammatory response to infection. While inflammation is good during infections it can cause a wide range of serious diseases if the inflammation remains in place.

“This is exciting, one of the biggest discoveries we have had,” said Prof Luke O’Neill, the chairman ofbiochemistry based in the Trinity Biomedical Sciences Institute. “It is fantastic, the thing we have been looking for for 30 years. This could be the missing compound.”

Their main discovery is being able to identify the pathway that allows the drug to block the action of a pro-inflammatory substance in the body called NLRP3.

They also confirmed that inflammatory diseases all share a common process, even though the parts of the body becoming inflamed might differ, he said.

Trinity’s collaborators included the Universities of Queensland, Michigan, Massachusetts and Bonn and their findings were published on Monday in the journal Nature Medicine. He was co-senior author with Prof Matt Cooper of Queensland, and lead author was Dr Rebecca Coll who worked with Prof O’Neill.

Journal Reference: A small-molecule inhibitor of the NLRP3 inflammasome for the treatment of inflammatory diseases.Nature Medicine, 2015; DOI: 10.1038/nm.3806

View more here on Irish Times/Science

New DNA mapping project to discover causes of MND launched

Today sees the formal launch in Ireland of Project Mine, an ambitious international effort to find the genetic causes of motor neurone disease. It will involve scientists in 14 countries, including Ireland, who will collect patients’ DNA, sequence it and then do comparative studies that will help provide answers about this terrible disease.

Ireland sees 110 new cases of motor neurone disease every year, says Prof Orla Hardiman, who will lead Project Mine activity in Ireland. Scientists are still learning about the disease, but there is undoubtedly a genetic component, hence the need for an initiative such as this.

“The project is based on looking at the population genetics of motor neurone disease; whole genome association studies, ” says Hardiman, a professor of neurology at Trinity College Dublin and a consultant neurologist at Beaumont Hospital. “It is based on looking at genes, but being able to do this is all about computers, maths and statistics.”

“Ireland is a really good place to do this work; it is good for gene research. It can be easier to do these studies in Ireland because we have larger families and can compare family genes against the wider population.”

The complexity of the genetic background here is also lower than in a country such as the US or mainland Europe.

Only an international effort could tackle such a huge job, says Dr Russell McLaughlin, a postdoctoral research fellow in Trinity’s academic unit of neurology.The genetic resource created by the project will be important not just for motor neurone disease but for research into other conditions. McLaughlin is also involved in a study of irritable bowel syndrome, and the genetic information could be used to study multiple sclerosis, diabetes or Parkinson’s, says Hardiman.

Members of the consortium will share data freely, and so, although we will only pay for a part of the project, we will benefit from having access to all of the resultant data. “We get the data back,” says Vajda. “It is not giving genetic information away, it is bringing it back to us. It represents a new era in gene research. We need to be be involved. We need to be a partner in this to avoid missing out.”

View more here

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