Severe skin rash is a symptom of a family of rare autoinflammatory diseases that can occur at birth and persist throughout life.
Severe skin rash is a symptom of a family of rare autoinflammatory diseases that can occur at birth and persist throughout life.
17 February 2015

Scientists in Brisbane and Ireland have developed a small molecule that blocks a key driver of inflammatory diseases – a finding that could inspire new treatments for arthritis, multiple sclerosis and a family of rare autoinflammatory diseases.

University of Queensland scientists worked with an international team, including experts from Trinity College Dublin, to study the molecule known as MCC950.

The molecule can suppress activation of a key process in inflammation caused by inflammasomes, crucial drivers of many autoinflammatory diseases.

The researchers have identified inflammasomes as promising therapeutic targets, and hope MCC950 will help develop better treatments for inflammatory diseases.

Dr Rebecca Coll, from UQ’s Institute for Molecular Bioscience, said current inflammatory disease treatments could be improved.

“Inflammatory diseases result when our immune system is unable to switch off and so causes chronic inflammation in the body,” Dr Coll said.

“Current therapies for inflammatory diseases, such as asprin, ibuprofen and steroids, don’t work well in severe cases and are not targeted, which can limit their effectiveness and cause side-effects.

“We now know that MCC950 can block an important component of the immune response — an inflammasome called NLRP3 that ‘switches on’ inflammation in our immune cells.”

The Institute’s Professor Matt Cooper said there were many advantages to MCC950 as a potential therapy.

“MCC950 is able to be given orally and will be cheaper to produce than current protein-based treatments, which are given daily, weekly or monthly by injection,” he said.

“Importantly, it will also have a shorter duration in the body, allowing clinicians to stop the anti-inflammatory action if the patient ever needed to switch their immune response back to 100 per cent in order to clear an infection.”

Researchers hope the finding may have a significant impact for treating patients diagnosed with Cryopyrin-Associated Periodic Syndromes (CAPS), a family of rare and severe autoinflammatory diseases caused by a genetic mutation to NLRP3 and estimated to occur in one in one million people worldwide.

For many patients diagnosed with CAPS, symptoms such as rashes, fevers, joint pain and headaches are often present at birth and persist throughout life.

Trinity College Dublin’s Professor Luke O’Neill said the findings confirmed that many autoinflammatory diseases developed the same way, regardless of which part of the body they affected.

“What we have found is a potentially transformative medicine that targets what appears to be the common disease-causing process in many inflammatory diseases,” he said.

“We are excited about MCC950, which we believe has real potential to benefit patients suffering from several highly debilitating diseases, where there is a dire need for new medicines.”

The study is a collaboration between seven institutions, including UQ, Trinity College Dublin, the National Human Genome Research Institute, the Walter and Eliza Hall Institute, and the Universities of Michigan, Massachusetts and Bonn.

The research findings have been published today in the world’s leading preclinical medical journal, Nature Medicine.

Media: IMB Communications Manager Gemma Ward, +61 7 3346 2134, 0439 651 107,