Research News

World-first centre opens for research into individual gene therapies for immune disorders

2.0 from 13 votes
Monday, April 28, 2014

A pioneering research centre to develop individual genetic therapies that will treat rare immune disorders is opening in Canberra.

The Centre for Personalised Immunology at the Australian National University (ANU) is the first centre of its kind in the world.

The researchers will focus on immune deficiencies, where the body's natural defences are dampened, and auto-immune disorders, where the patient's immune system attacks itself.

Centre co-director Professor Carola Vinuesa says the field of personalised immunology will revolutionise the way immune disorders are treated.

"Up to very recently, diseases like auto-immune diseases, so we're talking about diseases like Lupus, Rheumatoid Arthritis, Multiple Sclerosis, were all treated as if they were a single disease," Professor Vinuesa said.

"The only treatments available therefore were treatments that basically dampened the entire immune system.

"By knowing precisely what is the mechanism of disease in each patient we can start to tailor treatments specific for each patient. And we find that each patient, even though they might be diagnosed with the same disease, might need a completely different drug.

"So the treatments are now more effective and would have potentially less side effects."

Rapid advances in technology paved the way for the centre

Co-director Professor Matt Cook says many of the disorders are only vaguely diagnosed and are treated with broad, generic medicines.

"Up until now, a lot of progress has been made in treating these diseases but essentially it's based on therapies that can be applied to a large number of individuals," he said.

"Not everyone responds in the same way to treatment, some respond better than others, some get more side effects than others.

"So we think that if we can identify the pathway that leads to an individual's version of a particular disease, then that will lead inevitably to a specific treatment."

Adam Friederich was aged 19 when he was diagnosed with Common Variable Immune Deficiency - one of the disorders the researchers are focusing on.

The condition destroyed his immune system, making him susceptible to infections and viruses, and preventing vaccinations from having an effect on his body.

Mr Friederich says the disease prevents him from playing with his three young children.

"My body doesn't make the antibodies that normally tell your immune system what to do and what's good and what's bad and what to attack and what to leave alone," he said.

"I have to try not to be too involved when they're very sick, but you've also got to trade off that you have children to try and spend time with them.

"So you just have to take the risk and do it."

Mr Friederich has had his spleen removed because of the disorder and has to constantly take antibiotics and other injections.

Professor Cook says although each specific disorder only affects a small proportion of the population, these types of disorders are taking their toll.

"Individually each of these diseases can be quite rare, but collectively they account for a lot of illness in the community."

He says rapid advances in technology that allow sequencing of the genome, alongside more refined techniques for sorting that data intelligently, have made the centre's work possible.

The researchers have already found some successful treatments for individual patients and believe the new centre will speed up the process of finding more.

Author: Carl Smith
Source: ABC news online
2.0 from 13 votes
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