Cairns-based researchers contribute to promising type 1 diabetes treatment discovery

A Far North Queensland couple who started their research journey in Cairns are behind "promising" early research on a new type 1 diabetes treatment.

Rafid Al-Hallaf and his wife, Zainab Agha, migrated to Cairns in 2013 to study immunology at James Cook University (JCU).

More than a decade later, they are part of new research published in the Journal of Immunology from Johns Hopkins University (JHU) in America targeting type 1 diabetes.

According to Diabetes Australia, about 134,000 Australians are currently living with type 1 diabetes, an autoimmune condition.

Current treatment replaces the insulin that the body can no longer make.

"Our approach is different because we are trying to treat the cause of the disease, which is the autoimmune attack," Dr Al-Hallaf said.

"Rather than simply replacing the insulin, we are trying to stop the immune system from destroying the cell that produces it."

What did they discover?

During an immune response like diabetes, the immune system produces a variety of antibodies, including some that scientists do not fully understand.

JHU scientists, led by Abdel-Rahim Hamad, found immune cells from type 1 diabetes patients were making a specific, previously unrecognised antibody, dubbed "x-mAb".

Dr Hamad said current immunotherapy treatment worked "like a sledgehammer", suppressing large parts of the immune system and leaving patients vulnerable to infections.

However, he said the x-mAb antibody worked like a "guided missile".

"It selectively disarms only the rogue T cells destroying the pancreas, leaving the rest of your immune defence fully functional."

In a trial on mice in the early stages of type 1 diabetes, 16 were treated with the antibody and five with a placebo.

All 16 mice had their blood sugar return to normal levels, while none of the placebo mice went into remission.

In another experiment of pre-diabetic mice, 10 received the antibody and 10 received a placebo.

About 70 per cent of those treated mice then stayed disease-free for the rest of the three-month study.

"This is really, really promising," Dr Al-Hallaf said.

'Holy grail' of treatment

St Vincent's Institute immunologist Tom Kay, who is not involved in the research, said it had promising results.

"It's a creative and out-of-left-field idea that I think brings new ideas to the field," Professor Kay said.

He said scientists had long chased precisely that kind of highly targeted treatment.

Professor Kay said the earlier a patient was treated after diagnosis, the more of their own insulin production could be preserved.

His own team had worked on identifying a key driver of the autoimmune attack in type 1 diabetes and trialled an existing rheumatoid arthritis drug, baricitinib, in newly diagnosed patients.

Unlike x-mAb, which aims to precisely target only the specific rogue immune cells, the St Vincent's Institute's drug blocked the whole immune-signalling pathway driving the disease.

However, Professor Kay said there were limitations in how short the JHU mouse trials were and he would have liked to see them carried out longer to see if the drug had a lasting impact.

He said he would closely watch the research as it moved from mice towards possible human trials.

To undertake human trials, the JHU team will first need clearance from the US Food and Drug Administration (FDA).

"You're talking about definitely a decade, probably more, to go from successful mouse studies to the end of clinical trials," Professor Kay said.

Home in Cairns

As the research moves forward, Dr Al-Hallaf said his family ultimately wanted to return to Cairns where they still own a home.

He hopes the JHU discovery could help treat skin cancer in Far North Queensland, where some of the highest rates occur.

"We want to further characterise these cells … to see if this antibody can also have an impact on animals that have cancer," he said.