World-first human trial aims to treat underlying cause of type 1 diabetes

ABC NEWS (Australia)

A world-first human trial of an immunotherapy drug to treat type 1 diabetes patients has begun after decades of work by an Australian scientist. The drug is designed to rebalance the body's immune response to protect insulin-producing cells in the pancreas. It's early days but if trial is a success, it could be rolled out to a wider cohort of people.

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00:00It's really managing every minute of every day, trying to ensure that the blood sugar

00:07is under control, that the doses of insulin are correct, that you can manage your food

00:13and your exercise.

00:16And then there's the ongoing problems of potential issues arising with health as time goes on.

00:22Professor, you've been developing this new treatment for type 1 diabetes for, I believe,

00:26two and a half decades now.

00:28How does it work?

00:29What does it aim to do?

00:31So it's a problem of the immune system where the immune system attacks the cells that make

00:39insulin in the body, in the pancreas.

00:41So we've taken one of the proteins from the cells that make the insulin and combined it

00:46with an immune calming drug, vitamin D, into a little package.

00:50And then the package goes under the skin as a skin injection to patients.

00:54And the idea is that it's a very specific treatment for type 1 diabetes to calm the immune system

01:00at the onset of the disease.

01:01So is that essentially looking at preserving these pancreatic cells?

01:06Correct.

01:06So once they're killed, they can't be brought back.

01:10But I think if we try to go for the very early stages of the disease, or even before the onset

01:16of the disease, when you've got a larger number of these cells in reserve, the aim is to try

01:23to keep them and to keep that patient in as good control or remission if possible.

01:28Okay.

01:28Currently a phase 1 trial, so it is early days.

01:32But what are the early results telling you?

01:34So we've dosed five people so far and everything's going well so far.

01:38So we're blinded.

01:40Nobody knows who's got what treatment so far.

01:42All right.

01:43So is this medication designed to slow the onset of disease or stop it entirely?

01:49Yeah.

01:50So it's to slow the progression of the disease.

01:53And that, you know, we hope will stop the, or really slow down the progression.

01:57Okay.

01:57And so is this just for now, just for adults, or could it be extended towards a wider cohort

02:03of individuals?

02:05Yeah.

02:05So we're recruiting adults now at the moment who've been diagnosed with type 1 within

02:09the last five years.

02:11And with a good outcome from this trial, we aim to go into a childhood trial and then

02:18to expand out so that we can really test the efficacy.

02:22So looking at what it actually does for the diabetes.

02:25And so, I mean, of course, trial 1, you're looking at efficacy and safety for the patients,

02:30et cetera.

02:31But could something like this apply to type 2 diabetes or are they two very different diseases?

02:37Yeah.

02:37So they're different diseases.

02:38Type 1 is the immune type and type 2 is different.

02:43So we've really designed this to be very specific for type 1 diabetes.

02:47Okay.

02:48And from a timeline perspective, Professor, where do you see this going and how long might

02:54it actually become a reality for a greater cohort of Australians?

02:59So we're at the very beginning of the trial journey.

03:01And if we're successful, as I said, we roll it out into progressively more intense trials

03:09so that we can really see whether we can actually prolong that remission.

03:13And in the next phase, we hope to be able to get into people who are just about at the

03:20onset of disease as well as those who have just been diagnosed.

03:23You

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