June 30, 2024 • 4 mins
A young kiwi patient will be one of the first to be treated with a radical new treatment for a rare disease.
The drug will be used to target muscle cells and silence a gene which causes Facioscapulohumeral Muscular Dystrophy.
Neurologist and trial lead Richard Roxburgh says the method is promising.
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Episode Transcript
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Speaker 1 (00:00):
We've got a key. We as a first person in
the world to trial the groundbreaking gene therapy. So it's
a gene silencing drug targets a form of muscular dystrophy
known as FSHD. It if it's one in eight thousand trials.
Principal Advisor describes the drug as being as significant as
having antibiotics versus not having antibiotics. Associate Professor Richard Rocksbury
as well, it's Richard, morning to you.
Speaker 2 (00:20):
Oh, good morning.
Speaker 1 (00:21):
What's the technology that's allowed us to get here? Has
something happened? Have we discovered something changed something?
Speaker 2 (00:27):
Yeah, In the last ten years or so, various drug
companies have been working very hard on finding ways to
interrupt the messenger RNA at that level and do that safely.
So it's been a big and also finding ways to
(00:49):
direct that technology to the muscles where the problem is.
Speaker 1 (00:53):
Right, So that was ten years ago. They've been working
on that since they got that breakthrough. Has that been
a complicated journey?
Speaker 2 (00:59):
Yes, As you know, there's been a series of experiments
in the lad doing that first with cells and then
with animals of course, then and now we're ready for
human trials. Yeah, And what is.
Speaker 1 (01:13):
It about FSHD that's different from any other form of
muscular dystrophy, and that this particular drug may well work
for it.
Speaker 2 (01:22):
Well, it's unusual because the whole our bodies actually do
everything we can to suppress the expression of a gene
called Ducks four. So we're basically by doing something where
we silence this gene, we're actually doing the job that
the body is trying to do for itself. But with
people who have these conditions have a genetic abnormality, so
(01:44):
that the Ducks four escapes from the surveillance and suppression.
Speaker 1 (01:49):
Is gene silencing the future and all sorts of things.
If we can isolate one, isolate out the gene for
whatever it may be, and then go about silencing it,
you can solve pretty much anything, not.
Speaker 2 (02:00):
Just about everything, because some things are the problem is
a lack of you know, the genetic abnormality causes a
lack of something. But there are some diseases, such as
f SHD, where there's an over expression of something toxic.
Another example would be my atonic dystrophy. That's that's that's
probably the commonest muscular dystrophy in New Zealand, and that
(02:21):
we've already got trials, which is showing amazing benefits for
the patients.
Speaker 1 (02:26):
And when you talk about gene silencing, so you're not
I mean, you're not solving the problem. It's like a headache.
You've still got the head out. You just can't feel.
Is it that simple or not?
Speaker 2 (02:35):
Yeah, it's well, it's no, you don't. Don't you don't
have it's gene silencing in that we're not taking away
the gene. So you're right, it's not getting we're not
doing crisper at this stage. Maybe that's for the future,
but we're not changing the gene itself. But we're changing
the very first message from the DNA. Your body makes
(02:58):
messenger RNA, which goes out to that the cells to
make proteins. And it's at that very first step where
the DNA makes messenger RNA. We're interrupting at that very
fundamental level. So really getting to the root of things
that would.
Speaker 1 (03:11):
Not about side effects at this point and whether or
not it works with everybody, Oh.
Speaker 2 (03:18):
Good question. I mean, the whole of this this thing
where the New Zealanders are having the first doses and
last week and this week, the whole purpose of this
study is to make sure it's safe.
Speaker 1 (03:28):
All right, will we when will we know? And when's
the path to commercialization?
Speaker 2 (03:32):
Huh, Well, it's early days, but I was if it
works as well as this other study that we've just had,
I would think over the next three years, it may
may maybe in clinical practice and within about three years time.
Speaker 1 (03:47):
It's amazing, all right, Richard go well, appreciate it very much.
Richard Rocksborough, the trials principal investigate. It's amazing what science
can do, or will do, or is about to do.
For more from the Mic Asking Breakfast, listen live to
news to Books at b from six am weekdays, or
follow the podcast on iHeartRadio
We've got a key. We as a first person in
the world to trial the groundbreaking gene therapy. So it's
a gene silencing drug targets a form of muscular dystrophy
known as FSHD. It if it's one in eight thousand trials.
Principal Advisor describes the drug as being as significant as
having antibiotics versus not having antibiotics. Associate Professor Richard Rocksbury
as well, it's Richard, morning to you.
Speaker 2 (00:20):
Oh, good morning.
Speaker 1 (00:21):
What's the technology that's allowed us to get here? Has
something happened? Have we discovered something changed something?
Speaker 2 (00:27):
Yeah, In the last ten years or so, various drug
companies have been working very hard on finding ways to
interrupt the messenger RNA at that level and do that safely.
So it's been a big and also finding ways to
(00:49):
direct that technology to the muscles where the problem is.
Speaker 1 (00:53):
Right, So that was ten years ago. They've been working
on that since they got that breakthrough. Has that been
a complicated journey?
Speaker 2 (00:59):
Yes, As you know, there's been a series of experiments
in the lad doing that first with cells and then
with animals of course, then and now we're ready for
human trials. Yeah, And what is.
Speaker 1 (01:13):
It about FSHD that's different from any other form of
muscular dystrophy, and that this particular drug may well work
for it.
Speaker 2 (01:22):
Well, it's unusual because the whole our bodies actually do
everything we can to suppress the expression of a gene
called Ducks four. So we're basically by doing something where
we silence this gene, we're actually doing the job that
the body is trying to do for itself. But with
people who have these conditions have a genetic abnormality, so
(01:44):
that the Ducks four escapes from the surveillance and suppression.
Speaker 1 (01:49):
Is gene silencing the future and all sorts of things.
If we can isolate one, isolate out the gene for
whatever it may be, and then go about silencing it,
you can solve pretty much anything, not.
Speaker 2 (02:00):
Just about everything, because some things are the problem is
a lack of you know, the genetic abnormality causes a
lack of something. But there are some diseases, such as
f SHD, where there's an over expression of something toxic.
Another example would be my atonic dystrophy. That's that's that's
probably the commonest muscular dystrophy in New Zealand, and that
(02:21):
we've already got trials, which is showing amazing benefits for
the patients.
Speaker 1 (02:26):
And when you talk about gene silencing, so you're not
I mean, you're not solving the problem. It's like a headache.
You've still got the head out. You just can't feel.
Is it that simple or not?
Speaker 2 (02:35):
Yeah, it's well, it's no, you don't. Don't you don't
have it's gene silencing in that we're not taking away
the gene. So you're right, it's not getting we're not
doing crisper at this stage. Maybe that's for the future,
but we're not changing the gene itself. But we're changing
the very first message from the DNA. Your body makes
(02:58):
messenger RNA, which goes out to that the cells to
make proteins. And it's at that very first step where
the DNA makes messenger RNA. We're interrupting at that very
fundamental level. So really getting to the root of things
that would.
Speaker 1 (03:11):
Not about side effects at this point and whether or
not it works with everybody, Oh.
Speaker 2 (03:18):
Good question. I mean, the whole of this this thing
where the New Zealanders are having the first doses and
last week and this week, the whole purpose of this
study is to make sure it's safe.
Speaker 1 (03:28):
All right, will we when will we know? And when's
the path to commercialization?
Speaker 2 (03:32):
Huh, Well, it's early days, but I was if it
works as well as this other study that we've just had,
I would think over the next three years, it may
may maybe in clinical practice and within about three years time.
Speaker 1 (03:47):
It's amazing, all right, Richard go well, appreciate it very much.
Richard Rocksborough, the trials principal investigate. It's amazing what science
can do, or will do, or is about to do.
For more from the Mic Asking Breakfast, listen live to
news to Books at b from six am weekdays, or
follow the podcast on iHeartRadio
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