The Centre for Eye Research Australia invites people living with inherited retinal diseases and their parents/guardians to share their views and opinions about new potential gene therapy treatments.
We speak with principle investigator Associate Professor Heather Mack about the importance of the study and how the research will be used.
https://www.cera.org.au/ird-survey/
See omnystudio.com/listener for privacy information.
Episode Transcript
The Centre for Eye Research Australia is inviting people living
with inherited retinal diseases, I.D. and their parents or guardians
to share their views and opinions about potential gene therapy treatments.
New potential gene therapy treatments and to talk with us
(00:21):
about this. Today, I'm chatting with clinical associate professor Heather Mack,
who is leading the project. Thanks for your time today, Heather.
S2 (00:33):
Thanks very much for talking with me.
S1 (00:36):
Now, before we talk about the research in the survey,
can you tell us a little bit about yourself and
your involvement in IDI?
S2 (00:50):
Well, I'm a I'm a Melbourne based ophthalmologist. I practice
mainly in medical retina. And I see quite a few
patients with I do in my clinical practice. I'm a
research associate honoree at the Centre for Research Australia, and
(01:13):
that's enabled me to work more on on the science
of patients with biology. I've been working on and off
in this field for my whole career. I did a Ph.D.
in marketable ideology so that I could understand the genetic
basis of these diseases. And I did training with Elliot
(01:36):
Burton in Boston, who your audience might know was a
world leader in this field.
S1 (01:43):
So at the moment, the prognosis for retinal disease, there
isn't currently huge treatments are around I'd.
S2 (01:56):
But I do know in the past 10 years there's
been a transformation in treatments of many of the medical
retinal diseases. So for example, patients with memory generation and
diabetes now have really effective treatments with injection of anti-VEGF
agents into their eyes. And so that allows us now
(02:20):
to turn our attention to the patients with idea to
try to improve their outcomes, the equivalent of the other
medical retinal diseases.
S1 (02:29):
Now we were talking earlier that gene therapy is actually
coming to Australia. You were saying it's been approved in
other countries in the USA.
S2 (02:43):
Yes, gene therapy has been approved in the USA and
in Europe for one gene aged 65. And the retinopathy
associated with abnormalities of that. The drugs called Luxturna, and
it's passed through the many regulatory barriers in Australia. It's
approved and we're hoping that treatment will begin later this year.
S1 (03:08):
But before you begin treatment, what you want to do
is hear from people with inherited retinal diseases and what
their understanding of gene therapy is is that am I
getting this correct?
S2 (03:24):
Yes, absolutely correct. In some ways, there's a disjunction we
have the science which has come up with this fabulous,
world leading treatment. But we have a patient community and
we really don't understand what they know about the treatment.
And it's very, very important that we're able to understand
(03:46):
that and give them education, allow them to make informed
choices about the new treatment.
S1 (03:52):
So let's talk about the survey. What kind of questions
on the survey? Are people going to be asked?
S2 (04:01):
Well, as is common with any survey, they be asked
a few demographic details. The main guts of this survey
is a new instrument that we developed specifically specifically for
this purpose, and the questions revolve about understanding of the
mechanism of gene therapy, what the outcomes of the treatment are.
(04:27):
What the cost to the government might be, not to
the individuals, to the government, and also questions how it
might differ from clinical trials because there's quite a difference
between an experimental clinical trial and an approved treatment. Our
survey respondents are also going to be asked standard questions
(04:48):
about their visual function and their quality of life and
their understanding of clinical trials, and will correlate those those
responses with their understanding of gene therapy. I'd like to
reassure all people who would consider replying to to the
(05:09):
survey that their responses are confidential. They're not able to
be identified and the outcomes which they can receive if
they request a completely de-identified, they won't be able to
recognize themselves in the outcome.
S1 (05:24):
Because I was going to ask. It's not a test.
Then with this information, what will you do with this information?
Why is it important for the Center for AI Research
to have this information?
S2 (05:40):
It's important so that we can understand what education our
patients need. So that we can assist with that and
assist our patients when they're making an informed decision about
whether or not they'd like to have this treatment. It's
not as simple as having a COVID vaccination. This treatment
is as a lifetime treatment, and it requires a lifetime
(06:04):
of monitoring. We'd also like to be able to use
the information when we're dealing with the government, the regulatory
bodies and those who will be paying for the treatment.
S1 (06:15):
Now we're told the standard number for people in Australia
with D is about 15000 people. How many respondents would
the Centre for iResearch need to make informed or create
the information needed? And who would you like to hear from?
S2 (06:38):
We're estimating that we'd like to hear from at least
500 individuals who were living with IAG that would make
a statistically significant sample. So far, we have about 270
completed replies, so we're getting there. But so far we
don't have a statistically significant sample. We're looking for people
(07:03):
to reply to this survey who people with IAG living
in Australia, not internationally, living in Australia. We also have
the capacity to take responses from the parents or the
carers of people who are living with IAG.
S1 (07:25):
If you'd like to participate in the survey, there's a
number of ways that you can do this. You can
head to the Center for iResearch website Sierra dot org
dot AUC right dot org dot u. You can also
choose to complete the survey on hard copy paper form,
(07:46):
and you can also contact the researchers if you have
low vision and want to dictate your answers to a
researcher who will enter the data on your behalf. And
to do this to do either one of those you
can call zero three nine two nine eight six two
one eight zero three double nine two nine eight six
(08:09):
two one and those details, plus an email, is also
available on the Center for AI Research, a website and
I have been speaking with Clinical Associate Professor Heather Mack,
who is a lead of the Center for AI Research Project.
(08:30):
The survey for people living with inherited retinal diseases. Heather,
thank you so much for your time and thank you
so much for your work on this, and I hope
you get a good response.
S2 (08:45):
Well, thank you so much for speaking with me today.
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