well welcome welcome welcome everyone to the John Curtin School of medical research uh my name is Ricardo natoli and I’m the head of the Clear Vision research lab here at the John Curtain school and the associate director of research development at the school of medicine and psychology uh it’s a real privilege to be here for for a number of reasons uh but first um to be representing a little bit of rner Australia who I’ve actually joined as a director uh at the start of the Year BR Australia was one of the first funding agencies to actually ever give me any funding and Seed funding able to use some of that money actually to turn it into a successful uh nhmrc Grant so without the support of rner Australia we wouldn’t have had any of that support and also at the local era people such as Jan James who would did a remarkable job with the local rner Australia community in the ACT uh really helped support the Inception of CLE Vision research so before I begin I just wanted to really thank you and thank patients and the community for all the support that they’ve showed our research over the last 10 years so thank you very much and this is an attempt to sort of reinvigorate a lot of the stuff that was being done in the local community and largely driven by Julia Hall who will be uh chairing this session today uh who is the new CEO of Retina Australia um one last thing I just want to tell you a little bit about John Curtain and what we’re doing here so the John Curtain School of medical research It’s a Wonderful research environment where we bring together different disciplines including immunology and genomics and RNA biology and Neuroscience together to solve some really really complex problems please come on in guys um and so having those collection of researchers coming from different areas to address really complex problems is vitally important and we can only do that through concerted effort of research working together and with that I’d like to introduce as well Thomas PRI who’s the center director for the shin Oran Center for RNA Innovation who’s working alongside groups such as Retina Australia to bring new Therapeutics in the RNA space to Market um with all that said I’d like to welcome you here I’d like to also begin by saying I’d like to acknowledge the traditional lands on which we meet and pay our respects to the eldest past present and emerging with that said I’d like to hand over now to Julia who will take us through the rest of the session thanks everyone for coming thanks so much Ricardo I’d also like to extend a very warm welcome to everyone here today it’s so nice to see so many people um for an live in person event and also to have everybody online as well thank you so much for your interest I’d also like to thank our key sponsor Apellis thank you so much for your support and our event hosts today C Vision research the John Curtain School of medical research and the Australian National University uh we have three speakers today uh Dr Carla Abott who will talking who will be talking about discovering emerging treatments in Geographic atropy our second speaker is Nick barf who will be taking us through his three minute Fe on using the message of exercise to prevent blindness and Dr Adrian cioanca who was our 2023 research Grant awarde and he’ll be reporting on his research report and we’ll learn about how targeting inflammation can protect against retinal inflammation after each speaker we’ll have time um for some questions so if you’re in the audience here today just please raise your hand and someone will come around with a microphone um and you can ask your question if you’re online uh there is a button you can press to raise your hand or you can just type your question in the chat box if we don’t have time to get to your question today please feel free to contact our office um you can email at info retin australia.com toau and we’ll we will follow up with the research about your question to kick up our presentations I’d like to introduce Dr Carla Abbott Dr Abbott is a senior research fellow at the center for I research Australia um and the department of surgery Opthalmology at the University of Melbourne and she’s also an optometrist at the Australian College of Optometry her current research focuses on identifying new treatment strategies for retinal diseases including aged related macular degeneration and inherited retinol disease Dr Abbott trained is an optometrist and obtained a PhD in Vision research at the University of Melbourne prior to completing postdoctoral fellowships at the Australian National Vision Research Institute the saite Institute and Divas Eye Institute in the United States she’s been a part of the senior leadership teams of the bionic eye Research Unit and the macular Research Unit at SI since 2013 Dr Abbott was awarded Fellowship of the Australian College of Optometry in 2014 the Dr hitesh pesha varah award for outstanding contribution to S by an early to mid-career researcher in 2020 and the macular disease Foundation Australia Excellence award in research award in honor of Richard gr’s am in 2023 Dr Abbott is also a current appointment on the Optometry Board of Australia Today Dr Abott will be talking about the emerging treatments in Geographic atrophy a late stage form of age related macular degeneration for the first time there are now approved treatments available in the USA and it’s expected that these treatments will now be considered by Regulators in other jurisdictions including Australia in the coming months this talk will cover diagnosis the potential new treatments on the horizon the patients that these treatments are best suited for and opportunities for clinic to be involved in clinical trials let’s welcome Carla to the stage hi everyone um so thank you very much for that very warm welcome and I’m really pleased to be here and be given this opportunity by the organizers to come and have a chat today so um really Keen to get your questions at the end but I’ll try and make sure that I’m speaking for those who aren’t able to see the slides um clearly as well so uh yeah as introduced I will be talking um mainly about geographic atrophy which is a late stage of age related maculate Generation Um but first I would just like to acknowledge the traditional owners of the lands we are all meeting on today and pay my respect to Elders past and present and I’d also like to extend that respect to any traditional owners joining us here today as well um before we start just a couple of disclosures and acknowledgements so um I did get my flight paid for from Melbourne to attend um by rner Australia today but I have no other disclosures and um I work within the macular Research Unit at Sierra which is headed by Professor Robin gimer who’s a medical retina specialist and she has also contributed to this presentation and has a number of disclosures as listed so first just a little bit of the basics about age related macular degeneration and its clinical classifications um so we know that at late stage age related macular degeneration or I’ll call it AMD for short causes central vision loss um so there is an image up there showing what things look like clearly um with a normal vision compared to Vision with um AMD where you have central vision um missing um and in terms of what you see at the back of the eye at the retina um there’s a gradation between or a clinical stage progression between when you have no aging changes at the retina at all um to when you start to get early signs of AMD which um to start with its very tiny little yellow dots um called drusen that start to develop they get bigger um when you get diagnosed as a clinical stage of intermediate AMD but both the early and intermediate stage don’t usually cause a lot of symptoms and it’s not until the late stages where you actually have photo receptor loss which are the cells that um detect light in the back of the eye um where you actually get that central vision loss and there’s two types of late um AMD both what we call Geographic atrophy or I’ll be using GA for short since that’s what we’re going to mainly talk about today and that’s the atropic type or you also get the neovascular type which is when there’s bleeding in the back there um so both stages and the uh Geographic atropy type can also change to the neovascular type as well so that’s just a little bit of an introduction to AMD and I’ll be focusing on the ga um dry type um today so although AMD is a leading cause of vision loss um affecting about one in seven people over 50 historically there has been quite limited treatment options to slow the vision loss um so neovascular AMD um had first intravital anti F treatments approved way back in 2004 in the US to start with and Then followed through in Australia um and so those treatments have been available now for 20 years um but Geographic atrophy the other late stage um has not had a treatment at all until the first ones were approved in the US last year um which is not actually approved yet in Australia but we’ll be talking a bit more about that today and in early and intermediate AMD there’s no Treatment available um currently to prevent people from progressing to the late stages either neovascular or atropic
disease so today we’re going to be talking about the emerging treatments for Geographic atrophy or GA so in February last year in the US the FDA approved Peg seta Co plan um or also called ciorra as the first treatment to slow down the progression of ga and six months later in August they also approved um another um drug a vasin caped pegol um as another um approach so these companies will also be applying in other jurisdictions including Australia um and the update on that at the moment is that um in Europe it’s still a under um evaluation and in Australia there hasn’t been any announcement yet about either Medicine by our regulating system which is is the therapeutic Goods Administration um so we’re waiting um eagerly to hear what’s going to happen there and we’re going to talk about all this in a bit more detail um there’s also several other novel interventions currently in late stage clinical trials for GA so what are these um new emerging treatments doing so um the most common pathway that’s being looked at is the complement pathway and trying to suppress inflammation so the complement pathway is a group of proteins that are helping the immune system to work and I do have an image up there of um the roundabouts of that system um but essentially there’s strong evidence that implicating overactivity of the complement pathway in um how AMD develops and hence there’s many trials for GA now that are um targeting potential treatments um that work on trying to inhibit the complement pathway so reduce that overactivity that’s um known and um the most common point of uh targeting that is by what we call the C3 and C5 inhibition um which we’ll talk about um in terms of the medications so we’re going to go through some of the clinical trial data firstly that’s resulted in the approval of these um medications in the US so firstly um the peg a CO plan um there was two trials called The Oaks and the Derby trials and these are what’s called phase three trials which is the later stage um clinical trial development so there was over 12200 participants with GA across 200 sites internationally um that looked at um this Peg seta Co Coop plan um intervention and this is an anti C3 intervention um and it’s using inital injections either ly or every other month so that’s injections into the eye itself to um try and reduce the growth of the um atropic lesions so you can there is an image here that’s trying to point out that the macula is quite a large area at the back of the eye in the retina and the fobia what we call the fobia is the very center of the macula which has um the absolute finest Vision responsible for our absolute central vision so um and you can see there’s black areas in there in that image that are indicating the areas where the photo receptors are lost or we call it atrophic area and so that’s where there’s no vision so these studies were looking at the 12 and the 20 month 24mth change in the total area of these GA lesions to see if the drug was able to slow down the growth of the lesions and this trials also looked at a subanalysis of the lesion area change away from the fobia as well so so this is called extra fovial lesions um compared to lesions which are under the fobia called sub fovial lesions um and as I mentioned the f is that very central part of the macular and the trial has also been extended out to 60 plus months um ongoing but we’ll show you the 12 and 24 month data now so for the um Oak study this is the 12 month leion area data um in graph format here and for the whole data set so both the suboval and what we call the extra fovial lesions there was a 22% reduction in the size of in the lesion growth um compared to the people that didn’t get any of the um drug and um that’s for the monthly injections and for the every other month injections there was a 16% reduction in the growth of the lesions and then very interestingly when we looked at the pre-specified analysis of the extra fobal lesion so the outside of the um fobia there was a 35% reduction um in the growth of the lesions in the monthly group and a 21% in the every other month so it seems to work a bit better for the to slow down the extra fobal lesions um but I will come back to this a little bit later as well in the derby study um again looking at both uh all the data Al together there was a 12% reduction in those that are receiving monthly injections and 11% reduction in those receiving every other month um injections and then when they look just at the extra fobal lesions there was a bit higher um rates so 25% reduction in um the every other month and a 16% reduction in the monthly
injections uh when we’re looking at both the Oaks and the Derby studies at 24 month 24 months you see a continuation in this um Trend so there was a 22% monthly reduction in those receiving the monthly um injections 18% in the every other month for Oaks and similar numbers um 19% reduction in the monthly group for Derby and a 16% in the every other month um for
derby um so just on to the other um drug as well that’s now been approved in the US the avasin captained pegol so this was a study with 448 participants internationally and they were getting um vital injections um as well and looked at the 12 month change in the total area of the um lesions so um the data you can see a similar trend line where you get a 14% reduction in those that are receiving the monthly injections um compared to people not receiving the injections um with one statistical method and they had another statistical method that showed very similar outcomes 177% reduction um now they did not um have an every other month option they just had a monthly injection option um this trial is also being extended up to 42 months ongoing um and they did not have an evaluation of the extra fobal versus Sub fovial data because anybody that had a suboval lesion was actually excluded from the study to start with so a little bit diff of differences in the study design in terms of so that’s the efficacy data how well it works um in terms of the Adverse Events stter or the safety of these medicines um both the Oaks and Derby compared to the gather two studies um were similar so there’s very small numbers of Adverse Events that are similar to other intravital injection studies of course you’re putting a needle into the eye so there is a small risk very tiny risk of infection and so forth um just because that’s the approach um the main thing to note is there was seen an increased prevalence of the neovascular type of AMD onset in the treated groups compared to those that um did not get treated and that was a 12% um onset in those receiving monthly inital injections 7% in those receiving every other month compared to 3% that got a sham injection um for the Oaks and Derby studies um and in the ga 2 study there was a 7% in those that were treated compared to 4% in in the Sham now all these people were then treated with the anti-f injections which is the common um treatment for those with the wet type of AMD or neovascular AMD um so it was just a matter of treating those people with with that and as I said there is a small risk anyway of developing going from Geographic atrophy to um neovascular AMD the other thing of note that was actually not found in the trials um presumably because the cases are so low but is under um ongoing investigation is the American Society of Retina Specialists reported 13 cases um from Real World data of retinal vasculitis within 14 days of injection that was not found in the trials um and there’s also been one case that was reported as well um using the um vasin caped pegol um drug and so there’s ongoing um investigation into that there’s no cause that has been completely clarified at this time and the exact rate of cases is unknown but it’s very tiny however because this um vasculitis can have a severe visual impact um so you can have uh great loss of vision um this risk needs to be explained to patients at consent just comparing the two studies a little bit more The Oaks and Derby to The Gather 2 so they did have the same inclusion criteria for the lesion size Baseline so the same people going into the studies with the same types of lesion size um but they did have a different inclusion criteria for vision so the Oaks and Derby participants had to have what we call 69ine or better Vision which is up to three lines of central vision loss on the chart um whereas The Gather two could actually be up to what we would call um 695 Vision which is actually greater than legal blindness in this country so there was quite a difference in who was included in terms of their um vision and then um for the leion location there was also a different criteria as we’ve already talked about a little so the Oaks and derby um participants could have the sub fovial um or extra fovial lesions while gather two could only have the extra fobal lesions so they actually excluded if anyone with the suboval lesions um and so the implication is because over half the eyes in the Oaks and Derby studies had suboval lesions and these gener have a slower growth rate than the extra fobal lesions it’s unsurprising that they had a slower reduction in lesion growth compared to the Sham group as the Sham group will also have a lower rate of growth in its in itself just to do with the underlying knowing how those lesions change and the other thing that was different between the studies was the criteria for neovascular AMD in the fellow ey or the other eye so the Oaks and Derby studies did allow people with neovascular AMD in their fellow I to um be enrolled and the other two did not so um this means that the risk of neovascular AMD development is different between the studies because it’s known that people with neurovascular AMD and the fellow eye are at higher risk of developing neurovascular AMD in their other eye already so just the key messages on this um so the um monthly or every other month injections of both of these medications have been shown to decrease the rate of GA lesion growth especially for extraoral lesions overall the medicines are generally well tolerated however there was an increased rate of neovascular AMD which can be managed and there are ongoing investigations into post study reports of retinol vasculitis which is in very small numbers and we still need to understand more um it’s important for patients to note that there was no noticeable day-to-day effects of visual function and that patients won’t notice an improvement in Vision when you get these injections which is quite different to the neovascular AMD where um you do get this Improvement in Your Vision after you receive the injection um so I’ll talk a little bit more in a moment about what this means in terms of clinical management but it’s just important to understand that um and of course there’s ongoing clinical trials to assess the longer term safety and efficacy of the um medications um for longer periods of of time so just to recap the regulatory side so the regulatory approvals for these were obtained in 2023 in the US um both the European medicines Authority and the therapeutic Goods Administration in Australia are still considering um their decisions so it’s not currently available in Australia I’d just like to very briefly touch upon other emerging interventions in advanced stage clinical trials for GA so this is um a table I’ve got up here that was published in retina today in November and basically it’s just to indicate there’s a lot of studies happening I’m in late stage clinical trials and there’s also a range of targets and delivery methods um that are being looked at including oral subcutaneous so under the skin or also oneoff sub retinol approaches meaning under the retina um however most are still targeting this complement pathway that we talked about at the start I’m just going to very quickly touch on two of those to give you a flavor so the beauty of potential subcutaneous injections is there’s potential for self- administration so not having to go into the opthalmologist office every month to get your eye injection um so this one is under the golden Phase 2 trial um which is looking at an inhibitor of complement Factor B targeting the liver because the liver is um responsible for the production of that complement Factor um it would be able to provide simultaneous treatment for bilateral GA you wouldn’t have to get injections in both eyes you just need an injection Under the Skin it has over 300 participants and they get their subcutaneous injections every four weeks this is still under trial um meant to be finished this year um so there’s no um regulatory approvals for this at the moment the other one to highlight is Gene and cell therapies which have a potential for a once off treatment so none of this every month having to do anything you get one treatment once um and then that’s it so the um thing with this is it does require a sub retinol injection which means under the retina so you need a vitro retinal surgeon and an operating theater but then you only need that once so what are these for so Gene therapies are to modify a gene that not functioning properly and it’s basically bringing in a viral Vector to bring in a healthy copy of the gene to act on this complement pathway for example cell-based therapies um are aimed at replacing the retinal pigment epithelium um which is an important layer below the retina known to be affected in AMD and this can be um applied as a suspension or otherwise on sheets or a scaffold and it can also it’s made using stem cell technology um either human embryonic stem cells or otherwise induced Pur potent stem cells um and these are of course talks in their own right with a lot of detail about how all this works but just to give you a flavor the trials um for this is due for completion in 2029 these um later stage trials happening internationally um but at the moment there’s no regulatory approval anywhere so a little bit on how to get involved in clinical trials if you’re interested um firstly why why why would we do this so I guess advantages are the potential to receive a successful intervention um knowledge that you’re contributing to the finding of interventions or improving the understanding of ey disease and also access to a Cutting Edge research team with regular reports sent back to your primary eye care practitioner however the points to keep in mind because trials aren’t for everyone um the randomized nature of trials mean that you don’t know and you’re treating um researchers don’t know because it’s masked if you’re in a group receiving interventions or not so um you have to be prepared that you might not be getting the um intervention and often um the way a lot of clinical trials are at the moment and again there’s there may be sort of changes in this is that at the moment you have to usually get into a central um location such as I Hospital regularly which involves regular travel um at the center for I research Australia there’s currently 13 trials at on offer for AMD and I’ve sort of just listed them all there um the trials are for all stages of AMD and sometimes the participation just involves a one-off research visit up to Interventional studies requiring two or three years of visits um but clinical care is always maintained with your referring practitioner and um letters are sent back um you can directly register your interest for a trial on our website so there’s a big button there saying register your interest when you go out and it just takes you through a guided process of filling in an online form um so it’s quite easy to do that if you’re interested of course you can also talk to your optometrist or opthalmologist um if you’re interested to register for trials and there’s usually trials available in each major um capital city um in terms of the changes in clinical practice so what are the implications of these emerging treatments becoming available for GA going to have on clinical practice so it really is a dawn of a new era because this is really the first time there’s ever been treatments available for GA um which is incredibly exciting and um opthalmologists and optometrists in Australia even though it’s not approved yet have been preparing new clinical guidelines for GA when the treatments become available so ransco which is the governing body of Opthalmology have just um published their new referral pathway um last month online and um the optometrist one is about to be released very shortly as well so um that’s fantastic um it’s important to still recognize that the treatment is not suitable for everybody um and that it needs to be talked through on an individual basis to work out is this something that’s going to be right for this person or not um the clinician will need to be considering the progression risk factors for that person the treatment risk factors for that person and the treatment buring burden including that it at the moment at least it involves lifelong monthly in ritual injections um and also the fact that the patient um just to look after their expectations it’s going to reduce the amount of vision loss due to GA over time but it’s not going to cause a day-to-day noticeable difference in the vision so the patient needs to be prepared for that because that’s different to wet AMD for example um Imaging of the retina over time is going to be key to clinical decision making so it’s important to identify those at high risk of progression from early stage AMD to GA and our labs done a lot of work in this space identifying biomarkers and also it’s going to be important to those with GA lesions to work out who’s going to be most likely to progress to underneath the fobia and cause that fobal vision loss which is a really Central um central vision and as I’ve already said there’s a great importance on individual counseling on the risks and benefits and talking it through with each patient so just to sort of show you how we would look at the rate U the risk of Le leion growth over time so the Blackness that you can see in these images of the retina is the bits with no vision the parts that have atrophy and then you can look in different patients so we’ve got a patient up the top with extra fobal lesion starting out outside of the phobia how it changes from year one to year two to year three and the risk that it might come in underneath the fobia um versus someone down the bottom there who’s had um a suboval ga and how that changes over time from each year so this is the type of equipment that we’ll be using to make those decisions and to talk with patients about the risks and benefits okay last slide take home messages so there’s currently two proven interventions to slow the progression of G that are now approved and freely available in the US um there’s many more Trials of Novel interventions for GA underway those targeting this complement pathway are the most numerous and in later stage clinical trials um and these trials are international and are available in Australia including where I am at SI um and whilst the frequent inital injections are the mod of delivery of many new interventions other routs are also being explored which is really exciting and um uh you can rest assured that your um clinicians the optometrist and opthalmologists in Australia are preparing for this time when GA treatments become available here and um we are recognizing that conversations with our patients about individual treatment potential and risks is going to be very important so that’s um really what I wanted to share with you today and of course I’m happy to take any questions thank
you the question yep so the question is what is the uptake of these um new drugs by patients in the US um I’m not really I mean I guess because it’s real world data it’s very hard I mean so we’re getting some real world studies um starting to come through but um I guess I wouldn’t have the answer for that necessarily offand I’m not sure if um if would you have more information about that Mary thanks Mary I’ll just repeat a little bit that for those online so there’s been 250,000 um injection well people being uh choosing to uptake um the Pega Co plan in the US since it’s been available um since February 2023 and um that the um rates of compliance and um so forth has been quite high in the trials and also most people are opting for a six to8 week um in conjunction with their clinicians um injections rather than every month so about 70% of people are um using the six to eight week regimen rather than monthly I think I got all that right yep so that’s there we go that was um Mary from a palace uh giving you the information from
directly uh yes just yeah yeah yeah I’ll just um just m kind of question um you got this current practice in the US U looking here when it gets to here what do you expect the cost will be will it be PS kind of produ um cost um yeah do do you want to just down and speak into the do you just well because there’s people on um there’s another doubling of the audience online if you don’t mind so I’ll just repeat the question the question was basically once it’s actually approved in Australia this new drug what is the cost to patients going to be is it going to be on the PBS or is it going to be user pay yes so the company has plans to seek reimbursement through the PBS um so we plan to submit next year following from on from getting um regulatory approval by the therapeutic Goods Association Administration sorry TGA in the US what’s the position um the in the US there’s a different market so it’s they have a completely different model so in Australia you know we have a universal healthc care system um whereby the majority of therapies are hopefully funded and available on PBS and patients pay co-pay um in the US they’re largely funded by um Private health insurers there is a different kind of a fund called Medicare which is completely different to Australia it’s not the same Universal type of an arrangement um so it’s a completely different Health Care system in terms of paying for therapies so patients usually have to have Private health insurance if they’re not um able to afford um therapies out of pocket should start by say sounds
I hope that helps
thank probably have time for one more question if there’s one more question that yeah while you come coming down so essentially the question from the audience was uh what why was it approved in the FDA and not approved in the European
market um it’s a good question but each country has its own um regulatory body as you know so FDA in the US EMA in Europe in Australia it’s the TGA um these countries do um conduct their um regulatory evaluation of the submission in different ways um they are independent um but they do look to what’s happening in the rest of the world so for example Australia will be the the Department of Health in Australia will be very interested in what’s happening in Europe but they operate completely independently make their own um decisions as to whether or not a they consider a product to be um efficacious safe and they also look at quality as well from a regulatory
perspective thank you just in the interest of time we might move on to our next speaker and thank Carla for a wonderful
presentation thank you Carla that was a great presentation next up we have a special short Pres presentation by Nick barf Nick is a PhD student at the Clear Vision research lab here at the John Curtain School of medical research after completing his bachelor’s degree in biomedical science from the University of cber in 2019 he moved on to an advanced master’s degree in neuroscience at the a&u graduating with first class honors in 2021 Nick’s research at Clear Vision research is focused on identifying and explaining how the active physical exercise conveys protective messages to the retina and to see if we can harness these for the treatment of retinal degenerations last year Nick presented his PhD research in the three minute thesis competition entitled using the message of exercise to prevent blindness in this competition you must present your PhD research in under three minutes to a non-expert audience with only a single static PowerPoint slide to support your talk Nick ended up placing second at the a&u Grand Final let’s bring on Nick to hear his three minute
presentation thank you so much Julia uh it’s always fun having getting the opportunity to present this uh presentation without a timer that’s ticking down and then so it’s always good so may go over three minutes if someone’s timing it don’t yell at me please so for my three minute talk this is the P the PowerPoint slide that I chose and it will make sense in just a sec so when I was 15 years old I remember waking up in hospital not being able to see anything out of the bottom left of my vision I had a stroke and because of that my vision now looks like this now I want you to just imagine that that blind spot is actually in the center of that image because that’s what people see every day when they suffer with a devastating disease called age related macular degeneration or AMD AMD is caused by damage to the important piece of tissue that lines the back of your eyes called your retina which is responsible for your vision but this damage only worsens with age and even worse the particular part of your retina that actually damages is responsible for pretty much all your useful Vision including your central vision so when you suffer with AMD the center of everything you see disappears as the leading cause of blindness in Australia AMD will affect one in seven people in their lifetime meaning at least one person in each row here today will eventually lose their central vision what if we could stop this from happening to you using something as easy as exercise I mean we all know how good exercise is for us going to the gym makes you feel stronger running makes you feel fitter but despite these physical benefits the precise mechanisms as to how exercise protects our bodies yet alone our eyes from disease especially AMD still not understood we’ve shown that just four weeks of physical exercise can actually protect your eyes against the same damage that causes AMD my research has found that when you exercise your body produces small molecules called extracellular vesicles which act like molecular text messages inside your bodies not only does your body already use these messages as a normal way of talking to one another but it’s only when you exercise that the messages that are sent to your eyes are changing what they are saying and that’s what’s protecting your retina from damage when we took a handful of those messages from an animal that had exercised and put them into the eye of an animal that didn’t exercise that animal showed almost the same protection to damage as the exercised animal so can we use this molecule as the message of exercise and practically prescribe it to someone for whom exercise isn’t an option imagine being able to use your body’s natural response to exercise using what’s already inside our body to stop AMD and stop you from losing your eyesight later in life I want you to take a moment to look at the image on the screen and just imagine waking up every day and seeing that because of my stroke this is what I see when I look at this memory of me and my beautiful Mom this is what fuels my passion for finding a cure for AMD because if we don’t do something now for one in seven of you that blind spot will be in the center that image thank you are there any
questions what sort of EX so the model that we’ve shown protective effects of exercise is just aerobic running just voluntary
exercise yes so you said that it could um recreate those
messages yeah how would you do that well if we can find what’s inside those messages when we uh took messages from animals that had exercised and then we took the same messages from animals that didn’t exercise and we saw a functional difference in the degree to which they protected the retina in between those two groups so if we could find out what’s actually inside those extracellular vesicles find out what rnas what lipids what proteins are inside them could we make what’s stopping us from making our own message of exercise and giving it to someone who physically can’t and like so the exercise extracise of vesicles the contents are different to what’s inside the the 7ry ORS we’ve tried we’ve um tried to extend the experiment so what we actually tried to do is we tried to like eliminate these extra vesicles all together but we saw some interesting finding so it’s still on the on the the cards to F the experiments that need done this two wonderful questions online so I’ll just try and repeat them the first one is Mick how much exercise yeah so how much exercise how much exercise needs to be different so we’ve shown that four weeks of exercise provides functional protection to the retina um following this the damage that causes AMD but two weeks of exercise didn’t actually provide that degree of functional protection we did start to see molecular changes in the retina um that hinted at changing levels of inflammation in the retina but two weeks didn’t provide that level of functional protection um but four weeks I’ve very curious to see whether 8 weeks or 16 weeks or a years worth of exercise would be enough to provide that level of functional protection and the the second question uh was what what are extra cular of vesicles so extra cellular vesicles are as I mentioned in my talk they’re like molecular text messages inside your body so every cell in your body normally uses them to talk to one another exchanging information so the cell is like I I need this give it to me I’ll give you this back it’s another way it’s a way that your cells Ed to talk to it’s they like text messages inside your body and yourselves use them normally but when you exercise the number of these messages actually increases by two threefold and it’s yeah we think that’s what it’s what’s inside them that’s conferring The Beneficial effects of exercise think there was one more question from Daisy thank
so when we question yeah so the question was how do we administer these extra veses so what we did to show the the protective effects of these exercised extracise vesicales was we exercised some animals for four weeks and then we didn’t exercise anals for another four weeks and we took the EVS from just their blood so just a systemic response of these EVS uh we isolated them by spinning them very fast and we put them straight into the of an animal that didn’t exercise and then we exposed them to a a damage Paradigm that mimics the effects of AMD and we saw that the exercised EVS actually were more protective than the cry EVS so that leads us to think it’s what’s inside those EVS that’s what’s confering the molecular and beneficial effects of exercise so answer your question yes interjection so straight into the eye ideally it would be a the dream scenario to give like an oral administration of these EVs and somehow they make their way to the eye and that would be the dream goal we do have a question from Ryan top I just say
yeah so the question was when we measure the amount of exercise over two four weeks do we have certain animals that don’t exercise as enough how do we quantify the amount of exercise um because we do these in animals we have a bunch of uh running data metrics that we have and it is a voluntary exercise Paradigm so we’re not actually forcing these animals to run uh and they tend to do a lot of their running at night and overnight these mice run I’ve seen a mouse run 38 kmers in one day some of them run a lot more than other mice but they they run from like 10 to 20 to even 38 km in one day so over four weeks they run four or five marathons worth of running but what we’ve actually found from some interesting data we have is that it’s not actually the distance that is a a measure as to the degree of protection that they might as well have it’s actually the amount of times that the animals engage in exercise that is the most influential Factor on what’s happening in the
eye MUSC cells that’s what I would I would love to know that because a a key drawback with the research of extra vesical is cell surface markers I that identify which cells these EVS are coming from because all the cells in your body like every cell produces EPS just normally it’s hard to identify what cells these EVS are coming from um there’s been a lot of re research uh hypothesizing they coming from platelets sprad blood cells muscle cells this have been some people that have shown that they don’t come from muscle cells but other people saying they do so I’d love I’d love to know
that go right ahead just in terms of like tested it in terms of protection the Y is there any other effects you noticed on other
they get theit of
do that’s that’s a very common question um that comes up with this the EV story is could you get the same sort of physical benefits of exercise by injecting the EVS not that I know of um we were obviously taking the the systemic responsive exercise from the blood EVs and putting them straight back into the eye the more the effect we were hoping to see was just the less neuro degeneration um we wouldn’t see any other sort of physical effects by injecting them into the eyes but no I I’m I’m unaware of people taking EVS injecting them like from athletes and seeing if they run
faster would be
interesting uh we do have a question online I believe uh is there research show that people who exercise more have less incentives at aod yes there are a lot of a few population studies that have been done that have shown that people who report higher degrees of physical activity are less likely to develop AMD um but there are limitations in reporting of physical activity because not many people want pivot to know what they’re
lazy sorry follow up number of people in yes so I’m a
worried I’m also
worried when you said running are there other exercis you do we believe that you could do it come
on um so that’s a good question question are there other forms of exercise there is a lot of research into which form of exercise provides the highest degree of protection but unfortunately no one really looks at the eyes in that aspect usually it’s to do with memory memory loss uh motor impairments and all that other sort of stuff but there is a lot of research going into high-intensity interval training which correlates well with our running data um shows the number of times you engage in exercise it’s not actually the degree or like the amount or the load of exercise that you’re actually doing um obviously like strength athletes will be a lot stronger but less fit than a running athlete but will they have the same level of protection in the eye will they have lower inci of incidences of developing AMD I’m not sure on aspect but the data we have goes show it’s voluntary exercise um so just running and whether 20 30 km in a mouse translatable to a human that’s like an ultramarathon a day keeps the blind
look if there aren’t any further questions can you please help me in thanking Nick for wonderful thank you so much Nick we’ll all start our marathon training tomorrow I’m sure our final presentation today is by Dr Adrien chanka Adrian is a postdoctoral fellow here at the Clear Vision research lab at the John kurtain School of medical research his research is centered on the development of new treatments using RNA to Target certain genes involved in retinal degenerations Adrian’s experience in this area stms from training in molecular biology during his PhD the retina is the part of the eye responsible for capturing images and sending them to the brain his thesis explored why and how it starts to deteriorate or fail by closely examining the activity of many genes at once Adrien was awarded the Frank fenom medal for the most outstanding PhD thesis submitted in the John Curtain School of medical research for his PhD thesis in 2022 Adrian is also involved in the establishment of EOS site a biotech Venture emerging from the cision research group he teaches the genetics and cell biology component of the a&u medical program and lectures on retinal physiology within Neuroscience courses offered by the Eis Eis Institute of neuroscience at here at John Curtain School of medical research in 2023 Adrian was awarded with a research Grant from RE Australia the project using RNA silencing to tackle neuroinflammation in retinal de in sorry in retinal degeneration we’ll now hear about the results and outcomes of this project and learn about how targeting inflammation can protect against retino degeneration thank you rre well thank you thank you for the introduction Julian and thank you for the kind words so really today’s presentation is trying to describe to you guys how we spend the money that we’ve got from bre now Australia and what research we’ve done in this project so the key uh area of resear that we focused on was inflammation but specifically how can we target inflammation to actually slow down retino degeneration and what I’ll explain at the beginning of this presentation is how inflammation is really embedded in the context of and the um pathogenesis move the monitor so I can see that side so the motivation for doing this work is very simple it really can be summarized in four facts that I’ve got on this slide 200 million people right now are affected by AMD 1.5 million of them are currently in Australia and here we have a couple of patients that that have attended this presentation so really we welcome you and it’s always amazing to actually be able to hear the patient perspective and what it’s like to live with a disease because that really motivates us to do this work but it also enables us to probably optimize our research and really build better Therapeutics in fact 177% of Australians over the age of 50 experien vision loss as a result of age related macula degeneration and weer all about what really happens in the Rea in the previous presentation when someone has AMD we’ve also heard that there are two drugs now that are approved for AMD and it’s amazing to actually be able to see that you know patients to have access to drugs right now but also as as it is with any drug there is always room for improvement there is signs to be done to be able to improve how well these drugs actually work now the main topic for today is really inflammation and there are many definitions for inflammation and you know may some of you may be familiar with the idea that you know if you fall and you have a scratch or something like that that area which it is damage becomes inflamed if we look at the definition of inflammation it’s just take it from the dictionary is a biological response of the of body tissues to harmful stimuli such as pathogens damaged cells or irritants so for today really we talking when we talk about inflammation is the retinal response to damaged cells so again just to sort of make that very clear when I when I refer to inflammation is what happens in the retina when certain cells within the retina are damaged so there are many many reasons why cells can be damaged in the retina we F we F about some of the factors that may cause AMD and really we don’t have to focus on exactly what this what this stems mean here but things such as you oxidative stress there is accumulation of juen these are lipids within the eye they can accumulate certain cells within the eye may not function properly there are changes the to the mcure within the eye so blood vessels can change and a lot of those elements that occur within the eye many which are actually um stemming from aging and aging processes a lot of those can actually cause cell damage and what the retina does when there is damage it begins to respond to the damage and one of the responses that occurs within the retina is inflammation so what inflammation is supposed to do is actually attenuate or resolve some of the cell damage which happens within the eye the problem is that for many reasons that we don’t really understand when inflammation happens within the ROM it can actually damage even cells that are healthy they are undamaged they can actually be damaged by this inflammatory response and the rational for current drugs is that if we can slow down inflammation then maybe we have a chance to essentially slow down how the retina actually degenerates so really here’s the sort of the main idea carbon Therapeutics what they try to do and what we try to do through our research is slow down inflammation if we slow down inflammation then the disease should not progress as quickly and if it does not progress as quickly then maybe those patients would actually have this site saved so that’s sort of that’s sort of the main science that we have to cover today is just looking at how inflammation happens in the reum and how can we actually slow that down what you’re looking at here is a picture of a mouse R now and what we do in our lab is usually we try to understand when there is inflammation in the rum what really happen and what are some of the molecular factors what are some of the molecules that really Dr inflammation so while you’re looking here on on the left hand side here you can look at a retina of a healthy Mouse and the mouse retina and the human retina are very much the same there are some differences but really they very comparable and there’s one specific part of the retina that we want that we need to focus on here which is the photo receptor layer the photo receptor layer sits here in the retina and the photo receptor layer is really the cells that actually can respond to light so light comes into your eye it passes through your reum and then if eventually it hits the photo receptor cells when the photo receptor cells are detecting light they send a response to the brain and that is what Vision ultimately is but these cells are the ones that are actually damaged along with other cells in age related macular degeneration and if you look at this particular layer here there’s one feature that I want you to to sort of notice here you can see that there aren’t any of these other red cells within this area so what the red cells in this picture are called micrum but these are cells that in the Rea drive that inflammation so when we talk about slowing down inflammation what we really try to do is slow down how active this micral cells are and how much damage they can cause to the retina okay so then if we actually cause degeneration in the retina of a mouse and here what we do in our is we use animal models that can have degeneration occurring in the retina so here what you can see is a retina from an animal that actually has de generation and if you look at the same layer here the photo receptor layer which again is the layer that responds to light this particular layer here actually now has a lot of these immune cells here you can see in red which infiltrate into the photo receptor layer and because the infiltrate in the photo receptor layer is really why there is a lot of damage down to the photo receptors and if you lose photo receptors you lose vision it’s that simple now why is it so hard to actually slow down inflammation the reason why it’s very very difficult to slow down inflammation is because inflammation is a complex biological process we don’t have to go into the details of what happens at the molecular level but what you’re looking at in this picture is the molecules within the retina that we have identified to actually drive inflammation So within this within this Square here what we have is every single line here is one molecule or one Gene and if they show if they show up in blue it means that that particular gene or molecule is really not that it’s not found in the retina at high levels but if it is in red it means that it expression goes up and what you what we identify in the work that we’ve done for this and also some also previous work in the lab is that there’s at least 700 or so molecules or genes that actually can drive inflammation we’ve heard a little bit about how drugs or AMD actually work they target one one pathway and that pathway probably comprises of you know up to 10 molecules so current drugs affect you know up to let’s say about 10 molecules that can cause inflammation we know that there are at least 700 of them that can drive inflammation so if you want to improve how we actually treat namd what we need to do is be able to come up with a theraputic that actually can Target more than the more can Target a very large amount of proteins or genes that can actually drive inflammation and this was really the premise of our work is there a way that we can Target a large subset of these molecules that actually cause degeneration so we can improve how effective our drugs are in terms of slowing down inflammation so that’s the that’s sort of the rationale of our work now there are some ways that you can actually Target a large amount of molecules within a biological system and one way to do it is to actually identify special molecules called transcription factors so this this kind of molecules what they can do is they can go into a cell and they can actually make their way to the DNA of the cell in the nucleus and the nucleus is like the brain of the cell right so this transcription Factor can go into the brain of the cell and essentially orchestrate which genes or molecules are increased or decreased in expression so what we wanted to do in our work is maybe try to identify a couple of these transcription factors which are known to be linked to inflammation and if we can identify those then it gives us a way to actually Target a large subset of molecules that can cause inflammation so that’s really the premise of our work can we then find some transcription factors that we know that they they drive inflammation and to see if we actually inhibit them or silence them then would that actually be beneficial to the Rea that under goes degeneration so that’s exactly what we’ve actually done now before I actually describe the results of the work I just want to illustrate the power of transcription factors what can this molecule really do if you can find the right transcription factors in a biological system that controls a process you can really reverse that process and in fact some people have shown that in the retina of old mice these are old mice that have lost their vision the age of retinal cells can actually be reversed if you can’t find the right transcription factor to use in that process so so in other words transcription factors if you know which ones if you can identify the right ones can really reverse the age of all cells and then make those cells young again so this is how powerful transcription factors can be the challenge is though trying to identify the right transcription Factor now so because transcription factors can really reverse biological age we really believe that they can actually slow down inflammation I would argue that is a little bit more difficult to reverse age than it is to slow down inflammatory processes but maybe some immunologist if we have any in the audience probably will disagree with me but we can chat about it so how did we find these transcription factors okay so what we have the advantage of in our lab and in many many Labs now is we can take a biological system we can take a ream and we can basically look at all the molecules that change in expression if there is damage so we can so so what we’ve done is we’ve taken our animal model of degeneration and just to explain a little bit what this animal model is all we do is we have mice and then we just place them in a in they’re regular housing boxes but there is a bright light in there and being exposed to light for about five days it does actually cause some damage to the ream much in the same way that actually our eyes get damaged by light across our our time our time across our life so what what we’ve done is we’ve taken them that R and we looked at what is this gene expression signature in other words what what molecules really change in the retina that now has damage and then we applied some statistical methods and and a number of other the mathematical tools many of my coworkers call them Adrian ner things but we’ve taken that data we and then we’ve done a little bit of mats on that and then from that matth we’ve been able to basically make some predictions on like here may be some of the transcription factors some of these very powerful molecules that may drive inflammation and then once we have identified those then we can work on ways of actually controlling the action ways to inhibit them so essentially just to summarize this pipeline we looked at you know large data we’ve applied some statistical methods and then we found some key Regulators of inflammation in the retina it’s that simple so here what you’re looking at is we’ve identified three three of these transcription factors I willon mention their name because they’re horrible and they really don’t mean anything but well let’s just call them transcript Factor one two and three so tf1 TF2 and tf3 and what you looking as here at here in these graphs is very simple what is the expression of that molecule in the retina as the retina becomes damaged in our model so on the x-axis here what you’re looking at is just you know the time since damage so here we’ve got the time since we initiated the damage and here you can see that even within six hours after the damage we can see that all these three molecules are increased in the expression and if you look all the way down to five days you can see that these molecules are now expressed at very very high levels and we knew from our predictive analysis that I summarized in the previous slide these three molecules together all three of them actually control can control many other molecules in the retina that drive inflammation in fact I said that it’s about 700 different genes we identify that can drive inflammation and really together these transcription factors can actually together Target over half of them so now now we have the potential therefore of developing a therapeutic that not only targets five or six different molecules but over three to 400 of them which in principle it should really improve how effective a therapeutic might be in the retina so with that now for a particular like therapeutic or a drug work in the retina it really needs to Target the right cell types in the retinal and I won’t give a description of you know retinol Anatomy here it’s really not a of this presentation the key point is that you know as I said before there are some cells in the retina that drive inflammation they’re called Moga and microa the names don’t matter the point is there are specialized cells in the retina that drive inflammation so after we identify our key molecules we just look to see which cells are they in hoping that they are in the cells that drive inflammation and sure enough they will so here what you’re looking at is the morphology or the anatomy of the retina and in blue we’ve got the cells which actually have very very high levels of these transcription factors so now what we so what do we know we know that we’ve got three transcription factors that can Target hundreds of molecules that can drive inflammation and those three transcription factors are found in the cells that drive inflammation so we are working towards a potential way to now target cells that drive inflammation in the reum which really is the purpose or the Crux of our work so of course all all of our findings were from animal models and I brief describe what is the animal model that we use to actually drive inflammation and to cause degeneration but of course if you want our data to be valid to be solid we have to make sure that what we find find in animal modules really also happens in the RNA of people that have AMD so for that what we’ve done is we looked at the gene expression signature same idea we looked at all the molecules in the right now of people that have amm and this is tissue that comes from people that have donated their eyes for research and predominantly coming from The safite Institute and then here what we found is that the very same three transcription factors the same these three powerful molecules increase in expression in people that have AMD so the exact findings that we saw in our animal model also we see in people that have AMD which really is a great finding to have because what it means is we can take our animal moduls and really build on that data to be able to actually develop treatments and be able to test them in animal models before hopefully one day many many years from now make their hands into the patients that actually need them most now I’ll summarize just some of the findings that we’ve got from this work in terms of whether or not our approach to treatment works so here what we what we’ve done is we had out three different molecules that we knew if we inhibit them we should be able to slow down ratino degeneration and then what we’ve done is we made a cocktail of Inhibitors that can actually Target these three molecules so what these are so these are different set of molecules and if you inject those into the eye what they supposed to do is basically slow down how active these transcription Factor factors are so we have this cocktail of molecules that can Target our three key three transcription factors and we injected those into the eye then after that we we had our damage uh Paradigm initiated so this is where we again expose our mind to some to Bright Light and their retina sustains damage and then we assess that retina basically we look to see can this mice actually see better so in other words if we take this cocktail of Inhibitors and we inject that cocktail of Inhibitors into a mouse’s eye does that Mouse see better at the end of our degeneration Paradigm and then here’s what you here’s what you’re looking at what you’re looking at here is basically the response of the Rea to light and put simply what we found is that mice that received our injection with our Inhibitors could respond to light better and what we can say about this mice is that they can therefore see better so really this shows that you know our therapeutic at least has the potential to slow down how degeneration occurs in the retina and potentially allow for now mice to see better but hopefully in the future patients to see better we’ve also looked at another key factor here so I talked about that this approach to treatment really is a way to reduce inflammation so for that we also wanted to make sure that if we look at how much inflammation there is in the reum in response to our treatment that should be reduced and that’s why you’re looking at here here is the Ron of a mous that did not receive the drug and what that received the drug and remember initially in in my second slide I talked about like how these immune cells come into the outer retina here into the photo receptor layout and their presence there really causes a lot of damage so what we’ve done next is very very simple we said well okay if we inject our drug into the eye are the less immune cells into the photo receptor layer where they shouldn’t be and that’s what we found here you can see a large number of these immune cells found in the area of the retina that’s undergoing degeneration and if you look at the animals that received the treatment we did not see that many cells so what this indicates is that our approach to treatment really slows down inflammation and it prevents immune cells making their way to the to the photo receptor layer where where they can cause damage and therefore uh destroy the photo receptors and then basically that leads to blindness on the right hand side you’re just looking at the analysis of the data and and just to summarize it very simply if we if we inject our cocktail of drugs into the eye not only do we not only the animals can see better but they have less inflammation and we’ve Al we’ve done a number of other experiments to make sure that this actually works I won’t go through all of that by the point of of this presentation was to summarize what we have done this year which is that our cocktail of Inhibitors that we know which molecules they target can slow down inflammation which therefore can preserve sight but there’s one other one other sort of point that I want to touch on doing the research in the lab is amazing we we can actually you know inform the community on how to for example Target inflammation but if you want to get a drug from the lab into the patient’s hands we need to do much more than then the research and really what we need to see is that there is interest in this kind of therapeutic from actually large corporations such as aelis but but really any other large pharmaceutical company because to get a drug to Market it’s not cheap it requires a lot of work many many millions of dollars and it is a long and arduous journey and what what appears to happen in the market right now is that many large companies such as F IL noat they really try to invest into this idea of like using transcription factors as the Next Generation toties for many many many diseases they’re not really working on age related mular degeneration they mainly are focusing on cancer because in cancer if you identify the right transcription factors you can prevent cancer but there is a shift in using the same approach that we have worked on for the for the last two years and then because we see interest from other companies working in this area maybe this might be just the right time to actually try to form new Partnerships and really try to get something like this to Market and it’s amazing to be able to have in one room today researchers patients and also representatives of Biotech companies this is the kind of event that we have to have more often to be able to actually expediate the development of drugs and also bring transformative uh drugs to Market so the patients can actually benefit from them so that’s all I’ve got for now but I’m happy to take on
questions thanks aan please um yes I know um David Singler over the states has been doing work on biolog that was the work I presented
yes were you actually say that old BL could regain their yes that’s that’s exactly what they found so so they had a specific model which is a glucoma model which affects a retino gangion cell within the eye and what they found is that on the surface the gangan cells appear to be dead not functioning but when they injected three transcription factors they actually those cells became functional and they looked at the biological age so there are ways that you can actually say how old a cell is but just by looking at its DNA and what they found is that that cocktail of transcription factors essentially like you know not only did made those cells functional again but now they looked more like cells from a younger animal they’ve actually taken that to a next level where they basically had very old mice EX exercising running similar to what Nick presented and then after tuning the activity of specific transcription factors the old mice ran way more than the younger mice and I think one day David sinair would probably turn himself back into a baby he needs to be really careful like he just looks much younger than he used to I don’t know what he’s doing but we want some of
that so I guess if you lose if you lose the cell there’s probably no way to bring them back but in many in diseases such as AMD you’ve got let’s say many like Photo receptors in RP cells they are still there but they appear to not be functional so so long as the cells are not gone and completely atrophied this might be a way to actually kind of like reinvigorate them bring sort of bre life into them if you if you will any further questions from the audience here online Daisy repeat the question the question was whether or not this transcription factors have different levels of expression in different types of AMD such as early AMD intermediate and then late stage Geographic atrophy or neurovascular AMD the answer to that is when we actually look in samples from like early AMD intermediate and late and then what we found was that even in patients with the earliest form of disease actually had heightened levels of these transcription factors not only were these transcription factors very highly elevated in the retina they also found at high levels systemically and actually one of them was actually was one of the best predictors of early AMD so I guess to summarize that they appear to be switched on early in disease and then stay switched on all the way to the latest stage of disease we haven’t looked into neovascular MD we’ve only looked at early intermediate and Geographic atrophy my prediction is that they probably found at high levels in the in NE md2 and I guess to add to that we found them to be highly elevated in the central vetnam but also in the peripher in the periphery so I guess they’re very good markers of like early stress which probably happens across the entire Rea not just in the macula while people are thinking of their question so I might ask you a quick question and then I’ll come to you Michelle if that’s all right yeah thank you thank you just so less about the science and more about sort of rner Australia and what it meant to actually get some funding from R Australia to do some of this work as a as a young scientist what did that mean to you it was my first large brand that I’ve got and it really not only enabled us to do the work but it’s really a good way for me to be able to actually continue that work and then essentially to show to people that you know I can probably drive this research and then use this as a platform for larger funding which we are planning to apply for this year a couple of things are in the way at the beginning of the year but really the plan now is to basically use the data that we’ve got here and also the expertise that we actually have been able to get with bre austal’s help then take this fed by applying for larger funding and then probably try to somehow get this treatment to become a real treatment so patients can have access to it Michelle
[Music] question do you think
so we have looked in a couple of different other degenerations in um retin pigmentosum and then we actually found that they also expressed at very high levels and also at the same time I looked at some of the previous um funding that R now Australia has actually provided to forgot the name of the person actually got the money but the molecule they looking at has just been revealed to be one inhibitor one of these transcription factors I think at that time we probably you probably thinking it works in a different way but at least we’ve seen like intin Pigmentosa and also in glucoma that we seee elevated levels of the same transcription factors so I think it’s just the very very good markers of stress and inflammation and inflammation essentially is found in any type of degeneration not just AMD but inhabited inhabited diseases
to thank you might just add one follow-up question so Adrian uh we’re talking about random degenerations but what about neurod degeneration uh in general are there any lessons that we can learn from the eye that could be applied for other diseases like Alzheimers or Parkinson it’s possible I mean I’m not you know super familiar with the pathophysiology of some of the other ones but a key feature there say it again it’s inflammation and particularly there’s like there’s inflammation driven by asides there’s asides in the retina too there’s another type of cell in the retina called the mam which works similar to an asroy and then we know that these transcription factors in fact are found in the exact same same cell types that are also found in the brain they become very very active in other types of neurod degeneration such as alimer and also Parkinson we have looked through the literature a little bit and yes in some models of alam disease that appears to again be highter levels of the same molecule so maybe it seems like they’re going to treat everything we probably have time for one more question which has just come online which is more about Ai and the role that AI might actually play in trying to identify new molecules including potentially these transcription factors can you talk a little bit about how AI might be affecting your work well so with so with AI so you know so we’ve got essentially what we do these days is we take a damaged tissue and then we can really look at all the molecules that change in the damage tissue but it’s increasingly difficult to Bas to basically take that very large data set and find a way to then sort of get down to what is really the central element of that data and what should we really Target to be able to control this very large set of molecules soting with the advant of AI what we probably can do is basically make better predictions as to what are the right transcription factors to really Target and potentially be better at guessing what we need to Target to be able to develop new treatments so I think already in cancer that’s been happening and also with that as we get more and more data available from many many patients so we’re not limited by just by animal models but really get out from thousands and thousands of patients then using that sort of large data with AI and its predictive power could really inform what molecules to Target next and then if we build the right infrastructure for producing those molecules which I think it’s happening because of covid-19 AI with a sort of investment in infrastructure and then dedication from scientists and patients we can probably do
better on that can you help me thank Adrian for wonderful presentation back to Julia who will close out today’s thank you so much Adrian and congratulations on your very successful research project uh we look forward to how that research progresses um and hopefully in the future to Future treatment uh thank you to all our speakers today Carla Nick and Adrien such amazingly informative um and interesting presentations I think we can all be very encouraged by the progression of research and um and increasing our understanding by um being here to to today to hear directly from the researchers and also encouraged by the fact that there are treatments um which are approved overseas and hopefully will come to Australia soon thank you to our key sponsor again aelis for your support um and really allowing us to hold this inperson event today um and also to Ricardo thank you so much Ricardo and your amazing team for supporting us to have and hosted this event here today um all the volunteers who’ve assisted have been absolutely amazing thank you to all of you in the room for attending um it’s really so lovely to see so many faces and all all of you who are online today thank you so much for your interest um and joining us today for those who are here uh if you would like to take um a tour of the research facilities uh there will be volunteers just outside that we’ll be happy to take you um on your tour um right after the um this this close thanks so much and good afternoon
Retina Australia hosted a Research Update Event on 16th May 2024 at the John Curtin School for Medical Research (JCSMR) at the Australian National University in Canberra. It was also streamed live for viewers around the country who could not attend in person.
The event provided attendees with the opportunity to :
- Discover emerging treatments in geographic atrophy, a late stage of age-related macular degeneration (AMD) – by Dr Carla Abbott, Centre for Eye Research Australia and University of Melbourne
- Learn about how targeting inflammation can protect against retinal degeneration – by Dr Adrian Cioanca, Australian National University
- Hear how using the message of exercise may prevent blindness – by Nick Bariesheff, Australian National University
Emerging Treatments in Geographic Atrophy
Presenter: Dr Carla Abbott, Centre for Eye Research, University of Melbourne.
Dr Carla Abbott presented on the emerging treatments in geographic atrophy, a late stage of age-related macular degeneration (AMD). For the first time, there are now approved treatments for geographic atrophy, also known as atrophic AMD, available in the USA, and it is expected these treatments will also be considered by regulators in other jurisdictions including Australia in the coming months. Having access to potential new treatment options means it is important for clinicians to be thinking about who these treatments are best suited for and how best to discuss these new options with patients. This talk covered the diagnosis of atrophic AMD, the potential new treatments on the horizon, the patients that these treatments are best suited for and collaborative management approaches between optometry and ophthalmology. The talk also discussed opportunities for patients to be involved in cutting-edge clinical trials for AMD.
Using RNA-silencing to tackle neuroinflammation in retinal degeneration
Presenter: Dr Adrian Ciaonca, John Curtin School of Medical Research, ANU.
Dr Adrian Cioanca was awarded a Retina Australia grant in 2023 for the research project “Using RNA-silencing to tackle neuroinflammation in retinal degeneration”. Its aim was to develop a new treatment for retinal degeneration using a type of RNA called silencer RNA. Now that the project is completed, Dr Cioanca provided us with an update about his findings and project outcomes.
Using the message of exercise to prevent blindness
Presenter: , Nick Bariesheff, John Curtin School of Medical Research, ANU.
Nick Bariesheff, PhD student at Clear Vision Research Lab at the Australian National University, presented his PhD research talk that he made in the 3-Minute-Thesis (3MT) competition last year titled “Using the Message of Exercise to Prevent Blindness” for which he was awarded the runner up prize. In the 3MT, you must present your PhD research in under 3 minutes to a non-expert audience, with only a single static powerpoint slide to support your talk.
Vivienne Kaiser is a PhD student working at the Children’s Medical Research Institute under the supervision of Dr. Anai Gonzalez-Cordero. She completed a Bachelor of Science/Bachelor of Advanced Studies at the University of Sydney with First Class Honours in 2021.
She currently works on Usher Syndrome, an inherited disease that causes deafness and blindness in children. Her work aims to develop AAV-gene therapies for this disease using stem cell organoids derived directly from patients with Usher Syndrome.
Past Webinars
October 2024
Cell therapy, genetic research and a patient’s perspective
October 2023
Vision Loss Priority Setting Partnership and an Introduction to Stem Cell & Gene Therapies
August 2023