Understanding Clinical Trials for Vision Loss

Hadley

Understanding Clinical Trials for Vision Loss

Presented by Ricky Enger

Ricky Enger: You may be wondering what's available, like eye drops, vitamins, or even new treatment options for preserving or improving your remaining vision. In this episode, Rachel Retajczyk and Alex Homman of Ray Therapeutics join us as we discuss clinical research and vision loss. I'm Ricky Enger, and this is Hadley Presents. Did you know you can listen to our podcast plus find how-tos, discussion groups, and more, right from your phone or tablet with the new Hadley app? Search for Hadley Helps in the app store or text APP to 52545 and we'll send you a link to download. And now on with the show. Welcome to the show, Alex, Rachel.

Rachel Retajczyk: Thank you, Ricky.

Alex Homman: Thank you, Ricky. Excited to be here.

Ricky Enger: So happy to have you both, and I think this is going to be a really informative episode. There's a lot I'm curious about, so I’m looking forward to learning a little more. Before we get into our questions though, why don't we just do a couple of quick intros. Tell us a bit about yourselves and what you do. So, Alex, let's start with you.

Alex Homman: Yeah. Hello everyone. My name is Alex Homman and I am actually an optometrist by training and I now work on the medical affairs team with Ray Therapeutics.

Ricky Enger: Fantastic. And Rachel?

Rachel Retajczyk: Yes. Hi everyone. My name is Rachel Retajczyk, and I lead patient advocacy at Ray Therapeutics. And I've spent about 20 years in the biotech industry with much of my career working with patients and advocacy organizations to really ensure their voices help shape the development of new treatments. And I'm so excited to be here today and share more about clinical trials and the work we're doing at Ray.

Ricky Enger: Excellent. So, I think a good place to start is maybe a lot of us have heard the term clinical research, but it sounds very academic and sort of not something that I at least would be familiar with just by hearing the term. So why don't we start by just defining what clinical research is and why it's important for people living with vision loss?

Rachel Retajczyk: Sure. At its simplest, a clinical trial is really a research study involving people and it's how researchers learn whether a potential new treatment is safe and whether it might work. And depending on where a treatment is in development, researchers might be looking at different things like safety or the right dose or whether they're seeing early signs of the treatment is having an effect. And one thing that's really important for people to understand is that these treatments are still being studied. They're not yet approved and there's no guarantee that participating will improve someone's vision.

Ricky Enger: Yeah, that makes a lot of sense. I think that is really important to know going in because you may have some preconceived notions like, oh, I'm really excited about this and this is definitely the answer, but it isn't necessarily. So, when we talk about clinical research and clinical trials, are those terms kind of interchangeable or is there a difference?

Alex Homman: So, when we talk about clinical research, it really is how we move the field forward and the two terms are really interchangeable. So, you have research and then usually what comes out of research is progressing into that clinical trial where it's studied in patients. And what both of those together really try and help us do is better understand different retinal diseases and explore potential new ways to help treat them. And our patients are really such an important part of that process when they participate in research and share not only their experiences, but they're helping people also who are suffering with vision loss and they're also allowing the researchers to better understand not only the disease itself, but what really matters to those individuals in their everyday lives.

Ricky Enger: Gotcha. Yeah, that makes a lot of sense. So, when someone is thinking about participating in a clinical trial, maybe they've done some digging and they found something that looks kind of promising like, "Oh, I have this eye condition and I meet whatever criteria." What are some things that people should think about in terms of deciding whether or not to participate in a clinical trial and whether it might be right for them?

Rachel Retajczyk: Yeah, I think the first thing I'd say is that it's a very personal decision and what's right for one person might not be right for someone else. So, if someone's thinking about participating in a clinical trial, I'd encourage them to ask a lot of questions. So, for instance, what is the study trying to learn? What would be expected of you? How often would you need to come in for visits and what are the potential risks and possible benefits by being part of the trial? It's also important to understand what other options might be available and what might benefit you. And I really want to emphasize that it's okay for people to take their time and ask questions, talk with your doctor, involve your family or someone you trust, and make sure you feel comfortable with your decision. Participating in research doesn't guarantee a personal benefit and choosing not to participate is absolutely okay as well.

Ultimately, the goal is to make sure you have the information you need to make sure the choice feels right for you.

Ricky Enger: Right. And are there things like, I know it's difficult to speak in generalities, but certain clinical trials take place in a venue that is maybe not close to you. So, can you speak in generalities about, is it usually you must live in this particular geographic area to participate or just if you don't, you have to find a way to travel there on your own or is that really an example of those are questions that you should be asking the researchers before you decide to participate?

Alex Homman: Those are all great questions, Ricky. And a lot of times it does depend on, I would say, where the phase of the clinical trial is. So typically, a phase one is a little bit smaller in terms of patient populations that are needed because it's really meant to emphasize and evaluate safety. So sometimes those clinical trial sites aren't as spread out across the United States, whereas you progress into the phase two and phase three trials, those are usually a lot more expanded and can cover a lot more area. It also depends on the clinical trial site, whether or not they are open to accepting patients who maybe have to travel a greater distance, just because there are typically so many visits associated with the clinical trial because there's always ongoing safety as well as efficacy evaluations. And then also looking at the company as well, what they are researching, are they also open to patients having to travel further distances?

And sometimes that is required because there's only maybe certain specialists in certain areas, but I wouldn't say there's a one size fits all answer to that question.

Ricky Enger: Gotcha, gotcha. Well, I appreciate that. I think that helps to put things in perspective because I know a lot of times transportation is already a challenge, and so that might be one of those questions among many that you would ask if you're thinking about participating in a trial. So, we've talked a little bit about the patient side of things. When it comes to the researchers though, how are they going to know when they begin this trial and they're getting this data, how are they going to know if this is actually helping or not? Because I know that there could be very subjective reports from patients you want to feel like, oh yeah, this is definitely helping and maybe there's not that data to back it up. So, what is that approach like?

Rachel Retajczyk: Yeah, that's a really interesting question because for specifically the retinal space, vision is much more complicated than simply being able to read letters on an eye chart. Researchers look at vision in a number of different ways. They might measure how well someone can read letters, but they also see how they can see in low light or how easily they can navigate their surroundings. They may also take images of the retina to see what's happening inside the eye. But something I think that is especially important is hearing directly from patients about what they're experiencing because ultimately we want to understand not just whether something changes on a test, but whether those changes could make meaningful difference in someone's everyday life.

Ricky Enger: Yeah, that makes a lot of sense because you could see something that changes, but for the patient it's not having a practical everyday effect or maybe you see a slight change, but it turns out for whatever reason to have a significant effect. So, I think it's fascinating to think about that, and I like that it is both data-driven and also you do take some of that subjective feedback into account. We have mentioned a number of times the retina and retinal research in particular, and I think that's something that Ray Therapeutics is working on, and I think it's an exciting time. There's a lot going on in this particular space. So, can you talk a little bit about the kinds of advancements that are happening particularly in retinal research?

Alex Homman: Yes, absolutely. There is so much happening right now, Ricky. It's actually, I think, inspiring, not just for clinical researchers, but hopefully for patients as well. Research is studying several innovative approaches that includes gene therapies, cell therapies, regenerative approaches, as well as optogenetics. And what's encouraging is that as we continue to research these various modalities of treating these conditions, we continue to learn more and more about retinal diseases and also then explore potential new ways to treat vision loss. It's a very different playing field than what it was even 10 years ago. And again, as a former optometrist, it's exciting to see this advancement. I think also for people who are living with vision loss, it's very important to stay informed and keep talking with your eye care team and just learning about research that may be relevant and available to you.

Ricky Enger: Yeah, absolutely. There's one thing that stood out in your explanation there, it kind of caught my attention, which was optogenetics. And I'm like, "What in the world is that?" So, can you tell us a little bit about what that is? And I think Ray Therapeutics is doing some things in this space, so maybe just expand on that a bit.

Rachel Retajczyk: Yeah, absolutely. At Ray Therapeutics, we're developing investigational therapies with the goal of restoring vision for people living with degenerative retinal diseases. And I think that's really differentiating in the fact that we're helping to restore vision versus stopping progression of vision loss. And one of the approaches we're exploring is what Alex mentions is called Optogenetics. And I know that sounds very technical and new age, but the basic idea is actually pretty straightforward. And in many retinal diseases, the photoreceptors or the cells that normally sense light have been lost or aren't functioning normally. And our approach at Ray is designed to help those surviving cells in the retina respond to light, essentially working around those damaged photoreceptors to help send light signals to the brain. And what's particularly interesting about this approach is that it isn't designed around one specific disease-causing mutation. Ray's current clinical programs are focused on retinitis pigmentosa, Stargardt disease, and geographic atrophy.

So, it's a really exciting time to what Alex had said.

Ricky Enger: Yeah, absolutely. And so, for people who are looking for information about what Ray Therapeutics is doing, as well as maybe any other clinical research going on, I guess first share how they could find out about Ray Therapeutics, and then also if you have some resources that you might point people to in terms of here's where you can look at what's going on for clinical research with vision loss. I know Foundation Fighting Blindness has portion of their site dedicated to that, and we'll have that in the show notes. Anything else to share on that?

Alex Homman: Yeah, I think one of the best ways to also stay involved and up to date is also, again, like we've mentioned earlier, just having open and honest conversations with your providers in what are they aware of? There’re multitudes of various patient-focused organizations out there. Ricky, you mentioned Foundation Fighting Blindness. Hadley's another one that really attempts to keep their patients aware of what's coming. Clinicaltrials.gov is always a great resource just to show what studies are currently active and enrolling. And then of course with Ray Therapeutics, I would highly encourage any individuals who are interested in learning more of going to our website, which is just raytherapeutics.com.

Ricky Enger: Excellent. And again, we'll have that in the show notes. This has been fascinating. I know that many listening have probably never been involved in any sort of clinical research, and so it helps so much to know what you might expect and also just to be aware that interesting things are happening. Science is amazing and improvements are happening all the time. So really exciting things are being investigated. As we wrap up, I know we've covered quite a lot. I'm wondering if there's anything that you hoped we would cover but didn't, or just anything that you want to leave our listeners with before we wrap up?

Rachel Retajczyk: Yeah, absolutely. I think the biggest thing I'd want people to take away from this conversation is that your voice really matters. Clinical research isn't just about scientists in a lab or doctors conducting studies. It's also about understanding the experiences of people who are actually living with these diseases every day. And there's a lot of ways you can contribute. You might choose to participate in a clinical trial. You can share your experiences with researchers, get involved with patient organizations like Hadley, or simply just stay informed. And I think every person's experience is valuable, and those perspectives can really help shape the future of research moving forward.

Ricky Enger: I love that. Thank you. Alex, anything to add?

Alex Homman: Yeah. And again, just kind of echoing what Rachel said, every voice does matter and clinical research in its totality is really, it's a team game. It's not just the researchers in the lab, like Rachel mentioned. It's not just companies looking into different treatment modalities, but it's also the patient. So, it really is a team effort to bring these exciting new treatments to the people that we serve most.

Ricky Enger: Excellent. Well, thank you so much, both of you, for sharing your time and your expertise. It's always fascinating to learn about this kind of thing, and I so appreciate your stopping by. Really exciting things happening with Ray Therapeutics. And if you'd like to learn more, check out our website for that link as well as links to the other places like clinicaltrials.gov that we mentioned as well.

Rachel Retajczyk: Thank you so much, Ricky. It's been a pleasure speaking with you today.

Alex Homman: Yes. Thank you, Ricky, so much, and thank you to everyone out there.

Ricky Enger: Got something to say? Share your thoughts about this episode of Hadley Presents or make suggestions for future episodes. We'd love to hear from you.

Send us an email at [email protected]. That's P-O-D-C-A-S-T at HadleyHelps. Org, or leave us a message at 847-784-2870. Thanks for listening.