(upbeat music) - Welcome to this week's episode of The Readout Loud, a weekly biotech podcast from STAB. I'm Allison D'Angeles. - I'm Adam Forrestine. - And I'm Elaine Chun. - It is Thursday, January 29th, and on this week's episode, we're going to speak with patient advocate Lauren Holder about her experience with Huntington's disease, and her hope to push the FDA to approve a therapy from biotech company Unicure. - But first, a word from our sponsor in a roundup of this week's biotech news. (upbeat music) - I'm Jesse McCorders, stepper and studio editor. And today I'm joined by Ken Keller, chairman of the board, president and CEO at Diichi Sankyo US, and head of the global oncology business at Diichi Sankyo. How do you see the standard of care for cancer evolving in the next five years thanks to antibody drug conjugates? - Diichi Sankyo, our focus has always been an advancing science in ways that meaningfully change the lives of patients. We're taking the next step in treating patients by moving our ADCs into earlier lines of therapy, including the curative intent settings of early breast cancer and treating more tumor types. Because of our game changing work, ADCs may become a potential replacement for chemotherapy in any setting where chemotherapy is currently used. Our scientists are also exploring new payload technology and antibody designs. This exciting progress is what drives us to continue innovating to transform standards of care and bring more hope to patients with cancer. - How can people learn more? - Visit diichiSankyo.us to learn more about our science. - So my friends, it's very, very cold outside. It's been cold for a while. Boston just had, we had like two feet of snow Allison, is that what we had two feet of snow in here? - 23 inches, yeah. - So I wanna ask you, I don't know if you guys do this. I am a big snack cake person during blizzards and storms. I don't know why, but I am. And judging by the empty shelves at the grocery stores, I'm not the only person, but I wondered if you guys were the same and if you are, what snack cake? - Is the snack cake just a cake that you snack on? I don't, I don't, like. - Yeah, what? - I am a ring ding fan. - Is this like purely, I'm talking about the hostess. - Also tweaking about the hostess. - Yes, we are talking about those. So I went to the grocery store to do priest and the shelves where they had all the hostess products were very empty. So I feel like I'm not the only person who does it. - Okay, I wonder, Elaine, is this a generational thing that like the millennials and the Gen Zs, like Elaine and I didn't. - It could be. - Didn't catch on to because I really have never been a hostess. - Snack cake person. - See that, I think that is very perceptive. I think you are probably right because growing up, my mom would put a ring ding or a twinkie in my school lunch every single day. - No, no, I. - Like that was, like that was literally every single day I had a sandwich, it's like some bag of chips and like a ring ding or a twinkie in my lunch. That is not my experience with American food culture. (laughing) - So okay, so we've now established this definition. So as a Gen Xer, I bet if I ask other Gen Xers they're out there scarfing up twinkies during blizzards. So okay, we got that in some time. - Elaine, what was your blizzard, like coping mechanism culinary-wise? - Well, the thing is like with us in Chicago, we actually didn't get as much snow. But I'd rather snow because when it snows it's actually not too cold. We were just really, really cold. It did snow here, but not as much. But honestly, on one of the days that was snowing, I just went to a bar and it was honestly so cozy. - Oh, that's a good idea, see? - Yeah, I love just like sitting in a bar and you can see the snow outside the windows. - Yeah, yeah. Should we talk about the biotech news folks? - Yeah, let's do that. - So this week, the FDA halted trials of two experimental gene therapies from Magenex Bio after one child developed a brain tumor. The therapies are designed to treat rare diseases called hurler and hunter syndrome, and they rely on injecting patients with adeno-associated viruses to carry replacement genes into the brains of patients. - So these adeno-associated viruses or AAVs, they're widely used for gene therapies, but there's long been this theoretical concern that AAVs could potentially drive cancers by slipping parts of themselves into the genome. - Yeah, regenex Bio said the investigation into the tumor was still ongoing, but an early genetic analysis of a tumor biopsy found that parts of the virus had integrated into a site that can lead another gene that is linked to cancer to be overexpressed. - I think there is a fair chance, and tell me what you guys think, that this could have implications for other gene therapies. I mean, over the last 12 to 18 months, we've certainly seen quite a bit of concern about side effects and potential deaths caused by or related to gene therapies. And as Elaine was saying, there has been this fear that gene therapies and the viruses used to transmit them could drive some other health effects in patients. So I think it's almost inevitable that we will see some implication on the larger and already kind of depressing therapy fields. What do you guys think? - Yeah, well, you can see, in the FDA's actions that it took against regenex Bio, you can kind of already see that in some way, Allison. So I agree, the patient who developed this tumor was a patient who had been treated four years ago with a regenex Bio's treatment for the hurler syndrome, but because their treatment for a hunter syndrome was sort of essentially it's kind of the same construct. You know, the FDA took the sort of proactive step of also placing a clinical hold on that trial. So, you know, there was some concerns that this could be sort of a broader issue. Now, I would also say that with the regenex Bio, they have a gene therapy that is being tested for Duchenne, a muscular dystrophy. That's actually their most advanced candidate right now. And it uses an entirely different construct. It's a different promoter. They haven't seen this kind of issues in that, and you know, the FDA didn't do anything, didn't place that on clinical hold. So, you know, whether it's like something that's very specifically related to, you know, these, you know, the design, the construct of this hunter hurler syndrome or not, I mean, it remains to be seen. But, you know, just broadly speaking, I mean, you're right in that, you know, these are the kind of sort of theoretical concerns that have been out there for a long time. So it's very concerning when you actually see a case. - So switching gears to what's happening in DC, it seems like the pharmaceutical industry is meeting the Trump administration's broad cuts and federal policy changes with a record amount of lobbying. A report from Politico highlighted that the Pharma Trade Group spent nearly $38 million on lobbying last year, which is up 22% from 2024, and the highest annual total on record. - Yeah, on top of that, nine of the 13 US drug makers on the Fortune 500 list, which includes Bristol Meyer Squibb, Eli Lilly, Johnson and Johnson and Merck, reported their highest spending in at least a decade. - So I guess maybe it's not too surprising given what we've seen with all the changes that have been occurring at the health agencies, the drug pricing policies, MFN, that the administration has been talking about. What I found interesting was that in contrast to Pharma's lobbying, the trade group representing biotech companies, bio, spent their lowest amount in almost two decades. And that was surprising to me given that the biotech companies are facing a lot of disruption at the FDA level, but perhaps this disparity reflects the fact that they don't have as many commercial assets, so maybe they're not as immediately, at least many of them maybe are not as immediately affected or concerned by some of the drug pricing policies. - But there is a concern that biotech companies in general will start to face some sort of challenges and push for MFN type policies in the kind of months in the end year or so to come, right? - Yeah, that's definitely true. MFN is definitely something that larger biotechs that do have commercial assets are concerned about. And of course if it does get codified into some kind of legislation, then that down the line could also affect some of the smaller biotechs that will eventually have products on the market. - And staying in DC, the Drug Administration on Tuesday released the next 15 drugs subject to Medicare price negotiation, which will go into effect in 2028. The list includes Gilead's HIV drug betarby, Cated's IBD treatment in Tevyo, Novartis's psoriasis drug, chocentix and even Botox. And please give me a shout out because I pronounced all of those drug names correctly. - I was literally just thinking that as you were making that statement, Adam. - Well done. - I mean, well done. - This news was interesting. We were, I mean, to take you behind the scenes into the stats Slack channel, we're discussing how, I mean, most of these choices, I mean, these are obviously like fairly, you know, well known brand name drugs, but not drugs that are considered really material to a lot of the pharma companies involved. The, you know, the analyst over at Lee Rank had said that Gilead's HIV was, which was kind of anticipated to be on the list, is one of the only drugs on that list that really has a huge Medicare exposure, or really notable Medicare exposure, they estimated about 8% of Gilead's 2027 estimated revenues would be, you know, potentially affected or, you know, would be exposed by this choice. And for the other drugs that we're really talking about fairly low sales volume on Medicare. So it was an interesting choice of drugs this time around. - Another kind of interesting part of this announcement of this new round of drugs for negotiation is that, in recent times, the Trump administration has been downplaying this Medicare negotiation program because it is one of the signature achievements of the Biden administration, and the Trump administration has instead talked up much more the voluntary agreements that it's made with pharma companies. In reality, the Trump administration has continued to carry out these Medicare negotiations without any major changes, despite the fact that they are perhaps reluctant to give credit to the Biden administration. And this time around, even when announcing this new list of drugs, CMS administrator, Mehmet Oz, still touted it saying that it's an example of, quote, "taking strong action to target the most expensive drugs in Medicare." - And lastly, you may have noticed, we haven't really been talking about a thought of biotech or biotech-y news in this week's podcast because it feels like we're kind of in a little bit of a post-JPM week, January, early February lull right now. You know, maybe the most noteworthy thing that was announced recently was a deal that's not going to happen apparently, which is, you know, Merck apparently is not in talks or is not going to buy revolution medicines, cancer drug developer for $30 billion or whatever the price tag might have been. That was a deal that, you know, we had talked about before that had been speculated about or reported in the press. This report that apparently that the deal is off was published by the Wall Street Journal. So, you know, again, as we've talked about in the past, how M&A sort of certainly drives the conversation, you know, so far, we really haven't seen very much in the way of deal-making in early in the year. (gentle music) (gentle music) - It has been an emotionally wrenching five months for the Huntington's Disease community. - Yeah, last September, the biotech company Unicure announced clinical trial results for an experimental gene therapy that demonstrated an astounding 75% slowing of Huntington's Disease progression after three years. The gene therapy data were described as groundbreaking and potentially transformative, raising hopes that people living with this rare neurodegenerative condition might soon have access to a one-time disease-modifying treatment. - But two months later, those hopes were dimmed in November, Unicure made another announcement. This time it was bad news. After conferring with the FDA, the company said plans to submit its Huntington's gene therapy for approval were on hold. In a reversal of its previous position, FDA officials told Unicure that its clinical trial design and data were not adequate for an approval submission. - In the months since the setback, Unicure has continued to engage with the FDA, trying to find a way forward for its gene therapy. The Huntington's community, angry and disappointed, has also made its collective voice heard. A petition with more than 40,000 signatures was recently delivered to the FDA, urging regulators to expedite and review Unicure's gene therapy. - Joining us today to share more from the Huntington's community perspective is Lauren Holder. Lauren is a caregiver and advocate and someone who is living with Huntington's. She is also a producer and host of the Hope for HD podcast. Lauren, fellow podcaster, welcome to the readout loud. - Thank you so much for having me. I'm so excited to be on here with you guys. I love the name of the podcast. It seems very fitting for this moment, so. - Well, thank you so much, Lauren. We will get into the specifics of the Unicure situation in a moment. But first, tell our listeners a bit about yourself and your advocacy work. - Yeah, sure, so I was a caregiver. My dad passed away in January of 2021 from Huntington's disease and I was his primary caregiver and legal guardian. I'm also an advocate and someone living with HD. As you mentioned, I'm the host and producer of the Hope for HD podcast and shout out to our sponsors, Tava Pharmaceuticals, NeuroCrim Biosciences and the Griffin Foundation for sponsoring that and making that possible. And it is through a nonprofit organization called Help for HD International. But on my podcast, we focus on elevating patient and family voices and helping people understand what this disease actually looks like in real life. HD has really shaped nearly every part of my adult life. On top of being the primary caregiver for my dad and his legal guardian, I'm also gene positive myself and have begun experiencing cognitive and behavioral symptoms, but do not currently have movements. So my advocacy work specifically really centers on one thing and that's urgency with integrity. HD is a fatal progressive neurodegenerative disease with no disease modifying treatments, just treatment for symptoms and families don't have time to wait decades for perfect data, but we also care deeply about safety and scientific rigor. So those things are not mutually exclusive and I fight very hard to show how they aren't. Lauren, can you describe a bit what it's like to live with Huntington's disease to someone who may not be familiar with it? You mentioned that you are experiencing some symptoms right now, I'm so sorry, but no physical symptoms right now. What is Huntington's disease in tail? - Well, it entails a lot. So if you had asked me that 20 years ago when I first tested, my answer would be completely different and the reason is because historically, we have just looked at HD as a movement disorder as just having movements and it's a lot more than that. So for myself personally, it's a little bit layered because physically, I'm doing fine. Cognitive and emotionally, it's more ideal with social anxiety and ideal with depression and then it's like developing ADD, all of a sudden in my life and all of those things became harder and I would only know that if my husband has ADHD. So, you know, I knew what he was dealing with and I started asking him questions of like, is this what you deal with every day? And learned that like it was not just part of ADD and ADHD, it's called executive dysfunction and is found in HD. So a lot of my executive function is affected so it's a lot harder being able to organize, prioritize, time management, all of that stuff. And so most people wouldn't see that. What's different is for me, my symptoms will progress and continue to get worse. So there's no way to learn how to manage that and I'll continue to experience short-term memory loss. I'll move into the movement part of HD. Not everybody has bad movement. We have a saying in the community that is, when you've seen one person with HD, you've seen one person with HD because it is so individualized in how it affects people. And where my grandfather had a lot of movement and really kept his cognitive function and stuff, my dad was opposite. For him, it was more cognitive and behavioral symptoms. And only 20% of people really experience behavioral symptoms. So it really is a mixed bag of symptoms and we classify symptoms based off behavioral, cognitive and movement now. And it's finally getting noticed, that this earlier part of HD is finally getting noticed as part of the disease, not the before the disease. - Is there anything you can do to treat either the executive functioning issues that you have right now or to try to like, stave off the onset of any movement symptoms? - No, there's nothing on the market to help with that. You know, obviously the best thing for somebody with any brain disease to do is to continue to work your brain, right? Like you wanna make sure that you do brain games and you're challenging yourself and you don't give up those things that are going to strengthen those areas, just like physical therapy, right? Like you would do for your body, you have to do for your brain. But there is nothing specific to HD on the market for cognitive symptoms, behavioral symptoms. The only thing we have is a treatment for the movements. - So one of the reasons that Unicorn has garnered so much attention is obviously because it's gene therapy would address the underlying causes of the disease. So Lauren, I wanted you to take us back to September when the company announced those three year results. What was your reaction to the news? And what do you think that meant to the Huntington's community? - It was overwhelming, right? Because here, you know, it's kind of like, do we dare to hope that this is actually 75% slowing of progression of disease? And so it was kind of this feeling of, oh my God, like they did it. That's so exciting. And do we trust this? So it was, I think it was overwhelming in a good way because the data showing 75% slowing was just extraordinary. And then to have also well known clinicians in the space saying, this is a big deal and everything. That made it that much more exciting. And they did a really big push to get it out there. You know, it was all over media for us, which was huge. So I think it was very overwhelming, but in a good way. And so for the first time, like really those in where I am, in my group of pro-dromals, what we call ourselves, pro-dromal HD, we really felt the possibility of changing the trajectory of the disease. You know, and we have experienced failed trials and disappointments. And so I think we were also a bit cautiously optimistic as well. So afraid to hope, but really hopeful because it was good data to have data after three years is incredible. - Lauren, I know you're active on TikTok and you share your reaction to that news with followers at the time. - We're not looking at a cure, but we are getting what we had asked for in that they have bought us time and they have slowed progression. - Actually, we watched that video. And I think what I was struck by something you said, you said that you were happy, you were celebrating, of course. But you also advised the HD community to be prepared for a fight and that there would still be barriers and challenges to overcome. And I wonder, given what we know now today, do you think you were anticipating issues with the FDA? - Not necessarily with the FDA. And I don't think it was really anticipating specific issues. It's more of, I'm realistic because I am a long time advocate. And, you know, I realize that we've had setbacks and we've had issues in the past. And so I wanted the community to be realistic and not just hop on this wagon of, oh my God, we've got this cure because it's not, first off, it's not a cure, you know? And there were certain people who were asking that, is this a cure, is this a cure? And that's very harmful to our community because we don't want people to see this as misinterpreted, right, like it is what it is. It is treating the disease, slowing progression, but it's not a cure. And so I wanted the community to be prepared and I didn't want them to just be all hope and no realism, knowing that, okay, great, this is now, you know, out there, we know we can slow progression, but if it does get approved, what are we looking at for, you know, availability to the community? Like, what do we have to do to make sure that it's gonna become available to us? And I think I was thinking more on those lines of, okay, what are the roadblocks are ahead beyond the accelerated approval? So it wasn't really specific issue with the FDA. - But then in November, when Unicure announced it's set back with the FDA, what were your thoughts then? How are you feeling? - I still feel it. It's shocking grief. I mean, truly, it felt like the rug kind of being pulled out from underneath. Not just emotionally, but logistically. I think families had started planning, you know, and we had already started talking about, you know, how do we work and collaborate with Unicure to make sure that the stroke is gonna be available and, you know, is it gonna be approved by insurance? Those were the thoughts going through my head. And I regularly talk with CPath and FDA and all of those people. So yeah, it really felt like the rug was kind of pulled out from underneath me and I'm still dealing with shock and grief from it. - You said at the time, this is the time to be loud with the FDA. And the Huntington's community has certainly not shied away from using their voice. As we mentioned, you know, up at the top, you guys collected more than 40,000 signatures for this petition that was delivered to the FDA and from what we understand, the agency has refused to accept the petition in person. The advocates who showed up at the agency weren't allowed onto the campus. Are you concerned that the FDA isn't taking patients' perspectives seriously? - So I was one of those advocates that was there. So we were allowed onto the campus. We were not allowed to the building. So we got to the security part and we were told, we could not deliver the petition in person, not even to the security guards. They told us that, no, that we would have to contact an employee and have them come down and escort us, which is not what any of us were expecting because that's not what we had been told. I mean, we wouldn't have just shown up there. We had been told, like, okay, no, you can actually go and actually deliver a petition in person, but apparently- - So you guys had been communicating with people at the FDA that you wanted to deliver this petition. - Yeah, we had been communicating with not necessarily with the FDA, like not with SEBER. We had been communicating with liaisons and who made the suggestion, and because we're not the first disease group to deliver a petition in person. So we were going off of information and off of that and the problem was that we didn't know that there had been changes to their protocols. And that wasn't communicated to us when we were also standing there. For me personally, because of my engagement with them, over the last year to two years and really collaborating with critical path institute and with the FDA, I mean, I did a panel with Michelle Campbell, the FDA, about walking the walk with the FDA for PFDB meetings and like how to at the recent CPATH conference. And so, you know, and I was talking to people from the FDA at that meeting and it's just like, when you have that experience and they're sitting there and they're saying we hear your urgency, we wanna help you and everything. And then this happens, it's just devastating. So it's not that I don't think that they were sincere and that they don't seriously wanna help us. I think that something is going on that we don't know. And I just wish that there was more effective communication. - You know, I wanted to get into that a little bit more, Lauren, because I wanted to be curious like what you're, you know, what your take or perspective is on the current FDA because, you know, you do hear top officials, you know, commission or Martin McCarrie, Seabird Chief, Finite Prasad, you know, they talk publicly about their desire, you know, they're pushed to accelerate development and approval of treatments for rare diseases. They talk about that a lot publicly. You know, at the same time, you know, Unicure has to set back other companies that have been developing cell and gene therapies. I've also had similar setbacks with the FDA. So I wanted you to think, is the FDA saying and doing the right things, they seem to be saying the right things, maybe not doing them, you know, are they doing enough with regard to rare disease? - Historically, FDA has never done enough for rare disease. I honestly was of the opinion that we were starting to move in the right direction because of the fact of my own personal, you know, conversations and collaboration. And so seeing that, I did feel that they had, they had moved to a more patient centric model and actually taking notice of rare disease. So I do believe that the FDA in general wants to support rare disease innovation. But what we're seeing on the ground doesn't always align with that rhetoric and particularly for cell and gene therapies. And so, you know, I feel like two HD has kind of been put on the back burner for a long time and when it comes to the FDA and something needs to change there, I do think that more can be done. But I feel like overall, we were moving in the right direction. - Lauren, kind of on the flip side of that, in recent years, it does seem like patients and advocates have exerted more influence over the FDA and drug approval decisions. The most recent example is probably in Duchenne muscular dystrophy. There are some people who are concerned that advocacy groups in their urgency to accelerate treatments may overlook safety signals or push for drugs to be approved without strong evidence. What do you say about that? - I don't think it's fair to advocacy groups and patient groups. And the reason is because personally, like I'm not looking for special treatment. I'm not looking for them to not do what's needed to make sure that it's safe. I'm looking for them to be fair and consistent across the board. And, you know, you look at what's going on with cancer treatments and how they get approved and the neurodegenerative space is a very different beast. I think that there just needs to be consistency. I personally am of the opinion that placebo control is very important, you know? We should absolutely have placebo arms for clinical trials in a setting where it makes sense. If we're talking about unicure specifically, the difference here and why I don't push for a sham procedure is as a patient looking at this it is an invasive brain procedure that, you know, is it worth risking my brain on a sham procedure when it could cause neurodegeneration and then in a year, I wouldn't be able to qualify for the treatment. Absolutely not. I'm not gonna risk my brain for sham procedure. And it's not ethical, it's not. I mean, you look at Europe, they wouldn't even allow it. It was not even a question of it not being ethical because of the risk of brain inflammation and neurodegeneration to the patients. Like at that time, where rare disease, you can, you know, find, you can do something like this without doing a sham procedure that is so risky to somebody and could cause further neurodegeneration in such a short period of time. But in the context of other clinical trials, such as a pill or even infusions or treatments, you know, we have the spinal cord injections, I absolutely believe there should be placebo control. I'm not against going, you know, going the right path. It's just we should be treated consistently and we should be, it should be remembered that in certain rare diseases, if it's going to be a risk to the population to do something, then we need to pivot and think of something better. And just to kind of clarify for listeners who may not know, Unicure's gene therapy involves brain surgery because it needs to be delivered directly into the brain. And so Unicure's trial used an external control arm to kind of show the efficacy of its treatment and that seems to be a point of contention with the FDA because the FDA is now saying that that approach is not sufficient. So just to give some background to listeners. - You know, getting back to your consistency comment, Lauren, you know, I guess that, I mean, that must be one of the things that irks you, right? It's because what we've been told to Unicure's perspective is that the FDA was okay with this external control arm. They weren't requiring a sham surgery to be included, incorporated into the trial design, but then now the FDA has reversed itself and they've seen now to not wanting to accept this external control arm. And it's not like it was just a sudden thing. You Unicure has been having meetings with the FDA on this for over a year. So it's not like it was the sudden thing. Unicure literally set everything up based off of this guideline from the FDA. And then we're told, oh, now it's not aligned and we're saying it won't be accepted. So, you know, you got to do something different. So that's where it's like, it's really upsetting for me because we're not asking for like all of a sudden approval. We're asking for them to honor their guidance and to go by what their guidance says because it truly sets up everything for us for the future, not just with Unicure, but our future treatments, you know, can we trust the FDA to go on their guidance for even future treatments? So that's the problem. It's not that we're asking for them to bypass everything and immediately approve. We're just asking them to honor what they said and go by their guidance. - Unicure is expected to meet with the agency this quarter to discuss the path forward for the gene therapy. The meeting at this point it may have already happened. We haven't heard anything about whether that's the case or whether, you know, there is any outcome. What do you think will happen in like, what are you hoping for? - I guess I'm hoping for clear guidance and, you know, basically for them to honor what they initially said in their guidance and providing a viable path forward. You know, I think my fear is basically a start over scenario, right, and okay, you have to get more data. Now we've got another five years on where we have to do before we see any type of approval. So I realize that's worst case scenario, but still that's the fear because this is, we've already experienced worst case scenario by them re-nigging on what they initially provided as guidance. And so obviously my mind goes to worst case scenario. And then, you know, I don't have the time. I'm one of those people who I literally could have the treatment and I'm pro-dermal, I'm in that range. There's a short window where my brain is healthy enough to benefit. And, you know, if it takes another three or five years, I won't be in that window. So it's looking at that. I don't have the time to wait for them to do worst case scenario because it will cost me my life. Lauren, if UniCure is unable to seek approval for its gene therapy with its current study and data, what do you think happens next from the perspective of the Huntington's community? I think that is a very devastating day, a day of severe loss for the community for treatment that literally shows slowing of disease progression by 75% over three years. And the FDA takes that away, that's a very bad day. And the suicide rate in our community is twice as high as general population for a reason. We know what our fate is, if we don't have a treatment. Even this news of a delay already has put some people into a depression and you've got to remember these are people with neurodegenerative disease who don't process the same always as they go further and further along in that disease. You know, it's one thing to be in the space I'm in and I'm still aware and able to process, but as the disease goes on, your insight believes and you can't process as well. And you can imagine having to tell somebody that that treatment is gone. It's not available any longer. It's not even on a blip on the screen any longer. And that's a very devastating day. So moving forward, I don't know. I don't have a great answer other than it moves us back into a grief cycle with a community that has a twice as high suicide rate and, you know, us trying to pick up the pieces as a community and support each other in remembering that there are other treatments that are behind this, that are significant and they are showing benefit. They're not showing 75% slowing of disease progression, which is okay. That I mean, it's not that I expect that high, but you can't say that that's not meaningful and for FDA to take away something like that to a disease community that literally we don't have anything. We don't have anything else except death. That is it. And, you know, how is that fair? So yeah, it would be a bad day, but we would pick ourselves up like we always do as a community and, you know, remember that there are other things and we've got to fight for that, continue to fight for treatments and hope that the next one won't be the same way that, you know, the FDA has not set a precedent for our treatments and that we at least have some hope for the future. - Lauren, thanks so much for sharing your thoughts with us today and please let our listeners know how and where they can listen to your podcast. - Yeah, of course. So really you can listen to it anywhere you listen to podcasts among Spotify, iHeart iTunes, Google podcasts, all of that. We also use speaker for our main platform. You just search up Help4HD, so H-E-L-P, the number four, and HD. You can also go to thehelp4HD.org website and all of our episodes are listed on there. We also have a YouTube channel called Help4HDTV and I'm currently working on uploading stuff, all of my episodes to YouTube, but you can find me pretty much all over along with my personal stuff on YouTube, which is under dying to live. So, thank you so much for this opportunity. - Yeah, thanks for joining us. - Thank you, Lauren. Keep your chin up. Keep going. - That does it for another episode of The Read Out Loud. - Thank you to Hyacinth and Bonato for producing this week's episode. Our senior producer is Alissa Ambrose. Our executive producer is Rick Burke and our theme music is by Brian Joel. - We'd love to hear from you, tell us what you'd like about this week's episode, what you didn't like and for the Gen X listeners out there, are you a ring-ding fan or a twinkie fan? You can do all that by sending us an email at
[email protected]. - And if you like what we do, leave a review or rating on Apple podcasts or whichever platform you use to get your podcasts. See you next week. (upbeat music)
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