Empowered Patient Podcast

Dr. Antonin (Tony) de Fougerolles, CEO of Evox Therapeutics, highlights that exosomes, small nanoparticles, are produced by every cell in the body.  They contain RNA and proteins that the cell decides to put into the exosome, and they're secreted out into the environment and act as a way that cells communicate with each other. Evox is excited about using exosome therapeutics to load genetic medicines such as RNA interference, small RNA drugs, gene therapy, or genome editing into exosomes and effectively delivering them inside cells. This approach provides a new way to treat rare diseases with an underlying genetic cause. Tony elaborates, "Exosomes were first discovered or noticed in the scientific literature in the '80s and the early '90s, and at first were thought really just to be vesicles that cells excreted full of what people at the time thought was garbage, things the cell didn't need. In more recent times, in the last 10, 15 years, it's become clear that these exosomes can in some cases serve a function in terms of helping, as I say, cells communicate and sense their environment."  "It wasn't until one of our co-founders, Professor Matthew Wood, used exosomes as a deliberate vehicle. He had the idea, "Well, cells are constantly producing these exosomes. What they put into them is not at all random, it's very directed, and can we make use of that knowledge to actually proactively engineer drugs into these exosomes and then use the exosome's natural ability to deliver cargos inside cells? Can we use that to actually deliver drugs of our interest?"  #EvoxTherapeutics #RareDisease #Exosomes #RareGeneticDiseases #ExtracellularVesicles #RNATherapeutics #Biotech #GeneTherapy #GeneEditing #ProteinTherapeutics #Innovation EvoxTherapeutics.com Download the transcript here

Dr. Eugene Chan, Chairman and Co-Founder of Abpro, talks about the next generation of antibody therapies that can target more than one target to activate the immune system in the fight against cancer. Abpro is also working on a prophylactic antibody therapy to help prevent immunocompromised patients from getting COVID. Eugene explains, "Abpro has developed the antibodies, in a lot of these cases, to be able to target more than one target. This basically activates the immune system more so that whatever you're targeting is cleared more rapidly from the body. This applies to the cases of cancer that we are focused on, and we've basically developed these antibodies called bispecific antibodies - bi for targeting two different sites. These antibodies essentially have more and greater activity than conventional antibodies." "We developed a program and antibodies that bind to HER2+, which is one of the molecular markers on breast and gastric cancers. This particular therapy brings the immune system, the body's own natural immune system, to these cancer cells in a very active manner, which then allows it to get cleared from the body much sooner than the other mono-specific therapies that are currently out there." "Monoclonal antibodies are the body's own natural defense system for a lot of diseases. The ability to take these and augment and make them better is really the way to go because, ultimately, the body is already accustomed to these types of molecules. So we know to some degree they're safe. When compared to things like chemotherapy or even radiotherapy, these things are going to be much safer because they naturally exist in some way, shape or form in the body when you're fighting a disease or when your body's exposed to a new antigen or some sort of new threat. We're taking this approach and enhancing the body's ability to fight cancer and infection by augmenting the structure of these molecules." #Abpro #Antibodies #MonoclonalAntibodies #Cancer #Tumors #COVID #HER2+ abpro.com Download the transcript here

Dr. Edith Perez, Chief Medical Officer at Bolt Biotherapeutics, describes the innovative approach Bolt is taking to bring together the precision of antibodies against the tumor with the power of the innate and adaptive immune system by activating the immune system at the tumor site.  This approach can minimize side effects from systematic activation of the immune system and reinforce long-term anti-tumor efficacy. Edith explains, "The myeloid cells are a wide range of cells in the body, and initially, the myeloid cells can start attacking the tumors right away. But as important, or even more importantly, is that they secrete the cytokines and chemokines to teach the T-cells to essentially develop an immunological memory to be able to fight these foreign bodies for a long time."  "First of all, there are the abnormalities of the tumor themselves, and we target those with monoclonal antibodies. Then we actually link that with what we call a non-cleavable and non-cell permeable linker. Again, the idea is to bring the activation of the immune system right at the site of the tumor, which could be dramatic for humans."  "So these monoclonal antibodies essentially find the tumor cells because the tumor cells are circulating, they're in the body, so they latch onto these proteins in the membrane and with our compound, with our Boltbody compounds. What we have here is the attachment of the monoclonal antibodies with what we call the linker payload. The payload is really the activation factor of the immune system. We bring that activation of the immune system, tied into the monoclonal antibody, to the site of the tumor. That way, we can be very precise related to where the activity of the compound will occur." @BoltBio #BoltBio #ISAC #Immunotherapy #Oncology #Tumors #ImmuneSystem boltbio.com Download the transcript here

Dr. Romain Micol, CEO, and Dr. Tom VanCott, Chief Scientific Officer, at Combined Therapeutics, are excited about the opportunities to design mRNA vaccines that target specific tissue cells to restrict protein expression and have stronger potency in the fight against infectious disease and cancer. They have designed their vaccine platform to keep the vaccine antigen expression at the injection site, protecting the liver, kidney, and heart.  Romain explains, "So mRNA vaccines are highly effective both for infectious diseases and, more recently, to treat cancer. It's an ideal technology that is relatively easy to make and to manufacture. It could be very rapid and can get expression in vivo where protein would be folded properly. We can currently use the mRNA vaccine to deliver multiple materials to prevent infectious diseases and also cure cancer." "The main challenge we see at Combined Therapeutics, both for vaccines for infectious disease and for cancer treatment, is in their biodistribution and potency. Our platform at Combined Therapeutics is a proprietary technology that enables us to focus protein expression in specific tissue cells and prevent off-target expression in selected cell tissue. At the moment, our R&D program covers both oncology and infectious disease." Thomas elaborates, "What we've done here at Combined Therapeutics is we're taking advantage of something called micro RNAs. Now, what are micro RNAs? A lot of people don't know about them, but they're really just small RNA molecules that are naturally present throughout the body. They're actually fairly small molecules. They're about 22 nucleotides in size." "But what's really important is that they're distributed differentially throughout the body. So, if you look at the lungs or the heart or the muscles, you'll see a different array of micro RNAs that are expressed in each one of those tissues, and that is exactly what we take advantage of. What we do in our mRNA is engineer into it what we call these blockers. And what's a blocker? A blocker is really a binding site for the micro RNA." @Combined_tx #CombinedTherapeutics #mRNA #Vaccines #Cancer #Oncology #InfectiousDisease  combinedtx.com Download the transcript here

Nick Hedges is the CEO of Raintree Systems, a provider of software-as-a-relationship to help therapy and rehab companies enhance their patient engagement, provide access to an electronic medical record system, and improve revenue cycle management. Using predictive models and a digital approach to billing and reimbursement, Raintree is reducing the friction for patients, providers, and payers. Nick explains, "A lot of people describe software these days as delivered primarily through the internet as software-as-a-service. However, service doesn't really tell you the full story about how we interact with our clients. We see our software as being a partnership. We work very closely with our clients to make sure that it delivers exactly what they need, and we build our product in unison with our clients. That's why we call it software-as-a-relationship." "Healthcare, in general, is quite far behind in terms of the digital patient experience or the digital consumer experience. One of the things that Raintree seeks to do is to be at the forefront of that within healthcare and bring some of the capabilities that you see in other industries like financial services and automotive and so on to the healthcare industry because that's what consumers who are patients want. They want a more digital experience." "The revenue cycle side of things is a really important aspect, both for the therapy provider and the patient. What we try to do is to reduce the friction to the maximum amount for both sides because it's very complicated. It's an arcane system when it comes to how provider reimbursements work. And frankly, neither the patient nor the therapist, wants to spend a lot of time trying to figure out how their healthcare plan matches with the needs that they have for their healthcare. So what we do is automate a lot of that process." @RaintreeSystems #PhysicalTherapy #TherapyRehab #OccupationalTherapy #PediatricTherapy #SpeechTherapy raintreeinc.com Download the transcript here

Whitney Stewart, the Director of Clinical Project Management at Curebase,  and Megan Coder, the Chief Policy Officer of the Digital Therapeutics Alliance, have worked together on a white paper Setting the Stage for a Fit-For-Purpose DTx Evidentiary Standard. The goal is to outline foundational principles that, in some cases, are unique to the digital therapeutics category. Their focus is on baseline expectations related to procedures and the type, quality, and timing of clinical trials necessary to evaluate and widely implement DTx therapies.   Megan explains, "Given some of their development pathways and processes, given how they're regulated as a medical device and not a drug, we've really started talking with Curebase and some of our other members. We are looking at, and we recognize that, these digital therapeutic products have great value to patient care. They can be used alongside or sometimes even in place of some of these traditional therapies. But what became evident to us pretty quickly is that these therapies really don't sit in line with all of the evidentiary standards that exist for the current existing treatments and therapies." "We wanted to talk about this idea of a fit-for-purpose standard that can pull in some of the aspects from traditional therapies into this evaluation framework. This is so a patient, clinician, payer, and policymaker can understand, "Oh, this is what this therapy does, this is how I know it's going to be successful, and this is how I know what the impact is going to be for my patient population."  Whitney elaborates, "The future implications include a better understanding of the clinical evidence dossier, which just means more effective and fair evaluations of the technologies. So, do they need to conduct the same trials and generate the same evidence as pharmaceuticals? No, I think we all are aligned with that. Do they need to go through the same processes as high-risk medical devices? No, they probably don't. Most of them are fairly low-risk. So we've been talking about it just to get everybody on the same page and with the same kind of evaluation criteria so that everyone knows exactly what sufficient looks like." #DigitalTherapeuticsAlliance #Curebase #DTx #DigitalTherapeutics #PrescriptionDigitalTherapeutics #DTxTrials #ClinicalResearch #ClinicalTrials  DTxAlliance.org   curebase.com Download the transcript here

Karen Smith is the Founder of Catie's Cause, a nonprofit charity dedicated to building awareness about the safe use of over-the-counter pain medications.  After the death of her daughter Catie from liver failure due to overuse of Tylenol, Karen founded this organization to shine a light on the unintended and intentional side effects of easy access to these medicines. Karen explains, "The research shows that there are four main groups of individuals who are impacted by acetaminophen poisoning. Young adults definitely are a large group, oftentimes because they're on their own and they don't realize the dangers of medication. Some of them do begin to abuse it and they take it way too regularly. Sadly, about 50% of the deaths are from suicide, so obviously intended, but 50% of them are unintended. So young adults are a big group."  "Another group is young children because parents tend to freak out when their little ones get a headache, and they overmedicate. The third group impacted are the elderly because they have difficulty reading the warning labels or are taking a lot of other medications, and their liver may already be compromised by that." "And then the last group are those with limited access to healthcare. That's primarily because they don't have someone to warn them about what could happen if they overuse that medication. Instead of going to a doctor, they tend to go to the pharmacy and pick up whatever's on the shelf." #CatiesCause #AcetaminophenPoisoning #AcetaminophenDangers #KnowYourMedications #LiverSafety #DrugSafety #Tylenol #SouthRampartPharma #DoIt4Catie #PaintTherapy catiescause.org Download the transcript here

Dr. Jing Wang is the Dean and Professor at Florida State University College of Nursing and is focused on bringing a high-tech, high-touch approach to patient care in the education of nurses. The idea is to leverage technology and digital health tools to support nurses so that they can spend more quality time with patients. The newest generation of students are digital natives, and one goal is to help them use that knowledge to smooth the transition for the less technology savvy. Jing explains, "I think it's all about how technology is introduced into the learning in our current nursing education and health profession education. That is critically important. That's what we are doing here at Florida State University College of Nursing. We would like to do it in the right way, to introduce technology as a tool to enhance care and facilitate care versus this is a fancy technology, we must use it. I think that's core, and that's the attitude that we are hoping our graduating students will have." "We have launched a new Institute on Digital Health and Innovation. It's very exciting that we are launching our first design sprint. We're bringing students all together for them to work 24 non-stop to focus on solutions with people who build homes, people who design homes, people who take care of seniors, and people who focus on wellness. This includes doctors, nurses, and social workers. How can we design this home that is a future aging-in-place home that would consider all of the aspects for our seniors or adults who would like age-in-place?" #FSUResearch #FSUNursing #DigitalHealth #AginginPlace #DigitalPrecisionHealth #Nurses Nursing.FSU.edu Download the transcript here  

Dr. Deb Ondrasik is a pediatrician based in Massachusetts, and the topic today is the CACNA1A Foundation and its work on raising awareness about developmental disabilities. Deb's daughter was diagnosed in 2015 as one of eight children worldwide with a CACNA1A gene mutation causing epileptic encephalopathy. This is a story about the need for whole genome sequencing to determine the root cause of childhood developmental delays and the value of this information for developing a treatment plan.  Deb explains, "CACNA1A is a genetic mutation that's a group of diseases that can present in a number of different ways. It's mutations along a gene that affects the calcium channels and the neurons, and it's on chromosome 19. And so, there are numerous hundreds of different point mutations that could present a little differently. But kiddos can have anything from developmental delays to autism to epileptic encephalopathy, which my daughter has. Also, there are other conditions like hemiplegic migraines. And so, it is a variety of neurodevelopmental disorders that can be presenting." "I work as a general pediatrician. I see a lot of kids that have delays and significant delays or autism or epilepsy. And oftentimes, the families are told that that's the diagnosis. Your child has epilepsy, and we're going to give them some meds and see if we can control it. Or, if your child has autism, we're going to get them into therapy, which is partly correct. Medication is super helpful, and therapy is super helpful. But I think what's really important is to try to find the root cause of the symptoms, which are epilepsy, autism delays. And oftentimes, for some reason, pediatricians and even neurologists don't always go ahead and do the genetic testing."  #CACNA1AFoundation #CureCACNA1A #RareDiseases #RareDiseaseAwareness #CACNA1AAwareness #PatientAdvocacy #CACNA1AResearch #Epilepsy #IntellectualDisabilities #CerebellarAtrophy #CongenitalAtaxia #CaregiverSupport #WholeGenomeSequencing CACNA1A.org Download the transcript here

Heather Ridenoure, Center of Excellence Segment Leader, and Maeve Ruggieri,  the Director of Product at Contigo Health, shine a light on the challenge of employee substance use disorder and the resources available for care. This chronic disease can be caused by adverse childhood events, genetic predisposition, stress, and environmental factors. Contigo addresses any substance that can be abused through their Centers for Excellence program with virtual care and in-person care options where participation is voluntary and can be accessed 24/7. Heather explains, "As far as how frequently this occurs, the newest statistics from the Centers for Disease Control said one in seven people have a substance use disorder. That's new data, and that's been increasing by 30% since the pandemic, which is pretty significant. So, this is one of the reasons we struck out on a journey to try to address some of these issues."   Maeve elaborates, "As Heather mentioned, substance use disorder being a chronic disease, is something that causes clinically significant impairment. So that may or may not be visible to an employer in the workplace. Their employees may seem like they are high performers or be high performers and not be showing the stereotypical signs of substance use disorder, things that we may see from TV or movies, like erratic behavior, things even down to missing deadlines." "But what's so important is having the member be ready to participate themselves. This program is rooted in shared decision-making between the member and the providers, and our team at Contigo, who's doing a lot of care and case management and support. And if the member is not ready to participate, to explore recovery and manage their substance use disorder, then it's not time yet." #ContigoHealth #SubstanceUseDisorder #HealthPlans #HealthBenefits ContigoHealth.com Download the transcript here