Empowered Patient Podcast

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

Kyle Filkins and Kris Napper were both born with SMA, spinal muscular atrophy. Meeting as kids at The Muscular Dystrophy Association summer camp, they have developed a friendship and a documentary. After beginning the use of the drug Evrysdi, their rare genetic disease stopped progressing, and the oral form of this medication allowed them to avoid spinal injections. Now in their thirties, Kris and Kyle are advocating for the community of those with SMA, working on their documentary, and looking forward to more adventures.  Kris explains, "Just shortly before Evrysdi was released and made available, there was another drug called Spinraza that was the first treatment ever for our disability. It was a spinal injection, which poses some complications as far as it's a procedure with some risk. It can be painful, and there's the risk of hitting nerves and causing damage. It's not the most fun thing to go through, but it was beneficial. So I was doing that." "So for young children, just being diagnosed can be life-changing because you can stop that progression before it really hits. For us, we've already had significant progression. Still, hopefully, this will stop it where it is, and we'll be able to continue our lives for the foreseeable future, a lot better and longer than originally anticipated." Kyle elaborates, "Before I was on any type of therapy, I would go to bed one day able to do something, and I'd wake up the day after not being able to do that. And it would never come back. And it's pretty great to be able to go to bed and sleep securely knowing that what I did today I can, for the most part, probably do tomorrow. It's definitely life-changing. That's for sure." "There's been a lot of people interested in this story. Kind of piggybacking here on what Napper said, at the outset, it was really kind of just a budget film and like, "Oh, maybe they'll do cool activities and make jokes and stuff." But then with the advent of this therapy and specifically Evrysdi, that's really changed the outlook of the scope of the film, and for the better, as Napper said."  @KKtheFilm #SMA #SpinalMuscularAtrophy #MuscularDystrophy #RareDisease #Evrysdi  KrisandKyletheFilm.com SnappyClothing.com Evrysdi.com Download the transcript here

Marlena Del Hierro VP of partnerships and commercial strategy at Seniorly, which is a resource for families looking for senior living options and seniors planning ahead. The Best of Senior Living Awards determined by Seniorly highlights the best 150 communities around the country based on feedback from families, senior living advisors, licensing reviews, and media searches that might reveal something problematic or terrific about a facility.   Marlena explains, "We have an algorithm that then shares the top five to seven options for your loved one. So that's a unique piece, because of the data and the technology that we're constantly gathering from a variety of sources to help these families make the best decision for their loved ones."  "The reason why we're doing these awards is that we should be pushing the envelope. The Baby Boomers are fundamentally challenging these senior living communities. And the awards are just a sense of preparing us for more of a modern era in senior living."  @Seniorly #SeniorLiving #BabyBoomers #IndependentLiving #AssistedLiving #FinancialPlanning Seniorly.com  Download the transcript here

Dr. Steve Marcus, CEO of Cantex Pharmaceuticals, is developing drugs based on Azeliragon that might slow the metastasis of cancer and extend the survival rate for patients with brain and other cancers.  This drug has been shown to suppress the RAGE receptor, which is expressed on the surface of cancer cells and some immune cells. When RAGE is over-activated, as it is in several cancers and inflammatory diseases, it can cause pathology. Steve elaborates, "Azeliragon inhibits something abbreviated as RAGE, the receptor for advanced glycation end products. Originally, it was thought that RAGE is very important for the progression of Alzheimer's disease. And because of that, a company called vTv Therapeutics initiated large-scale clinical trials of Azeliragon for Alzheimer's disease. Unfortunately, those trials failed to show effectiveness of the drug in Alzheimer's disease, but did demonstrate very robust safety. The drug is given once a day and was given to patients for periods up to 18 months, and it was very well tolerated." "At the same time, over the last several years, there has been extensive literature indicating that this receptor, RAGE, plays a role in the progression of cancer, both brain cancers, as well as cancers outside of the central nervous system. And with that information, I approached vTv Therapeutics after their clinical trial failed to show efficacy, and I proposed licensing the product and taking it into cancer indications." #CantexPharma #GBM #Glioblastoma #BrainMetastasis #RAGEReceptor #Cancer Cantex.com Download the transcript here

Dr. Spyros Papapetropoulos is the President and CEO of Bionomics, a biotech company focused on central nervous system disorders, particularly social anxiety, and post-traumatic stress disorders. Social anxiety is a disease primarily of adolescents and young adults, and the social isolation during COVID  contributed to a rise in the number of people experiencing this condition. The Bionomics drug in development is BNC210, a therapeutic that can be taken on schedule an hour before conducting an activity likely to induce anxiety.  Spyros elaborates, "Social anxiety disorder is actually known as social phobia, and it's very different, because everyday interactions cause significant anxieties. And they're very situational, so you become very self-conscious. You feel embarrassed and have a fear of being scrutinized or judged negatively by others. It is considered a very, very important chronic mental health condition that, if left untreated, leads to comorbid depression, even suicidality, and addiction down the road." "BNC210 acts on nicotinic receptors. These are receptors that are expressed or found in areas of the brain. The alpha-7 nicotinic receptors are found primarily in areas that control emotions, so in areas that are considered brain anxiety generators. I don't know how much your audience has heard about the amygdala, but the amygdala is considered one of the most important areas, also responsible for fight or flight, for instance. And it's an area of the brain that has received a lot of attention. So BNC210 binds to the alpha-7 receptors found in the amygdala and reduces the activity or the activation of the amygdala, which is abnormally high in anxiety-related disorders." #Bionomics #SocialAnxietyDisorder #SocialPhobia #CNSDisorders #PTSD #COVID  bionomics.com Download the transcript here

Matt D'Onofrio, Co-Founder, President, and Chief Operating Officer at Evoke Pharma, is developing a treatment to help patients with diabetic gastroparesis.  This little-known disease is thought to be due to damage to the vagus nerve, interrupting the communication between the brain and gut, stopping the stomach from functioning normally. Current treatments are a pill required to move through the stomach.  Evoke has developed a nasal spray to treat the condition that bypasses the stomach and provides predictable relief. Matt explains, "We developed a product called GIMOTI, or nasal metoclopramide. The interest behind doing so is to completely bypass the stomach to get a dose on board for patients. The unpredictable nature of the stomach makes dosing with pills very unpredictable. We know that a number of patients who take oral treatments don't find efficacy. We believe that's, in part, because of the stomach's problems itself." "By developing the nasal format, or GIMOTI, we are able to avoid that unpredictable issue and get the product on board directly by spraying it into the nose and getting absorption straight through the thin membrane in your nostrils, getting the product into the bloodstream from there. We found that this is very helpful for patients who do have issues with any absorption problems. We also found it helpful for patients who are nauseated and vomiting because you can't vomit up a nasal spray. There have been a number of different things we found as the product has come on the market that's been helpful for it." #EVOK #Evoke #FightGastroparesis #DGPAwareness #DiabeticGastroparesis  evokepharma.com Download the transcript here

Jared Bauer is the CEO of IONIQ Sciences which is exploring new ways to detect cancer at an earlier stage using non-invasive bioimpedance-based technology. This approach is not looking for the presence of cancer cells but rather changes in the interstitial fluid. Using a low level of electric current, the IONIQ AI-enhanced technology can identify where in the body the cancer is. Jared explains, "It's a bioimpedance-based technology, and the way the system works is we inject a very low level of electrical current, not enough for you to feel. You can't even recognize that it's there. Still, we inject electrical current using DC power, direct current. If you just think physics 101, electricity 101, whatever you want to call it, DC power, the path of least resistance, and that electricity is going to go through the interstitial fluid. Then we pull that back out with an EKG electrode on the other side. And we're scanning that interstitial fluid over distances, allowing us to see that immune response." "When someone doesn't have cancer, the ions or that electricity is going to flow freely. When someone does have cancer, a malignancy is present, and we see that cytokine storm, the changes in the chemokines, and the changes in the protein matrix. All of the interstitial fluid gets jumbled up, and those ions don't move as smoothly through. So over the course of a 20-minute, non-invasive test, just electrical current, it's all digital, we pull 10,000-plus data points from our scan, and we analyze that using our proprietary algorithm to give you an answer of malignant or benign." #IONIQSciences #EarlyDetectionofCancer #Cancer #LungCancer #Bioimpedance #CancerScreening ioniqsciences.com  Download the transcript here

Colin LeClair, CEO of Connections Health Solutions, shares his concerns about the lack of resources for people needing behavioral health crisis care and solutions for urgent and emergency psychiatric services in a facility-based setting. With a community-based approach, Connections partners with hospitals, care facilities, service providers, and law enforcement to better understand patients dealing with complex behavioral challenges and provide new alternatives for care.  Colin elaborates, "Right now, we have two of the largest crisis facilities, as we call them, in Arizona, one in Tucson and one in Phoenix. And those services include emergency psychiatric services, 24 hours a day, seven days a week. Crisis stabilization services for patients who need a few days to recover and get back on their medication regimen and for whom we can prepare a care plan. In addition, there is an observation unit, a 23-hour observation unit for patients who are very acute and require immediate stabilization in a very unrestricted environment to get them back on their feet and on the path to recovery. The facilities look like small hospitals. We operate in about 20,000 to 40,000 square feet. We see about one thousand patients a month in each of our facilities."  "We take a lot of pride in saying that we never say no to a patient. And so the crisis is very self-defined. A patient who's in crisis may be having an anxiety attack, they may be new to the neighborhood, and they don't have a primary care physician or a primary psychiatric medical home. They just need a prescription refill. Or it could be somebody having a psychotic episode. We will treat anyone who comes to us." "On the higher acuity end of the spectrum, about 60% of our patients arrive with first responders. They're often picked up in the community by first responders. And we're trying to be a better alternative for police and for the patient than going to an emergency department where treatment is not designed around a psychiatric patient or potentially jail. And so, again, 60% of our patients come to us involuntarily. We take a lot of pride in the fact that we are able to convert more than half of those patients to voluntary status. In other words, they agree to engage in their own treatment, and that's a very, very difficult thing to do." #ConnectionsHealthSolutions #MentalHealthAwarenessMonth #MentalHealthAwarenessWeek #MentalHealthisImportant #CrisisSupport #SelfCare #MentalHealth #MentalHealthMatters #988  ConnectionsHS.com  Download the transcript here

Dr. Shankar Musunuri is the Co-Founder and CEO of Ocugen, which is a biotechnology company focused on vaccines for public health, gene therapies targeting rare diseases in the retinal space, and cell therapies for regenerative medicine applications in cartilage repair. One key opportunity is to develop an inhalation vaccine that would be durable and efficient at preventing and controlling transmission of COVID and able to be combined with a flu vaccine.  Shankar explains, "There is a linkage if you take some of these vaccines and gene therapies. Some of the processes we use to make gene therapies can also use similar processes for vaccines. So there is a shared science, a shared manufacturing platform, and shared expertise. And then if you take the biotech itself, cream-of-the-cream in the pharmaceutical industry, they work in cell and gene therapies and vaccines, very complex science." "If you have a mucosal vaccine, which will create antibodies in the respiratory tract and control where the virus enters, it can neutralize it. And also, if you have neutralizing antibodies in your respiratory tract where the virus enters, you'll not only neutralize what is entering, it'll prevent transmission or control transmission of the disease." "If we can move COVID into this annual booster, just like flu, we thought we could provide solutions for COVID, flu, and combination. So, imagine yourself walking into a CVS, and what Ocugen wants to do is provide solutions for customers. If you want only flu, you'll get your flu inhalation vaccine. You want only COVID, and you'll get that. If you want a combo, you're going to get a combo. So we created three different vaccines, COVID, flu and combination, using our inhalation technology and novel vector platform." #Ocugen #Vaccines #InhalationVaccines #InhaledVaccines #COVID #Flu  ocugen.com Download the transcript here

Steve Glover, Co-founder and CEO of ZyVersa Therapeutics, focuses on rare kidney diseases and diseases with an inflammation component. Inflammasomes, the first response in the innate immune system, create an inflammation cascade.  The ZyVersa approach targets multiple inflammasomes to address diseases caused by the malfunction of this response. Steve explains, "Inflammation drives a lot of different diseases. And as we get into this a little bit more today, it's everything from kidney disease such as rare diseases such as FSGS and Alport syndrome and diabetic kidney disease all have an inflammation component. And in addition to that, when you look at diseases like Alzheimer's or MS, or Parkinson's, they're inflammation driven as well. So there is a connection between the two disease classes." "I've been in the industry a long time, and every so often the industry finds a new interesting discovery, and inflammasomes are clearly one of those new discoveries. In fact, one of the things I talk about quite a bit is that ten years ago, there were about two or so publications that were available on inflammasomes. Now there are over 20,000 publications available on inflammasomes. And this whole concept of inflammation driving certain diseases has become an area of an immense amount of research and development of products that can inhibit the inflammation you don't want in the human body." "When inflammasomes go into overdrive and are left uninhibited, they can cause a lot of damage to organs and cells in the body. And so, for example, they drive a lot of different diseases, everything from cancers to diseases such as Parkinson's and Alzheimer's disease, and a whole host of CNS diseases, as well as non-CNS diseases, that are driven by this inflammasome activation. So it's really important to find a way to inhibit those cytokines by inhibiting the inflammasome." #ZyVersa $ZVSA #RareDisease #InflammatoryDiseases #KidneyDisease #RenalDisease #FSGS #FocalSegmentalGlomerulosclerosis #AlportSyndrome #DiabeticKidneyDisease #Inflammasomes   ZyVersa.com Download the transcript here

Carly Newton is an apheresis expert at Terumo Blood and Cell Technologies, a medical device company that manufactures and designs systems that collect blood components and cells. Their software provides management tools to optimize procedures and help physicians better manage care. The largest patient group treated with red blood cell exchange are those with sickle cell disease and benefit from the Terumo iron-neutral therapy over a simple transfusion. Carly explains, "We've also got software technologies that many of our blood banks use and they're designed to get the most from blood donors when they go and present at a blood bank. So, for instance, instead of a blood donor just donating a bag of whole blood, our software can actually look at the starting counts of these donors and recommend what the blood donation center should collect from that donor to get the optimal cells from that patient." "The main goal of any transfusion therapy with sickle cell disease is to increase the oxygenation of the blood. There are different types of transfusion therapy and this is where we spend a lot of time educating physicians and patients on the different types of transfusion therapy. So probably the most common type of transfusion therapy for sickle cell patients in the US at the moment is something we call simple transfusions. And that's really sickle cell patients going into the hospital once or twice a month and having red cell infusions. The purpose of that is to increase the amount of healthy donor cells in these patients and increase oxygenation. " #Terumobct #RedBloodCellExchange #RBCX #SickleCellDisease #BloodBanks Terumobct.com Download the transcript here

Dr. Daniel Karlin, Chief Medical Officer at MindMed, is bringing an updated approach to developing an LSD-based drug to treat anxiety and depression. Instead of developing a psychiatric medicine to be taken every day, MindMed is changing the paradigm to a session-based therapy that uses psychedelic drugs and therapists to improve psychiatric health. As a trained psychiatrist, Dan works at the intersection of psychiatry and neuroscience with this psychedelic-assisted therapeutic approach to brain health.  Dan elaborates, "For quite some time, I think it feels like psychiatry hasn't been meeting people there. Treatments have been more focused on symptomatic resolution and these sorts of things, and visits with psychiatrists can feel too short and it can be really hard to find and afford psychotherapy. I think that what people are responding to about LSD and other drugs like it and their therapeutic potential is the sense that they offer something more than what people are able to access today. That there's real hope that these are drugs that can help people get at the real reasons they feel the way they feel and come to understand themselves better and their role in the world." "There's another school of thought that the mechanism of these drugs is largely neurobiological and that the perceptual experience may not even be necessary. Of course, anytime we start to do binaries in biology, we're probably wrong. And it's probably some of both. And we operate under that hypothesis too, that the mechanism by which these drugs cause a psychedelic experience is very well explicated. The mechanism by which they cause lasting change isn't." @MindMedco #MNMD $MNMD #PsychedelicMedicines #PsychedelicTherapy #LSD #Psychedelics #MentalHealth #BrainHealth #PsychiatricHealth  mindmed.co Download the transcript here

Dr. Waqaas Al-Siddiq, CEO and Founder of Biotricity, shines a light on the need for better data about heart rhythm throughout the day, not just for a limited time. Biotricity has developed a non-invasive, cost-effective, available over-the-counter monitor that provides patients and cardiologists with a new level of understanding of heart activity and enhances diagnosis and disease management. Waqaas explains, "I'll take a step back to try to explain the problem that we were trying to solve. Dexcom created a continuous glucose monitor, saying that there are these data points that are in between when you're pricking your finger and collecting your information, which is quite valuable and insightful." "In the continuous heart monitoring world, or in the heart world, patients don't really have anything. You have an implantable loop recorder, which records your ECG continuously. But you have to be a high-risk patient, it's a very high cost to get that implanted, and when it comes to a non-invasive solution, you have a 30-second ECG. If we look at the criteria, the patient issues are asymptomatic. They happen while you're sleeping. If it's a 30-second or 3-minute ECG and you're sleeping and don't feel the symptom, you're not waking up to collect that information. And even if you were, by the time you wake up and get the device, that information is gone." #Biotricity #Cardiology #Cardiovascular #HeartDisease #Wearables #ContinuousHeartMonitoring  biotricity.com  Download the transcript here

Dr. Eddilisa Martin is the CEO and Founder of M & B Sciences, which has created the Neighborhood Trials online platform and app available in the Apple Store and the Google Store. Their mission is to drive more diversity in clinical trial participation by providing accessible information to patients, providers, and caregivers looking for therapies and opportunities for trials.   Eddilisa explains, "When we talk about diversity, people most often think of race and ethnicity. And that's very, very important. But diversity means having representation across a whole spectrum of things. So whether that's age, whether it's your sexual preference, whether you live in the country versus the city, whether you have lots of comorbidities, lots of illnesses in conjunction with the disease that's being tested, or whether that's the only thing you have. There are a whole host of things that we're talking about when we say diversity." "But you hear people talk about their hesitancy to be a guinea pig. And so it really does help people to understand that things have changed from the way they used to be. People hear horror stories from years and years ago, but they don't realize all of the protections that are in place for patients now. So the FDA committee has oversight. There are all of these safety committees that are involved in every single clinical trial. There's a safety committee that's looking at results as they are going, so they're not waiting until the end of the trial and seeing if there are different side effects that are popping up. They're measuring that on an ongoing, continuous basis." #NeighborhoodTrials #ClinicalTrials #DecentalizedClinicalTrials #DiversityinClinicalTrials  NeighborhoodTrials.com Download the transcript here

Eric Shaff is the President and CEO of Seres Therapeutics, which is focused on the gut microbiome in order to develop bacteria as an oral treatment for bloodstream infections, cancer, and immune system diseases. The first therapy is SER-109 for recurrent C. diff infection an orphan disease with inadequate solutions for patients. The starting point for Seres is that the health of the microbiome is the starting point for preventing infections from occurring. Eric explains, "The real susceptibility of someone getting C. diff is not just the bad actor bacteria. But really the susceptibility of the microbiome and an injury to the microbiome, and susceptibility to infection. Typically what happens is if people get antibiotic therapy, which can be very effective in knocking back or killing all bacteria, it creates a significant niche in the microbiome, or an injury. There is what we call a race to repair, so either a patient will naturally repair and restore their microbiome, or C. diff may infiltrate, which can lead to an infection and a recurrence." "Typically, if a patient is going to have a recurrence, it happens quickly. If they get C. diff, they take antibiotics, and the hope is that C. diff will not infiltrate to cause an occurrence. If it does, then people typically recur quickly. What happens following antibiotic therapy, is we treat patients with an oral regimen of SER-109, which is a consortium of bacteria in spore form you put in the capsule. That was the approach we took to our phase 3 study, where we had an 88% reduction in recurrence, which is a significant impact for patients suffering from this terrible disease." @SeresTX #SeresTherapeutics #Microbiome #OralMicrobiomeTherapy #Cdiff #RecurrentCdiff #AMR #AntimicrobialResistance #InfectionProtection serestherapeutics.com Download the transcript here

Ashley Perry is the Chief Strategy and Solutions Officer at Socially Determined, a social risk analytics and solutions company working with payers, providers, and life science companies. Their Socialscope risk and data analytics platform helps organizations understand social determinants of health, identify and manage risk and advance equity for the populations they serve. These organizations are able to be data-driven and action-oriented to mitigate community and individual-level risk. Ashley explains, "The evidence is very clear from a multitude of sources that up to 80% of healthcare utilization, cost, outcomes, and, importantly also, equity are driven by non-clinical factors or non-medical cost drivers. That includes all aspects of everyday life. So, you think about factors like food insecurity and housing instability, transportation barriers, and health literacy challenges. We see consistently in the data that we look at every day the impact these factors have on those utilization costs and outcome measures. Our customers are ultimately focused on population health and the overall health and well-being of the communities and populations they serve." "Socialscape is a platform that assesses both community-level SDOH risk exposure for the entire United States as well as individual-level social risk factor scores for millions of adult Americans. Importantly, we do that across different domains. For example, on the community side, we look at factors like the economic climate, the housing environment, and the digital landscape. On the individual patient or member side, we look at factors like food insecurity, housing instability, and transportation barriers." "Across all of those different domains, we're able to quantify risk using a standardized one-to-five scale. One indicates little to no risk, five indicates severe risk. It empowers the organizations that we work with to be able to identify the contours and concentration of community-level risk for the markets that they're serving. As well as identify the segments of their overall population that they bear the risk for that face elevated risk across those different social domains." @SocDetermined #SDOH #SocialDeterminantsofHealth #ACOREACH #HealthEquity #SocialRisk #Data #DataAnalytics sociallydetermined.com  Download the transcript here

Rich Pascoe, CEO of Zevra Therapeutics, is working with a team with deep experience developing drugs and getting them approved by the FDA.  Having developed multiple prodrugs for pain and ADHD, Zevra is now developing rare disease therapies with an initial focus on Niemann Pick Disease Type C and idiopathic hypersomnia.   Rich explains, "Our lead product is a drug called arimoclomol. It's intended for the treatment of Niemann Pick Type C disease, or NPC for short. Niemann Pick primarily affects children. It's a lysosomal storage disorder condition, meaning patients lack the ability to break down certain things, including cholesterol. As a result, they develop a myriad of symptoms that ultimately lead to, in most cases, the patient's death. The issues they deal with revolve around loss of ambulation and the ability to swallow, and it affects their cognitive function. It's a progressive disease, and oftentimes, it can advance very rapidly." "A little history on arimoclomol. The drug was previously in the hands of a company out of Denmark. We acquired the asset last year from that company along with some of the individuals at the company that are critical for the success of the product going forward." "In that setting, Orphazyme, the company that had the drug before us, had submitted it for approval here in the US with the FDA. The FDA rejected that approval request based on a number of factors that we have subsequently been working to address. We believe that given our track record as a company of being able to work with the FDA to get drugs approved, in some cases under some very challenging circumstances, that we are well-equipped to handle and manage this submission."  @Zevratx #ZevraTherapeutics #RareDisease #RareSleepDisorders #NiemannPickDiseaseC #IdiopathicHypersomnia zevra.com  Download the transcript here  

Steve Reed, Co-Founder, President, and CEO of HDT Bio, has spent his life focusing on tropical diseases and points out how the cost of developing and manufacturing vaccines determines access for a global population. HDT Bio was founded to develop more cost-effective technology to induce broader immune responses to various diseases. Their advanced RNA platform AMPLIFY is a technology applied to developing mRNA vaccines for infectious diseases and a therapeutic vaccine to help prevent recurrence and prolong survival from cancer.  Steve elaborates, "COVID was something that came up right after we founded the company, and so the National Institute of Health asked us and funded us to develop a novel platform that could complement the platforms already in place for mRNA vaccines. Other indications that are incredibly important include other viruses. They're always coming up, and they're always mutating. But you probably read in the news about fungal infections on the increase in the United States and other countries." "The AMPLIFY technology is an RNA platform that expands in a limited way once it's introduced into the cell and then enables you to use a much lower amount of RNA. So that's significant in terms of cost, as well as safety. And then to be able to deliver the RNA, you have to protect it into a formulation, and the formulation we've developed is one that can be mass-produced at low cost and is very safe." "Back in the '90s and 2000s, when molecular biology, and monoclonal antibodies, those technologies became widely used and understood, we all naively thought that the road to a parasitic disease vaccine would be relatively straightforward. And in some ways, while the technology has enabled such vaccines, the practical limitations have stopped progress or severely limited it." #Vaccination #Vaccines #HealthForAll #COVID #mRNA #RNA #InfectiousDiseases #Cancer HDT.bio  Download the transcript here 

Jeff Liter is the CEO and Founder of Luminary Therapeutics, which is using gamma delta cells in their approach to CART-T cell therapy because of the ability of these cells to infiltrate solid tumors. With a multi-targeting system, Luminary has demonstrated promise treating multiple types of solid tumors, blood tumors, and autoimmune diseases. Their goal is to create a scalable model to manufacture allogeneic cells to drive down the cost and availability of effective cell therapies. Jeff explains, "We're focused primarily on three big challenges in the CAR-T industry. The first is the overall manufacturing approach and cost. The second is why do CAR-Ts, to date, not work in solid tumors. And then finally, the third is antigen escape and relapse of patients currently being treated by CAR-T therapies."  "We have moved into coming back to the manufacturing area. We have moved into an allogeneic platform, which for your listeners, if they're not familiar with that term, means that we can make multiple products from one healthy donor. And we do that in a very special way, using gamma delta cells and preserving both subsets of the gamma delta cells. Then we also add some immune cloaking with our non-viral gene engineering, and that makes us very unique on the allogeneic front." "If you look at those 800 companies, probably 80, 85% of them use what's called alpha beta normal white blood T cells. A fair number of companies, maybe 7% to 8%, use NK cells. And then there's a new group using a different kind of subset of cells called IPSC. But in the gamma delta, there are only 12 to maybe 15 companies. So we're very differentiated on that front from the 800. And then, as I noted, we are the only company to have both Vdelta1 and Vdelta2 subsets in our final product."   #LuminaryTherapeutics #CARTCellTherapy #CARTTherapies #Allogeneic #Autologous #CellTherapy #GammaDeltaCells #SolidTumors #Cancer #BloodCancers #AutoimmuneDiseases luminarytx.com Download the transcript here

Joe Tucker, the CEO of Enveric Biosciences, is discovering, developing and testing psychedelic-inspired therapies for the treatment of depression, anxiety, and PTSD. Current treatments target receptors in the brain to increase the concentration of serotonin in the brain by going through classic neurotransmitter pathways. The Enveric Psybrary portfolio and PsyAI drive drug discovery to create new molecules that synthesize chemistry and how nature makes molecules to target new receptors. Joe elaborates, "This one's completely different. This one is actually going through a receptor that's never been targeted before, 5-HT2A. And it's an adjunct to psychotherapy at this point. The leading molecules are an adjunct to psychotherapy. Although there's some interesting new work, including stuff that we're working on, no one has yet been able to separate those two in humans. But that's where we'd like to go next, is to separate them. But right now, it's really this 5-HT2A receptor, this induction of hallucination. No one else is doing that. No other molecules out there do that as part of a treatment program." "The issues with those classic psychedelics are, as you can imagine, they weren't designed as mental health treatment molecules. They came about for other reasons. And so they have a lot of side effects or safety issues that really make them suboptimal for patient use. So psychedelic-inspired says, let's take what we can learn from here. If you target the 5-HT2A receptor, you can induce some interesting effects in the patient, but can we then modify the molecules so they have better attributes?" "And in doing so, we also realized we're breaking new ground. I mean, we're making hundreds and hundreds of new molecules in an area where a handful of molecules had existed before. Five molecules, ten molecules, is what everyone else has looked at. We realized we were in this completely new field, and we had to come up with a way to identify prospectively which of these new molecules were going to be likely good drug candidates for various mental health indications." @Enveric_Bio #EnvericBiosciences #MentalHealth #Psilocybin #Anxiety #Depression #PsychedelicInspiredTherapeutic #PsychedelicBasedTherapeutic #Psychedelics enveric.com Download the transcript here

Dr. Joshua Schiffman is the CEO and Co-Founder of Peel Therapeutics and has taken the understanding of the natural resistance to cancer by elephants to develop PEEL-224 to attack tumors. Using a plant toxin and modifying existing approaches, Peel has created a drug that increases the amount of the drug that gets to the cancer cells with reduced side effects.   Joshua explains, "Peel is actually the Hebrew word for elephant. So that's why we call ourselves Peel Therapeutics. My Co-Founder, Dr. Avi Schroeder, is from the Technion-Israel Institute of Technology in Israel. While we were working on the elephant drug, we realized, you know what, it's more than just elephants. Looking around nature, we understand that evolution has had hundreds of millions of years to perfect, in some cases, perfect cancer-fighting protein or peptide or anti-inflammatory molecule. Our lead drug, which we call PEEL-224, actually works really well targeting the DNA repair mechanism in cancer cells." "This DNA repair mechanism is called topoisomerase 1. And by blocking it, we're able to actually ensure that cancer cells essentially self-destruct. At Peel, we say that we like to engineer nature better than evolution intended. So, in the case of PEEL-224, we've taken this plant toxin, it's called camptothecin. It comes from an ancient Chinese tree called the Happy Tree, and we've modified it so that it can avoid chemoresistance. So, in other words, you give this drug to the patient, which goes to the tumor, and the tumor cell can't spit it out anymore. And we're seeing remarkable early results in mice and now have begun a clinical trial." @peel_tx #PeelTherapeutics #PeopleofPeel #Cancer #Inflammation #PEEL224 #Nature #Biotech #NaturesMoleculesIntoMedicine  peeltx.com  Download the transcript here

Cristina Varner is the life science Practice Leader at Newfront, a technology-focused insurance broker. While digital health and telemedicine companies have robust technology infrastructure, they tend to be less medically focused and unaware of insurance requirements to cover bodily injury, financial injury, data breaches, cyber liability, and state regulatory requirements. Wearables and data collected by them are adding issues of privacy, security, and data accuracy. Cristina elaborates, "I'm not going to say I'm surprised because a lot of the founders of these companies come from technology backgrounds, from tech companies. And so insurance just is not, and risk management is just not top of mind for many of them. And so we're not surprised. They're moving very quickly. I think when COVID came to be, digital health definitely exploded. And during the COVID period is when we started to recognize where some companies were lacking in risk management infrastructure. And I think that many of the companies we see now are much better positioned from a risk management perspective and also are more aware." "There are more customers and patients with wearables that are recognizing that their privacy and their security are important. I don't think historically, a lot of owners of wearables ever really considered those types of concerns. But now there's a lot more scrutiny in this area. And the other issue that comes up in tandem with privacy and security is data accuracy. If your data is not accurate on your wearable, for example, and it allows a patient to make a decision based on inaccurate data, which can result again in potential bodily injury to the patient." @NewfrontHQ #DigitalHealth #Telemedicine #Insurance #Wearables #RiskInsurance  Newfront.com Download the transcript here

Seli Fakorzi, Director of Mental Health Operations at TimelyCare, and Gaurang Choksi, CEO and Founder of Violet, join me to discuss their partnership to bring comprehensive healthcare to college students from all socioeconomic backgrounds, genders, ethnicities, and physical abilities. Training healthcare providers with the tools to understand different communities allows clinicians to learn new ways to be more inclusive and build trusted relationships with patients. Seli explains, "Providing quality, equitable access to care is a foundational mission for Timely. We are aware that students from underserved and underrepresented populations many times possess unique barriers to care, and that has made seeking help increasingly difficult. Breaking down those barriers in as many areas as possible has been our goal. Forming this relationship and partnership with Violet will help in those areas."  Gaurang elaborates, "I'll briefly share exactly how we get to understand how a clinician is doing, and then I'll share more about how we educate. At Violet, when a clinician joins us, we go through a process called benchmarking their cultural competence. We take a look at five pillars of information, including who is that specific person, what communities their family or they belong to, we look at what are the communities they feel really confident serving. That's a clinical measure called self-efficacy that we've pulled in." "Then we also look at what are the communities they want to work with, the communities they've studied about and the communities they have worked with, using claims data and their voices. Then using all this information, we get to have clarity on is that specific provider doing well working with the BIPOC or LGBQ or TGNC communities." #TimelyCare #JoinViolet #Healthcare #VirtualCare #LGBTQHealth #TGNCHealth #BIPOCHealth #MentalHealth #HealthDisparities #HealthEquity #MinorityMentalHealth timelycare.com       joinviolet.com Download the transcript here              

Michael Axt, the Chief Member Empowerment Officer at Zelis, is focused on helping healthcare payers and clinicians provide more information to patients about expected costs for procedures and visits to providers and hospitals. A recent survey conducted with Hanover Research revealed insights about patients' challenges in understanding healthcare billing and correcting errors. Driven by the move to transparency, Zelis acquired Sapphire to offer the Sapphire Digital solution to enable patients to better understand insurance benefits and the correct amount the patient owes.  Michael explains, "We found some interesting insights. Over 40 percent of the consumers in our study indicated that they were significantly frustrated in trying to find the right person to resolve an error. So they might have found an error, but they didn't know how to do anything about it. For those individuals, it took a month or more for nearly half of our respondents. For 70 percent of them they spent over two hours of their own time trying to work to correct this." "To the extent that there's greater clarity for the member or the patients in understanding the bill and what they owe, they're more likely to pay it more promptly. So we all know that cash flow is a challenge, and collections on patient liabilities are frankly atrociously low in the healthcare space." "For the health insurance companies and payers, there are options as well. Obliviously improving the member experience helps to improve retention and reduce churn in the plans. Also, if they're bringing tools that help to accelerate payment to the provider, it has opportunities for them to improve their relationships with their provider networks, which is a huge strategic asset for the health insurance companies." @ZelisHealthcare #Healthcare #PaymentIntegrity #HealthcareCosts #ConsumerBilling #PriceTransparency #HealthcareBilling #MedicalBillingErrors zelis.com Download the transcript here