Empowered Patient Podcast

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