Empowered Patient Podcast

Sofia Elizondo, COO and Co-Founder of Brightseed, discusses the company's focus on bioactives and their potential to improve health outcomes. Bioactives are small molecules found in nature, such as plants, fungi, or bacteria, that activate human biological receptors and positively affect the body. Brightseed uses its AI platform, Forager, to identify and commercialize these bioactive compounds in foods, supplements, and over-the-counter medicine. As part of the food is medicine movement, Brightseed's goal is to make these superfoods more accessible to a broader population. Sofia explains, "To bring us back to the source, caffeine is a small molecule. It’s a natural chemical that a seed produces. And so we take these coffee seeds, grind them up, and then extract the caffeine and other tasty flavors with water every morning." "When we take a sip of our coffee, it’s actually very well understood what happens with caffeine. We absorb it in our stomach lining. The caffeine molecule goes into our bloodstream, goes into our brain while our blood is circulating around our body, and docks with a biological receptor called the adenosine receptor in our brain. What happens is it interacts with this receptor, and then we feel a bunch of downstream effects. We feel focus and energy, and maybe too much of an adrenaline rush sometimes." "But this is a great example that many of us can be familiar with of the power of such a small molecule. It’s just one bioactive in one bean of one plant that can have this very precise and recognizable effect. In this case, science knows very well exactly what it does." "In the big picture of bioactives, there are hundreds of thousands of them in plants and foods that we eat every day that we may not know or may not have cataloged. Science, in most cases, doesn’t exactly know what it does when we ingest these bioactives, at least not yet." #Brightseed #AppliedAI #Bioactive #Nutrition #FoodisMedicine brightseedbio.com Download the transcript here

Luke Piggott, principal scientist at Debiopharm, has a unique business model in the drug discovery and development to commercialization landscape. Focusing on rare cancers, they license drugs from smaller biotechs and conduct clinical trials before out-licensing them to larger pharmaceutical companies with marketing capabilities. The use of AI is expanding biomarker discovery and the identification of potential novel mechanisms for targeted therapies. Novel-novel combinations are being explored to provide therapies with more selective profiles with fewer side effects. Luke explains, "Rare cancers is an interesting area. I mean, it’s never an easy one to be developing in the space of rare cancers. With a business model like ours, we have the opportunity to take some of these assets that perhaps are not as, shall we say, financially attractive to investors because of the smaller market opportunity. As a privately owned company, we have the ability to develop these kinds of drugs and move them ahead with solid financial backing. And so I think that’s one of the areas that we have the opportunity where some other companies would not." "We do see overlap there with other diseases, although they may be morphologically different than the current specific sites that are more rare than other diseases. These underlying mechanisms are consistent across the different types of cancers. Now that we’re in the era of precision medicine and going after specific key oncogenic drivers of these tumors, it does allow potential opportunities to treat them in a more similar fashion, shall we say, to the larger populations or at least re-purpose some of those drugs and try them out on these rarer diseases." #Debiopharm #RareDiseases #RareCancers #DrugDevelopment #PrecisionMedicine #BiotechInvestment Debiopharm.com Download the transcript here

Tiara Green, President of Accessia Health, provides financial assistance for copayments, physician visits, durable medical equipment, radiology testing, and travel expenses to support individuals with rare and chronic healthcare conditions who cannot afford their care. They also offer educational services to help patients navigate the healthcare system and make informed decisions about their healthcare. They are reaching out to patients through physicians, pharmacies, patient advocacy organizations, and faith-based and community-based organizations. Accessia Health aims to improve overall healthcare outcomes by using online tools to reduce emergency room visits and help patients adhere to treatment plans. Tiara explains, "Accessia Health is a national nonprofit organization. We’re a patient assistance organization that supports individuals with rare and chronic healthcare conditions. I think we all have been impacted or have been in the healthcare system to receive care, and we know that the cost of care is extremely high. So, our mission is to serve as a safety net for those individuals who cannot afford their care. I love our tagline, "We speak health and human," because we realize the human side of healthcare in that we’re people first, and individuals are not necessarily defined by their condition. However, we also understand what it means to navigate the healthcare system. We want to do our part to stand in the gap for them and help them with their costs so that they can focus on their healthcare journey." "We also provide educational services. We have a webinar series, Amplify Your Voice, Control Your Care, where we want to give those individuals tools for success, helping them navigate the complex healthcare system to make informed decisions about their healthcare. So, it may be a topic on, "How do I properly communicate with my physician?" Or it can be a topic such as, "What does the Inflation Reduction Act mean for me?" However, we want to ensure that they understand their healthcare so that they can make the appropriate decisions." #AccessiaHealth #PatientAssistance #FinancialAssistance #RareDiseases #ChronicConditions AccessiaHealth.org Download the transcript here

Roger Rivera, psychiatric mental health nurse practitioner at Mente Suave Psychiatry, discusses the different types of bipolar disorder, the potential causes of the disease, available treatment options, and the importance of early detection and management. Roger emphasizes the need for a comprehensive approach to care and highlights the impact of bipolar disorder on patients' lives and society as a whole. While there are currently no biological markers for bipolar disorder, ongoing research and initiatives, including the use of AI, show promise in improving diagnosis and treatment. Roger explains, "So rather than bipolar disorder, it should probably be called bipolar disorders just because of the different manifestations that it has. One of those manifestations could be manic episodes. Manic episodes could be described as just expansive mood, inflated self-esteem, decreased need for sleep, more targeted flight of ideas, and all these other things, which have a very strong correlation with functional impairment. And that's just one of the variations of bipolar disorder." "We also have bipolar II, which has more hypomanic type features. So, if we look at the diagnostic manual that we use in psychiatry, we'll see that for bipolar I, it highlights we need at least one manic episode, but we didn't need a depressive episode. Yet, when we look at bipolar II, we usually need at least one depressive episode associated with that." #BipolarDisorder #MentalHealthAwareness #TreattheWholePatient #BipolarTreatment #PatientEngagement mentesuavepsychiatry.com Download the transcript here

Punit Dhillon, Chairman and CEO of Skye Bioscience, focuses on anti-obesity and developing a drug that targets the cannabinoid receptor 1, which plays a role in signaling the body to store fat. The next generation of CB1 inhibition moves from a centrally mediated pathway to targeting CB1 receptors outside the brain. Deploying the endocannabinoid system, their lead drug candidate targets the major organs where CB1 receptors reside in fat tissue to increase mitochondrial activity and burn more fat. Punit explains, "Skye is focused on exploring research modulating different therapeutic targets via the endocannabinoid system. Our research has narrowed in on the CB1 receptor, where we’re modulating it by focusing on this pathway of inhibition. There’s a big body of evidence, actually existing validation of this cannabinoid receptor 1 being a really important target to reduce caloric food intake and increase energy expenditure. It’s been a really important target for weight loss because of its particular relevance in that therapeutic area. So we’re advancing in a very interesting clinical pipeline and immediately a Phase 2 study that will look at that particular target. We hope that we will be able to show its relevance in the anti-obesity medication landscape." "I feel like the current landscape of drugs that have been getting the most attention has been focused on caloric restriction. Weight loss is driven by that particular pathway of restricting the amount you eat. We are trying to shift that paradigm to the opportunity to address the underlying issues relating to weight loss so you can essentially have this aspect of targeting fat and being able to address fat metabolism. That comes back to this link to the exercise comment you made because it’s driving the underlying mitochondrial activity, which is helping to improve the thermogenesis and the conversion of that fat into thermogenic energy." #SkyeBioscience #Obesity #EndocannabinoidSystem #WeightLossDrug #CB1Receptor #GLP1 skyebioscience.com Download the transcript here

Joe Ferra, CEO of Elevation Oncology, highlights the unmet needs in gastric cancer and the potential of targeting Claudins, proteins involved in cell adhesion. The current competitive landscape of antibody drug conjugates (ADCs) confirms the promise of a targeted therapy that delivers a cytotoxic drug directly to cancer cells. Targeting Claudin 18.2 with an ADC approach offers an opportunity to treat a broader range of tumors expressing a lower level of Claudin 18.2 than those currently treated by CAR-T therapy. This approach is being investigated as a single-agent drug with promise for use in combination for gastric cancer, pancreatic cancer and esophageal cancer. Joe explains, "As you likely know, in the continuum of cancer drugs, you have chemotherapy on one end that indiscriminately kills everything it touches. On the other end, you have uber-targeted therapies looking at specific drivers of what’s driving that specific tumor. In our case at Elevation Oncology, we’re focused on what we like to call selected targeted oncology drugs so that we are using the unique characteristics of the tumor to target and meet in a targeted way attempt to kill the tumor for a better outcome for the patient." "Gastric cancer in and of itself was an area where Claudin 18.2 is known to be highly expressed, but for gastric cancer, to your point, there’s a huge unmet need. For most patients, once they’re in second line or third line, the opportunity for drugs that are available to them today is, unfortunately for all of us, very dismal. We think there’s a significant opportunity to utilize a Claudin 18.2 antibiotic drug conjugate for overall better outcomes for patients living with gastric cancer." "We quickly saw an ADC approach as an opportunity to treat a broader range of Claudin 18.2 expression. Now, as I’m sure you’re aware, in any target in any tumor, there’s always a range of expression for Claudin 18.2. In gastric cancer, it’s known that as much as 80% of gastric cancer expressed Claudin 18.2 at some level. With that expression, we think an ADC approach will be able to treat a broader range of expression." #ElevationOncology #ADC #AntibodyDrugConjugate #SolidTumors #Claudin182 #Claudins #Cancer ElevationOncology.com Download the transcript here

Dr. Steve Goldberg, physician and Senior Clinical Advisor at DecisionRx, and Dr. Jamie Wilkey, pharmacist and expert on medication therapy optimization, explain that medications are the fourth leading cause of death in the country. They emphasize the need to consider genetic information and pharmacogenomics to design personalized medication plans to improve health outcomes. The DecisionRx solution is integrated into the pharmacist's workflow to improve medication safety, reduce avoidable costs, and enhance communication with providers, pharmacists, and patients. Steven explains, "Our goal is to help people get more benefits from the medications that they use. A couple of starting facts. The US spends about $500 billion-plus a year on medication, and sadly, there are hundreds of thousands of ER visits, hospitalizations, and deaths that occur to people attributable to medications. There are a number of reasons for it, but relative to what we do, it is often triggered by people having genetics that make them not do well with particular medication. It’s often the regimen that they’re on has too many drugs, and that creates a problem. Finally, and potentially most importantly, they’re not taking the time or not given the time to have their medication regimen carefully reviewed by a well-trained pharmacist to help organize things for the best outcomes. And best outcomes, reduce health risks, control chronic disease, and provide treatment." Jamie elaborates, "We also consider the genetics of people taking their medication, which has been overlooked significantly in the past. Specifically, the way our body metabolizes medication is so different based on ethnicity, background, and gender, and you can’t just tell by looking at a person how they’ll metabolize medication. We can finally understand what medication is likely to work for a person, which is unlikely to work, and what is probably going to give them a lot of terrible adverse reactions before the patient has to go home and try it." #DecisionRx #Healthcare #MedicationTherapyOptimization #PrecisionMedicine #Pharmacogenomics #HealthcareInnovation decisionrx.com Download the transcript here

Ryne Natzke is the Chief Revenue Officer for TrustCommerce, a Sphere company that facilitates electronic payments in the healthcare industry, helping ensure security and compliance in transactions between patients and providers. Healthcare payments present unique challenges, as patients have varying preferences and are impacted by economic factors on their ability to make healthcare payments. TrustCommerce aims to make the payment process easier for patients by integrating with electronic health record systems and patient billing systems, reducing friction in healthcare payments and providing patients with more options and transparency in pricing. Ryne explains, "Some people like paying online, and some people pay in person. There are payment plans. So what we do with our technology and our partnerships with EHRs like Epic and patient billing systems is help make sure that they can facilitate those patient financial experiences across the board and have that happen within systems they’re already using. So within MyChart, for example, or just while the patient’s checking in at the front desk. We help take that PCI compliance and security piece off the shoulders of our partners and our healthcare systems using our technology." "Nobody really plans to have a disease like cancer, and all of a sudden, you have a whole bunch of oncology appointments or plans on getting into a car accident and showing up in the emergency room. So it’s not really a budgetable expense the same way as I’m going to save up for a Peloton or a new TV or whatever it is. So you’re kind of balancing this unknown of what the balance is and being able to line up what that patient might be able to do. Somebody might be able to pay a $2,000 bill when they receive it, and a lot of people can’t. So how can we put them on a payment plan and be empathetic of where they are within their situations while also still having all of the compliance and security pieces that come with the transactions?" #TrustCommerce #TransparencyinPricing #HealthcareCosts #HealthcarePayments trustcommerce.com Download the transcript here

Dr. Andrew Dold, Co-Founder of RevMed, a B2B marketplace for hospitals and ambulatory surgery centers (ASCs) to address the problem of slow-moving or expiring inventory. This medical marketplace allows facilities to recoup lost revenue by listing medical devices for sale and offers purchasing options for smaller facilities. RevMed serves as a middleman, facilitating the interaction between facilities without taking custody of any items. The marketplace has an extensive database of millions of different SKUs and items, which are updated weekly. The goal of RevMed is to decrease the price of healthcare by getting as many facilities as possible to adopt the platform. Andrew elaborates, "So, full disclosure, this problem came into light during my fellowship at NYU. I did a sports medicine fellowship a few years ago in New York City, and it simply started with me interacting with one of the representatives, the orthopedic representatives at my hospital. I wanted to get a few suture anchors to practice tying knots in my apartment in New York City. And he simply took me into the back supply room. On one of the shelves, he grabbed a number of different boxes, just flipped them over, looked at the expiration date, and I think they were all expired. And he handed me ten of these boxes and said, here you go. You can practice on these." "There were brand-new implants that had simply expired on the shelf of the surgery center where I was operating in New York City. It shocked me at the time because of simply the cost of these implants. One of these boxes was in the vicinity of $800 to $1,000, and he handed me 10 of them to take home because they had expired. They were now worthless. They couldn’t be used on a patient. From that point forward, I became more aware of the problem of stale, slow-moving, and expiring inventory and the surplus of medical and surgical supplies on the shelves of hospitals and ASCs. And what a massive problem it is." #RevMed #supplychain #healthcare #medicalequipment #healthcaresupplychain #revolutionizeprocurement #medtechmarketplace #transparenttransactions #sustainablesourcing #healthcareinnovation #medicaldevicemarketplace revmedconnect.com Download the transcript here

Ed Esquenazi, Founder and CEO of Sirenas, is exploring the chemistry of marine organisms to accelerate drug discovery. Marine-based scientific elements are still poorly understood but have the potential to produce unique and useful compounds for drug development. There are stark differences between land-based organisms and marine-based organisms, which have evolved for much longer and, therefore, offer more diverse and complex chemistry. Using AI and machine learning, Sirenas has identified cancer-killing agents, anti-infective agents, and anti-inflammatory compounds from marine organisms and is developing marine-based personal care products. Ed explains, "We go out into the ocean, collect samples, we bring them back. We understand the different chemistry being produced in these organisms, identify and catalog it very carefully using instrumentation, and then figure out how that chemistry might impact biological systems, all with the goal of accelerating drug discovery. So the goal is to make the chemistry from marine organisms accessible to drug discovery." "We discovered one of the most powerful antimalarials working with the Gates Foundation. We’ve also identified quite a few anti-inflammatory compounds. These are things that modulate the immune system in ways to dampen inflammation or, in some cases, actually accelerate inflammation. So, we’ve identified some of those with very interesting therapeutic paths." "I think it’s a matter of diversity. A lot of the elements used in drug discovery traditionally have been based either on plants or bacteria, but land-based or synthetic, and they limit the diversity of the chemistry used in drug discovery. So, if you go into the ocean, you’re opening up a realm of very different components that can help accelerate the discovery of novel therapeutics." #SirenasMD #MarineBasedDrugs #MarineOrganisms #DrugDiscovery #Algae SirenasMD.com Download the transcript here

Kim Perry, Chief Growth Officer at emtelligent, focuses on using medical AI in the prior authorization process to structure unstructured medical text in patient records, making whole record healthcare possible. AI can be used to extract relevant information from faxed or PDF medical records, saving time and improving efficiency over existing manual processes. For payers, there is potential for medical AI to address other processes such as risk adjustment, care management, quality measures, and payment integrity in an environment still lacking interoperability and reliant on highly trained humans. Kim explains, "For prior authorization in particular, well over 50% of our prior authorizations here in this country are manual. And that means once a prior authorization is submitted for a request for a procedure or a specialty drug, it's a cue for a clinician to review. And part of that review process is to pull a patient's record." "Unfortunately, because of the lack of interoperability in the healthcare ecosystem, that record is often a fax and a PDF, which could be hundreds of pages. So, the clinician is tasked to manually read through that PDF to find the clinically relevant information to help them deny or approve the prior authorization. So, what they're doing here is using AI to pull out the relevant information at the time that's needed so the clinician doesn't have to manually read through all hundreds of pages looking for the needle in the haystack, if you will, of the information that they're looking for to solve that problem in that point in time." "I think people assume that with the adoption of EMRs, everything will be digital. But again, due to the lack of interoperability or the lack of payers, in this case, getting direct access into the EMRs for the clinical data, there has to be a way to transmit the information, the relevant information. And often, as I mentioned, it's fax-based. The health systems or the providers are faxing over medical records to the payers. Today, the payers are forced to do that manual review because they haven't had the tools available to them until now to help them apply technology to that manual review process." #emtelligent #PriorAuthorization #MedicalAI #ArtificialIntelligence #HealthcareInnovations #CMS #LLM #LargeLanguageModels #ClinicalData #EHR #FutureofHealthcare emtelligent.com Download the transcript here

Nolan Townsend, CEO of Lexeo Therapeutics, and Jen Farmer, CEO of FARA, the Friedreich's Ataxia Research Alliance, are working together to better understand and treat Friedreich's Ataxia. This rare genetic condition includes cardiomyopathy and scoliosis. Lexeo Therapeutics is studying gene therapy as a potential treatment for the cardiovascular component of the disease. The Friedreich's Ataxia Research Alliance (FARA) plays a role in funding research, understanding the natural history of the disease, and advocating for treatments. Nolan explains, "For most patients in Friedreich’s ataxia, cardiovascular disease becomes a very challenging component of the pathology, and it is what’s called a hypertrophic cardiomyopathy phenocopy. So, it mimics hypertrophic cardiomyopathy, which is a thickening of the heart wall. And that typically progresses as well, which, for many patients, ends up being the cause of mortality associated with the disease. This pathology does appear to be amenable to treatment approaches like gene therapy, and it’s one that we’re working on here at Lexeo." Jen elaborates, "As Nolan mentioned, it’s caused by mutations in a gene called FXN, and the mutation in the gene causes a silencing of the gene. So, individuals with FA have what we call frataxin deficiency. And frataxin is a highly conserved protein that is important in lots of different cells, which is why the disease affects multiple organ systems. So, while it’s a genetic disease, it’s this protein deficiency that causes the pathology within the cell. The cells just don’t make enough energy and don’t function properly, and that’s what contributes to the underlying cause of the disease." #FriedreichsAtaxia #CureFA #FAAwarenessMonth #FAAwarenessDay #RareDiseases LexeoTX.com CureFA.org Download the transcript here

Dr. Craig Joseph, Chief Medical Officer at Nordic Global, highlights the concerns and challenges healthcare professionals face when using health IT and the clinician burnout related to the administrative burdens of electronic health records. He addresses the obstacles to integrating IT into healthcare workflows, including cost, risk aversion, and resource limitations. He identifies areas where AI can have an immediate impact in reducing errors and biases. Craig elaborates, "In the past, we lived in a paper-based world, so it was pretty flexible. You could fill out a form or not fill out a form. You could give as much detail or as little detail. Often, especially physicians really couldn't read what they were writing anyway, so it was less important. All of that's changed now in the last 15 or 20 years since the advent of electronic health records." "What we've seen with large language models, like Chat GPT and other modern AIs, is the ability to do a lot of things with voice now, to be able to communicate directly with a patient, not have to look at the screen to either get information or to write a progress note. Lots of physicians have complained deservedly that they have to spend pajama time, time after they put their kids to bed at night, where they're not getting paid and writing their progress notes and office visit notes." "I think AI, like LLMs are going to really do a lot to help to rehumanize the interactions that we have. Although, there will be downsides. For that to work, there has to be a microphone in the exam room and it has to be listening to what you and the doctor are saying to one another. Folks are going to have to be comfortable with that to be able to leverage some of those things." #NordicGlobal #AI #MedicalAI #Hospitals #HealthIT nordicglobal.com Download the transcript here

Mike Kelly, CEO of NervGen Pharma, discusses the company's vision to build a startup biotech focused on repairing the central nervous system, helping patients regain function, not just slowing progression. Their first product, NVG-291, is currently in clinical trials for spinal cord injury. The drug candidate aims to promote natural repair by targeting molecules called CSPGs that inhibit nerve growth and repair. NerveGen is also developing NVG-300 for other indications such as stroke, ALS, MS, and Alzheimer's. He draws parallels between his previous experience developing the Narcan Nasal Spray for opioid overdose and the unmet medical need in spinal cord injury. Mike explains, "We are a company focusing on a group of products, more like a platform, that will enable the nervous system to repair itself. So the vision is to build a startup biotech focused in CNS and to get our first product over the line in spinal cord injury." "There are no drugs available. So this is something that happens in an instant and someone's life has changed forever. The patient typically will then get hospitalized, have surgery to decompress the area that's injured, and then they're on the road to recovery. The only treatment is rehabilitation and exercise, and they push themselves to remake as many connections as possible. Still, that repair typically plateaus around six to nine months after their injury. And the level of function they regained during that period is where they'll be for the rest of their lives, unfortunately." "NVG-291 is a product that aims to promote natural repair. When you damage, say, you cut your finger or break your foot, your body repairs itself very easily. When you damage your central nervous system, your body protects itself very well and inhibits repair, actually. Our drug targets one of those mechanisms that inhibit repair, turns it off, and allows the body to repair itself naturally." #NervGenPharma #SCI #SpinalCordInjury #SpinalNews #SpinalInjury #Paralysis #NerveRepair #CNS NervGen.com Download the transcript here

Matt Cronin, Founding Partner of House of Kaizen, discusses how patients' changing healthcare expectations are shaped by their experiences with products and services such as Netflix and Amazon Prime. Consumers now expect a longer-term relationship and personalized experiences with their healthcare providers. House of Kaizen emphasizes the importance of building better customer experiences in health and wellness to improve health outcomes and create more value for businesses. In the age of AI, the importance of trust and brand reputation is paramount. Healthcare providers, as the trusted source of information and a filter for web-based searches, play a crucial role in this landscape. Matt elaborates, "We're a specialized consulting firm for recurring revenue products and services. We build loyalty and advocacy for better lifetime value through customer experience optimization. We do this for all kinds of businesses that operate on a recurring revenue model, which includes many products and services in the health and wellness space. It's a space we've been working in for over 20 years. It's an area where we see a ton of leadership but also a ton of potential in understanding a longer-term customer journey. A true relationship, as opposed to a singular point with the transaction, actually can be a real advantage and an opportunity to set yourself apart from the competitors in the market space." "When it comes to health and wellness, we have, of course, the healthcare scenario where people have recurring relationships with their physicians, they have recurring relationships with the healthcare systems in which they operate with their insurance companies. But they also have recurring relationships with the products that they may use, like software technology or hardware technology, the news and information that they consume related to their health and wellness, as well as replenishable products like the foods, drinks, supplements, medication, all of those things can make up their portfolio of health and wellness that is a long-term relationship." #HouseofKaizen #PatientFirst #PatientExperience #HealthcareInnovation #HealthcareMarketing houseofkaizen.com Download the transcript here

Dr. Mollie Leoni, Executive Vice President for Clinical Development of Kura Oncology, discusses the company's ziftomenib program, a menin inhibitor for acute myeloid leukemia (AML). The company initially allowed all AML patients to participate in the clinical study, but later found that specific subtypes, such as those with NPM1 mutation or KMT2A rearrangement, were more likely to respond to the menin inhibitor. They also discovered other subtypes that were responsive, expanding the potential patient population. Understanding the role of menin and menin inhibitors in addressing abnormal gene expression and promoting healthy cell development opens the door to potential combination therapy, where menin inhibitors could be layered onto existing treatments for various cancers related to menin independence. Mollie explains, "In oncology, we have gotten good at treating the end state, but cancer has many, many causes with a common final endpoint to the way the cells look. So, it's many, many different diseases all at once. We've learned and are learning that with better technology and a better understanding of cancer in general we can identify not just how to stop the end state but how to stop it from starting." "That's where ziftomenib comes in. Ziftomenib addresses key mutations that when they occur, are what cause the development of cancer. So you're able to shut down the development and force the cells to develop normally rather than waiting until they're already diseased and killing them with chemotherapy or some other cytotoxic agent." "There is a protein complex that we refer to as the menin MLL complex, which goes rogue when something unusual happens in the cell. For example, that could be an NPM1 mutation. That could be a KMT2A rearrangement. Those are two things that are well-known to happen within AML diseases. It could also be a SETD2 RUNX1 mutation. So many types of mutations could happen that could cause this machinery to go rogue within the cell. And that complex causes genes to become active at levels and at times that are abnormal." #KuraOncology #Ziftomenib #MeninInhibitors #PrecisionMedicine #BTD #AML KuraOncology.com Download the transcript here

Dr. Steffen-Sebastian Bolz, Chief Scientific Officer of Swiss-based Aphaia Pharma, shines a light on the excitement around weight loss drugs due to the discovery that GLP-1 agonists and points out the shift in the perception of obesity as a root cause of various health issues. Aphaia Pharma's approach involves stimulating hormone production in the small intestine by reinvigorating the mechanism that has become dormant due to the ingestion of highly refined foods. The company is also exploring the effects of the circadian rhythm on weight loss and maintaining a healthy weight. Steffen elaborates, "What is understood now is obesity is the root cause of many sequel pathologies like type 2 diabetes, diabetes, fatty liver disease, and a whole lot of cardiovascular disease. So, the idea is that if we get obesity under control and the obesity pandemic under control, we might also lower the incidence of all the other diseases. There’s a massive rush into this field because there are already blockbuster drugs in the market, as you just said, and there are already 1 billion people worldwide who are obese and our projections predict that rapid growth of these numbers. So this is kind of the gold rush." "Let me start with briefly outlining the mode of action of GLP-1 agonists. A common denominator of all metabolic diseases, either obesity or a related disease, is a lack of hormone output from the small intestine. What’s behind this is that the small intestine is capable of producing hormones in response to food intake." "All these hormones act in an orchestrated fashion to allow digestion, proper immune control, and all these things. It was found that all these hormones are reduced in patients with obesity, and GLP-1 agonists replace one of those hormones, GLP-1. They do this by injecting very high doses to yield the effects we’re seeing, such as type 2 diabetes control and weight loss. But that’s just the hormone replacement therapy. That’s just one hormone out of a large portfolio, out of hundreds of hormones produced by the small intestine. And that is exactly where our thought process started." #AphaiaPharma #Obesity #GLP1Agonists #SmallIntestine #WeightLoss AphaiaPharma.com Download the transcript here

Rick Vanzura, CEO of Freight Farms, and Dr. Amber Orman, Chief Wellness Officer at Advent Health, discuss their partnership in bringing hyperlocal, sustainable food to healthcare facilities. Freight Farms uses modified shipping containers to grow crops, allowing for year-round production with minimal land and water usage. Advent Health recognized the importance of nutrition in whole-person care and saw the opportunity to provide their team members, physicians, patients, and the public with fresh, nutritious food and donate to local food pantries. The partnership has demonstrated the power of food as medicine and the potential for sustainable farming practices in healthcare settings. Rick explains, "We grow about two and a half to three acres worth of crops out of an 8 by 40-foot shipping container. It looks like any shipping container you would see on a dock or a boat, but it's highly modified to grow food using LED lights, airflow, nutrients delivered through the farm, and controlled through software. It's really designed so you can grow 365 days a year, very clean food, with temperatures from minus 40° to 120°, and do that consistently. And again, get all of the value of it being hyperlocal and fresh and using minimal land and water." Amber elaborates," We have a doctors’ lounge where the doctors, nurse practitioners, and some leaders eat. We also have a public-facing cafeteria, so anybody who visits the hospital can eat in the cafeteria. It's served in there. It's served in our patient meals, so we have certain meals containing the produce, so all of our salads, sandwiches, and wraps. Then we're also supplying anything extra to a local food pantry, and then they also donate to Kingdom Kids Academy in Kissimmee. We are able to sustain all of these things and donate some extra when we have it, even out to the community. It certainly provides better nutrition within a given day to those patients and team members and the people who receive the donations. It's also a way to start educating people about the benefits of eating more plants. We all need to eat more plants for better health, and so that's one of our guiding missions here." #AdventHealth #FreightFarms #VerticalFarming #Healthcare #FoodAsMedicine #AgTech adventhealth.com freightfarms.com Download the transcript here

Guang Qu, Co-Founder of NGGT Corporation is focused on developing gene therapy products using dual functional vector strategies. Guang highlights the importance of distinguishing between recessive and dominant mutations in rare diseases and explains how their approach differs from other gene therapy approaches. The two leading indications caused by a gene mutation that NGGT is working on are Bietti's Crystalline Dystrophy (BCD), a rare ocular disease, and phenylketonuria (PKU), a metabolic disease. Guang explains, "For our strategies and our product development strategies, I think we are leveraging our experience in the gene therapy field. Later, I will talk more about myself, Dr. Lixin Jiang, and our team. The other thing we are leveraging is our fully integrated team in gene therapy product development, which involves R&D, research and development, CGMP manufacturers, from tox development and clinical regulatory functions and the medical teams. So with all of the teams we built up in the last couple of years, we're in very good positions in developing our gene therapy products." "So, of the two currently leading indications, one is involved in ocular disease, what we call the BCD, Bietti's Crystalline Dystrophy. This disease is caused by a gene mutation. The gene mutation causes lipid metabolic problems. The particular gene is called the CYP4V2 gene. The mutation directly leads to the lipid metabolic process being interrupted. Therefore, lipids are precipitated in the different ocular cells, such as the cornea and the retina. That's most of the disease cell layers affected." #AAV #GeneEditing #GeneTherapy #PKU #BCD Download the transcript here

Jennifer Jones-McMeans, divisional vice president of global clinical affairs at Abbott’s vascular business, and Dr. Brian DeRubertis, a doctor at the New York-Presbyterian Weill Cornell Medical Center, shine a light on peripheral artery disease and critical limb-threatening ischemia where artery blockage restricts blood flow to the lower legs. Abbott has developed the Esprit, a dissolving scaffold that opens up the artery in the blocked area and elutes a drug that slows disease progression. Their LIFE-BTK trial demonstrated success in a diverse patient population and is a significant new treatment option for CLTI. Brian explains, "One of the things that I’ll say is our group is starting to explore doing additional efforts at patient education with our own patients, patients who have actually been diagnosed with this disease, and educating them a little bit more about what it means to have this disease, how they can be involved in the process of trying to prevent their disease from getting worse. And the reason we’re doing this is exactly what you said. Everyone knows what a heart attack is, but very few people understand that the same process that leads to clogged arteries in the heart or blocked arteries in the heart can lead to blocked arteries in the legs. Those blockages can ultimately lead to amputation. And so, the education of patients and keeping them informed about the disease process is one important part of this." Jennifer elaborates, "This is really a landmark moment for people with this severe form of peripheral artery disease, which we’re calling CLTI. When you think of these minimally invasive procedures to treat this disease, currently in the United States, you only have plain balloon angioplasty. And this is what the LIFE BTK trial was measured against. The Esprit was measured, which is our dissolving stent, and tested against the standard of care, plain balloon angioplasty. And it’s been decades since there’s been any innovation in this, what they sometimes call the endovascular first space. The Esprit is made out of a polymer material similar to what you would think about with dissolving stitches, and it also has a drug on it." #Abbott #CLTI #PeripheralArteryDisease #PAD #LegArteries #PlainBalloonAngioplasty #DiversityinClinicalTrials abbott.com Download the transcript here