Empowered Patient Podcast

• Strategy of the Global Leader in Rare Disease Drugs with Scott Pescatore Recordati

  Scott Pescatore, Executive VP of the Rare Diseases Business at Recordati, is focused on rare and ultra-orphan diseases with high unmet needs by acquiring promising compounds from other companies and advancing them through development and approval. An example is the company's drug Isturisa, acquired from Novartis, which is an FDA-approved effective treatment for patients with Cushing syndrome, a rare endocrine condition. Raising awareness of rare diseases among physicians, patients, and the general public is a priority for Recordati to improve diagnosis rates and clinical trial participation, and to encourage more research and funding in the rare disease space. Scott explains, "We have two primary divisions at Recordati. One is our specialty primary care business, and the other is the rare disease business, which I have the honor and privilege to look after. And we have a very simple sort of work ethic or business mantra, if you will, and that's focused on the few. And we really dedicate ourselves to focusing on disease areas and patient groups and therapeutic areas that have a high unmet need and really low or limited options for patients. And really focusing on diseases and areas that are rare and considered ultra-orphan by the definitions in the US, where really there's a very small patient base. And that's where we began back in 2007, when the rare disease business was formed. And that's really what our focus has been since then. And we continue to focus on this segment of the market." "So Isturisa is really a fantastic product. We acquired this product through a deal we did with Novartis Pharmaceuticals back in 2019, and this product has FDA approval for patients who have endogenous hypercortisolemia with Cushing syndrome. So it's quite a nasty disease, but it's a very efficacious product. The product is what's considered a cortisol inhibitor in the blocks in a particular enzyme to help normalize hypercortisolemia in patients with Cushing syndrome. And Cushing syndrome, for those who aren't familiar, is a rare endocrine condition that really has a significant impact on patients' quality of life, on the caregivers, on the families. And the indication I mentioned was supported by quite robust phase 3 trials."  #Recordati #RareDisease #FocusedontheFew #CushingSyndrome #IMCD #CastlemansDisease recordati.com Download the transcript here  

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• Scanner Detects Pressure Injuries Before Visual Symptoms with Martin Burns Bruin Biometrics

  Martin Burns, CEO of Bruin Biometrics, is focused on preventing pressure injuries, which are often not detected through visual and tactile assessments because damage starts invisibly under the skin. The Bruin scanner technology identifies pressure injuries by measuring sub-epidermal moisture, which can predict later tissue death, highlighting the condition that can be treated before it develops further. This handheld device is skin tone agnostic, addressing a significant health disparity and providing an objective number indicating the presence of excess fluid, allowing clinicians to act quickly. Martin explains, "They are surprisingly common, and I emphasize surprisingly because pressure injuries are typically not talked about in the mainstream, but actually they affect about three to three and a half million patients a year in the US. And really shockingly, of those three to three and a half million, about 60,000 people die from them every year. So think about that as in the list of the top 10 leading causes of mortality in the United States, which is the surprising part. When you speak to friends and relatives, they've often heard of them potentially as bedsores, but nobody really has an appreciation of just how significant they are. How widespread or how deadly."   "The initial stages of it are imperceptible to any practitioner, but are actually measurable by objective technologies, and they don't become visibly manifest or physically manifest until much later. And what happens is they end up breaking the skin surface. As you can imagine, every time the skin is broken is an opening for infections and significant complications, which actually is the thing that ends up causing huge amounts of additional lengths of stay and costly treatments. And to the extent that those don't work, mortality." "Thankfully, our scanner is one in which the mechanism it uses disregards skin tone entirely. In other words, skin color doesn't matter for the scanner. What we are measuring is an increase or a decrease in fluid at the specific site that we're measuring. And so it's skin tone agnostic, which is rather brilliant because dark skin tone patients die at a rate four times more than any other cohort, which is an absolute travesty and one in which it simply doesn't need to happen. And our scanner is a leading reason why it doesn't need to happen." #BruinBiometrics #Prevention #PressureInjuries #HospitalSafety #PressureInjuryPrevention #PatientSafety #Hospitals bruinbiometrics.com Download the transcript here

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• Targeting Elevated Cortisol Seen as a Hidden Driver of Treatment-Resistant Type 2 Diabetes with Robert Jacks Sparrow Pharmaceuticals

  Robert Jacks, President and CEO of Sparrow Pharmaceuticals, identifies that an elevated cortisol level is a newly recognized cause of treatment-resistant type 2 diabetes. A significant portion of patients with diabetes who do not respond to standard treatments, including GLP-1 agonists, have underlying high cortisol. Sparrow has developed a drug designed to lower cortisol levels inside cells, directly addressing the underlying driver of the disease, and to be used as a complement to existing treatments. This concept of targeting cortisol-driven resistance could be extended to other conditions, such as treatment-resistant hypertension. Robert explains, "I feel as though Sparrow has come full circle, actually, with the mechanism of our drug. Originally, we have a drug that targets HSD-1. We can talk about what that is, but it's involved in intracellular cortisol regulation. This was a class of drugs that was originally developed targeting cardiometabolic diseases like type 2 diabetes. And the drugs had some moderate efficacy, but they weren't well differentiated in a broad population and largely were just discontinued for commercial reasons." "Our company was founded a number of years ago based on the idea that these drugs had real potential but hadn't been used in the right patient population. And that being the patient population with the disease that we know is driven by excess cortisol toxicity, because that's aligned with the mechanism, as I was mentioning. So we generated some really interesting data in a rare disease called Endogenous Cushing syndrome. This is a very severe orphan disease with patients who have very severely elevated cortisol, showing in fact that yes, this mechanism does seem like it could have a very major impact in the right patient population." "Simultaneously, another company published some data showing that actually there's a very large population of people with treatment-resistant type 2 diabetes, a very high level of medical need, and that their underlying disease actually is being driven by elevated levels of cortisol. And so when you bring together the data that we generated and what appears to be a large amount needed in a large population, it seems like we may have the perfect solution for that. So we've refocused our efforts on a broad population of treatment-resistant type 2 diabetes in patients whose disease is being impacted or driven by elevated cortisol levels."  #SparrowPharmaceuticals #Type2Diabetes #CardiometabolicDisease #CortisolRegulation #Cortisol #GLP1 #RareDisease sparrowpharma.com Download the transcript here

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• Focus on Health Span Rather Than Lifespan Using AI to Cut Costs Expand Access to Therapies with Jesse Levey Longevity Health

  Jesse Levey, CEO and Founder of Longevity Health, has a focus on extending the health span not just the lifespan of more people by providing access to tools and preventative health information. Lessons learned from expensive concierge medical services are being applied to a broader population, driving down health costs and democratizing longevity medicine. Using AI to build a scalable and personalized approach to wellness not just sick care, Longevity Health aims to make an AI doctor available around the globe.  Jesse explains, "Our mission is to help a billion people live to a hundred in good health. That's the vision. And the way that we get there is by building an AI doctor trains of ed on longevity medicine and distributed around the world. We have a three-phased business model. Phase one is to take the sort of hundred-thousand-dollar-a-year longevity concierge experience and deliver it for $10,000. So we've taken this really high-end experience that combines an executive physical with a longevity concierge physician and a team to help you implement the recommendations. And we've delivered that for $10,000. That's been around for about two years."  "Phase two takes that down to $1,000 with the help of AI. So it reduces the time spent by humans and the need to spend a lot of time with clinicians. And so it's a mix between clinician time and AI interactions. And then eventually, as the regulation allows and as the technology improves, we believe that we'll be able to deliver this experience for as low as $10 a month or $100 a year via an AI doctor. And so that's the future. That's what we're building towards." #LongevityHealth #Longevity #HealthyAging #HealthSpan #FunctionalMedicine #HealthAI #Aging longevityhealth.me Download the transcript here

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• Clinical AI Drives Early Detection of Undiagnosed Diseases with Sean Cassidy Lucem Health

  Sean Cassidy, CEO and Co-Founder of Lucem Health, is applying AI to identify patients at high risk of undiagnosed conditions like cancer and diabetes.  The goal is to facilitate earlier diagnosis and treatment by flagging patients that need screening based on the AI plaform's ability to analyze EHR data and demographics of diverse patient populations to ensure broad scalability. This technology was designed to integrate into existing clinical workflows for established screening procedures rather than making direct treatment recommendations.  Sean explains, "The origin of the company, the idea for the company, originated within Mayo Clinic in about 2020. Mayo Clinic has faced a challenge, and I think sometimes continues to face a challenge that a lot of researchers in AI have faced, which is how do you get promising AI in a clinical context from the so-called bench to the bedside? How do you get it from the lab into clinical practice? And what they realized was that while the data science and the AI part of it is really interesting, what was needed was scaffolding around the AI to facilitate integration with data and integration with workflows, a measurement and monitoring system, and so on and so forth." "We are trying to facilitate, and you're going to see us begin to expand the aperture, if you like, or open the aperture of how we position the company. Because as we've gone on, we have realized that the opportunity here is to actually help healthcare provider organizations, health systems, and so on, create really high-impact care delivery programs that have at their core or feature at their core earlier diagnosis, accelerated treatment, earlier treatment and therefore better outcomes for patients and hopefully even saved lives. So that's the generic approach that we take."  #LucemHealth #AIinHealthcare #HealthcareAI  #HealthTech #EarlyDiseaseDetection lucemhealth.com Download the transcript here

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