Drug Targets Iron Dysregulation in Rare Neurodegenerative Disease Multiple System Atrophy with David Stamler Alterity Therapeutics TRANSCRIPT

Jean-Philippe Vert, the Co-Founder and CEO of Bioptimus, is building a foundational AI model for biology to solve the problem of siloed biomedical research. Key goals are to bridge the translational gaps between drug discovery and development, and between clinical research and real-world patient outcomes, and to redesign clinical trials for greater efficiency and improved results. Creating digital twins of patients is a way to simulate treatment outcomes and create synthetic control arms for clinical trials, ultimately lowering the risk and cost of drug development and enabling the creation of new medicines for a broader range of conditions, including rare diseases. Jean-Philippe explains, "So at its core, what we try to build at Bioptimus is the foundational AI infrastructure for biology. The problem we're trying to solve is that biology is complex and operates across different scales, from genes and proteins to cells, organs, patients, etc. And historically, lots of research, lots of biological, biomedical research has been very siloed, has been focusing on specific aspects of biology, like studying only genes or studying only cells. What we are building at Bioptimus is an AI-intelligent system that can see across all these layers, all of these cases, to get a holistic picture of biology. And it's not only a scientific endeavor, but the reason why it's hard to make a drug today, why so many diseases remain untreated, is that the siloed nature of biomedical research has created difficulties in how we move from research in discovery, like understanding a disease, to making a treatment for the patients."   "So we have indeed a model called H-Optimus, which is a foundation model for one type of modality, which is one thing you see in an image. It's for histopathology slides. This is when someone has, for example, a cancer, you take a biopsy and then typically a pathologist looks at the biopsy under the microscope to characterize the disease, to see if there are cancer cells, to see the shape, to see the organization, and so to pose a diagnostic and suggest a treatment. We have trained an AI system that helps pathologists be better because our systems have been trained by looking at billions of such images, and so have a very detailed understanding of the subtle variations that can be observed in images."  #Bioptimus #ArtificialIntelligence #DrugDiscovery #Biotechnology #PrecisionMedicine #FoundationModels #BiologyAI #ClinicalTrials #CancerResearch #RareDiseases #DigitalHealth #Innovation bioptimus.com Listen to the podcast here

Jean-Philippe Vert, the Co-Founder and CEO of Bioptimus, is building a foundational AI model for biology to solve the problem of siloed biomedical research. Key goals are to bridge the translational gaps between drug discovery and development, and between clinical research and real-world patient outcomes, and to redesign clinical trials for greater efficiency and improved results. Creating digital twins of patients is a way to simulate treatment outcomes and create synthetic control arms for clinical trials, ultimately lowering the risk and cost of drug development and enabling the creation of new medicines for a broader range of conditions, including rare diseases. Jean-Philippe explains, "So at its core, what we try to build at Bioptimus is the foundational AI infrastructure for biology. The problem we're trying to solve is that biology is complex and operates across different scales, from genes and proteins to cells, organs, patients, etc. And historically, lots of research, lots of biological, biomedical research has been very siloed, has been focusing on specific aspects of biology, like studying only genes or studying only cells. What we are building at Bioptimus is an AI-intelligent system that can see across all these layers, all of these cases, to get a holistic picture of biology. And it's not only a scientific endeavor, but the reason why it's hard to make a drug today, why so many diseases remain untreated, is that the siloed nature of biomedical research has created difficulties in how we move from research in discovery, like understanding a disease, to making a treatment for the patients."   "So we have indeed a model called H-Optimus, which is a foundation model for one type of modality, which is one thing you see in an image. It's for histopathology slides. This is when someone has, for example, a cancer, you take a biopsy and then typically a pathologist looks at the biopsy under the microscope to characterize the disease, to see if there are cancer cells, to see the shape, to see the organization, and so to pose a diagnostic and suggest a treatment. We have trained an AI system that helps pathologists be better because our systems have been trained by looking at billions of such images, and so have a very detailed understanding of the subtle variations that can be observed in images."  #Bioptimus #ArtificialIntelligence #DrugDiscovery #Biotechnology #PrecisionMedicine #FoundationModels #BiologyAI #ClinicalTrials #CancerResearch #RareDiseases #DigitalHealth #Innovation bioptimus.com Download the transcript here

David Stamler, CEO of Alterity Therapeutics, is developing a drug to treat multiple system atrophy (MSA), a rare and rapidly progressing neurodegenerative disease that often presents as Parkinson's disease but is distinct and more aggressive. There is no single genetic cause or specific biomarker, making accurate diagnosis a significant challenge. The lead drug is a novel small molecule designed to manage excess reactive iron in the brain, which drives the disease, and may be effective for other neurodegenerative diseases involving iron dysregulation. David explains, "Multiple system atrophy is a rare disease, and that's part of the reason people may not know so much about it. It is a neurodegenerative disease, and as the name implies, there are multiple regions of the brain that are affected, hence the term multiple systems that are governed by those regions of the brain. And as the disease progresses, some of these regions degenerate, and you get abnormal function in various areas."   "Now, we like to characterize the disease as a Parkinsonian disorder, which means early on, it can look like Parkinson's disease. And that's kind of a good descriptor to help people understand what it might look like, but it's distinct from Parkinson's disease, and it progresses a lot faster, a lot more rapidly. So it's a disease that people don't know about, probably because no one famous has been diagnosed with MSA, although I'm sure various famous people have probably had the disease and maybe didn't know it."  #AlterityTherapeutics #MultipleSystemAtrophy #MSAAwareness #NeurodegenerativeDisease #Biotech #Phase3 #Neurology #MSA #ClinicalTrials #AlterityTherapeutics #ATH434 #Biotech #RareDisease #Neurodegeneration #DrugDevelopment #MedicalBreakthrough #IronChaperone Alteritytx.com Listen to the podcast here

David Stamler, CEO of Alterity Therapeutics, is developing a drug to treat multiple system atrophy (MSA), a rare and rapidly progressing neurodegenerative disease that often presents as Parkinson's disease but is distinct and more aggressive. There is no single genetic cause or specific biomarker, making accurate diagnosis a significant challenge. The lead drug is a novel small molecule designed to manage excess reactive iron in the brain, which drives the disease, and may be effective for other neurodegenerative diseases involving iron dysregulation. David explains, "Multiple system atrophy is a rare disease, and that's part of the reason people may not know so much about it. It is a neurodegenerative disease, and as the name implies, there are multiple regions of the brain that are affected, hence the term multiple systems that are governed by those regions of the brain. And as the disease progresses, some of these regions degenerate, and you get abnormal function in various areas."   "Now, we like to characterize the disease as a Parkinsonian disorder, which means early on, it can look like Parkinson's disease. And that's kind of a good descriptor to help people understand what it might look like, but it's distinct from Parkinson's disease, and it progresses a lot faster, a lot more rapidly. So it's a disease that people don't know about, probably because no one famous has been diagnosed with MSA, although I'm sure various famous people have probably had the disease and maybe didn't know it."  #AlterityTherapeutics #MultipleSystemAtrophy #MSAAwareness #NeurodegenerativeDisease #Biotech #Phase3 #Neurology #MSA #ClinicalTrials #AlterityTherapeutics #ATH434 #Biotech #RareDisease #Neurodegeneration #DrugDevelopment #MedicalBreakthrough #IronChaperone Alteritytx.com Download the transcript here  

Greg Mayes, President and CEO of Reunion Neuroscience, and Dr. Manish Agrawal, the Co-Founder and CEO of Sunstone Therapies, shine a light on adjustment disorder, a disproportionate emotional or behavioral reaction to a significant life stressor such as a cancer diagnosis. This under-recognized condition lacks FDA-approved treatments and is often managed with SSRIs or talk therapy.  The REKINDLE study evaluated the use of a psilocybin analog drug in development for treating adjustment disorder and has shown positive results by integrating emotional treatment into the standard of care for serious illnesses. Greg explains, "In fact, there are no approved investigational assets that had been approved by the FDA for adjustment disorder. But it was an area of exploration that the FDA encouraged us to move into in light of people's disproportionate and really unfortunate reactions in terms of a depressive or anxiolytic reaction to cancer diagnosis or other major medical illness like Parkinson's disease, MS, ALS, or pulmonary fibrosis."   Manish elaborates, "Well, usually you have to have had some stress-related event. For example, it could be things like divorce or job loss. But here, specifically, this study is targeting an illness, and the ones that Greg had just listed out, the neurologic conditions, as well as the cancer diagnosis. And then usually it leads to symptoms, intense feelings of sadness or anxiety or hopelessness. You can have a depressed mood with its subtypes with depressed mood. Some people have more anxiety. And so both a stressor and the symptoms that present are associated directly with that. So, for example, for the study, the symptoms of sadness need to be tied to the illness, such as cancer or MS." #ReunionNeuroscience #SunstoneTherapies #AdjustmentDisorder #MentalHealth #REKINDLEStudy #Psychedelics  #PsychedelicMedicine #CancerCare #MentalHealth #ClinicalTrials #Psilocybin #Oncology #PatientCare #MedicalInnovation #Neuroscience reunionneuro.com sunstonetherapies.com Listen to the podcast here  

Greg Mayes, President and CEO of Reunion Neuroscience, and Dr. Manish Agrawal, the Co-Founder and CEO of Sunstone Therapies, shine a light on adjustment disorder, a disproportionate emotional or behavioral reaction to a significant life stressor such as a cancer diagnosis. This under-recognized condition lacks FDA-approved treatments and is often managed with SSRIs or talk therapy.  The REKINDLE study evaluated the use of a psilocybin analog drug in development for treating adjustment disorder and has shown positive results by integrating emotional treatment into the standard of care for serious illnesses. Greg explains, "In fact, there are no approved investigational assets that had been approved by the FDA for adjustment disorder. But it was an area of exploration that the FDA encouraged us to move into in light of people's disproportionate and really unfortunate reactions in terms of a depressive or anxiolytic reaction to cancer diagnosis or other major medical illness like Parkinson's disease, MS, ALS, or pulmonary fibrosis."   Manish elaborates, "Well, usually you have to have had some stress-related event. For example, it could be things like divorce or job loss. But here, specifically, this study is targeting an illness, and the ones that Greg had just listed out, the neurologic conditions, as well as the cancer diagnosis. And then usually it leads to symptoms, intense feelings of sadness or anxiety or hopelessness. You can have a depressed mood with its subtypes with depressed mood. Some people have more anxiety. And so both a stressor and the symptoms that present are associated directly with that. So, for example, for the study, the symptoms of sadness need to be tied to the illness, such as cancer or MS." #ReunionNeuroscience #SunstoneTherapies #AdjustmentDisorder #MentalHealth #REKINDLEStudy #Psychedelics  #PsychedelicMedicine #CancerCare #MentalHealth #ClinicalTrials #Psilocybin #Oncology #PatientCare #MedicalInnovation #Neuroscience reunionneuro.com sunstonetherapies.com Download the transcript here  

Russ Nix, Consulting Associate Director, and Dr. Stacey McCoy, Pharmacy Clinical Program Manager for the Clinical Surveillance and Compliance business at Wolters Kluwer Health, highlight the problem of drug diversion in healthcare environments and the shared responsibility to prevent this breach. AI-enabled software is becoming crucial in detecting suspicious patterns, the types of individuals most likely to steal drugs, and gaps in the supply chain from ordering to delivering drugs to the patient. While opioids are the most commonly diverted drugs, motivated by substance abuse and addiction, other medications, including non-controlled substances, insulin, and high-cost cancer drugs, are also at risk. Effective prevention programs focus on a culture in which staff feel safe reporting concerns and seeking help. Russ explains, "Drug diversion is basically when you're in a healthcare system where the medications in that facility are not going to their intended destination. And that's typically what we see most of, a deliberate taking of those medications, whether it was a substance abuse issue or your healthcare practitioners or staff outside of the facility, are taking those medications and basically denying your patients that medication. And it is a pretty significant issue since the opioid crisis, again in the early 2000s or late 1990s."   Stacey elaborates, "So ideally, we want to be in a position where we're able to utilize software applications or a mixture of software applications to have oversight of what's being ordered, what's coming in, and what's going on inside our pharmacies, what's inside the machines on each hospital floor. Just imagine you have such a varied audience. Every single hospital floor has 15 or so nurses working. Those nurses need to grab medications from machines, like a vending machine. So the pharmacist is responsible for making sure that's taking place properly." "Then that same team or person was also responsible for making sure that what's removed from the machines truly makes it to the patients in a safe and sound manner. So there are a number of breakpoints within the process that someone has to oversee. Ideally, we'd like to make sure that drug diversion prevention takes place using the most up-to-date software applications that are AI-enabled, and that we have multidisciplinary governance on these teams." #WoltersKluwerHealh #AISurveillance #DrugDiversionPrevention #Sentri7DrugDiverison #PatientSafety #DrugDiversion #HealthcareSecurity #OpioidCrisis #PharmacySafety #HealthcareCompliance #PatientCare #MedicationSafety #HealthcareLeadership #AIinHealthcare wolterskluwer.com Listen to the podcast here

Russ Nix, Consulting Associate Director, and Dr. Stacey McCoy, Pharmacy Clinical Program Manager for the Clinical Surveillance and Compliance business at Wolters Kluwer Health, highlight the problem of drug diversion in healthcare environments and the shared responsibility to prevent this breach. AI-enabled software is becoming crucial in detecting suspicious patterns, the types of individuals most likely to steal drugs, and gaps in the supply chain from ordering to delivering drugs to the patient. While opioids are the most commonly diverted drugs, motivated by substance abuse and addiction, other medications, including non-controlled substances, insulin, and high-cost cancer drugs, are also at risk. Effective prevention programs focus on a culture in which staff feel safe reporting concerns and seeking help. Russ explains, "Drug diversion is basically when you're in a healthcare system where the medications in that facility are not going to their intended destination. And that's typically what we see most of, a deliberate taking of those medications, whether it was a substance abuse issue or your healthcare practitioners or staff outside of the facility, are taking those medications and basically denying your patients that medication. And it is a pretty significant issue since the opioid crisis, again in the early 2000s or late 1990s."   Stacey elaborates, "So ideally, we want to be in a position where we're able to utilize software applications or a mixture of software applications to have oversight of what's being ordered, what's coming in, and what's going on inside our pharmacies, what's inside the machines on each hospital floor. Just imagine you have such a varied audience. Every single hospital floor has 15 or so nurses working. Those nurses need to grab medications from machines, like a vending machine. So the pharmacist is responsible for making sure that's taking place properly." "Then that same team or person was also responsible for making sure that what's removed from the machines truly makes it to the patients in a safe and sound manner. So there are a number of breakpoints within the process that someone has to oversee. Ideally, we'd like to make sure that drug diversion prevention takes place using the most up-to-date software applications that are AI-enabled, and that we have multidisciplinary governance on these teams." #WoltersKluwerHealh #AISurveillance #DrugDiversionPrevention #Sentri7DrugDiverison #PatientSafety #DrugDiversion #HealthcareSecurity #OpioidCrisis #PharmacySafety #HealthcareCompliance #PatientCare #MedicationSafety #HealthcareLeadership #AIinHealthcare wolterskluwer.com Download the transcript here

Meghan Gutierrez, CEO of the Lymphoma Research Foundation, and Jennifer Branstetter, the Executive Director, North America Corporate Affairs at BeOne Medicines, join me to discuss the growing use of AI in patient care with a specific focus on individuals with lymphoma and other blood cancers. Emphasis is on the necessity of a collaborative, patient-centric approach to developing AI tools, bringing together patient advocacy organizations, healthcare professionals, and pharma partners to provide accurate information to patients. The goal is to serve a diverse population by providing personalized, accessible information to help patients have more meaningful discussions with their healthcare providers, not to replace the clinician's role. Jen explains, "We know that tens of millions of people are using AI tools in general, like ChatGPT, for health questions, including patients with blood cancers like chronic lymphocytic leukemia or CLL, they're all turning to the different AI tools that are out there. And from our perspective, we see this continuous growth of use, we want to make sure that the right healthcare information is getting fed into those AI resources and tools so that patients are getting the most accurate information possible."   Meg elaborates, "Many patients are using these AI tools to make sense of everything from their PET scans to their blood results, to some of the treatment language they receive in their clinician's or doctor's office. And one of the things that I heard recently from a cancer patient who had uploaded his scans and some test results into an AI platform. And he called me incredibly concerned. He was very concerned about his prognosis and the limited treatment options that he believed were available to him." "So when I pressed further, I found out that this AI platform was the only source of information that he had. So I became concerned when I recognized that the information he received was antiquated. So the treatment results and some of the information he was working from in this incredibly anxious and anxiety-provoking moment were 10 years old. And so in this case, antiquated information was as dangerous as inaccurate information. And both of these remain top concerns at the Lymphoma Research Foundation. And I think across our sector, as we see more and more patients using these tools and platforms to help educate them about their disease and their treatment options." #LymphomaResearchFoundation #LRF #BeOneMedicines #LymphomaAwareness #AIinHealthcare #PatientAdvocacy #BloodCancer #HealthcareInnovation #PatientEmpowerment #PrecisionMedicine #CLL #HealthTech #CancerCare lymphoma.org BeOneMedicines.com Listen to the podcast here

Meghan Gutierrez, CEO of the Lymphoma Research Foundation, and Jennifer Branstetter, the Executive Director, North America Corporate Affairs at BeOne Medicines, join me to discuss the growing use of AI in patient care with a specific focus on individuals with lymphoma and other blood cancers. Emphasis is on the necessity of a collaborative, patient-centric approach to developing AI tools, bringing together patient advocacy organizations, healthcare professionals, and pharma partners to provide accurate information to patients. The goal is to serve a diverse population by providing personalized, accessible information to help patients have more meaningful discussions with their healthcare providers, not to replace the clinician's role. Jen explains, "We know that tens of millions of people are using AI tools in general, like ChatGPT, for health questions, including patients with blood cancers like chronic lymphocytic leukemia or CLL, they're all turning to the different AI tools that are out there. And from our perspective, we see this continuous growth of use, we want to make sure that the right healthcare information is getting fed into those AI resources and tools so that patients are getting the most accurate information possible."   Meg elaborates, "Many patients are using these AI tools to make sense of everything from their PET scans to their blood results, to some of the treatment language they receive in their clinician's or doctor's office. And one of the things that I heard recently from a cancer patient who had uploaded his scans and some test results into an AI platform. And he called me incredibly concerned. He was very concerned about his prognosis and the limited treatment options that he believed were available to him." "So when I pressed further, I found out that this AI platform was the only source of information that he had. So I became concerned when I recognized that the information he received was antiquated. So the treatment results and some of the information he was working from in this incredibly anxious and anxiety-provoking moment were 10 years old. And so in this case, antiquated information was as dangerous as inaccurate information. And both of these remain top concerns at the Lymphoma Research Foundation. And I think across our sector, as we see more and more patients using these tools and platforms to help educate them about their disease and their treatment options." #LymphomaResearchFoundation #LRF #BeOneMedicines #LymphomaAwareness #AIinHealthcare #PatientAdvocacy #BloodCancer #HealthcareInnovation #PatientEmpowerment #PrecisionMedicine #CLL #HealthTech #CancerCare lymphoma.org BeOneMedicines.com Download the transcript here