Empowered Patient Podcast

Dr. Charlotte McKee, Chief Medical Officer at Sionna, describes the nature of cystic fibrosis (CF, a genetic disease caused by a mutation in the single CFTR gene. While current CFTR modulator therapies do not address the most common mutation, Sionna's novel oral medicine is designed to target the previously undruggable NBDI domain of the CFTR protein. This new therapy aims to lead to better lung function and prevent the accumulation of permanent damage to other organs like the pancreas, gut, and liver.  Charlotte explains, "Cystic fibrosis is a genetic disease. The gene was actually discovered in 1989 for cystic fibrosis. It's considered a rare disease, but it's a relatively large rare disease. And one of those rare diseases that is potentially fatal, as you mentioned, it's thought of as a lung disease. And most patients, if their life is shortened, it's typically because of lung disease, because the lungs can be very severely affected. But the protein is caused by a genetic mutation in a gene called CFTR, a

Mark Bertagnolli, Chief Operating Officer at ViroMissile, has developed a modified vaccinia virus that can be delivered intravenously to seek out and destroy solid tumors throughout the body and is not dependent on a specific genetic driver of a cancer. The virus has been engineered to be resistant to the body's complement system, allowing it to survive in the bloodstream longer to infect the tumor. This action also turns the tumor microenvironment from cold to hot, signaling the immune system to attack the tumor and potentially working in combination with other treatments like PD-1 inhibitors to make them more effective. Mark explains, "ViroMissile has a modified vaccinia virus, and viruses tend to like to infect tumor cells. So the trick has been how to harness this. And we're on our third generation of virus, and we have a virus that you can inject intravenously that searches for tumors throughout the body, infects them, the body clears the rest of the virus, and the virus embeds itself in the tumors, rep

Dr. Ronald Lane, Founder of HOPE-Neuron Therapeutx, is developing an approach to treating ALS by rebalancing the body's immune system, which becomes dysfunctional in neurodegenerative diseases. The mechanism involves treating a patient's blood outside the body to activate the immune system and then infusing it back into the patient. The theory is that traditional drugs often fail because they target static genetic mutations and symptoms, while the source of the disease lies in the ongoing immune imbalance.  Ronald explains, "Well, in a broad sense, the goal of HOPE Neuron is based on the fact that our challenge here is to bring hope. And we use a neuronal basis to do that. We're not about drugs. There are no chemicals involved. We don't go that direction. But basically, it cuts across. We deal with memory, we deal with dementia and Alzheimer's, but our target initially is ALS."   "As I mentioned a moment ago, we're not doing drugs, we're not using chemicals. So what are you doing? Well, we're using the cell'

Dr. Galia Schwarz, CEO and Co-Founder of C8 Health, has developed a platform that addresses the gap between hospital best practices and their implementation across all departments. This approach consolidates medical knowledge from numerous disconnected systems and uses AI to proactively deliver relevant information to a clinician. C8 Health does not create best practices; it ensures that a hospital's vetted standards are communicated consistently to provide optimal patient care. Galia explains, "Hospitals are struggling to implement their best practices effectively, or in other words, control the quality and cost of care delivery. And let me explain this. There is tons of medical knowledge out there, and now with AI coming into our lives, there is even more. And hospitals create this knowledge through clinical trials, or they license this knowledge through platforms like Open Evidence or UpToDate. And every hospital takes these global best practices and turns them into local best practices, things like proto

David Klein, a longtime healthcare innovator and Founder and CEO of Click Therapeutics, is a pioneer in the field of prescription digital therapeutics, PDT, which are defined as software as medicine. Click has received FDA authorization for an app to treat depression and migraine and had a successful pivotal trial for an app treating negative symptoms of schizophrenia. These digital therapeutics are designed to fill significant gaps in current care and there are opportunities to combine software with drugs and biometric data from wearables to determine personalized treatment plans. David explains, "Click's approach towards digital therapeutics is, I would say, probably the most rigorous from a clinical evidence perspective in the industry. So we really take a drug-like clinical approach to these programs where we discover and then validate these programs in multiple randomized control trials, all culminating usually in a pivotal trial. And on top of that, you'll often see in some other areas of the world or

Tony Baldassarre, President and CEO of UniDoc Health, has a mission to provide accessible healthcare through remote technology and a Health Cube that can be deployed in a wide range of settings. This approach integrates into existing EMR/EHR platforms and includes a healthcare professional who works with the patient in the Cube to interact with medical devices and consult remotely with a doctor. There have been successful deployments in Ukraine, Italy, and Alaska, showing a significant reduction in emergency room visits and demonstrating capabilities for specialized care and management of chronic conditions.  Tony explains, "With the UniDoc solution, what we do is not just enable a conversation between a doctor and patient - it is not just a phone or video call. We actually provide real diagnostic medical devices for doctors to conduct a comprehensive medical visit remotely, just as if the patient were in their office. The important thing is that number one, we only use real medical devices. These are medical

Dr. Jennifer Perusini, CEO and Founder of Neurovation Labs, is developing a precision medicine platform to create targeted therapeutics and diagnostic tools that treat specific brain regions to address central nervous system diseases. For the treatment of PTSD, the approach focuses on the overactive amygdala due to dysregulated glutamate signaling. This objective, brain-based approach of treating a discrete part of the brain rather than the whole brain moves mental health treatment beyond the current one-size-fits-all approach, reducing side effects and improving outcomes. Jennifer explains, "What we are doing is really taking an objective, precision approach to treating mental health disorders, something like an oncology model. So what we really care about is that mental health disorders and psychiatric disorders are rooted in brain dysfunction, yes, but critically that dysfunction occurs in very discreet brain regions and circuits, not necessarily uniformly across the entire brain. So we have really develop

Dr. Paul Testa, Chief Health Informatics Officer who helped create the About Me Initiative at NYU Langone Health, and Dr. Katherine Hochman, Director of Hospital Medicine at NYU Langone Health, who envisioned the initiative, discuss the benefits of encouraging patients to provide non-medical information. The goal is to help care teams see patients as whole people beyond their diagnoses and to give patients an opportunity to share interests, hobbies, and fun facts that can help build trust and meaningful human connections. This About Me Initiative is being expanded from inpatient care to outpatient settings, such as infusion centers. Paul explains, "The About Me section in our EHR Epic is something that NYU Langone developed with the inspiration of Dr. Hochman, and it's essentially a dedicated discreet space in the medical record to allow patients to declare themselves, allowing them to give them a space that is transparent to all the users of the electronic health record, which are many and vast in their rul

Dr. David Kirk, Chief Medical Officer at Regard, discusses the role and benefits of real-time clinical AI to help clinicians navigate patient charts and find critical information to prevent medication management errors and misdiagnoses. The Regard platform can also generate proactive documentation, allowing physicians to complete their documentation more accurately and efficiently, reducing note-bloat and the need for after-hours charting. One key goal of using AI is to free physicians to spend more time on the art of medicine, directly working with patients.   David explains, "So, Regard with real-time clinical artificial intelligence software, is helping clinicians propose the right diagnosis and information while they're working in the chart. Our mission is to make patient care as clear as possible. At the same time, we make sure that the doctors are getting the support they need so they can better support the patient. The charts of patients are getting bigger and bigger every year because more and more d

Daniel Hart, CEO of Sentact, describes the need to integrate technology, analytics, and services for hospitals and health systems to improve patient safety, patient outcomes, and regulatory readiness. One of Sentact's key goals is to help organizations gain insights from data to allow them to move from a reactive posture using lagging indicators to a proactive environment supported by AI and real-time data to generate early warning signals. Successful implementation of these insights also reduces operational costs and potentially eases clinician burnout. Daniel explains, "Sentact is a healthcare technology, services, and analytics company. Three core pillars of our business are across tech analytics and services. We serve the hospital and health system market primarily, although we do have all types of healthcare organizations in our customer base. But as an example, a representative example, we service 60% of the top 20 health systems in the market with our solutions. And we are focused on patient safety, q

Matt Holland is Chief Operating Officer at Healio, a company that has been producing high-quality, vetted information for healthcare professionals for over 100 years. The company has focused on personalization of data as it has evolved from print to digital to AI-driven access to on-demand information. Healio AI is also transforming continuing medical education from a static activity to a dynamic, personalized experience based on real-time information gaps. Matt explains, "We're a little bit unique in this space. We're family-owned and privately held, and have been for over a hundred years. So there are a lot of companies in this market right now that are not. So for decades, the focus has really been on developing and delivering quality information to healthcare professionals through all sorts of different channels. Obviously, prior to the internet, it started with good publications and journals. We also have a series of live meetings and conferences, but the primary focus really is the Healio AI, which we

 Ryan Clarke, Co-Founder and CTO of Syntax Bio, is focused on solving manufacturing challenges in producing stem cell-derived therapies, specifically the process of stem cell differentiation. The Cellgorithm platform was designed to make differentiation an engineering problem by using a modified form of CRISPR to turn genes on and off in a specific, controlled sequence, reducing processing time from months to days and resulting in cost savings. One goal is to use AI and machine learning to build models capable of predicting optimal gene regulation sequences accelerating the discovery of new differentiation protocols and treatments for degenerative diseases. Ryan explains, "The key problem that we are focused on applies to stem cell-derived cell therapies. And so, just for a little bit of context setting, we use induced pluripotent stem cells or IPS cells. And about 20 years ago, when these were first derived, everybody was very excited because this is the platform where you could then have an infinite supply

Dr. Daniel Sodickson is Chief Medical Scientist at Function Health and author of the new book, The Future of Seeing: How Imaging Is Changing Our World. Building on the drive to improve medical imaging, AI and neural networks are now reshaping image interpretation and how and what data are collected. This approach produces high-quality results with minimal additional data and is inspiring innovative scanning techniques and equipment design. The future of medical imaging is the everywhere scanner, enhancing a single-shot session with a large hospital machine with continuous health monitoring through wearables and devices integrated into everyday life.  Daniel explains, "One of the things that imaging can do is peel away all of the obscuring layers of skin or skull or whatever else there is, without having to make a single cut, and show us the inner workings, show us inner space, what's inside. I think that means being able to detect tumors early enough that they can be cured, to guide surgeries, to try to unde

Dr. Howard Federoff, Scientific Co-Founder, Chief Medical Officer, and Executive Vice President of Corporate Medicine and Science at Kenai Therapeutics, is developing a cell therapy for Parkinson's disease that involves transplanting IPSCs into the brain to replace lost dopamine neurons.  The goal is not to cure the underlying cause of the disease, but to restore motor function and reverse the disease's progression. Initial clinical focus was on patients with moderate to moderate-severe idiopathic Parkinson's, showing efficacy and safety, leading to expanding the study to include earlier-stage and familial forms of the disease. Howard explains, "Kenai was formed about three and a half years ago, and the intention is to develop a cellular product, which means that what is manufactured will be eligible in the right patient groups who have a diagnosis of Parkinson's to be placed into the brain. And consequent to its placement, the cells will then form new circuits, dopamine circuits that are lost owing to the d

Brent Ness, CEO and President of Aclarion,  highlights the challenges of diagnosing and treating chronic lower back pain, a leading driver of healthcare costs and opioid addiction. Traditional MRI and CT imaging do not reveal the biochemical source of pain within spinal discs, leading to misdiagnosis and unsuccessful treatment. The Aclarion technology uses MR spectroscopy to measure pain-causing biomarkers and, through a cloud-based, AI-powered SaaS model, analyzes the raw data and sends the physician a report within minutes. Brent explains, "The diagnosis and accurate treatment planning of back pain are incredibly complex. And when you think about pain management physicians, rehab, all the way up to spine surgery identifying the source of pain accurately leads to better treatment and then obviously better outcomes.There are 266 million people around the world who suffer from chronic low back pain. So I'm not talking about the kind that you had a rough weekend skiing, golfing, or hiking, and you're a little

Dr. Alanna Morris-Simon, Senior Medical Director for US Medical Affairs at Bayer, describes the symptoms and diagnostics used to classify heart failure and the key at-risk populations for this condition. The rapidly evolving landscape of heart failure treatments now includes the Bayer drug KERENDIA, a non-steroidal MRA approved to reduce cardiovascular death and heart failure in adults with an ejection fraction of 40% or more. This drug is part of an emerging trend to treat multiple related conditions simultaneously  and could prevent the onset of heart failure and treat established heart failure. Alanna explains, "At a basic level, heart failure is a clinical syndrome, and that's important. I'm actually a heart failure cardiologist as well. And so this is important because patients have to have signs and symptoms. And those signs and symptoms really result from the heart being unable to either fill with blood properly or squeeze that blood out in a way that meets the body's demands. Either way, patients exp

Steve Brown, Founder and CEO and Lisa Booth, Vice President of Operations of CureWise, are both cancer survivors, which led them to develop an AI-powered tumor board platform to advance cancer diagnoses and treatment. This approach to precision medicine provides patients with the opportunity to better understand their specific condition, educating them about possible courses of action that may be more appropriate than the standard of care.  The platform also helps patients find relevant clinical trials and manage the side effects of their treatments. Steve explains, "So the mission really is to harness AI to advance cancer care. And ultimately, for that reason, anybody who's touched by cancer is hopeful that we will be making more progress toward cures for cancer. And we believe that that's going to happen through precision medicine, which really means recognition that everybody's cancer is unique. And at some point, we're going to treat cancer that way because we're going to know enough about how it all wor

Nayla Chaijale, Medical Strategy Lead for Rare Syndromes at UCB, describes Dravet syndrome, a rare, medication-resistant epileptic encephalopathy that involves seizures and significant co-morbidities like cognitive and developmental delays. Their approved drug FINTEPLA has a unique mechanism of action that modulates pathways in the brain and has demonstrated a significant reduction in the frequency of convulsive seizures in those with this condition. Nayla notes that the good news is that most patients with Dravet syndrome have a mutation in the SCN1A gene, a critical biomarker that enables accurate diagnosis. Nayla explains, "So, Dravet syndrome is a rare condition in epilepsy, and what we call it is neurodevelopmental and epileptic encephalopathy. I'm just going to call it DEEs, to say that it's a rare epileptic syndrome. Even though it's very rare, it's also very burdensome for the patients and their families. So, people living with these conditions start having the symptoms at a very early age, between tw

Vanessa Lemarié, Chief Operations Officer at myTomorrows, which is a platform for connecting patients, physicians, and clinical trial sponsors using a Trial Search AI tool designed to improve the accuracy of matching patients with suitable trials. The technology aims to speed up trial recruitment, increase diversity by reaching underserved populations, and ultimately shorten the drug development timeline. There is a particular focus on patients with no treatment options or who have not responded to current therapies. Vanessa explains, "We are all about patients. First and foremost, we want to help patients know their options. Patients who turn to us either have no treatment options for their conditions or have exhausted the treatment options. They are looking for alternatives and insights into whether we can help them find, for example, a clinical trial. Physicians who treat those patients turn to us."  "And last but not least, we work with biopharma companies that plan and conduct clinical trials and that s

Paul Pruitt, Chief Growth Officer at SHARx, which is a company that partners with employers to provide access to drugs not covered by insurance, such as specialty medications. The SHARx model acts as a complementary service to existing Pharmacy Benefit Managers and allows employers to opt out of covering the most expensive drugs. Paul's personal experience managing his sons' rare condition and navigating the administrative burden and frustration of the insurance system gives him deep insights into the factors driving up the cost of medications. Paul explains, "When we started SHARx eight years ago, the really highest cost biologics and what we call specialty medications were about 35% of the dollars that were being spent within a pharmacy program. And this year, we rarely see it under 50%. So it's really become a huge issue that we're noticing and realizing why many employers have to make the jump to do something different."   "The highest cost medications are 1% to 5% of how a plan is used on the pharmacy s