Empowered Patient Podcast

Andrea Pfeifer, Co-Founder, CEO, and Director of AC Immune, is developing vaccines for neurodegenerative disorders such as Alzheimer's and Parkinson's. Unlike vaccines for infectious diseases, these vaccines target internal proteins that are misfolded and contribute to the disease. These new vaccines are active immunotherapies against the Abeta protein. Trials are showing significant progress in overcoming safety concerns and lack of strong immune response for prevention, treatment, or maintenance therapy.   Andrea elaborates, "When you speak about vaccines, you always think about infectious diseases where you have this external virus or bacteria coming in, and you develop an immune system response against this virus or this bacteria, something external. In our area, in Parkinson's and Alzheimer's, we are activating the immune response of the body against an internal protein, but a misguided or misfolded protein."   "This is why it's so difficult because you have to overcome the tolerance of the immune syste

Wael Hashad, Chief Executive Officer of Longeveron, is focused on developing cell therapy for aging-related conditions such as aging frailty, dementia, and Alzheimer's disease. Using mesenchymal stem cells, also known as medicinal signaling cells, obtained from healthy donors 18-45, Longeveron is seeing promising results in clinical trials with improvements in brain volume and cognitive function. This approach is also being explored in the pursuit of a treatment for hypoplastic left heart syndrome, a rare congenital heart disease. Wael explains, "We believe there is a huge unmet need for bringing safe and effective products to try to slow the progression of the disease, improve the quality of life, and reduce the burden on the caregivers of such a disease. As for the aging frailty in general, as you know, with aging, there are a lot of things that affect the vascular system, muscle wasting, and all of those things that affect the quality of life during the aging years of an individual. Anything we can impact

Joe Tucker, CEO of Enveric Biosciences, discusses the use of neuroplastogen therapies for the treatment of neuropsychiatric disorders, including depression, anxiety, and post-traumatic stress disorder. Enveric has developed the Psybrary, a portfolio of compounds inspired by psilocybin that bind to the serotonin receptor in the brain and induce neuroplasticity without the hallucinations. The lead candidate EB-003 is intended to be administered orally and taken regularly as a maintenance therapy. Joe explains, "This whole neuroplastogen space was recently discovered, and it was discovered through academic research that we're looking at the effects of these agents we call psychedelics, psilocybin being the active ingredient in magic mushrooms and a very well-known psychedelic agent. It was in looking at psilocybin and other molecules known to be psychedelic agents like ayahuasca and LSD and others that scientists realized that what's going on is you're activating a particular receptor in the brain - something c

Vish Charan, Divisional Vice President of Product Development at Abbott's Cardiac Rhythm Management Business, explains some of the limitations of traditional pacemakers implanted in the chest with long wires connecting the pacemaker to the heart. Abbott has developed the AVEIR DR system, a dual-chamber leadless pacemaker that uses implant-to-implant technology to send electrical impulses between the upper and lower chambers of the heart.  The i2i technology allows these capsule-size devices to be implanted directly in the heart with the ability to communicate to synchronize the heart rhythm. Vish explains, "What we can do right now, is to get a very, very small access into the veins in the leg, and from the veins in the leg with a very small access, we're able to deploy these capsule-size pacemakers directly into the heart with a long tube. We call that a catheter. But that is how they're deployed. So, you don't have a surgical pocket. You don't have a big cut open in your chest, and the device is implanted

Laxminarayan Bhat, Founder, President, and CEO of Reviva Pharmaceuticals, is focused on developing next-generation therapies for neurological diseases, particularly schizophrenia. Their lead candidate, brilaroxazine, acts on multiple neuro receptors to balance imbalanced neurotransmitters in the brain that are responsible for causing schizophrenia. This new molecule shows positive results in efficacy and a low discontinuation rate, indicating the drug can be used for acute and long-term maintenance therapy. Lax elaborates, "Reviva is a late-stage pharmaceutical company. We are focused on developing next-generation therapies for diseases of neuro targets. That's our primary focus. So, currently, we are focused on CNS, inflammatory, and cardiometabolic diseases. We use chemical genomics-driven technologies to develop new chemical entities. Currently, we have two molecules in development. One is brilaroxazine, which is also called RP5063. The second molecule is RP1208. Both molecules were discovered in-house." "

Robert Maxwell, Founder and CEO of ClinConnect, has created a platform that simplifies the process of patients finding clinical trials, shortens enrollment times, and focuses on providing support to ensure patient retention in the trials. The platform works in collaboration with sponsors, healthcare providers, hospitals, and other stakeholders to streamline communication and more quickly identify qualified clinical trial participants from a broader universe. Robert explains, "We make the process of not only finding the trial very, very simple, but we also shorten the enrollment times, which in the case of acute disease is distinctly important. But generally speaking, it's usually more advantageous to get into a trial more quickly than waiting unnecessarily. And so, while we don't claim to solve all clinical trial recruitment issues, we've found that our patients generally see about a 60% to 70% reduction in enrollment time by working with us and letting us be their advocate through the enrollment process."

Chris Paradise, VP of Research and Development at Rion, is taking an acellular approach to developing next-generation regenerative medicines to treat degenerative diseases. The proprietary Rion system PEP, purified exosome product, uses extracellular vesicles or exosomes isolated from human blood platelets to send regenerative messages to cells to stimulate faster healing. This regenerative therapeutic allows for the administration in various forms, such as nebulization for pulmonary conditions and topical application for wounds. Chris explains, "What our focus has shifted towards at Rion is now an acellular approach, so it doesn't involve the use of cells or other components that have been proposed in the past. But we're focusing on extracellular vesicles or exosomes, and we can discuss those in more detail in a minute. But the point of emphasis is that it overcomes many of the limitations the field has previously faced. We're excited to offer reproducible, consistent, and, most importantly, accessible and a

Fady Boctor, President and Chief Commercial Officer of Petros Pharmaceuticals, discusses the process of transitioning drugs from prescription to over-the-counter status, which includes conducting studies to determine label comprehension.  In this situation, the FDA reviews the package wording to determine if the consumer can self-select the drug safely without a physician's involvement. With a focus on men's health issues like erectile dysfunction, Petros also sees the opportunity to educate consumers, reduce healthcare costs, and potentially identify related conditions that might require further investigation. Fady explains, "Switches had been occurring, those prescriptions to OTC switches, have been occurring since really 1974, 1975. Over the past many years, about 106 products have been switched from prescription to OTC. The common denominator among those switches is that they're relatively safe products. We're talking antihistamines, allergy medications, and smoking cessation products. These are products

Dr. Holly Miller, Chief Medical Officer at MedAllies, discusses the importance of data interoperability and usability to provide clinicians and patients with information that can lead to better-informed decisions.  The Sequoia Data Usability Project focuses on ensuring that data is trustworthy and useable. MedAllies is involved in the related Taking Root Movement, which promotes the adoption of the Sequoia Data Usability Implementation Guide. Holly emphasizes the need for solutions to provide a friendly user interface and software design that improves the providers' workflow.   Holly explains, "Medallies offers multiple interoperable networks, ensuring the secure transmission of critical health information among healthcare constituents. It's important that the listeners understand that we've moved to a world where a lot of data is being captured electronically. To optimize clinician-patient decision-making and patients being able to make decisions for themselves, that data has to move across the healthcare en

Dr. Mark Lomax, CEO of PEP Health, a new way to listen to the patient's voice. PEP, the Patient Experience Platform, collects and analyzes patient comments from various digital platforms to gain insights into the quality of care provided by healthcare organizations. Using AI to interpret and understand the sentiment of the comments, the company is providing real-time data that can help organizations make better management decisions and improve patient care.  The technology can also be used for risk management and early detection of problems within healthcare facilities to improve patient safety and outcomes. Mark explains, "We've got a few arms to it, but the fundamental part of what we do is collect all the comments that patients might post anywhere on the digital landscape from social media, online review sites, doctor review platform sites. We aggregate it all together and then make sense of it so that we can listen to a whole population. We listen to the whole US, and every location, every hospital, and

Dr. Fiona Elwood, VP and Neurodegeneration Disease Area Stronghold leader at J&J Innovative Medicine discusses the challenges in developing new therapies for neurodegenerative diseases in part because of the heterogeneity of the patient population.  She highlights the difficulty in defining the subsets of these diseases and identifying the right patients for clinical trials. Emphasizing the importance of early detection, J&J Innovative Medicine is developing strategies to support the move to precision medicine in treating diseases like Alzheimer's and Parkinson's to prevent patients from moving to a symptomatic stage of disease.   Fiona explains, "For example, in Alzheimer's disease, as many people know, one of the primary symptoms is dementia, but dementia is a symptom. There can be other reasons why people develop forms of dementia. Alzheimer's disease is a specific disease where now we know patients can be defined by the pathology in the patient's brain. So specifically, the patients develop amylo

Dr. Ping Cao, Co-Founder and CEO of BridGene Biosciences, has developed IMTAC, a platform that can identify molecule ligands for hard-to-drug targets. This platform allows for proteome-wide screening for drug candidates from a small molecule library and has identified over 4,000 protein targets providing numerous opportunities for drug development. The lead candidate, BGI-9004, is a TEAD inhibitor of multiple cancers. BridGene is also in partnership with Takeda to focus on neurodegenerative disorders.   Ping explains, "For small molecule discovery, the major challenge right now is the limited druggable space. Human proteome consists of over 20,000 proteins, and approximately 12,000 of them have been confirmed to play a role in human diseases and may become targets for precision therapy."   "However, statistics analysis shows drugs so far approved by the FDA only target less than 10% of proteins. In other words, most potential drug targets within human proteome do not have associated drugs. Those targets are c

Bronwyn Spira, Co-Founder, and CEO at Force Therapeutics, provides a comprehensive digital platform connecting patients to their rehab and recovery care team for musculoskeletal therapy.  The platform includes personalized treatment plans and allows continuous monitoring and real-time intervention to help reduce readmissions and complications. While patients have come to expect a digital alternative, providers have become more comfortable with digital tools, recognizing their ability to engage with more patients and reduce burnout by automating repetitive tasks.   Bronwyn explains, "Force is a comprehensive tool that patients access during their recovery to get all their personalized treatment plans, which might include exercises, education, videos, outcome forms, and anything they need when they're not in the presence of their providers. The platform is almost like an extension of the patient's care team, allowing patients to provide feedback that enables real-time intervention. We've seen that this feedbac

Dr. Michael Gao, Co-Founder and CEO of SmarterDx, discusses the challenges hospitals face in getting paid for their services. He explains that the revenue cycle, which involves generating accurate receipts for patient care, is a complex process that requires manual work from healthcare professionals. SmarterDx is streamlining the process by using AI to scan and analyze medical data, allowing professionals to focus on making judgments based on clinically validated data to report and bill more accurately for actions taken.   Michael explains, "One of the biggest challenges in the healthcare environment today is that hospitals have a hard time getting paid to provide the services they do for sick patients. And we've all seen through COVID, and certainly, in modern-day, these organizations are operating off of 1% operating margins or even losing money year over year. I think, very fundamentally, the problem that we're trying to solve is how do we make sure that the hospital can tell a complete patient story and

Karen Schatten, the Associate Director of the National Board of Physicians and Surgeons, is encouraging consideration of alternative paths for continuous certification for board-certified physicians and surgeons. Since 2000, a bureaucratic process has been mandated for this ongoing education.  NSPAS aims to modernize and disrupt this process by offering an alternative path for continuing education that addresses physician burnout and provides relevant training to stay current in their medical specialties.   Karen explains, "The reason why the bureaucracy persists is, despite it not being really proven and physicians really having to collectively protest it, that requirement we now know after studying it for a number of years is it is actually more expensive. It's more burdensome. It creates a lot of irrelevant administrative burdens. Those forced programs are not a great match for physicians and what they do in day-to-day practice. It's often not clinically relevant and plays a critical role in exacerbating

Phoebe Souza is the clinical lead for mental health at OncoHealth, a digital telehealth platform focusing on personalized oncology-specific support for patients with cancer and their caregivers. Advanced care planning is how individuals can communicate their wishes for care that aligns with their values. Phoebe highlights the role of healthcare providers, including oncologists, in initiating conversations about advanced directives to ensure patients understand their options. She emphasizes all individuals over 18 should document their desires for medical care in the event they are unable to do so themselves.  Phoebe explains, "Advanced care planning, also called ACP, is an umbrella term that includes both communication skills, so conversations with loved ones and a person's medical team, as well as completing documents such as an advanced directive, and that helps a person make plans about their future healthcare. An advanced directive and a care plan serve as a roadmap for medical providers and loved ones t

Dr. Steve Marcus, CEO of Cantex Pharmaceuticals,  emphasizes the importance of the RAGE receptor in diseases like cancer and inflammation.  Their lead compound, azeliragon, blocks the activation of the RAGE receptor and shows effectiveness in inhibiting cancer metastasis and enhancing the impact of radiation. Clinical trials are underway for breast cancer, pancreatic cancer, glioblastoma, and COVID-19. This approach addresses the cancer cells and the microenvironment in which they reside to reduce disease progression and alter the immune environment. Steve explains, "RAGE is a receptor on the surface of cells that sends signals to the cell that can either enhance or harm health. When over-activated, RAGE has been implicated in a wide range of inflammatory diseases and cancers. In cancer, in particular, over-activation of RAGE has been associated with both the invasiveness of cancer, with the progression of cancer, and with metastasis of cancer."   "Azeliragon blocks the binding of anything that can activate

Alex Nemiroff, General Counsel at Praxis Precision Medicines, focuses on developing life-altering treatments for pediatric patients with epilepsy, particularly those with genetic epilepsies known as Developmental Epileptic Encephalopathies- DEEs.  Praxis targets specific gene mutations SCN2A and SCN8A with therapies that precisely target the source of the disease. Lead drug candidate PRAX-562 is a precision small-molecule treatment that inhibits seizure-causing activity, and PRAX-222 is a type of RNA therapy that shows potential for complete disease modification. Alex explains, "There are a number of types of epilepsy. At Praxis, we focus on a few. We have a broader focal epilepsy program, and then we have a number of programs targeting these genetic epilepsies that are referred to as DEEs, Developmental Epileptic Encephalopathies. These are epilepsies that start in infancy, and they're tied most commonly to a mutation in a gene that is known to cause epilepsy. Specifically, we have at Praxis right now in th

Dr. Stephanie Lahr, President of Artisight, is promoting the concept of Smart Hospitals and ambient technology's role in improving healthcare delivery.  Hospitals can use sensors, cameras, speakers, microphones, and screens to address the increasing complexity of patient care while allowing clinicians to focus on patient care. These technologies are designed to integrate seamlessly into the clinicians' workflow with a focus on process automation and applying AI to drive the convergence of technology and medicine.   Stephanie explains, "A Smart Hospital is a hospital that is equipped with sensors and other kinds of elements that can capture information based on what's happening in real-time and leverage that information in real-time to help automate communication, automate documentation, and send signals. So, in your own home, you might think about a smart home where your thermostat has different elements that can sense when there's no one in the house, and it will change the temperature to be more economical

Arnon Chait, President and CEO of Cleveland Diagnostics, discusses the role diagnostics play in accurately identifying those patients most at risk for cancer. The current PSA and related tests are used to diagnose prostate cancer to determine if additional diagnostics are necessary. By identifying the stage and treatment options with a more specific blood test and AI-aided identification, doctors and patients can make better informed decisions about further testing or imaging.   Arnon elaborates, "A long time ago, in the late '80s and more in the '90s, a new test called prostate-specific antigen came to the market. It is a simple blood test, which is great and offered a glimpse at the important information for the first time. Before that, it was never available to either a PCP, to a private GP or a urologist, whether you have potentially prostate cancer or not."   "The biggest issue in the industry today, and in the clinical world especially, is what to do with the information. Because some of this informati