Empowered Patient Podcast

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

Lora Sparkman, an RN and VP and Partner of Clinical Solutions, Patient Safety, and Quality at Relias, highlights the high maternal mortality rate in the US and the factors that contribute to it.  Lora emphasizes the need for standardization of definitions and a better understanding of the social determinants of health that influence the success of a pregnancy, including the need for pre-pregnancy and post-pregnancy healthcare. Digital technology is bringing advancements to healthcare education and helping overcome geographic barriers to improve outcomes and reduce maternal morbidity and mortality. Lora explains, "In some cases, we're getting our hands around the real story versus some variation across states and definitions, and how death was categorized, those types of things. Putting that all behind us, there's been a lot more work and effort at the federal and national levels to standardize definitions and categories."   "There's been kind of a shift we've seen over the last couple of years, which is we'v

Mei Mei Hu, the CEO and Co-Founder of Vaxxinity, focuses on an approach to vaccines that aims to break immune tolerance to get the body to produce the antibodies to target self-antigens causing chronic conditions. Initial trials are being conducted to slow the progression or prevent the onset of Alzheimer's, Parkinson's, and hypercholesterolemia. Motivating this effort is the desire to democratize health and make transformative medicines available to a larger population at a lower cost.   Mei Mei explains, "Interestingly, in 1900, the average global life expectancy was 32. Today, when you hear someone died at 79, you're like, oh my gosh, what happened? We get these two bonus lifetimes now, and those three lives total for three main reasons. The first is the Green Revolution feeding us all. The second is hygienic plumbing, and the third is vaccines. But when we think of vaccines, they're usually crying babies or kids pre-COVID. What they did was able to banish the biggest diseases of our early lives." "What w

Dr. Karlene Kerfoot, Chief Nursing Officer at symplr, points out the challenges nurses and doctors face when dealing with technology in the workplace.  The disparate systems and lack of data integration make it difficult for clinicians to perform necessary duties and have moments that matter: time to provide the kind of care patients require. Karlene highlights the potential for AI and robots to alleviate some of the inefficiencies in the workflow and the need for frontline staff to be part of the decision-making process to ensure advanced technology solutions meet real needs.   Karlene explains, "We do have to deal with the technology to make it easier for people to work. For example, the nurse manager, when they want a staff and schedule, sometimes they have to go through six to eight disparate systems that don't talk to each other. So they have to go into one system and then out, and another system and out, and another system and out. So, at the end of the day, they're quite fatigued and have a lot of tec

Retired Brigadier General Dr. Steve Xenakis is Executive Director of the American Psychedelic Practitioners Association, a group of clinicians who are interested in developing and promoting the mainstream use of psychedelic and plant medicines for the treatment of mental health conditions. APPA works to educate practitioners on best practices and explores AI's potential role in identifying patients who could benefit from psychedelic therapies.   Steve explains, "This is a multidisciplinary group. As the treatments unfold, we're seeing it's multidisciplinary. We have psychiatrists, psychologists, social workers, advanced nurse practitioners, and physician's assistants because it's a team approach. When we look at the best treatments and therapies, these people come together. They can collaborate in terms of not only prescribing the medications but also conducting the therapy and supporting the patients who come to them for help." "They've come into this organization because they realize that to promote these

Jeffrey Madden, Co-Founder and CEO of ADvantage Therapeutics is developing therapies for neurodegenerative conditions, particularly Alzheimer's disease.  Instead of pursuing the amyloid hypothesis, ADvantage is focused on neuroinflammation as the primary driver of Alzheimer's pathology. Trials for their lead compound AD04 show promising results in slowing hippocampal volume loss and stabilizing cognition.   Jeffrey explains, "The assumption that has been in play for more than 20 years is the amyloid hypothesis. And the idea behind that is if you could clear amyloid beta, this toxic protein, you could stop or even reverse Alzheimer's. The results are in. We're reading about Biogen and Lilly, the monoclonal antibodies that do a great job of clearing amyloid but only have modest slowing of cognitive decline." "What we offer is that the pathology of Alzheimer's is just incomplete. We take a biology of aging worldview and we know the biggest causation for Alzheimer's is aging. We know a lane of aging is chronic i

Dr. Sam Lee, Co-CEO of Cocrystal Pharma, discusses novel antiviral therapeutics to treat norovirus. This highly contagious virus causes acute gastroenteritis, and there is no known treatment or vaccine.  Cocrystal is developing a compound that targets the essential enzymes of the virus, showing promising results in inhibiting norovirus replication and showing possible use in treating human rhinovirus and enterovirus. The goal is to develop a protease inhibitor therapeutic for norovirus that can alleviate symptoms and potentially prevent infection spread in high-risk environments. Sam explains, "Let me step back and tell you how we approach the noro antiviral drug development. So, we're focusing on what we call direct-acting antiviral therapeutics. We're targeting viral essential enzymes, and we initiated this noro program looking at the essential genes such as RNA-dependent RNA polymerase. We have really exciting hits, but we also look at the protease. Norovirus has this essential protease, which is absolute

Dr. Ruben Mesa, Executive Director, Atrium Health Wake Forest Baptist Comprehensive Cancer Center, discusses advancements in the treatment of myelofibrosis, chronic leukemia, a blood disease characterized by scarring in the bone marrow.  Current therapies include stem cell transplants and ruxolitinib, a drug that helps with symptoms and spleen enlargement but does not improve anemia. A new once-a-day pill, momelotinib, known as Ojaara, has been approved, effectively reducing spleen size, improving symptoms, and effectively addressing anemia.   Ruben explains, "What we've learned over time is that the origin of the disease is a chronic leukemia that occurs in the bone marrow that is driven by different changes in the genes that control the function of the bone marrow. And then there's a secondary scarring that occurs." "Patients with myelofibrosis might be afflicted with a drop in the blood counts and have anemia or a drop in the white blood cell count or the platelet count. They may have symptoms associated

Beena Wood, Senior VP of Safety and Medical at ArisGlobal, highlights how artificial intelligence is revolutionizing patient-centricity in drug development, pointing out the factors drawing more interest in this challenge. The LifeSphere drug development platform aims to drive patient-centric guidance by providing access to real-world data and enabling patient collaboration. Beena emphasizes the importance of engaging a diverse universe of patients and leveraging AI, data analytics, and machine learning to reveal hidden insights from the data to improve drug testing and safety. Beena explains, "Before specifically diving into how AI helps, I want to step back a bit on what the forces that drive patient-centricity are. I don't think these forces are new. They had existed, but I think they have been accelerating that patient-centricity conversation, especially with patients increasingly taking charge of their health and becoming more empowered. I think that drives the life sciences companies to focus on that an

Frank Forte, CEO of AnatomyIT, provides cybersecurity, IT, and value-based healthcare services to organizations that need help making an intelligent transformation to healthcare IT. Despite the prevalence of new technology, many providers and payers still rely on paper and fax machines, and data security risks and privacy issues still need to be addressed. Frank points out the need for change management and training to integrate generative AI and automate manual tasks to improve efficiency, clinician acceptance, and patient outcomes.   Frank explains, "But look, traditionally, when people thought of IT, they thought of a help desk. That was what came to mind. Networks, routers, cables, hardware, all those things that lived in the basement or the closet. And that's how I thought of IT. I would say that most IT initiatives were not received well, specifically from people who needed it the most, like clinicians, caregivers, and nurses, where it required a change in workflow, adopting new technology, or even rem

Dr. Barnaby Balmforth, Co-Founder and CEO of Biofidelity, has a mission to make genomic analysis simpler, faster, and more reliable for treatment decisions. Biofidelity addresses genome sequencing challenges by using a targeted approach and unique technology that allows for local testing using PCR rather than DNA sequencers. Thanks to COVID, an existing global infrastructure of PCR equipment provides an opportunity to target specific biomarkers and deliver actionable information to physicians and patients.   Barnaby explains, "The challenge as we see it is that in this modern oncology era, there is now a wide need for multi-gene genomic profiling of patients. The only solution that has been available to enable that is DNA sequencing. The media has quite widely covered how the genomics market is growing, and the cost of sequencing is shrinking. But sadly, in the clinical space, oncologists and patients are still failing to fully experience the benefits of this technology." "It is not a simple process to perfor