Empowered Patient Podcast

Anne Meneghetti, Executive Director of epocrates, is a leader in medication management. She discusses the challenges of polypharmacy, especially in older adults, and emphasizes the app’s capabilities like quick drug lookups and dosing calculations. Anne highlights the dangers of unintentional medication errors, including interactions with over-the-counter drugs and the importance of medication reconciliation. She also shares practical tips for clinicians and patients to improve adherence and communication, advocating for proactive healthcare strategies.

Dr. Adam Kilian, Director of the Rheumatology Fellowship Training Program at the St. Louis University School of Medicine, focuses on IgG4-related disease, a rare multi-organ disease that has only recently been recognized due to significant diagnostic challenges. The MITIGATE trial is a landmark study that demonstrated that the first FDA-approved treatment, UPLIZNA from Amgen, provides an effective, targeted, steroid-free therapy. The approval of this drug is driving awareness in the medical community about IgG4-RD and whether the disease should be considered after inconclusive results for other suspected conditions. Adam explains, "IgG4-related disease is a rare disease that affects many organ systems. It's a chronic systemic, fibroinflammatory disease that can affect almost any organ system. And it will usually present with these tumor-like inflammatory masses that can cause scarring and lead to organ failure." "Our understanding of it continues to evolve, and it's had a really interesting story over the last century because IgG4-related disease affects so many different organ systems. Over the past century, there were actually many different diagnoses that were recognized, which now all fall under the umbrella of IgG4-related disease. Different scientists and physicians around the world over the years recognized the different manifestations of the disease in the pancreas, in the lymph nodes, in the head and neck, in the kidneys, or in the blood vessels. And it's only been in the last 20 years or so that we have recognized that actually all of these different disease entities from the last century are actually all manifestations of this disease, IgG4, IgG4-related disease." "The clinical trial is called the MITIGATE trial, and it's a very seminal clinical trial in rheumatology from the last several years. This was the first-ever phase three double-blind, placebo-controlled, randomized controlled trial in IgG4-related disease. This was a huge trial. It was global. It was conducted in 22 countries with multiple specialties coordinating this trial. It was 52 weeks long, and its purpose was to evaluate the effectiveness and the safety of UPLIZNA in patients who have IgG4-related disease." #MITIGATETrial #IgG4RelatedDisease #IgG4RD #UPLIZNA UPLIZNA Download the transcript here

Dr. Thomas Wisniewski, Director of NYU Langone's Alzheimer's Disease Research Center, shares insights from a recent study that projects new dementia cases in the US will double by 2060. New diagnostic tools have made it easier to identify early signs of Alzheimer's, and disease-modifying therapies are being approved, which can be effective if patients are treated in the earliest stages of the disease. AI is seen as a tool to help identify at-risk patients and routinely screen patients to manage the growing need for access to dementia care. Thomas explains, "This was a study that was led by the NY Optimal Institute, which is directed by Dr. Joe Resh, who's really a leader in this area and many public health issues. He did a very thorough analysis along with his team that the annual number of incident dementia cases in the United States is projected to increase from current estimates of 500,000 to around 1 million in 2060. So basically doubling in white adults. Furthermore, in African Americans, this incidence of dementia rate is expected to triple, with the largest absolute increases in dementia cases going to be in the oldest old population." "There was perhaps a lack of awareness of the prevalence of dementia in past decades. But now the importance of making an accurate diagnosis and recognition of dementia is becoming much more prominent in the medical literature and in the medical profession. And that message, I think, is permeating to the lay public as well. So there hasn't been a change in the definition so much, but there is an increase in knowledge in the medical profession about the importance of making this diagnosis, and people living alone." "It's really been a dramatic change. So, apart from being a cognitive neurologist, I'm also a board-certified neuropathologist, and it used to be that making the definitive diagnosis of Alzheimer's disease required a chunk of brain. I'm delighted I don't need those chunks of brain anymore to make a diagnosis of Alzheimer's disease. There are now very clear clinical criteria and biomarker definitions for making a very accurate diagnosis of Alzheimer's disease." #NYULangoneAlzheimers #AlzheimersDisease #DiagnosingAlzheimers #AlzheimersResearch #MedAI med.nyu/centers-programs/alzheimers-disease-research/ Download the transcript here

Pedro Sanchez de Lozada, CEO of Canid, highlights the significant financial risks that pediatricians face in managing their vaccine inventory. There are administrative burdens associated with ordering, patient demand, tracking, reporting, and filing for insurance reimbursement. The Canid platform was designed to be a vaccine-as-a-service model, removing risk and administrative work from pediatric practices, allowing small and independent practices to achieve economies of scale, and freeing up more time to spend with patients and their parents. Pedro explains, "When managing a vaccine program, the expectation is that the pediatrician will buy and maintain the stock in their office until a patient comes in and gets vaccinated. So, the refrigeration, the temperature logging, the stocking up, and the cost of the vaccine always fall as liability to the pediatrician. And what happens there is that you have a fairly small margin on these vaccines. Now I'm sure we're going to get into this, but when people say pediatricians make money on vaccines, they're usually cherry-picking." "But in reality, as a pediatrician, you actually have to stock all the vaccines. And not all of them have decent margins. Some of them have negative margins. And so as a portfolio, you end up making a pretty thin margin, and then that doesn't even include all the different challenges you might have along the way. So let's take an example: a vaccine that expires or is dropped, and unfortunately needs to be wasted." "So the way that we like to think about it is we want to make it so that the pediatrician can just give vaccines. That's all they need to think about. And what that means is, let's just imagine for a second, a refrigerator that automatically restocks itself, and you just take out the vaccines, you give it to a child, and you go on your merry way. That's obviously a little bit of an idealistic way of viewing things, but it's the experience that we want to accomplish." #Canid #Vaccinations #Pediatricians #Vaccines #PediatricVaccineManagement canid.io Download the transcript here

Alicia Silver, senior director at ADVI Health, highlights the evolving landscape of cell and gene therapy and the need to improve patient access and payment for these treatments. Availability of these therapies for solid tumors and genetic diseases like sickle cell disease is expanding due to the transition from inpatient to outpatient and community settings. The FDA's decision to remove REMS requirements for specific therapies has accelerated the growth of facilities to provide care, particularly for vulnerable populations. Alicia explains, "We work with a number of different cell and gene therapy clients throughout the sector. So we work with manufacturers who have commercialized cell and gene therapy products. So they have products that are currently on the market, manufacturers who are going through the process of clinical trials right now, working with the FDA to get approved products. But we also work with trade organizations that are working behind the scenes at the sector level, trying to get different policies and access changes for patients." "To date, there's probably close to a couple of dozen FDA-approved cell and gene therapies, and they treat everything from blood cancers, which were the first approvals in something called CAR T. We saw blood cancers as the first approvals, and then everything through to solid tumors in oncology. But also, we have newer gene therapies for conditions like sickle cell disease. And that's an area that's been incredibly underserved and definitely will benefit from a durable gene therapy that hopefully corrects some of the issues that patients with sickle cell disease have, like pain crises that end up in a hospital. So from that perspective, we see a really wide range of treatments available to patients today and many more on the horizon." "I think the price tag is definitely somewhat of sticker shock for people who don't understand how cell and gene therapy products are valued. And so what we do a lot of times, educating on, is helping payers understand that it's not necessarily $2 million for a treatment that's going to be a recurrent payment, but something that's kind of an investment in the patient's and the plan's future." #ADVIHealth #CellTherapy #GeneTherapy #AcesstoCellGeneTherapy #ClinicalTrials advi.com Download the transcript here

Dr. Stacy Lindborg, President and CEO of IMUNON, has developed a DNA-based immunotherapy candidate for the treatment of ovarian cancer. This therapy works by administering the drug, which utilizes IL-12, a powerful anti-cancer cytokine, directly into the cavity where the cancer resides, thereby affecting the tumor microenvironment. Trials are showing that the drug can produce significant life extension when used in combination with standard chemotherapy. Stacy explains, "There are about 300,000 women who are newly diagnosed with advanced ovarian cancer every year, about 20,000 in the US, and the frontline standard of care hasn't seen a change in the treatment in about 25 years. So this is for newly diagnosed women. The very first treatment that they would have, which we call frontline treatment, is a platinum-based chemotherapy. So carboplatin and Paclitaxel are both administered through IV over an hour for carboplatin, about three hours for Paclitaxel. So most women go directly to chemotherapy and then surgery, and then chemotherapy. Some women will go straight to surgery and then have this chemotherapy afterwards." "Our approach is a DNA-based immunotherapy candidate that we have in Phase 3. We refer to the lead candidate as IMNN-001, and this is a non-viral nanoparticle that is administered directly into the cavity of interest. So, what we call the micro-tumor environment is the peritoneal cavity, where the cancer actually resides, and it's delivered through a catheter. And it basically has a very powerful anti-cancer fighting cytokine, IL-12, that is encoded in this immunotherapy. And it causes the cells that are within each woman's body, both cancer and non-cancer alike, to start producing activities that will help fight these complex cancer cells that exist." #IMUNON #OvarianCancer #DNABasedImmunotherapy #WomensHealth #CancerAwareness imunon.com Download the transcript here

Donovan Campbell, CEO of Medbridge, empowers clinicians through a platform that is focused on movement-based medicine and a holistic approach to patient care. MedBridge has developed AI tools to perform triage, act as a care coordinator, and serve as a patient-facing coach, offloading administrative tasks and providing better support to patients. These tools are designed to support clinicians by flagging potential issues and increasing patient engagement by delivering immediate digital access to care. Donovan explains, "We serve primarily physical therapists. And those physical therapists can work either in what's called a private practice outpatient setting or in a hospital setting, whether that's inpatient rehab or outpatient rehab. We also service physical therapy and adjacent practitioners, such as athletic trainers, speech and language pathologists, or occupational therapists. With the release of our new hybrid care platform, we're moving into different care categories, which is really exciting. So we're increasingly working with advanced primary care companies that service the employer health market. That's exciting. And we're exploring other opportunities within women's health and within orthopedics." "Movement-based medicine is relevant to a really holistic approach to patient care, and it spans multiple clinical specialties. So think about the elderly patient who's just had a right knee replacement. Movement-based medicine is critical to getting them back up and on their feet. For a woman postpartum, pelvic health and movement-based medicine are really important. And pelvic health, movement-based medicine that she can access at home, is really important in her recovery and meeting her where she's at in her phase of life. And then physical therapy obviously is all about movement-based medicine." #Medbridge #DigitalHealth #HealthcareAI #FutureAI #MovementBasedMedicine medbridge.com Download the transcript here

Dr. Matt Goldstein, CEO of jscreen, focuses on the importance and power of preventive genetic testing for reproductive carrier screening and hereditary cancer screening. All genetic tests include analysis to inform specific medical management plans and provide genetic counselors to explore options. Of particular interest is testing for the gene mutations that cause Tay-Sachs disease and helping families take proactive steps to effectively eliminate the disease in future generations. Matt explains, "So, jscreen is a nonprofit that focuses on providing access to education and high-touch support to individuals, families, and to clinicians in the area of preventive genetics and more specifically for reproductive carrier screening and hereditary cancer screening. "These types of tests actually have been around for a really long time with the advancement in genomic technology and our knowledge generally of genetics. The capabilities we have around these panels—what we can test for and what we can do with them—have grown tremendously over the last two decades. And so, what we're offering now is completely different than what you may have been offered five or 10 years ago." "What I will say is I think that this technology is the most powerful medical intervention that we have. Maybe second to vaccines, which we know obviously can have profound impacts on saving lives, preventive genetics, the ability to screen an individual or a couple or a family, and identify diseases that they may be at risk for, with things that you can do to prevent those diseases from happening or catch them early. That's the holy grail. That's the most amazing thing: being able to intervene." #jscreen #GeneticTesting #TaySachsDisease #ReproductiveCarrierScreening #HereditaryCancerScreening jscreen.org Download the transcript here

Dr. Yuri Maricich, Chief Medical Officer at CAMP4 Therapeutics, describes regulatory RNA, a new area of biology that recognizes the role of Reg RNA in the production of proteins from specific genes. This technology is well-suited for haploinsufficient diseases such as SYNGAP1-related disorders, in which there is a lack of healthy protein and both parents carry a copy of the mutated gene. The goal is to create disease-modifying treatments that correct the underlying genetic cause rather than treating the symptoms. Yuri explains, "What was really unique about CAMP4's scientific approach is that we're focused on a whole new and emerging area of biology called regulatory RNA. And these are control elements for the expression of genes. In other words, how much protein we get from a particular gene. And there's been a lot of work in the past on how to have less protein made, particularly if it's a protein that has a mutation that causes a problem. But in medicine, there have been very few opportunities to actually increase the amount of protein, but there are many diseases that need more healthy protein." "The backdrop of CAMP4 is that there was work done just over eight years ago at the Whitehead Institute at the Massachusetts Institute of Technology in a lab by Rick Young, and he was working with a colleague at Boston Children's Hospital, Lenson, and they noticed that there was this group of so-called regRNAs. These were non-coding regions that historically have been really ignored. And as they looked and explored their function further, what they discovered was that, in fact, these regRNA elements play a critical role in controlling how much protein is produced. And so the story of CAMP4 has been to continue to understand and map different cell lines so that we could take a particular target gene and, by using tools or established medicines, for example, like antisense oligonucleotides, we could actually increase the amount of a gene's protein back up to normal." #CAMP4 #CAMP4Therapeutics #SYNGAP1 #CuresSYNGAP1 #regRNA #RegulatoryRNA camp4tx.com Download the transcript here

Phil L'Huillier, CEO of Scancell, has developed an off-the-shelf DNA immunotherapy designed to generate a potent and durable immune response against advanced melanoma in patients who are unresponsive to current therapies. Their lead candidate showed significant benefit when added to standard of care checkpoint inhibitors, improving duration of response without adding side effects or toxicity. Patient selection in future trials will use a blood test to identify the immune types that can be expected to respond best to the therapy. Phil explains, "Perhaps first and foremost, Scancell is a clinical-stage company developing novel active immunotherapies for patients. And our objective for patients is to pick up the patients that are unresponsive to current therapies or that respond for a short period of time to improve the overall survival through developing therapies that give a good duration of response, potent immune responses, but are also safe for patients." "Perhaps before I share results from the studies, I should step back a little bit and just tell you about the platform and the product that the data has arisen for. At Scancell, we're developing these off-the-shelf. The data that we're about to talk about comes from our lead program, which is an off-the-shelf DNA immunotherapy called Immunobody, that's the name that we use for it. And it's different from the personalized therapies, the personalized vaccines that require an individual to give a tumor sample. And then there's sequencing and manufacturing for the individual there. This is off the shelf." #Scancell #Immunobody #Immunotherapy #Cancer #Oncology #Melanoma #Biotech scancell.co.uk Download the transcript here