Empowered Patient Podcast

Emee Baldwin, Founder of Poppy's Purpose, is a nursing student and patient advocate with a success story about getting the attention of care providers and elected officials to re-evaluate the rules regarding patient visitation rights, particularly in the age of COVID-19. Emee and her family worked with their local representatives to make a law in Alabama where no patient will be alone every day.  Emee elaborates, "So, last November, November of 2020, my grandfather, known to me as Poppy, was diagnosed with COVID-19. This was new to us, and it was new to everyone at the time. But to start with the background story, I was used to seeing my Poppy every single day. I grew up on a small family farm, so we were very close. If I didn't see him every day, I at least heard from him every day, so when he was first admitted to the hospital with COVID-19, that first week, he was just on a regular floor up here in Huntsville, Alabama, which is where I live and where I go to school. So it was really odd being two miles away from him and not being able to get in to see him." "Ultimately, he ended up passing away after a little over three weeks, and he was in the hospital just a little over three weeks, completely by himself. My grandfather was very involved in politics here in Alabama, so at his funeral, we had a lot of politicians there, a lot of legislators there. Right after the service was over, they came up to us, and they were like, "We've seen the hurt that your family went through. We saw it firsthand through you, and we want to make a change. We want to help y'all to overcome this. That way, other families don't have to battle what you went through." @Emee_Elizabeth #PatientAdvocate #COVID #COVID19 #PatientRights poppyspurpose.org Download the transcript here

Hamid Khoja is the Chief Scientific Officer of FibroBiologics which is developing regenerative medicine using fibroblasts which are one of the most abundant cells in the human body. While stem cell therapy has gotten significant attention, fibroblasts are easier to source and grow. Hamid elaborates, "Just to give you a sense of some of the capabilities of fibroblasts. They behave very similarly in many aspects to stem cells, which we're very well aware of. What makes fibroblasts significantly interesting for us and for therapeutic purposes is that they can also differentiate into many different cell types." "So the CybroCell is the product name that we've given our tolerogenic fibroblasts, cells that have been specifically cultured for use in degenerative disc disease. But we've also tested this same product in a clinical trial for multiple sclerosis. So we have done limited trials for safety for both degenerative disc disease and multiple sclerosis, and the results have been very promising." #FibroBiologics #Fibroblasts #DegenerativeDiscDisease #DDD #MultipleSclerosis #MS #MultipleSclerosisTreatment #StemCellAlternative FibroBiologics.com Download the transcript here

Scott Gray is the President and CEO of Clincierge with a mission to support participants in clinical trials, particularly for patients with rare diseases. Since many rare diseases require a patient caregiver to accompany a trial participant, Clincierge also manages the logistics for both of those people. The result of these patient-centric efforts is greater diversity in recruitment, higher retention rates, and higher participant satisfaction. Scott explains, "The purpose for Clincierge is to improve clinical trial performance by improving the patient experience. Depending upon the disease state, different patients require different types of support to enable them to participate in a clinical trial. It may be a geographic location for a family so that a pediatric patient can participate in a trial. It may be language barriers, so we provide interpreters. Along with that relocation may come the need for housing that's funded, prepaid, meal stipends, ground transportation, visa services." "So what we do is look at each individual patient's need and design the services that support them, enable them to get to the clinical trial visits so that they can participate, and the research team can get their outcomes data." @Clincierge #Clincierge #RareDisease #RareDiseaseDay #RareDiseaseMonth #ClinicalTrials #ClinicalTrialResearch #PatientCentricity #PatientEngagement #PatientExperience #PTEXP Clincierge.com Download the transcript here

Sandy Macrae Ph.D. is the CEO of Sangamo Therapeutics which has a unique technology called zinc finger. This allows them to find and modify a particular DNA either to delete a faulty gene or to help enhance one that isn't producing enough or turn one down when it's producing too much. While currently focusing on rare diseases like hemophilia, Sangamo is also moving into treating neurodegenerative diseases, multiple sclerosis, inflammatory bowel disease, and kidney transplants with genomic medicine and cell therapy. Sandy explains, "Sangamo has been at the forefront of genomic medicine for 26 years now, and the world has changed so much and is now embracing genomic medicines as one of the key parts of the tools that we have to address serious diseases." "I think in the future, there'll be three kinds of medicines. There will be the medicines you take or inject, there'll be the vaccines that prevent disease, and then there'll be genomic medicines that will help solve some of the long problems from our genomic DNA that so many patients struggle with. So I think it's going to be a fundamental core part of what we do. It's important, though, to say these are early days, and every patient that volunteers we are immensely grateful to. We learn from each patient on a daily basis, and it helps us all move these medicines forward." @SangamoTX #CellTherapy #GeneTherapy #GeneRegulation #ZincFinger #GenomicMedicine #RareDiseases #RareDiseaseMonth Sangamo.com Download the transcript here

Chris Calhoun is the CEO of Paracrine Cell Therapy which has a different approach to regenerative medicine. This biologic approach uses an autologous cell therapy approach, taking a mixture of a patient's own cells, not just one cell type to address underlying pathologies through multiple mechanisms of action. Using a device that sits at the point of care, fat is extracted and the cells are processed and put back in the patient in about an hour. Chris explains, "It's interesting that if you look at the evolution of most of the therapies out there today, drug therapies and so forth, they generally treat the symptoms, and they target one mechanism. We're taking a very different approach. We're looking at treating complex conditions and diseases where there are multiple underlying issues or conditions or pathologies, depending on how you want to look at it. We think that this sort of silver bullet approach doesn't necessarily work to address more complex diseases where you've got these multiple underlying conditions." "Well, it's an emerging field, so I think there are various approaches. We're really the unique company out there that's focused on this heterogeneous or this mixed cell population approach to regenerative medicine via device. Probably the most important distinction ... well, there's two. One, I think the clinical benefits of this mixed cell population are becoming clear, and second is the economic aspect of it. We are not isolating these cells and growing them in a laboratory, and engineering them in any specific way that ultimately ends up costing orders of hundreds of thousands of dollars per treatment or sequence of treatments for a single patient." #Paracrine #RegenerativeMedicine #CellTherapy #AutologousCellTherapy Paracrine.com Download the transcript here

Jan D'Alvise is the President and CEO of Acasti Pharma, a late-stage specialty pharma company addressing rare and orphan diseases. The topic today is the treatment of the rare disease SAH, otherwise known as subarachnoid hemorrhage with GTX-104, a reformulation of the existing drug nimodipine. We also talk about reformulating and repurposing drugs for the rare neurodegenerative disorder ataxia-telangiectasia A-T that affects young children and for treating postherpetic neuralgia, the severe nerve pain that persists in some patients after they have a shingles infection. Jan explains, "So an aneurysm in the brain is known as subarachnoid hemorrhage. Now you could have bleeding on the brain caused by other things, but if it's caused by a burst aneurysm, it's known as subarachnoid hemorrhage or SAH." "So I mentioned that nimodipine is the standard of care today, and all patients who present with SAH are put on nimodipine post-surgery. The nimodipine is very potent, so you have to carefully monitor it. The problem today is that it's only available as an oral drug. Most of these patients are unconscious, so the nursing staff has to take a capsule, mix it with some liquid and actually give it to the patient through a nasogastric tube. So they literally put it through the nose, down the throat to deliver it into the stomach." "What we've done is we have figured out a very clever way of solubilizing nimodipine, so providing it in a formulation that can be delivered via an intravenous infusion. These patients are very sick, they're in an intensive care unit, so they're all already on an IV. They're getting other drugs via IV, they're getting fluids, so it's very easy to hook our drug up to an IV." "The key thing about it is because the nimodipine is then going directly into the bloodstream, you can get much tighter control of the amount of nimodipine that's actually going into the patient and being absorbed." #AcastiPharma $ASCT #SubArachnoidHemorrhage #SAH #GTX104 #Nimodipine #AtaxiaTelangiectasia #AT #Bupivacaine #PostherpeticNeuralgia #RareDiseases acastipharma.com Download the transcript here

Ted Karkus is the CEO of ProPhase Labs, which is in the battle to provide rapid results from PCR tests for COVID-19. While expecting a winter spike, Ted has been surprised at the record-breaking demand the latest Omicron variant has created. ProPhase Labs has recently acquired Nebula Genomics to provide whole genome sequencing direct to consumers to help guide medical treatment and educate people about diseases they may be predisposed to. Ted explains, "So, the issue with regard to turnaround times really has more to do with the number of employees you have, and it's very difficult, particularly in this environment, to find qualified lab techs, particularly in the state of New York, if not across the nation. So, we had these tremendous out-of-the-blue spike COVID incidents. The amount of testing, the request for testing went through the roof. All the labs in the country, and certainly in the state of New York, were overwhelmed." "As you can see, it's not just about the amount of time it takes to process a test with PCR, which is probably four to six hours. It's what the capacity for your lab is. We have three shifts working 24/7, but understand that there was a period in December where the number of specimens that came in, each of our customers, the amount of specimens they sent into us was like double or triple what it had just been a few weeks earlier." "What we do is provide whole genome sequencing direct to consumers. The future of medicine is all about precision medicine or personalized medicine. It's all about using your genetic makeup to understand what are the best drugs and therapies that will optimally work to treat your condition. We provide whole genome sequencing for $300. So, we're the low-cost provider. Right now, our turnaround times are about six weeks." #ProPhaseLabs #COVID19 #COVID #COVIDTesting #PCRCOVIDTest #PCRTest #Omicron ProPhaseLabs.com    Download the transcript here

Wim Souverijns is the Chief Community Engagement and Commercial Officer at Pharvaris and is focusing on HAE or hereditary angioedema a rare disease that impacts the cells as recurrent angioedema or swelling episodes. These patients go through unpredictable swellings in their body at the extremities, in the abdomen, and in the facial area.  Wim explains, "The cause of HAE is a deficiency or dysfunction in a particular enzyme called the C1-inhibitor, and there are tools to detect this deficiency or dysfunction. The levels of C1 inhibitor are measured and, based on these levels, patients with this condition are identified. That deficiency leads to a cascade of biochemical events, whereby the ultimate culprit for these swellings is a molecule called bradykinin. Bradykinin will connect to a receptor called a B2-receptor, and by doing that, it will signal to the cell that it should vasodilate - the cell basically opens its pores - meaning fluid is being taken up into the cell, into the tissue, leading to the swelling."  "The last approach addresses the penultimate event in the cascade by preventing the actual bradykinin in the plasma to connect to the B2-receptor. This is where Pharvaris is focused. We're trying to avoid bradykinin from activating the receptor, thereby avoiding swelling in the tissue. So, there are three different categories, and we are focusing on really on that bottom one, the bradykinin, because our therapy competes for the receptor." #Pharvaris #HAE #HereditaryAngioedema #Bradykinin #RareDisease Pharvaris.com Download the transcript here  

Amit Kumar is the President and CEO of Anixa Biosciences and is working on a prophylactic vaccine to prevent the onset of breast cancer rather than a therapeutic vaccine to fight the solid tumors. Anixa is also working on CAR-T therapy for solid tumors and using artificial intelligence and machine learning to discover compounds to treat COVID-19. Amit elaborates, "We believe that trying to destroy cancer cells as they arise in a patient's body is much easier than waiting until that cancer becomes a large tumor mass with trillions of cells. As you know, cancer starts off with one single cell that becomes aberrant. Then it reproduces, it becomes two cells, four cells, eight cells, and so on, eventually becoming a tumor that is comprised of millions to trillions of cells. So if we try and destroy a tumor that's millions and trillions of cells, it's very difficult." "You have a tumor microenvironment, you have a very immunosuppressive environment in that tumor, and it's very hard to address that tumor. However, if you could destroy those cells as they arise at the two-cell, four-cell, or handful-of-cell stage, then you could eliminate the tumor from ever arising. The cells never have a chance to become a tumor. That's what we're trying to do with our breast cancer vaccine." "This is a therapeutic as opposed to a vaccine against coronavirus. We believe that the coronavirus is going to be an endemic disease, and there will always be a need for therapeutics. We want to be able to have a therapeutic that can be easily distributed, it's room-temperature-stable, unlike the vaccines, which require a cold chain, and something very inexpensive that can be taken by mouth." @AnixaBio #CancerVaccine #CART #Immunotherapy #BreastCancer #COVID19 #COVIDTherapeutic Anixa.com Download the transcript here

Chris Young is the Head of Product at Force Therapeutics and is focusing on presenting information to patients before and after surgery, particularly those undergoing musculoskeletal related treatments. The key to patient engagement is presenting the right information in the right form at the right time to keep patients on track with their medications and rehab and to help manage their expectations about pain and recovery time. Chris elaborates, "And so when we talk about digital health 2.0, what we want to do is take advantage of the fact that the majority of patients have a behavior-changing device in their pocket. So a lot of patients are going to be walking around with an iPhone or an Android in their pocket. And the way that we can present information in real-time based on their needs, we feel like that with that behavior-changing device in their pocket, we can truly help them ingest information. We can do more than just kind of present this cold, clinical kind of information in front of them, and actually try to create true behavioral change. So I think that's really where the importance of design and health tech comes into play." "I would say that the preoperative window, particularly around up to a week, especially when it's surgical care, a week before surgery, is really, really important. You want to do a couple of things, especially in the move towards same-day discharge. You'll often see, especially with hip and knee replacement, that there is a move towards same-day discharge. You're leaving the hospital or the ASC after about six hours. We want to make sure that you feel adequately prepped for when you get home. So a lot of what we're going to do is present that information." @ForceTherEx #ForceTherapeutics #DIgitalHealth #PatientEngagement #CareManagement ForceTherapeutics.com Download the transcript here