Empowered Patient Podcast

Dr. John Dobak is the President and CEO of DermTech which is leading the genomic revolution in dermatology to more accurately determine the presence of skin cancer. Their noninvasive platform changes the protocol for assessing a suspicious mole from visual review and surgically removing tissue to genomic analysis and using broader objective data before treatment is determined.  John elaborates, "Genomics is fundamentally changing the way medicine is practiced, and it is particularly changing cancer care and how cancer is detected and treated. We're really bringing that revolution to the dermatologist. We do that with our noninvasive genomics platform. We have something called the Smart Sticker, which is a special adhesive patch that can collect the sample of the skin noninvasively. And it feels a lot like scotch tape, you just press it on the skin, and you lift it off. And from that skin sample, the tissue we've collected, we can run a variety of genomic analyses, and that allows us to determine whether or no

Tom Isett is the CEO of iBio which is pioneering the next generation of biopharmaceuticals. Their goal is to reduce the cost, time, and risk of failure involved with drug discovery and development by using a plant-based recombinant protein manufacturing platform to create small and large batches of desired proteins. Tom shares his career journey from working with the USDA to iBio where he is back to working with the little leafy green guys again. Tom explains, "We are a biotech, so we're a developer of biopharmaceuticals, specifically focused in oncology, fibrosis, and infectious diseases, where we're really trying to address some of the bigger unmet medical needs out there. But what I think really makes us unique is how we develop these new medicines, molecules, and candidates. We use plants to produce these new drug candidates." "And as we move forward, we also use artificial intelligence for the design of the molecules. So the combination of the two winds up putting us in the position where we have this re

Paula Ragan is the CEO and President of X4 Pharmaceuticals, which focuses on the rare disease WHIM Syndrome. Patients have a deficiency in their immune system, and as a result, this diminishes their ability to fight chronic infections, and they have a higher risk for severe cancers. Paula explains, "If one thinks about doing the right test, it's easy to diagnose, and the right test is doing genetic sequencing. There are commercially available panels, so it exists for the patient and physician communities. The current challenge is getting the physicians to recognize the test is needed. And then to also enable payment. X4 has done a great job in providing free testing through our PATH4WARD program." "Our lead candidate is called mavorixafor, and it's an oral once-daily capsule. And the amazing thing is its mechanism. What it does is binds to a receptor called CXCR4. And it basically shuts it down a little bit. It's kind of blunting the signaling that normally exists in the cell. And for these patients with WHIM

Dr. Luca Benatti is the CEO of EryDel, a biotech company developing and commercializing cell-therapy treatments for rare diseases using autologous red blood cell encapsulation technology to deliver drugs. With a focus on the rare genetic disease Ataxia Telangiectasia (AT), Erydel has just completed a multi-center, multi-national phase III trial for the disease. Their symptomatic treatment has been shown to delay the progression of the disease and could be part of a regular immunoglobulin therapy session for these patients. Luca explains, "The first characteristic of our technology since there are similar technologies that utilize red blood cells to deliver therapeutics, is that we are the only one that has developed a technology that can work at the bedside. So, it's really the only one that can use the patient's blood, and we don't need to have a centralized lab. The beauty of this technology is that it is the only one that can use a sustained release formulations technology - that is, the one that is actual

Eliav Shaked, CEO, and Catherine Bornbaum, Head of Clinical Operations and Partnerships at RetiSpec are using hyperspectral retinal imaging to find more accurate and earlier biomarkers of neurodegenerative diseases. As part of a regular eye exam, this non-invasive test can be administered by an ophthalmologist or optometrist using existing retinal cameras with the data being analyzed using AI to identify unique molecule signatures. Eliav explains, "What's unique about hyperspectral imaging or specifically hyperspectral retinal imaging is the fact that it's a tool that measures different coloring of elements in a much more detailed way. So, while we humans are limited to seeing and interpreting only three colors, hyperspectral retinal imaging can cover more than a hundred different colors in a very precise way. And it's really our way to get much more content from the back of the eye. And what RetiSpec is doing with that data is we're integrating our very own artificial intelligence that allows us to recognize

Dr. Sam Asgarian is the Chief Medical Officer of Scipher Medicine which is predicting drug response with precision immunology by building on the work of the Human Genome Project. Now that the human genome has been mapped, the next challenge is how to turn that information into use cases that are applicable in treating specific diseases. Sam explains, "The goal for us is to figure out which part, very specifically, of the autoimmune system is overacting and analyze that through a simple blood sample. We can take the blood in just a simple lab draw, just like other blood work, and analyze areas of perturbation or areas where the immune system specifically is overacting. That gives the physician enough to then treat it with a prescription that focuses only on that specific area which, of course, in the immune system is critically important because it's still serving a very important goal of protecting that person against disease. You don't want to shut down the overall immune system. You just want to quiet the a

Sunny Jain is the Founder and CEO of Floré by Sun Genomics and is driven to better understand the microbe cells that live inside of our intestinal tract and all around our body.  With a history of working at Illumina, Sunny is studying the DNA from those cells, the microbiome, to understand their function and how they contribute to our overall health. Sunny explains, "Sun Genomics has created the world's only precision probiotic, And it utilizes AI, DNA sequencing, biome chromatics, and e-health technology. Our technology helps us build the largest database of microbiomes that are sequenced via metagenomics, or whole genome sequencing, to understand the modulatory effects or the changing effects that food ingredients and probiotics can have for 80 plus million Americans that suffer from gut health issues every year." "So, you truly are unique, from a gut microbiome perspective. Our human DNA is 99.9 percent similar. So, we're very identical from a human DNA perspective. But the DNA from microbes of the gut, y

Tim Clackson is the President and CEO of Theseus Pharmaceuticals which has a mission to outsmart cancer resistance by developing pan-variant tyrosine kinase inhibitors (TKIs) that are designed to hit different types of cancer cell mutations. The goals of the therapy are to delay treatment resistant cancer as well as effectively deal with the variety of cancer mutations once they have occurred. Tim explains, "The unfortunate truth is that nearly all types of tumors at some point are going to develop resistance. We are at this stage now where some tumors that are caught early can be really cured and go into long-term remission. Still, there's a substantial portion of tumor types and certainly those that are caught a little bit later that unfortunately show an almost inevitable evolution of resistance. So, then the challenge becomes, can you delay how long that resistance takes to emerge? And then can you come up with therapies or approaches that will hopefully substantially prolong the post resistance treatment

Dr. John Krakauer is the Chief Medical Advisor at MindMaze, pioneers in digital neurotherapeutics focused on brain health and recovery from neurological diseases and injuries. John explains, "What we really mean by digital neurotherapeutics is the belief that one can actually administer a medicine in a way that's different from pharmacology, different from surgery or devices. But in fact, consider software as a pill so that you can actually have a software experience, an immersive gaming-like experience that can actually have an effect on a disease, in particular, neurological conditions in a way comparable to other medical approaches." "So MindMaze really would like to create immersive gaming software that allows the nervous system after injury or disease to undergo such change. We're different, I think, in so much that we realize that you can't just be an app on a phone or a tablet. You have to be in an immersive full-body experience that combines exercise, cognitive challenge, and skill learning. And so, w

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 awa

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

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

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 D

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 condit

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

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 tes

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 swelli

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, i

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 actual

Matt Murphy is the EVP of Network and Clinical Content at Cohere Health, innovators in utilization management and prior authorization for tests, services, and drugs. By taking a patient-first approach means reviewing cases on behalf of the Cohere health plan clients as well as with an eye to the health outcomes of patients. Matt explains," Utilization management really evolved as an adversarial exercise with health plans pitted against physicians. And if you think about the key areas where it's impacting patients and physicians more broadly, really it's three things. First is delays in care and access. Really there shouldn't be anything coming in between a patient and the care they need as long as it's clinically appropriate. So I'd say that's probably the first issue." "A few weeks ago, we reported out jointly with Humana, one of our first clients, on the impact we had in year one, and it was tremendous in terms of medical expense savings. But I think it goes back to the philosophical differences people have