Empowered Patient Podcast

Dr. Simrit Parmar, Founder of Cellenkos, is developing T regulatory cell therapies from cord blood to treat aplastic anemia, myelofibrosis, and acute lymphoblastic leukemia. Their three-prong strategy focuses on resolving inflammation and alleviating the burden of transfusions. Treg cells from cord blood are naturally tolerant and do not risk rejection, meaning they can be administered to patients without the need for matching. The cells can be consistently manufactured in a scalable way and distributed globally. Simrit explains, "Tregs, the T regulatory cells, are actually regulators of our immune system. So if you think about it every day as a human being, we face many challenges. We face many insults and injuries to our body, both externally and internally, but our body has a way to maintain that balance. The T regulatory cells are the cells that are the mastermind of making sure that any response by our body, for example, to get rid of an antigen or to get rid of an irritant does not overstay the welcome because our body utilizes the mechanism of inflammation to get rid of these injuries or these insults." "So umbilical cord blood is, or the umbilical cord is, the connection between the baby, the fetus, and the mother. And if you think about it, the baby is 50% mismatched to the mother, and nature allows it. Nature allows a permissive environment where the baby can grow and eventually give birth, and the whole species is advanced. What happens is that nature has allowed the tolerance, and one of the mechanisms by which this tolerance, the maternal-fetal tolerance, is induced by the very T regulatory cells that populate the conduit between the mother and the baby, is the cord blood. So what we did is harness the power that nature has bestowed upon these cells to just do one job and one job only, which is to resolve inflammation. This is the fundamental reason we went after cord blood as a starting material because as a physician and drug developer, we wanted to make sure that the product, the cells we're giving into the patient, are doing its job."  #Biotechnology #DrugDevelopment #LifeSciences #AplasticAnemia #Myelofibrosis #GVHD #ALS #Tregs #CellTherapies #TargetedTherapies #AutoimmuneDiseases   cellenkosinc.com   Download the transcript here  

Jay Hartenbach, the President and COO of Diakonos Oncology, is developing a dendritic cell therapy to treat late-stage and aggressive cancers like glioblastoma and pancreatic cancer. This approach uses the patient's cells and tumor tissue to generate a personalized treatment. The resulting vaccination tricks the immune system to attack cancer cells by making them look like a virus which the immune system knows how to eliminate. Jay explains, "Our vaccine works by using something called a dendritic cell from the patient. And so the dendritic cell is what we like to think of as the master of the immune system and somewhat the general that directs immune responses against threats, whether a bacterial threat or a viral, fungal threat, whatever it may be. The dendritic cell sits on top of the immune hierarchy. So, we take a patient's dendritic cell, and we also take a sample of that patient's tumor. We replicate effectively a viral infection with those tumor-derived antigens, the tumor-derived specific genetic material." "By mimicking how a virus would infect those dendritic cells with the specific tumor antigens, we're able to trick the body into thinking that the cancer cells are virally infected cells. And from there, the body's good at eliminating those cells because we've all fought viruses before, and we're all standing and walking around and alive today. We just take over or hijack into that immune response except instead of killing infected cells, the body is now targeting those cancer cells."  #Diakonos #Vaccines #Cancer #Oncology #Glioblastoma #PancreaticCancer #Tumors #DendriticCells diakonosoncology.com Download the transcript here

Dr. Pol Boudes, CMO of Rectify Pharmaceutical, highlights the importance of membrane proteins, specifically the role transporters play in how cells interact with their environment. Rectify is working on developing positive functional modulators (PFM) to address diseases related to dysfunctional transporters the first being primary sclerosing cholangitis, a rare liver disease with no current treatment options. The PFM can potentially restore the normal function of transporters involved in bile composition and secretion, which are key mechanisms in PSC.   Pol explains, "So you have different types of membrane proteins, but what we're working with are called transporters, so they’re proteins that are based on the membrane but channel components out and in the cells. So basically, it's a way for the cell to interact with its environment, and you have multiple types of transporter proteins. We're specifically working or started to work with one group of family of proteins that are called the ABC transporters. They're a very important function, and they can either be disrupted because there is a mutation in the protein, so it's a genetic disease, or they are potentially disrupted because there is just a functional deficit of this transporter. The protein is normal, but it's not functioning properly. So it's a functional deficit of what we call the wildtype protein." "So with the PFMs, what we're doing is to use small molecules that you can administer orally, and they bind to the transporter, and by doing so, they can modify the three-dimensional structure of the transporter and consequently the way this protein behaves within the cellular environment. So we correct the function of the transporter by changing the size, if you want, of the shape of the transporter. So we started to focus on this type of proteins and you have many potential diseases due to transporter deficit."  "We're focusing on one disease, liver disease. That's our lead PFM for a disease called primary sclerosing cholangitis, which is a disease of the liver. This very severe liver disease is also a disease that has no treatment available, which is a little bit frustrating because the progression of this disease leads to liver cirrhosis. And the only thing you can do at this stage is liver transplantation. As you might know, liver transplantation is problematic because it's a very complex process, it's also very expensive, and unfortunately, there is a shortage of transplants. So we're trying to address this problem." #RectifyPharma #PrimarySclerosingCholangitis #PSC #RareDisease #LiverDiseases #BileProduction #Transporters rectifypharma.com Download the transcript here

Neetu Rajpal, the CEO, and Co-Founder of Lilac Software, brings data and analytics tools to healthcare payers with a specific focus on addressing health disparities and improving benefits for Medicare Advantage members. The Medicare Advantage Star Rating program is a key incentive for health plans to be more innovative about data analysis to improve quality measures and receive financial incentives. Timely, reliable information in an accessible format and better data analytics improve decision-making, patient outcomes, and engagement with providers and patients. Neetu explains, "Health plans tend to have lots of data. They have lots of valuable tools, but those tools have data that is locked inside those tools themselves. So if you're, for example, part of an actuarial team, you may actually be getting PDFs, you may be getting spreadsheets, you may be getting access directly to a CSV file. You may get all these things on a one-off basis, and you have to make sense of all this data. The burden of cleaning and making use of this data falls on you." "This is exactly where lots of energy is lost, lots of labor costs are lost, and lots of efficiency is lost. So with Lilac, we're trying to make sure that all of this stuff of banal value is just available behind the scenes. This is exactly what tech is supposed to be doing for you. Help you operate at the top of your license. If you're an actuary, do actuarial things and let tech make sure that the data you need to do your work is available to you when you need it, and you can just rely on and trust it." "Yes, unfortunately, there is still a lot of paper in the process. Some of the paper is regulatorily required. So if you're a payer, you're required to send any of your members a directory of providers that are available. If they ask for it in paper, you are required to send them ID cards that can be paper. We all know about the fax machines and all of those things on paper." #LilacSoftware #DataAnalytics #HealthAI #AI #MedicareAdvantage #MedicareStarRating #HealthPlan #HealthcarePayers #HealthcareInsurance lilacsoftware.com Download the transcript here

Dr. Sean Ianchulev, CMO, previously CEO of Iantrek, is developing a new approach to treating glaucoma, which involves a biologic implant that can be customized to the patient's disease progression. This technology is designed to be more durable compared to traditional glaucoma treatments, which often require lifelong use of eye drops. This treatment is a combination of microinterventional technology with biologic tissue technology to transform the approach to eye care. Sean explains, "So we are a first not only for ophthalmology but also for medicine. We are the first, made of biologic technology, and we're to implant that tissue without any hardware, which is important because it minimizes implantable response. We can treat glaucoma patients who need intraocular pressure opening by opening and stenting the outflow pathway in a very unprecedented way. So, we've created a new categorical treatment, which we call bio-interventional."   "I think in cataract, we can say we've probably been able to cure that disease by cataract surgery and have a permanent cure. Unfortunately, glaucoma is difficult, affecting the optic nerve. And when you talk about neurodegenerative diseases, we're not yet there where we can cure them, but if we can slow them down, and we can slow them down to the point where it does not impact somebody's well-being and vision until the end of their lifespan, I think that's almost equivalent to cure because we've been able to enable them to have a productive life." #Iantek #Glaucoma #Innovation #Biointerventional #Microinterventional #Biotissue #EyeCare #Ophthalmology  iantrekmed.com Download the transcript here

Robert Den, Chief Medical Officer at Alpha Tau Medical, is changing cancer treatment using their Alpha DaRT technology to deliver a potent form of alpha radiation directly into solid tumors with minimal side effects. There is potential for the Alpha DaRT to be combined with immunotherapy and chemotherapy to further invoke an immune response. This one-time alpha radiation treatment has shown the ability to treat all solid tumors, only limited by the ability to deliver the Alpha DaRT directly to the tumor. Robert explains, "So alpha radiation is one of the three types of radiation that occur naturally, and it's been known as a very potent form of radiation for several decades now. The challenge with alpha radiation in the treatment of patients with localized disease and with solid tumors, meaning tumors not like leukemias or lymphomas but more like pancreas, lung, and prostate cancer, is that the alpha particles themselves aren't only able to travel a very short distance inside tissue or inside the cancer itself." "Before Alpha Tau and the Alpha DaRT technology, there was no pragmatic way to use this super potent and very safe type of radiation because you could not put enough alpha particles inside a tumor to cover the tumor with the radiation dose. What Alpha DaRT technology does is rely on the movement of what's called alpha-emitting daughter atoms." "So essentially, we take a biocompatible inert metal seed, which means that if you were to put this seed into the body, there would be no immune effect. Patients could have it inside them for the entirety of their life. Essentially, what we do is we cover this seed with a radiopharmaceutical called Radium-224. We just put Radium-224 on the outside of the seed. Then, we insert it either through a minimally invasive approach or using a different endoscopic approach inside the tumor directly. So now we have the seed covered with the radium inside the tumor, and this is basically where the magic happens."   #AlphaTauMedical #Cancer #TargetedRadiation #ClinicalResearch #RadiationOncology #Oncology #CancerTreatment #AlphaRadiation  alphatau.com Download the transcript here

Dr. Daniel Vitt, the CEO of Immunic Therapeutics, is taking a different approach to treating autoimmune diseases, focusing on neuroprotection, gut barrier restoration, and anti-inflammatory effects. These dual-mechanism small-molecule drugs are initially developed for multiple sclerosis and inflammatory bowel diseases. The lead candidate for MS aims to reduce relapses, slow disease progression, and help prevent reactivation of Epstein-Barr virus due to the antiviral properties of the oral drug.   Daniel explains, "If you look back over the last 20 years, maybe even longer, you figure out that there have been limited improvements in treating big chronic indications like autoimmune diseases. Specifically, looking from the patient’s point of view, there are a lot of things missing. So, there are a lot of indications that are untreatable or not sufficiently solving the medical need or a lack of convenience for use, for example. Another thing that is highly underestimated usually is the safety and tolerability of the drugs. And it's our mission to work on having better drugs with better safety and tolerability profiles to offer patients more convenient and easy-to-use therapies."   "That is something currently not addressed by the drugs available for patients today. Therefore, our drug is making a difference because our mode of action is a dual mode of action. On the one hand, the molecule is a potent anti-inflammatory drug by inhibition of a protein called DHODH. Secondly, we also have our molecule, a very good activator of a protein called Nurr1. This is a neuroprotective factor and plays an important role in the different areas of the brain, directly in the neurons in the brain, but also in the environment of the brain. That is why we believe Nurr1 activation is a key step forward to treating patients and offering a better benefit for slowing down the disability." "This is because quality of life is one of the important parameters for judging whether a drug does its job or not. We need to address this more in our industry, and therefore, I agree that it's an important piece. And with gut wall repair, you're more on the side of ok, maybe we can fix the problem in general and the body returns to normal behavior. So, in a perfect world, in further clinical trials, we can see cases of patients who maybe flip back to healthy status or disease-free status or something like that, for example, in celiac disease or other GI indications." #ImmunicThera #MultipleSclerosis #MS #AutoimmuneDiseases #InflammatoryDiseases #EpsteinBarrVirus #UlcerativeColitis #IBD  imux.com Download the transcript here

Kent Dicks, CEO of Life365, has extensive experience in the remote patient monitoring industry and is focused on developing wireless and cost-effective RPM technologies to connect patients to their healthcare providers and improve patient engagement and care. The mission is to support the shift from reactive to proactive care by using RPM data fed into AI and machine learning systems to identify trends and the need for early intervention. Working with partners and large enterprise clients, Life365 is connecting patients in rural areas with RPM technology. In an arrangement with the US Department of Veterans Affairs, the company is part of a remote patient monitoring initiative to identify biomarkers and early warning signs so patients can stay compliant with their therapy plan. Kent explains, "I've been in the industry for about 18 years now and dealing with remote patient monitoring. We've been fortunate to have built the vision when there wasn't reimbursement and when remote patient monitoring had been started by previous companies that were using wired technology. Our strategy was to use wireless and cost-effective technology to try to get into see patients or to connect with patients who were especially in hard-to-reach places like rural locations, trying to connect them to their doctors." "So Life365 is about making sure that we can help support the new models of care that are emerging, that are out there, value-based care, the ability to connect people in rural locations. The ability to get data cost-effectively from patients used to mean feeding the data directly into their HER. It's now really important to be able to feed the data into artificial intelligence and machine learning systems that are going to be used a lot to be able to monitor patients and see trends occurring. This is way before a patient decides that they need to go to the emergency room and be admitted to the hospital."  #Life365 #CareAccess #CareatHome #ConnectedCare #CommunityCare #Datasharing #Healthcare #HealthEquity #HospitalatHome #PopulationHealth #RemotePatientMonitoring #RPM #ValueBasedCare #VBC #VirtualCare #VeteransCare Life365.Health Download the transcript here

Dave Rosa is President and CEO of NeuroOne, a company focused on advancing neurotechnology by developing thin-film minimally invasive electrode technology that can perform diagnostic and therapeutic functions for neurological conditions. The company's OneRF ablation system allows doctors to identify and ablate problematic brain tissue in a single hospitalization. NeuroOne is expanding its technology beyond the brain to include the spine to treat pain and is addressing various neurological conditions through ablation, stimulation, and drug delivery.   Dave explains, "The OneRF ablation system is an electrode that looks very similar to the electrode that we got cleared shortly after our last discussion. So that was in October of 2022. But that device only had diagnostic capabilities. So what doctors would do is they would implant those electrodes, and they would leave the patient in the hospital for, say, up to a week on average." "The goal was for the neurosurgeon and neurologist to be able to identify the parts of brain tissue that were triggering seizures or causing neurological issues. Then, after that procedure was done, the devices would be removed, and patients would go home. So that was kind of the old way of doing it."   "What we did was to have the ability to reduce the amount of hospitalizations a patient would have to experience. We did develop this electrode technology that looks very similar to the diagnostic one approved in April but with the capability of not just doing the diagnostics but also the ablation. So the difference here is that when the patient comes in for the diagnostic procedure after that concludes, the doctor will wheel them into the patient's room, not the operating room."   #NeuroOne #Neurosurgery #ElectrodeTechnology nmtc1.com Download the transcript here

Michael Demurjian is Chairman and CEO of Aspargo Labs, a drug development company focused on improving drug delivery through suspension-based formulation and smart device technology. Traditional pill-based drug delivery has significant limitations, with poor absorption rates and difficulties swallowing for many patients. The company's initial focus is on an oral spray formulation of sildenafil to treat erectile dysfunction that has shown rapid absorption. Suspension technology can be applied to reformulate many prescription and over-the-counter drugs to improve drug delivery, adherence, the onset of action, and user experience. Michael explains, "Here at Aspargo we have a proprietary technology, both in the suspension and in how we coat it, so that we can mask the taste of the drug itself. And so I know you're going to probably go down the path of our first drug to market, and I'll answer the next question, but masking is key because there are two things that happen with the masking. One is you make it more of a pleasant experience. People are more willing to adhere and comply than administering the drugs. If you don't, people are going to say, my goodness, that tasted horrible, and I'm not going to do that again. Or they're going to be resistant in particular children, they won't do it."  "Well, 30 years in the drug development business, and I will tell you, you bring an ED product to market where you're showing a five-minute absorption time, it captures a lot of people's attention. So we've been very well received. Our first drug to market is an oral spray, sildenafil, which is the active ingredient in Viagra. And what we've shown is a five-minute absorption time. So, it has a lovely peppermint flavor. Now, here's the key there. Sildenafil is extremely bitter. It's one of the top 20 most bitter drugs that is manufactured. Our taste is peppermint. Through the proprietary technology that we have, we've successfully bonded the flavor agents right to the molecule without damaging it. I'll tell you, it's an interesting experience. Typically, it's the medical community. But when you're looking at a drug like Sildenafil, it's a consumer experience as well." "You have to remind yourself the efficiency of absorption is critical. We look at patients, particularly in oncology, where we know that if they miss a dose of a medicine, cancers rapidly reproduce. So, missing one dose can set a patient back. What we look at and focus on is two things. One is our device to remind patients to dose their medicine. And if they forget that their physician, the physician assistant, or the office manager can intervene and text or call them saying your device just said you didn't dose. That's key." #AspargoLabs #DrugDelivery #ED #ErectileDysfunction #Sildenafil #DrugAdherence #MedTech aspargolabs.com Download the transcript here