Empowered Patient Podcast

Scott Gray is the President and CEO of Clincierge and supporting the activities related to the observation of Rare Disease Day on February 28. Clincierge is focused on coordinating logistical, financial, caregiving, and translation services to facilitate participation in clinical trials by patients with rare diseases. With a personalized, local approach, they support sponsors of research in the recruitment and retention of patients by removing obstacles and reducing stress. Scott explains, "The intent is to provide an energy and a focal point that enables rare disease advocacy work to progress on the local, national, and international levels. The intent of improving access to treatment and medical representation for the many individuals who struggle with a rare disease and include their families who support and care for them. Since it was created in 2008, Rare Disease Day has played a critical part in building an international rare disease community that is multi-disease, global, and diverse but truly united in its purpose of expanding access for these unique patients." "From what we've observed in the rare disease space, there hasn't been a great effect by technology. Monitoring can happen, but a lot of the observational reviews that have to happen during the visit are only able to happen with going to the site. During the pandemic, as you mentioned, we were asked in some instances to move the healthcare provider to the home of the rare disease patient so that they didn't have to travel, especially if it was a patient who was immunocompromised. And if that wasn't possible, there were also instances where the sponsor budgeted for private jet travel so that an immunocompromised patient could visit the site." @Clincierge #RareDiseaseDay #ShowYourStripes #RareDiseases #ClinicalTrials  clincierge.com Download the transcript here

Sandy Macrae, CEO of Sangamo Therapeutics, talks about the promise and limitations of genomic medicine to find the most effective and affordable treatments for patients with autoimmune and inflammatory diseases.  Their CAR-Treg cell therapy platform allows T cells to be tracked throughout the body and activated to release chemicals and signals to reduce inflammation. Small molecule oral pills will always be used to treat diseases and vaccines to prevent them. The goal of genomic medicine is to eradicate the disease. Sandy explains, "We co-opted what's a very natural process and attached a whole series of things to it. The zinc finger gets us to the right bit of the DNA, and then we can either cut the DNA. We can turn it down, turn it off, replace a base, or replace a chunk of it. That's the advantage of the zinc fingers. It's natural. It's small. It's easily delivered and has a whole range of functions that allows us to choose the right tool for the right disease." "But there's going to be a growing space for diseases where there is a clear genetic influence or genetic participation in the disease, where a simple injection of a virus that takes the editing technology to the DNA can change the patient's course of their disease. I'm thinking of Sickle Cell Disease, where patients who were going into the hospital 10, 12, or 15 times a year are now not having any of those painful episodes. I'm thinking of Hemophilia, where they used to have to inject two or three times a week and have bleeding episodes, and they can now walk free and not worry about their bleeding." "But really, the excitement within Sangamo is the next-generation projects, the Tregs that we just spoke about, and a whole platform of brain-active conscription factors that we're working on." @SangamoTx #GenomicMedicine #GeneTherapy #CellTherapy #ZincFingers #Tregs #Tcells sangamo.com Download the transcript here

Professor Arik Eisenkraft is the VP of Clinical and Regulation at Biobeat, which has developed a short-term disposable chest patch that collects and transmits vital signs data to a cloud-based platform. They have also developed a rechargeable wrist device that collects the same kind of data and is designed for long-term use at home, in care centers, or for clinical trials. Using the optical technique photoplethysmography, they collect reflected light from the tissue not using the light that goes through the tissue, allowing for these form factors. Arik explains, “I can tell you that specifically at Biobeat, we had several very exciting clinical studies in which, for example, we managed to show that we can detect unique patterns of flu two days before people started to feel ill. That's amazing because now it can help you direct treatments or use all kinds of measures like isolation, as we saw early in the COVID pandemic.” “Biobeat is using an optical method, photoplethysmography, or PPG, which is widely used in all the past oximeters. I think that everyone now knows that because of COVID. We developed our own in-house sensor and the algorithms that are related to it. With most devices, you can measure pulse and blood oxygen saturation.” “With our device, we get a much stronger and clean signal. And by that, we can now track not only these two parameters but actually a total of 13 different parameters, including cuffless blood pressure, cardiac output, stroke volume, respiratory rate, temperature, all the five basic parameters that nurses have to collect wherever the patient is, whether in the hospital or in the clinic or at home.” @BiobeatT #RemotePatientMonitoring #RPM #MedicalDevice #Wearables #AI #Cardio #Noninvasive #HospitalatHome #DigitalHealth #Telemedicine #Healthcare #Medtech #PatientExperience #BloodPressure #CardiacOutput #StrokeVolume #HealthcareInnovation #Health #PersonalizedMedicine #PrecisionMedicine bio-beat.com Download the transcript here

Dr. Rakesh Suri, President and Chief Medical Officer at Fountain Life, reflects on the impact COVID has had on our understanding that, as a society, we are only as safe as the least protected member in our global community.  He emphasizes the need to diagnose pre-symptomatic and symptomatic conditions and the long-term value of advanced diagnostics to address individual and population health needs. Rakesh elaborates, "It's leveraging the latest science, shortening the preclinical latency gap between new diagnostic tests, which we know are scientifically impactful. Yet, it takes 10 to 15 years to become embedded in mainstream clinical practice. Fast-forwarding those things, curating them in a thoughtful manner so that not everybody gets the same suite of tests, but they're actually tailored for age, gender, and other types of, what we say, comorbidities or other sorts of things that are going on in people's lives." "We understand, through our decades of research, that fixing the heart valve, if it's leaking, it’s way too late. The heart never recovers despite the fact that they now have a normal heart valve. The same is true with any disease process in the body. If we wait until a disease manifests itself symptomatically, with symptoms, whether it's a heart attack or metastatic cancer or dementia or osteoarthritis, debilitating or arthritic conditions, we know that we oftentimes can never restore a normal human performance and normal human healthspan." #FountainLife #PreventativeMedicine #PrecisionMedicine #EarlyDetection #FunctionalHealth #ProactiveCare #Healthcare #DiseasePrevention #Longevity #Healthspan  FountainLife.com Download the transcript here

Anat Cohen-Dayag is the President and CEO of Compugen, which uses its computational discovery platform to identify new drug targets allowing them to develop new treatments based on these new targets. While most immunotherapies are based on proteins PD-1 or PD-L1, Compugen has discovered two other proteins involved in stimulating the immune system response against cancer. Anat elaborates, "In 2009, we discovered the protein called TIGIT. We discovered it computationally, and we sent it to publication back-to-back with Genentech. And this is the protein that is being now developed by Compugen and by others, Genentech being the leader in this field with phase three studies in the clinic. Their phase two data showed improvement for patients with non-small cell cancer." "Following this TIGIT discovery, we decided that we're not going to compete in the TIGIT space, per se, but that we would like to focus the company on a different type of family of proteins, and we discovered PVRIG. And PVRIG is, again, a protein that we believe has a role in stimulating the immune system response against the cancer. But it is not working only alone. It is a pathway that is working in parallel and in complement to TIGIT and to PD-1."   #Compugen #TIGIT #PVRIG #ImmunoOncology #OvarianCancer #ColonCancer #MachineLearning #ML cgen.com Download the transcript here

Wally Gomaa, the Co-Founder of SimplePay Health, provides employer-sponsored health insurance plans that bring price certainty to patients. The plan aligns lower costs with higher quality care, giving the individual transparency about the best providers in their community and out-of-pocket cost, which motivates them to choose higher quality care.  Wally explains, "If you think about what we're doing, we are simplifying the payment model and the plan design model not only to create a better experience. Health insurance is the most complicated industry on the planet. We're using insurance simplification to naturally self-motivate individuals to choose higher-quality providers on their own by getting rid of the complexity of the plan design. Giving the patient member, the plan participant, whatever name we're going to give it, a price out-of-pocket for every covered service before they go." "We make our model available on a national level in partnership with Aetna and their TPA company Meritain. And partnering with a Fortune four organization of their size and scope allows us to have legs and reach anywhere we want to in the country." "The result is not only better affordability, but more importantly, we're getting individuals connected with the best providers in their community and extending the quantity and quality of people's lives. We do find ourselves on the mission of being the life preservation company. We want to get people the best care that extends the quality and the quantity of their lives. And we use insurance simplification to be able to get there. " #SimplePayHealth #HealthInsurance #HealthcareInsurance #PriceTransparency #QualityTransparency #EmployerSponsoredHealthInsurance simplepayhealth.com Download the transcript here  

Dr. Joslyn Kirby is the President of the HS Foundation, which focuses on improving the lives of those who suffer from Hidradenitis Suppurativa, a skin condition caused by an overactive immune system. Generally people with this condition are misdiagnosed with skin infections and treated with ineffective antibiotics. As a member of the DermTech Scientific Board, Joslyn sees the promise of this non-invasive technology to make a diagnosis for someone with the disease or help make a precise therapeutic selection.  Joslyn explains, "This condition is not new. It's just that we've gotten better at giving it a name. It's gotten a lot more attention, which it deserves because it's so devastating to some patients but mildly annoying to others. Still, either way, it's impactful to people's quality of life. So being able to find it is number one. Number two, give it a name. And then, number three, really pursue treatments that are going to be more effective than just antibiotic courses now and again." "The nice thing about the DermTech technology is that it is non-invasive, and people with HS are already going through so much discomfort because of the inflammation of this condition. I really like a technology like this where I can promise them I don't have to hurt you to help you, but to be able to get clues from what's happening in the skin cells to help. We can either make the diagnosis for somebody who's been struggling or, to help make a therapeutic selection, to take an injection that deals with one part of the immune system rather than a pill that might affect a different part of the immune system. To be able to look at what is happening within these cells at a molecular level, I think, would really help both patients and physicians. So I'm looking forward to what we can do with this technology."  @HSOrg #HSFoundation #HidradenitisSuppurativa #HS #DermTechStratum #SmartSticker #PrecisionMedicine #PrecisionDermatology HS-foundation.org Download the transcript here

Shelley Davis, MSN, RNC, CCM, is the VP of Clinical Strategy at Lightbeam Health Solutions expert in moving from the traditional fee-for-service model to a value-based care system. Working alongside providers, Lightbeam is identifying opportunities to provide education and better care to underserved communities with less administrative burden. As part of the ACO Reach model, providers must develop annual health equity plans to identify gaps in access for the most at-risk patients. Shelley explains, "We look at population health management as a way to identify patients, identify members, identify those at the greatest risk to fall through the gaps, those that have the most complex conditions. We offer solutions through patient and provider engagement tools, leveraging technology to be successful in value-based contracts. We are continuing to offer new services as we evolve as a company to ensure that we are meeting the needs of the next generation of value-based contracts."  "What we've done at Lightbeam and certainly other population health vendors are doing or should be doing is leveraging analytics, identifying those opportunities to surface those patients who are experiencing or those most at-risk members. Then developing initiatives around that, identifying those who are facing transportation barriers, those living in a food desert, those most likely to have utility instability. You can partner with a community-based organizations, you can develop organizational initiatives, and you can address educational gaps to make sure that they're getting the care that they deserve." #LightbeamHealthSolutions #PopHealth #PopulationHealth #ACOReach #CareDelivery #HealthEquity #RiskBearing #PatientOutcomes #SDoH #SocialDeterminantsofHealth lightbeamhealth.com Download the transcript here

Dr. Adam Mendelsohn, the CEO of Vivani Medical, seeks to enable people to receive the maximum potential benefits from medicines while minimizing the associated burden with taking those medicines. Drug implants overcome forgetfulness, procrastination, and other human behavior that impacts medication adherence. They anticipate expanding partnerships with various drug molecules and indications around their nano portal drug delivery technology. Adam elaborates, "When these medicines are evaluated in randomized clinical trial settings, they produce fantastic results. But these results don't translate into the real world in the way that they possibly could, and the primary reason for this is, as Charles Everett Koop, former US Surgeon General, once said, "Drugs don't work in people that don't take them." So, we are aiming to address this by developing miniature subdermal implants that can provide steady therapeutic doses over many months, guaranteeing medication adherence for these patients and, hopefully, improving real-world outcomes for chronic diseases." "The underlying technology we've demonstrated works with peptide therapeutics and some of the larger protein-based therapeutics. These are larger hydrophilic molecules that some of the other implant technologies have not been shown to be compatible with. We do believe that the technology could work with smaller hydrophobic molecules as well." "Down the road, we anticipate developing a portfolio of drug implants that can address adherence across a wide range of chronic diseases. But we have been pretty focused on a few different peptides and one in particular that we are preparing to begin a phase II clinical study early next year with around the type 2 diabetes indication. But the underlying technology is a platform that we think does have broad applicability." #VivaniMedical #MedicationAdherence #DrugImplants #ChronicDiseases #Type2Diabetes Vivani.com Download the transcript here

Dr. Alejandro Dorenbaum is the Chief Medical Officer of Reneo Pharmaceuticals, developing treatments for primary mitochondrial myopathy, a type of rare metabolic muscle disease. Now with genetic testing, there are special panels devoted to diagnosing PMM that can provide quick results for an accurate diagnosis. Unfortunately, no treatment options are currently available, and physicians only treat the complications. REN001, a drug developed by another pharmaceutical company, is being tested in clinical trials to address muscle strength. Alejandro explains, "The myopathies and, specifically, our lead program is in primary mitochondrial myopathy. Primary mitochondrial myopathies are a group of diseases where genes that code for proteins involved in mitochondrial function are affected. So these patients can have defects both in the nuclear genes of the muscle cells or in the mitochondrial genes, and then there are defects in the ability to manage fats in the cell as a source of energy." "We then did a Phase 1 trial in patients with PMM and treated patients in an open-label fashion for 12 weeks. We used several measurements to see if the drug could potentially work in these patients. One of the measurements we used was a twelve-minute walk test. This is a test where you ask the patient to walk as fast as they can for twelve minutes and you measure the distance they walk." "Now, people may have heard about the six-minute walk test as one that is used frequently, but we use the twelve-minute walk test because, remember, these patients have a problem in the metabolism of the fats. And the fats are usually metabolized when you move, late in the onset of exercise. When you first exercise, you use phosphocreatine, and then you use glycogen. It's only around five to six minutes into exercise that you start using oxidative phosphorylation, which is the mechanism by which you break down fats as a source of energy. So that's why we did a twelve-minute walk test. And interestingly, we saw increases in the distance patients can walk during the twelve-minute walk test." #ReneoPharmaceuticals #MitochondrialDisease #Mitochondria #RareDisease #Myopathies #MetabolicMuscleDisease reneopharma.com Download the transcript here