Empowered Patient Podcast

Dr. Michael Racke is the Medical Director at Quest Advanced Neurology, which is extending the options for neurologic diagnoses by looking for biomarkers in blood tests. Michael explains, "And now, we're very much involved in developing plasma-based biomarkers to help diagnose Alzheimer's disease. Alzheimer's disease was a disorder that was initially diagnosed pathologically. You had to find the plaque and tangles in the brain at autopsy. Over the last 20 years, they've been able to show that with spinal fluid testing, you're able to identify changes in the cerebral spinal fluid suggestive of Alzheimer's disease. And now, we're also able to do that with a blood test." "And for example, one of the ways that people were first able to do an evaluation for Alzheimer's disease in living people was to do a PET scan and actually demonstrate that you could do an imaging test that would show the beta amyloid plaques." "So, Alzheimer's disease was diagnosed by finding these plaques. The plaques are caused by aggregation of peptides of beta amyloid. And then the other thing was the neurofibrillary tangle. And that's actually the aggregation of a molecule called tao within neurons. And the combination of these two things results in the death of neurons and the loss of synapses, which eventually leads to the cognitive dysfunction that you see in Alzheimer's disease." #QuestDiagnostics #Alzheimers #Dementia #Biomarkers #BloodBiomarkers #PlasmaBiomarkers #NeurologicalDiseases #LewyBodies #LewyBodyDementia questdiagnostics.com Download the transcript here

Dr. Guenther Koehne is the Deputy Director and Chief of Blood and Marrow Transplant and Hematologic Oncology at the Baptist Health South Florida Miami Cancer Institute. They are part of a clinical trial for an approach that protects healthy cells from the toxic side-effects of targeted blood cancer treatments with a focus on acute myeloid leukemia (AML). Guenther explains," And then, we have the opportunity to administer medications or CAR-T cells, for example, post transplantation, that are specifically targeting CD33, which then presumably allows us to specifically target residual leukemia cells." "So, my institution, Miami Cancer Institute, and before that, I was at Memorial Sloan Kettering Cancer Center, where I implemented the purification of stem cells from the stem cell product, from the donor stem cell product, allowing to deplete the T cells and other cells that were not necessarily needed for a successful transplant while we have a purified stem cell population. And on this clinical trial, we can now use the purified stem cells and send them to a specified laboratory to silence or down regulate the expression of CD33, using CRISPR technology so that the end product after this step will include a donor-derived stem cell population that is CD33 negative." #MiamiCancerInstitute #VorBio #CellTherapy #BloodCancers #AML #AcuteMyeloidLeukemia #AML #Leukemia #ClinicalTrial #Miami miamicancerinstitute.com Download the transcript here

Dr. Judy Chou is the President and CEO of AltruBio which is developing novel immunomodulators to treat immunological diseases such as autoimmune and inflammatory diseases. Their first-in-class agnostic antibody has a unique mechanism to regulate T cell homeostasis. Currently, there is a clinical trial for SR-aGVHD, steroid-refractory acute graft-host disease and an investigation is underway into using their second-generation molecule to treat ulcerative colitis. Judy explains, "So we have this specific approach, only targeting those activated or the so-called over-activated T cell. So when the T cell is supposed to come down to quieten and to bring back to homeostasis, go back to balance, it did not. It keeps going and going and going. And we have identified the key target to turn it down to the balance point to tell those T cells it's time to rest, to downregulate this group of T cells. And that's the approach you are taking. That's why AltruBio is so unique in addressing immunological diseases." #AltruBio #ImmuneModulators #ImmuneDiseases #TCells #GVHD AltruBio.com Download the transcript here

Jeff Cleland is the Chairman, President, and CEO of Ashvattha Therapeutics, initially focusing on demonstrating the ability to deliver drugs that cross tissue barriers like the blood-brain barrier and the blood-retinal barrier to attack inflammation. The company is part of the emerging era of Hydroxyl Dendrimer Therapeutics, HDTs. While starting with ophthalmology and treating wet age-related macular degeneration and diabetic macular edema, they are also running trials with long haul COVID patients. In addition, they are investigating neurological diseases like Alzheimer's and Parkinson's with the ability to measure the amount of the drug that gets into the brain. Jeff explains, "The secret is the platform that was developed by our founders at Johns Hopkins. It's a husband and wife team that really focused on trying to figure out how to get across tissue barriers. So, they empirically went about looking at different nanomedicine platforms, and they came across what's called a hydroxyl dendrimer platform." "What's unique about this is the density of hydroxyls on the surface makes it look like water to the body, so when it goes into the body, it doesn't get taken up by cells or tissues unless there's inflammation present. Then as it goes by those areas of inflammation, the cells grab the hydroxyl dendrimer and take it inside. Then we could treat intercellular targets, unlike other approaches where it hits all cells." #AshvatthaTherapeutics #HDT #HydroxylDendrimerTherapeutics #WetAgeRelatedMacularDegeneration #DiabeticMacularEdema #Inflammation #LongCOVID #COVID #Alzheimers #Parkinsons AVTTX.com Download the transcript here

Dr. Todd Johnson is the Chief Growth Officer at M2GEN an oncology-focused health informatics company. Its mission is to connect cancer patients to a precise cure using its data platform to analyze longitudinal clinical and genomic data. With a permission-based approach to data collection, M2GEN looks at the information related to the patient and from a tumor biopsy. This data analysis allows M2GEN to understand how patients respond to existing cancer treatments and gain more knowledge about the science behind cancer so that innovations and new precision medicines can be developed. Todd elaborates, "Let's call the first the natural genome or the normal genome of a patient, also often called the germline genome. We provide information on germline as well as on the genome of the cancer. The reason why that's really important is that cancer is a disease of DNA, and it's a disease of how DNA changes over time or mutates. Those mutations are what cause a cancer to actually occur in the first place, for it to be able to evade the immune system, which is an important element of cancer progression. Then cancers can continue to mutate during the course of their life as they grow. That access to what we call serial specimens, that access to additional information about the cancer and how it continues to change from the body's normal or the patient's normal DNA, is a really important part of unlocking the mystery of what's happening here." @M2GEN #DataScience #DataSolutions #Genomics #CancerResearch #CancerTreatment #PrecisionOncology M2GEN.com Download the transcript here

Laurent Levy is the CEO of Nanobiotix, which is applying nanophysics to direct radiation therapy more efficiently to cancerous tumors while reducing the side effects of damage to surrounding tissue. This intratumoral injection of nanoparticles maximizes the concentration in the tumor and minimizes the rest of the body exposure. Starting with soft tissue sarcoma, they are expanding to head and neck cancer with plans for other indications like pancreatic, liver, and breast cancer. Laurent explains, "We thought, at Nanobiotix, that we may find something different. Rather than making better radiation therapy equipment, we thought about the problem in a different way. We did develop a product that is an injectable that you will inject directly into the tumor. And, when the product is in the tumor -- they are based on nanoparticles, tiny objects -- these objects will go into the cell, and they will sit there. They'll do nothing until the patient is getting the usual radiation therapy." "And we've been designing those objects to be able to absorb the energy of radiation. Therefore, when the patient is getting radiation, the x-ray will cross the cancer cell, and the nanoparticle in that cancer cell will absorb the energy and will deliver around them a high quantity of damage and will kill the cancer cell more efficiently." @Nanobiotix #Nanomedicine #Nanophysics #Nanotechnology #Nanoparticles #RadiationTherapy #Cancer #SolidTumors #HeadandNeckCancer Nanobiotix.com Download the transcript here

Dr. Jacob Donoghue is the CEO and Co-Founder of Beacon Biosignals, a computational neurobiomarker discovery and deployment platform taking EEG readings and applying their algorithms to identify novel neurobiomarkers that might be related to a disease that can be modulated by a new therapy. Jacob drills down, "We anchor these biomarkers that we are able to identify off the world's largest annotated EEG database, which we have at Beacon, to be able to really put things into context. When we make a discovery, we can really see, even in a small cohort of patients, how does that relate to both other patients with that related disease or also healthy normal patients? "Beacon was born by looking at the massive opportunity and unmet need in patients with neurological and psychiatric diseases and to take in all these long data sets of electrical brain activity, EEG data, to be able to provide the same types of tools that were transforming digital pathology, radiology, and GI to better characterize diseases of the brain and help novel therapies make it through the clinical development process into patients." "One of the traditional endpoints that persist in rare diseases for epilepsy is often the seizure diary, where you ask the parents, in the case of pediatric genetic epilepsy, you ask the parents to annotate either in a paper notebook or in an app when they see a visible event that resembles a seizure or is a seizure event." "In some of the kids that we've started analyzing, we see that the patient reported seizures, the seizure diaries, will show only 10 or 20 seizures a day, but when you put an EEG on, you actually capture hundreds of seizures per day. And similarly, we have other kids where they're actually having 0 seizures a day, but the patient diaries often will report 10 or 20 seizures a day." @biosignals #BeaconBiosignals #EEG #Epilepsy #RareDiseases #SleepDisorders #Neurology #NeurologicalDiseases #NeuroDegenerativeDiseases #PsychiatricDiseases #ML #MachineLearning beacon.bio Download the transcript here

Dr. Jennifer Jones-McMeans is a Divisional Vice President of Global Clinical Affairs at Abbott Vascular Business and she sheds light on the FDA guidance to encourage sponsors of clinical trials to include all genders, ethnicities, and under-represented patients to participate. New guidelines are more organized and provide better guidance for what sponsors of clinical trials should consider during their submissions to the FDA. Jennifer explains, "If you think about where clinical trials sit, clinical trials are part of the overall healthcare ecosystem. And if we consider that the healthcare ecosystem is to be inclusive, it is to make sure that there are appropriate treatments for women and different ethnic groups. Also understanding that possibly there could be differences in maybe therapeutic responses, or even considering access too. Clinical trials have to then move forward as the medical ecosystem is moving forward to ensure that there's treatment available for all patients. And so this is where again, this guidance is just a guideline to help support that pathway for clinical trials." "We do know that there are specific populations that, based on their access to health and lack of trust that can occur with the healthcare system, when it comes to clinical trials, they may actually not know and have an awareness of the clinical trial. But they also may not be connected with the appropriate medical system or within a medical system to even have that access. So that's some of the work that we know that the sponsors can do in reaching out." @AbbottCardio #ClinicalTrials #FDAGuidance #UnderservedPatients #UnderrepresentedPatients #MinorityPatients abbott.com Download the transcript here

Kathleen Aller is the Director of Market Strategy for Healthcare at InterSystems, a software company that focuses on high-volume data management and mobile applications to manage healthcare data. By taking advantage of the InterSystems capabilities on the AWS platform, young startup companies can use its technology to help them cost-effectively build interoperable applications or data-intensive applications and scale up as needed. Kathleen explains, "In fact, there are more than a billion records managed in our technology. One of the groups we've been working with a lot in the last few years are payer organizations who are responding to government regulation that says you have to make the information you hold about patients available to them. You need to do it using a standard called FHIR, Fast Health Interoperability Resources. They have to be able, then, to import that data into applications so that they can do things with it." "We've been involved in digital health practically since its inception. Some of that is managed in applications built by partners like Epic, like 3M, who use our data management platform at the center of what they do. Some of that is in applications that we build on our own platform, so our HealthShare Interoperability engine connects up records for citizens around the world and gives a seamless longitudinal record. Our TrakCare EHR is used outside the US to document and deliver care." @InterSystems #AWS #DigitalHealth #Healthcare #MedicalApps #HealthcareApps #FHIR InterSystems.com Download the transcript here

Keith Palmer is the Worldwide Vice President for Hip Reconstruction at DePuy Synthes, which is the orthopedic company of Johnson & Johnson. DePuy Synthes is focused on adapting their technology, techniques, and implants to provide stability, flexibility, and durability to an ever more active patient population. Keith reports, "When we think about our surgeons and our patients and keep them in mind, we have solutions that address unmet needs in patients that lead to improved outcomes. So, through our anterior advantage, our portfolio solutions, in particular, patients benefit from shorter hospital stays, quicker recovery times, and reduced use of pain medication when they're surgeon implants or comprehensive systems for hip replacement surgeries." "Our patients today are highly educated on different surgical technique offerings. They even think about what's best in terms of meeting their needs, the quicker recovery, and even going to outpatient surgery centers and ensuring that the time is reduced to help them get back to their active lifestyle quicker. But they're continuously involved in goal planning with the surgeon to be able to address some of these needs." @DePuySynthes #HipReplacement #HipReplacementSurgery jnjmedtech.com Download the transcript here