Empowered Patient Podcast

Craig Parker is the Chief Executive Officer at Surrozen. Their research in the Wnt pathway and proteins identified these are critical to maintaining tissues in our body and are found in tissues as diverse as the lung, intestine, liver, sensory hair cells, and the eye. The company is first targeting a patient population with a severe alcoholic injury to the liver who are not eligible for transplant but would benefit from regeneration in their liver. Craig explains, "It really starts with a signal outside the cell that gets transmitted inside the cell by this Wnt signaling pathway, which in turn activates a number of genes that are responsible for these processes, like tissue repair or embryonic development, or renewal of stem cells." "The Wnt proteins actually don't dissolve very well in the circulation, and they really only act within a few cells distance from where they're produced. What made this pathway very difficult to try to modulate for therapeutic use is that these proteins, you can't just copy one of these proteins and inject it and have it activate this path in a distanced cell. These proteins are quite unique in the way they only work one or two cells away from where they're produced." #Surrozen #wntpathway #wntproteins Surrozen.com Download the transcript here

Dr. Richard Watson is a Co-Founder of Motient and has taken on the challenge of creating an efficient solution to solve the puzzle of how to move patients from one place to another with a focus on rural healthcare and rural hospitals. Motient was founded as a medical transport company that did air and ground transport.  They have now created Mission Control which is even more critical in this time of the Delta resurgence to help manage decisions in the care of all patients. Richard says, "The sending facility environment is different than the receiving facility. That's different from the EMS transport people. And everybody's got their head down doing solid work, but the idea that we could actually link arms and make that work better, improve the outcomes, improve patient care. I don't think that was something that was the focus. I mean, we're too busy measuring other things in the system to understand how that affects patient care. But every aspect of quality and sustainability is affected how patients move in the system." #PatientTransfer #RuralHealth #RuralHealthCare #RuralHospitals #Healthcare #EmergencyMedicine #HealthIT Motient.io Download the transcript here

David Katz is the Chief Scientific Officer at Sparrow Pharmaceuticals. He is exploring the possibility of blocking the formation of intracellular steroids to reduce the side effects of steroid medicines and conditions related to the overproduction of cortisol in the body in such diseases as Cushing's Syndrome and autonomous cortisol secretion. David says, "Cortisol is made in an organ called the adrenal, and then it circulates throughout the body. And endocrinologists tend to think about the cortisol that's circulating as really being the effector of both the good effects, so immune suppression that you want in a patient who has a transplanted organ or an autoimmune disease, as well as all the bad effects." "What we recognize actually is that most of the receptors of cortisol are within the cell and that it's the steroid that's in the cell that matters. And most of that actually is made by a different pathway, by an enzyme called HSD1, which is the target of our drug." "And the magic of that is the immune suppressive actions of the steroids almost uniquely seem to be dependent on systemic steroids more than the intracellular steroids. And so, by blocking the formation of this pool of intracellular steroids by inhibiting HSD1, we can block many of the bad effects without necessarily meaningfully altering the desired effect in patients who are taking steroid medicines." @SparrowPharma #Steroids #Corticosteroids #Cortisol #CushingsSyndrome #PolymyalgiaRheumatica #IntracellularSteroids #RareDiseases SparrowPharma.com Download the transcript here

Hideki Garren MD Ph.D. is the Chief Medical Officer at Prothena with a mission to advance the pipeline of candidates for neurodegenerative and protein amyloid diseases, including Alzheimer's and Parkinson's.  We talk about the recent FDA approval of aducanumab and its impact on research. Hideki says, "As we've shown at the AAIC, the Alzheimer's Association International Conference last week in Denver, we highlighted two out of three of our Alzheimer's disease potential treatments. One of them is PRX012, which is our anti-Aβ antibody. The second one that we showed there was a dual, Aβ-tau vaccine. And then thirdly, we have an anti-tau antibody, which is being developed. It was not shown at the AAIC, but we're also developing that. So these three molecules we're developing for Alzheimer's disease. And this momentum has really applied to us as well." #Prothena #Alzheimers #aduhelm #ProteinAmyloidDiseases #NeurodegenerativeDiseases #Vaccines #AAIC21 Prothena.com Download the transcript here

Karen Chen is the Chief Executive Officer of the Spinal Muscular Atrophy Foundation.  With a background in drug development having worked in pharma and biotech in neurodegenerative diseases like Alzheimer's and Parkinson's, Karen brings a deep understanding of the value of treating rare diseases that are genetically defined. Karen explains, "The foundation is a little unusual as far as a nonprofit because we are really focused on our sole mission, which is to accelerate the development of treatments for SMA. And unlike other organizations, we're really not focused on raising awareness or raising funds as much as some of our sister SMA nonprofits. So we have really partnered with biotech and pharmaceutical companies to develop drugs for SMA. And SMA is one of the leading genetic causes of death in young children. It's actually the leading genetic killer of children." "So even though there are three types, four if you include the adult form of SMA, it's all caused by the same underlying missing gene. The differences in severity are really a result of this backup gene that I talked about, SMN2." #SMA #SpinalMuscularAtrophy #RareDisease #SMN2 #Evrysdi #Spinraza #Genentech #Biogen #SMAAwarenessMonth SMAFoundation.org Download the transcript here

Sanjay Shukla is the President and CEO of aTyr Pharma, focusing on the extracellular functionality and signaling pathways of tRNA synthetases. The lead clinical product candidate ATYR1923 is a potential disease-modifying therapy for patients with severe inflammatory lung diseases, including interstitial lung disease pulmonary sarcoidosis. Sanjay explains, "Dr. Schimmel, for many years, has been really interested in an enzyme in all of our bodies called tRNA synthetases. These are basic building block enzymes that help us make proteins. They work inside the cell by shepherding an amino acid to a tRNA, and this, in turn, helps us make proteins. For many years, it was thought to be a kind of well-understood class of enzymes." "Dr. Schimmel discovered, and he published this in both Science and Nature papers about 12 years ago, was for some reason these enzymes break apart into fragments. Those fragments migrate out of the cell, and they travel to different tissues and organ systems in our bodies, and there they play a very different role, a nonenzymatic role. In fact, they are mostly involved in controlling local immune environments. So our job at aTyr is to really look at this new protein class of therapeutics and determine where they might best fit in helping patients and different disease pathways." #aTyrPharma #PulmonarySarcoidosis #ILD #Cancer #Immunology #raredisease #tRNA #Bispecificantibody $LIFE aTyrPharma.com Download the transcript here

Dr. Elizabeth Marshall is the Director of Clinical Analytics at Linguamatics an IQVIA company, where she is responsible for clinical oversight of all healthcare projects with a focus on the application of Natural Language Processing in drug development and healthcare. NLP is a type of artificial intelligence that is concerned with the interaction between computers and human language with an ability to find linguistic patterns in unstructured data. Liz says, "I commonly work with teams of people that don't always understand the subject matter. There was one time when I was told certain corpora of patients' data had no mention of pain, which as a physician immediately made me suspicious, and a red flag." "So I dug a little deeper into the data and found the data came from rheumatology. So I knew there had to be some abbreviation or something. Something was missing. For some reason, this group decided to abbreviate pain as PX, which is not a common way of abbreviating it. So, we changed the algorithm, and as you can imagine, the pain was everywhere in the records." @Linguamatics #NaturalLanguageProcessing #NLP #ML #MachineLearning #AI #ArtificialIntelligence #ClinicalTrials #DrugDevelopment #SDOH #EHR Linguamatics.com Download the transcript here

Dan Temple is a patient advocate for those with Spinal Muscular Atrophy (SMA) and he shares his treatment journey and actions he has taken to raise awareness about SMA.  We also talk about the drug treatments that have come onto the scene since he was first diagnosed when Dan was 2 years old.  He is now 49. Dan says, "Like I said, very important was that my lung function improved. I probably gained about ten years back from what I lost. I'm still on a feeding tube, but I couldn't eat by mouth for seven years, and the drugs helped my muscles start working better. So I can eat and drink by mouth a little bit. I don't do it a lot, but it's nice to be able to do it occasionally and have some food that I enjoy. And my hand, of course. The one finger I have is kept pretty strong. Thank God because if I lose that, I've got nothing." "In general, it's kept me from getting worse, and when the drugs came along, I was at the point health-wise where I probably didn't have a lot of time left. My lungs were getting so weak that I was going to have to be on a respirator soon. So, basically, the Spinraza and Evrysdi both saved my life and gave me another 20 years, probably." #SMA #SpinalMuscularAtrophy #RareDisease #Evrysdi #Spinraza #MDA #MuscularDystrophyAssociation #PatientAdvocate #Genentech #Biogen #SMAAwarenessMonth  

Professor Florian Solzbacher is the Co-Founder and Chairman of Blackrock Neurotech and is focused on expanding the options, understanding, and marketing of brain computer interfaces and neuro devices. I asked Florian about their new project underway with Northwestern University and DARPA called the NTRAIN Project. He said, "The key driving force behind that project is to try and help counter the effects of jet lag and dysfunctions in the circadian rhythm. All those of us who have been traveling internationally a lot will know that it usually takes you a few days to adjust your inner clock to a different time zone that you're in." "What has been shown in initial research is that there are ways to help adjust the internal clock by essentially generating some of those same peptides that regulate our circadian rhythm. Then releasing them into the bloodstream on demand essentially sends clear clock signals through the body that allow you to adjust faster than you normally would." @BlackrockMicro #innovation #Neuroscience #Neurotechnology #BCI #Technology #Neurotech #RestoringFunction #TouchAgain #FeelAgain #TalkAgain #MoveAgain #BCIPioneer #braincomputerinterface #Research BlackrockNeurotech.com Download the transcript here

Dr. Oleg Bess is the Founder and CEO of 4medica and a practicing OB/GYN, and a strong believer in allowing patients to have easy access to their health data in order to equalize healthcare across all categories of patients. Oleg says, "4medica's mission is really to formulate a one patient, one record paradigm, where we are able to access a number of data sources for a patient. We have the engine that is able to, on-the-fly, real-time actually, add these records into the correct chart. Even in this country where there's no unique identifier for patients, we're able to look at all kinds of demographics data for the patient and on-the-fly place that record into the correct chart." @4medica #DataQuality #DataAccess #Healthcare #HealthIT #SDOH #Outcomes #DigitalHealth 4medica.com Download the transcript here