Empowered Patient Podcast

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 sup

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 differ

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 p

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

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

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 #Neuro

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

Dr. Steve Worland is President and CEO of eFFECTOR Therapeutics a pioneer in the Genomics 3.0 category putting to work the research on selective translation regulator inhibitors (STRIs) and the key ways that cancer cells change their behavior and also change the immune system. Steve says, "There are two revolutions in cancer in the last 10 to 15 years-- targeted therapies and immunotherapies. And we think STRIs is a third way to compliment one or both of those. And really with the idea here is that you could control a cancer for a long period of time. It's very hard to completely eradicate every tumor and every cancer cell in your body.  "But if you can restore control, your immune system can control the cancer. Hopefully, patients can live for years knowing they have the disease but not having it debilitate their daily life." #eFFECTORTherapeutics #NSCLC #STRIs #cancer #SPAC eFFECTOR.com Download the transcript here

Bret Larsen is the CEO and Co-Founder of eVisit, a virtual care platform that is serving the largest health systems in the country to establish remote access for a telemedicine solution that works for patients as well as hospitals, doctors, and administrators. Bret says, "One of the things the pandemic accelerated in a big, big way was the consumerization of care. It forced us, as consumers, not patients, but consumers, to look for more options. With many physical encounters being closed or highly limited, we had to start looking for other options." "I strongly believe that the future exam room will be your living room, or your bedroom, or somewhere where you can be diagnosed and suggested treatment, and then directed to the most appropriate point of care, whether that be an asynchronous interaction, a synchronous interaction, or the physical exam room at the hospital. So, the interoperability piece is enormous. It stands to accelerate the improvement of patient outcomes in a big, big way." The eVisit Enterpr

John Zaleski Ph.D. NREMT, CAP, CPHIMS is the head of clinical informatics at Capsule. The topic is medical device integration to track progress and improve hospital care with continuous patient data collection and surveillance with prompts for action. John says, "Aside from the physics of actually connecting and pulling the data, there are all sorts of IT-related issues of how to store the data, how to correctly format the data for communication. Now, there are standards that have been developed over the years that are in use today that tend to make those types of challenges less formidable. But having data access from any location in the hospital continues to be a little bit of a challenge in terms of lower acuity settings versus higher acuity settings." "And so you have a mix of devices that do communicate, a mix of devices that communicate in a proprietary manner, and then medical devices that communicate more ubiquitously using standards like Health Level 7, various flavors of the Health Level 7 standard.

Emily Parks is a behavioral and patient advocate and the Founder of POP! --Pissed Off Patients-- with a mission to educate healthcare professionals and patients about medical PTSD. Born with a rare intestinal illness called Myopathic intestinal pseudo-obstruction, her own medical journey has led to awareness about medical PTSD and the drive to pursue a Ph.D. in medical trauma and become a patient advocate. Emily says, "I find as an adult, a lot of the trauma that I've received in the healthcare system and interacting with the healthcare system has impacted me in how I socialize with both providers and non-providers as much as friends." "Socially, it's impacted my thinking style. It's impacted my ability to trust and connect with others. And so that is what POP! is about. What is Medical PTSD, and how does it manifest in you? What are some coping mechanisms?" #MedicalPTSD #PTSD #PTSDAwareness #MedicalTrauma #PatientSafety #PatientExperience #RareDisease POPMedicalPTSD.org Download the transcript here

James McArthur Ph.D. is the CEO of PepGen and talks about applying their enhanced delivery cell-penetrating peptide technology to treat rare genetic neuromuscular diseases. The lead diseases PepGen is focused on are Duchenne muscular dystrophy, treatable with an exon 51 skipping oligonucleotide, as well as myotonic dystrophy type 1 (DM1), both rare genetic diseases with high unmet medical needs. James says. "For a long period of time, we've known that we can fundamentally change the outcome of many diseases with oligonucleotide therapeutics. But the great challenge has been getting enough of these oligos, as they're often called, into the cells where they need to do essentially the job of changing disease outcomes." #PepGenPioneers #Oligonucleotide #Neuromuscular #RareDiseases #DuchenneMscularDystrophy #MyotonicDystrophy #DM1 #DMD Pepgen.com Download the transcript here

Glenn Mattes is the CEO of TFF Pharmaceuticals, with a hybrid business model developing an internal portfolio of products and also working with external partners to apply thin film freezing technology. The first compounds are voriconazole, an antifungal being developed for treating invasive pulmonary aspergillosis, and tacrolimus, for lung transplant patients. Other compounds are targeting COVID-19. Glenn says, "So our thin film freezing technology has the ability to transform pharmaceutical products, mostly solid pharmaceutical products, into dry powder formulations. One characteristic that is common to most pharmaceutical compounds is that they're rather poorly water absorbed. So when they are made into a drug, the ability to get enough product into the bloodstream or the plasma means you have to dose the compound at a pretty high level, and you butt up against sort of the adverse events profile." "So, by using thin film freezing, you ultimately can lead to a lower dose of the drug being administered direct

Alain Maiore is the Co-Founder and CEO of Mnemo Therapeutics and has a mission to change the future of cell therapy.  The Mnemo platform EnfiniT is an integrated drug discovery engine that combines T-cell engineering technologies with greater target identification to transform the body's immune response to overcome currently incurable diseases. Mnemo has just raised $90 million in a Series A round, the largest round to date in France. Alain says, "I think our job is to make sure that whatever technology we put together, everyone that is in need of having access to these drugs gets access to these drugs. So it is our responsibility to combine the technologies and medical development and the manufacturing of these drugs so that we can size the necessity of financial performance and shoulder reward with accessibility to every patient. That responsibility is increasingly important in the world that is opening to us." @MnemoTx #MnemoTherapeutics #EnfiniT #Cancer #BloodCancer #CellTherapies #SolidTumors #DrugTarget

Dr. Pier Vincenzo Piazza is Co-Founder and CEO of Aelis Farma, a biotechnology company targeting disorders induced by external cannabinoids, the chemical compounds contained in cannabis.  These cannabis-related disorders include addiction, cannabinoid hyperemesis syndrome, and psychosis and are related to how the CB1 receptor is stimulated by cannabinoids. Pier says, "The CB1 is a very interesting receptor for several reasons. First of all, is one of the neurotransmitter receptors the most expressed in the brain. So in your brain, one of the neurotransmitter receptors that you have the most of is the CB1 that is consequently involved in a large number of functions." "Another thing that is interesting about these receptors is that people are all different, you, me, even your son and daughter are different. However, we all share something that is absolutely identical in all of us--that is, the CB1 because the receptor CB1 has no variance between human beings. It's one of the few receptors that is 100% identical

Jim Joyce is the Co-Founder, Chairman, and CEO of Sigyn Therapeutics, and we talk about removing inflammatory targets from the bloodstream with advanced blood purification technology. Jim says, "The next transformative step in the recipe of blood purification is the advancement of devices that can address the sources of inflammation, which could be a viral or a bacterial source, in concert with the depletion of inflammatory cytokines and other modulators from the bloodstream." "You need the ability to quickly modulate down these inflammatory cytokines, but you also need to be able to address the source of inflammation.  The source of inflammation continues to stimulate the immune response. The source of inflammation in COVID-19 is obviously the viral pathogen itself and related bacterial infections. We're very focused on looking at bacterial toxins as well." #BloodPurification #COVID19 #Cytokinestorm #Cytokine #Sepsis  #Innovation #MedTwitter SigynTherapeutics.com Download the transcript here

Dr. Nick West is the Chief Medical Officer and Divisional Vice President of Global Medical Affairs at Abbott's Vascular Business. Nick points out the need to make sure the blood vessel is properly closed up at the end of a procedure to ensure no complications.  While the Abbott device Perclose™ has been around for quite some time, the ProStyle™ is the new version of it and is a unique suture mediated closure system. Nick reminds us that, "The old way of doing this is simply to press. Now pressing on the artery, usually, you have to press for at least 10 minutes. If it's done properly, that seals things very nicely, but there are many, many factors that affect the availability of what we call manual compression to seal these punctures. They include where the puncture was, how many times the physician needed to attempt to puncture the vessel before they got into it." Nick confirms this is the way bleeding has been stopped for hundreds of years and that pressing on the groin is medieval.  We talk about how this

Dr. Marc Semigran is the Chief Medical Officer of Renovacor and shines a light on BAG3-associated cardiomyopathy and the cardiac gene therapy in development to treat this rare genetic disease. Marc explains, "So, what we are focusing on are patients who have a genetic or familial origin of their dilated cardiomyopathy. Our first target is a gene described as BAG3, which is an important protein within the cardiac muscle cells. And some patients with heart failure have the heart failure because the gene that they have is mutated and is not able to fully express a functional protein." "We are studying our lead asset, which is REN-001, a modified virus that we believe will specifically target the heart. This will insert into the cardiac muscle cells a good copy of the BAG3 gene, which can then be expressed and allow those cardiac muscle cells to begin to function normally." #Renovacor #GeneTherapy #DilatedCardiomyopathy #DCM #BAG3 #RareDisease Renovacor.com Download the transcript here

Koenraad Wiedhaup, Founder and CEO of Leyden Labs, and his team have taken a proactive approach to overcome the challenges of protecting people from known viruses and new viruses. They are developing intranasal sprays containing biological molecules that can temporarily protect against a broad range of viruses.  This approach is between a vaccine that often gives long-term protection and a therapeutic necessary once someone is infected.  Koenraad says, "This actually gives people the autonomy to protect themselves and to take the nasal spray. And with that, they protect themselves but also others by not transmitting the virus. And indeed, therefore, the additional benefit is that you don't need a healthcare professional to do that. So, which of course, makes such a rollout also easier in the developed world, in places like the US and Europe. But also in more remote areas, where that might be even more important because of the limited availability of healthcare professionals." @LeydenLabs #LeydenLabs #Pandemic