Empowered Patient Podcast

Kyle Filkins and Kris Napper were both born with SMA, spinal muscular atrophy. Meeting as kids at The Muscular Dystrophy Association summer camp, they have developed a friendship and a documentary. After beginning the use of the drug Evrysdi, their rare genetic disease stopped progressing, and the oral form of this medication allowed them to avoid spinal injections. Now in their thirties, Kris and Kyle are advocating for the community of those with SMA, working on their documentary, and looking forward to more adventures.  Kris explains, "Just shortly before Evrysdi was released and made available, there was another drug called Spinraza that was the first treatment ever for our disability. It was a spinal injection, which poses some complications as far as it's a procedure with some risk. It can be painful, and there's the risk of hitting nerves and causing damage. It's not the most fun thing to go through, but it was beneficial. So I was doing that." "So for young children, just being diagnosed can be life-

Marlena Del Hierro VP of partnerships and commercial strategy at Seniorly, which is a resource for families looking for senior living options and seniors planning ahead. The Best of Senior Living Awards determined by Seniorly highlights the best 150 communities around the country based on feedback from families, senior living advisors, licensing reviews, and media searches that might reveal something problematic or terrific about a facility.   Marlena explains, "We have an algorithm that then shares the top five to seven options for your loved one. So that's a unique piece, because of the data and the technology that we're constantly gathering from a variety of sources to help these families make the best decision for their loved ones."  "The reason why we're doing these awards is that we should be pushing the envelope. The Baby Boomers are fundamentally challenging these senior living communities. And the awards are just a sense of preparing us for more of a modern era in senior living."  @Seniorly #SeniorL

Dr. Steve Marcus, CEO of Cantex Pharmaceuticals, is developing drugs based on Azeliragon that might slow the metastasis of cancer and extend the survival rate for patients with brain and other cancers.  This drug has been shown to suppress the RAGE receptor, which is expressed on the surface of cancer cells and some immune cells. When RAGE is over-activated, as it is in several cancers and inflammatory diseases, it can cause pathology. Steve elaborates, "Azeliragon inhibits something abbreviated as RAGE, the receptor for advanced glycation end products. Originally, it was thought that RAGE is very important for the progression of Alzheimer's disease. And because of that, a company called vTv Therapeutics initiated large-scale clinical trials of Azeliragon for Alzheimer's disease. Unfortunately, those trials failed to show effectiveness of the drug in Alzheimer's disease, but did demonstrate very robust safety. The drug is given once a day and was given to patients for periods up to 18 months, and it was very

Dr. Spyros Papapetropoulos is the President and CEO of Bionomics, a biotech company focused on central nervous system disorders, particularly social anxiety, and post-traumatic stress disorders. Social anxiety is a disease primarily of adolescents and young adults, and the social isolation during COVID  contributed to a rise in the number of people experiencing this condition. The Bionomics drug in development is BNC210, a therapeutic that can be taken on schedule an hour before conducting an activity likely to induce anxiety.  Spyros elaborates, "Social anxiety disorder is actually known as social phobia, and it's very different, because everyday interactions cause significant anxieties. And they're very situational, so you become very self-conscious. You feel embarrassed and have a fear of being scrutinized or judged negatively by others. It is considered a very, very important chronic mental health condition that, if left untreated, leads to comorbid depression, even suicidality, and addiction down the ro

Matt D'Onofrio, Co-Founder, President, and Chief Operating Officer at Evoke Pharma, is developing a treatment to help patients with diabetic gastroparesis.  This little-known disease is thought to be due to damage to the vagus nerve, interrupting the communication between the brain and gut, stopping the stomach from functioning normally. Current treatments are a pill required to move through the stomach.  Evoke has developed a nasal spray to treat the condition that bypasses the stomach and provides predictable relief. Matt explains, "We developed a product called GIMOTI, or nasal metoclopramide. The interest behind doing so is to completely bypass the stomach to get a dose on board for patients. The unpredictable nature of the stomach makes dosing with pills very unpredictable. We know that a number of patients who take oral treatments don't find efficacy. We believe that's, in part, because of the stomach's problems itself." "By developing the nasal format, or GIMOTI, we are able to avoid that unpredictabl

Jared Bauer is the CEO of IONIQ Sciences which is exploring new ways to detect cancer at an earlier stage using non-invasive bioimpedance-based technology. This approach is not looking for the presence of cancer cells but rather changes in the interstitial fluid. Using a low level of electric current, the IONIQ AI-enhanced technology can identify where in the body the cancer is. Jared explains, "It's a bioimpedance-based technology, and the way the system works is we inject a very low level of electrical current, not enough for you to feel. You can't even recognize that it's there. Still, we inject electrical current using DC power, direct current. If you just think physics 101, electricity 101, whatever you want to call it, DC power, the path of least resistance, and that electricity is going to go through the interstitial fluid. Then we pull that back out with an EKG electrode on the other side. And we're scanning that interstitial fluid over distances, allowing us to see that immune response." "When someo

Colin LeClair, CEO of Connections Health Solutions, shares his concerns about the lack of resources for people needing behavioral health crisis care and solutions for urgent and emergency psychiatric services in a facility-based setting. With a community-based approach, Connections partners with hospitals, care facilities, service providers, and law enforcement to better understand patients dealing with complex behavioral challenges and provide new alternatives for care.  Colin elaborates, "Right now, we have two of the largest crisis facilities, as we call them, in Arizona, one in Tucson and one in Phoenix. And those services include emergency psychiatric services, 24 hours a day, seven days a week. Crisis stabilization services for patients who need a few days to recover and get back on their medication regimen and for whom we can prepare a care plan. In addition, there is an observation unit, a 23-hour observation unit for patients who are very acute and require immediate stabilization in a very unrestrict

Dr. Shankar Musunuri is the Co-Founder and CEO of Ocugen, which is a biotechnology company focused on vaccines for public health, gene therapies targeting rare diseases in the retinal space, and cell therapies for regenerative medicine applications in cartilage repair. One key opportunity is to develop an inhalation vaccine that would be durable and efficient at preventing and controlling transmission of COVID and able to be combined with a flu vaccine.  Shankar explains, "There is a linkage if you take some of these vaccines and gene therapies. Some of the processes we use to make gene therapies can also use similar processes for vaccines. So there is a shared science, a shared manufacturing platform, and shared expertise. And then if you take the biotech itself, cream-of-the-cream in the pharmaceutical industry, they work in cell and gene therapies and vaccines, very complex science." "If you have a mucosal vaccine, which will create antibodies in the respiratory tract and control where the virus enters, i

Steve Glover, Co-founder and CEO of ZyVersa Therapeutics, focuses on rare kidney diseases and diseases with an inflammation component. Inflammasomes, the first response in the innate immune system, create an inflammation cascade.  The ZyVersa approach targets multiple inflammasomes to address diseases caused by the malfunction of this response. Steve explains, "Inflammation drives a lot of different diseases. And as we get into this a little bit more today, it's everything from kidney disease such as rare diseases such as FSGS and Alport syndrome and diabetic kidney disease all have an inflammation component. And in addition to that, when you look at diseases like Alzheimer's or MS, or Parkinson's, they're inflammation driven as well. So there is a connection between the two disease classes." "I've been in the industry a long time, and every so often the industry finds a new interesting discovery, and inflammasomes are clearly one of those new discoveries. In fact, one of the things I talk about quite a bit

Carly Newton is an apheresis expert at Terumo Blood and Cell Technologies, a medical device company that manufactures and designs systems that collect blood components and cells. Their software provides management tools to optimize procedures and help physicians better manage care. The largest patient group treated with red blood cell exchange are those with sickle cell disease and benefit from the Terumo iron-neutral therapy over a simple transfusion. Carly explains, "We've also got software technologies that many of our blood banks use and they're designed to get the most from blood donors when they go and present at a blood bank. So, for instance, instead of a blood donor just donating a bag of whole blood, our software can actually look at the starting counts of these donors and recommend what the blood donation center should collect from that donor to get the optimal cells from that patient." "The main goal of any transfusion therapy with sickle cell disease is to increase the oxygenation of the blood. T

Dr. Daniel Karlin, Chief Medical Officer at MindMed, is bringing an updated approach to developing an LSD-based drug to treat anxiety and depression. Instead of developing a psychiatric medicine to be taken every day, MindMed is changing the paradigm to a session-based therapy that uses psychedelic drugs and therapists to improve psychiatric health. As a trained psychiatrist, Dan works at the intersection of psychiatry and neuroscience with this psychedelic-assisted therapeutic approach to brain health.  Dan elaborates, "For quite some time, I think it feels like psychiatry hasn't been meeting people there. Treatments have been more focused on symptomatic resolution and these sorts of things, and visits with psychiatrists can feel too short and it can be really hard to find and afford psychotherapy. I think that what people are responding to about LSD and other drugs like it and their therapeutic potential is the sense that they offer something more than what people are able to access today. That there's rea

Dr. Waqaas Al-Siddiq, CEO and Founder of Biotricity, shines a light on the need for better data about heart rhythm throughout the day, not just for a limited time. Biotricity has developed a non-invasive, cost-effective, available over-the-counter monitor that provides patients and cardiologists with a new level of understanding of heart activity and enhances diagnosis and disease management. Waqaas explains, "I'll take a step back to try to explain the problem that we were trying to solve. Dexcom created a continuous glucose monitor, saying that there are these data points that are in between when you're pricking your finger and collecting your information, which is quite valuable and insightful." "In the continuous heart monitoring world, or in the heart world, patients don't really have anything. You have an implantable loop recorder, which records your ECG continuously. But you have to be a high-risk patient, it's a very high cost to get that implanted, and when it comes to a non-invasive solution, you h

Dr. Eddilisa Martin is the CEO and Founder of M & B Sciences, which has created the Neighborhood Trials online platform and app available in the Apple Store and the Google Store. Their mission is to drive more diversity in clinical trial participation by providing accessible information to patients, providers, and caregivers looking for therapies and opportunities for trials.   Eddilisa explains, "When we talk about diversity, people most often think of race and ethnicity. And that's very, very important. But diversity means having representation across a whole spectrum of things. So whether that's age, whether it's your sexual preference, whether you live in the country versus the city, whether you have lots of comorbidities, lots of illnesses in conjunction with the disease that's being tested, or whether that's the only thing you have. There are a whole host of things that we're talking about when we say diversity." "But you hear people talk about their hesitancy to be a guinea pig. And so it really d

Eric Shaff is the President and CEO of Seres Therapeutics, which is focused on the gut microbiome in order to develop bacteria as an oral treatment for bloodstream infections, cancer, and immune system diseases. The first therapy is SER-109 for recurrent C. diff infection an orphan disease with inadequate solutions for patients. The starting point for Seres is that the health of the microbiome is the starting point for preventing infections from occurring. Eric explains, "The real susceptibility of someone getting C. diff is not just the bad actor bacteria. But really the susceptibility of the microbiome and an injury to the microbiome, and susceptibility to infection. Typically what happens is if people get antibiotic therapy, which can be very effective in knocking back or killing all bacteria, it creates a significant niche in the microbiome, or an injury. There is what we call a race to repair, so either a patient will naturally repair and restore their microbiome, or C. diff may infiltrate, which can le

Ashley Perry is the Chief Strategy and Solutions Officer at Socially Determined, a social risk analytics and solutions company working with payers, providers, and life science companies. Their Socialscope risk and data analytics platform helps organizations understand social determinants of health, identify and manage risk and advance equity for the populations they serve. These organizations are able to be data-driven and action-oriented to mitigate community and individual-level risk. Ashley explains, "The evidence is very clear from a multitude of sources that up to 80% of healthcare utilization, cost, outcomes, and, importantly also, equity are driven by non-clinical factors or non-medical cost drivers. That includes all aspects of everyday life. So, you think about factors like food insecurity and housing instability, transportation barriers, and health literacy challenges. We see consistently in the data that we look at every day the impact these factors have on those utilization costs and outcome meas

Rich Pascoe, CEO of Zevra Therapeutics, is working with a team with deep experience developing drugs and getting them approved by the FDA.  Having developed multiple prodrugs for pain and ADHD, Zevra is now developing rare disease therapies with an initial focus on Niemann Pick Disease Type C and idiopathic hypersomnia.   Rich explains, "Our lead product is a drug called arimoclomol. It's intended for the treatment of Niemann Pick Type C disease, or NPC for short. Niemann Pick primarily affects children. It's a lysosomal storage disorder condition, meaning patients lack the ability to break down certain things, including cholesterol. As a result, they develop a myriad of symptoms that ultimately lead to, in most cases, the patient's death. The issues they deal with revolve around loss of ambulation and the ability to swallow, and it affects their cognitive function. It's a progressive disease, and oftentimes, it can advance very rapidly." "A little history on arimoclomol. The drug was previously in the hands

Steve Reed, Co-Founder, President, and CEO of HDT Bio, has spent his life focusing on tropical diseases and points out how the cost of developing and manufacturing vaccines determines access for a global population. HDT Bio was founded to develop more cost-effective technology to induce broader immune responses to various diseases. Their advanced RNA platform AMPLIFY is a technology applied to developing mRNA vaccines for infectious diseases and a therapeutic vaccine to help prevent recurrence and prolong survival from cancer.  Steve elaborates, "COVID was something that came up right after we founded the company, and so the National Institute of Health asked us and funded us to develop a novel platform that could complement the platforms already in place for mRNA vaccines. Other indications that are incredibly important include other viruses. They're always coming up, and they're always mutating. But you probably read in the news about fungal infections on the increase in the United States and other countrie

Jeff Liter is the CEO and Founder of Luminary Therapeutics, which is using gamma delta cells in their approach to CART-T cell therapy because of the ability of these cells to infiltrate solid tumors. With a multi-targeting system, Luminary has demonstrated promise treating multiple types of solid tumors, blood tumors, and autoimmune diseases. Their goal is to create a scalable model to manufacture allogeneic cells to drive down the cost and availability of effective cell therapies. Jeff explains, "We're focused primarily on three big challenges in the CAR-T industry. The first is the overall manufacturing approach and cost. The second is why do CAR-Ts, to date, not work in solid tumors. And then finally, the third is antigen escape and relapse of patients currently being treated by CAR-T therapies."  "We have moved into coming back to the manufacturing area. We have moved into an allogeneic platform, which for your listeners, if they're not familiar with that term, means that we can make multiple products fr

Joe Tucker, the CEO of Enveric Biosciences, is discovering, developing and testing psychedelic-inspired therapies for the treatment of depression, anxiety, and PTSD. Current treatments target receptors in the brain to increase the concentration of serotonin in the brain by going through classic neurotransmitter pathways. The Enveric Psybrary portfolio and PsyAI drive drug discovery to create new molecules that synthesize chemistry and how nature makes molecules to target new receptors. Joe elaborates, "This one's completely different. This one is actually going through a receptor that's never been targeted before, 5-HT2A. And it's an adjunct to psychotherapy at this point. The leading molecules are an adjunct to psychotherapy. Although there's some interesting new work, including stuff that we're working on, no one has yet been able to separate those two in humans. But that's where we'd like to go next, is to separate them. But right now, it's really this 5-HT2A receptor, this induction of hallucination. No

Dr. Joshua Schiffman is the CEO and Co-Founder of Peel Therapeutics and has taken the understanding of the natural resistance to cancer by elephants to develop PEEL-224 to attack tumors. Using a plant toxin and modifying existing approaches, Peel has created a drug that increases the amount of the drug that gets to the cancer cells with reduced side effects.   Joshua explains, "Peel is actually the Hebrew word for elephant. So that's why we call ourselves Peel Therapeutics. My Co-Founder, Dr. Avi Schroeder, is from the Technion-Israel Institute of Technology in Israel. While we were working on the elephant drug, we realized, you know what, it's more than just elephants. Looking around nature, we understand that evolution has had hundreds of millions of years to perfect, in some cases, perfect cancer-fighting protein or peptide or anti-inflammatory molecule. Our lead drug, which we call PEEL-224, actually works really well targeting the DNA repair mechanism in cancer cells." "This DNA repair mechanism is call