Raise the Line

“I know it's hard for people to think about holding on if they're in despair, but there's the possibility of healing coming ahead. Don’t give up hope,” says Dr. Rick Doblin, who has devoted his career to getting MDMA and other psychedelics approved by the FDA.  As you’ll hear in this episode of Raise the Line, he could be on the verge of seeing his decades-long dream come true.  On the day host Shiv Gaglani spoke to Doblin (January 5, 2023) successful results from a Phase 3 clinical trial of MDMA to treat PTSD were released by the Multidisciplinary Association for Psychedelic Studies – an organization Doblin founded thirty-six years ago. This sets in motion a process that could result in FDA approval of MDMA for this use in 2024. That’s very good news for the millions of Americans with PTSD – and hundreds of millions worldwide – whose disease is resistant to other treatments. One of the next big steps is training therapists to incorporate these drugs into their work, something MAPS and other organizations are moving quickly to accomplish. Tune into this fascinating conversation about the political and social factors that have held up legalization of psychedelics for decades, other promising applications of MDMA, what fuels Doblin’s passion for normalizing the use of these promising medications, and the largest ever conference on psychedelics coming to Denver, Colorado in June.  Mentioned in this episode:https://maps.org/https://psychedelicscience.org/ If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

The growth in skepticism about science that was fueled by disinformation during the pandemic has been a frequent topic on Raise the Line, with many insightful guests from medicine and academia offering analysis of the problem and possible solutions. On today’s episode, we’ll hear from someone who is very well-placed to actually make progress on this front. Max Bronstein, the Assistant Director for Health Innovation at the White House Office of Science and Technology Policy, joins host Shiv Gaglani to discuss the broad support President Biden has provided for science – elevating his science advisor to the cabinet level being a prime example – and also provide details about programmatic investments that tell the tale at a deeper level. At the top of Bronstein’s list of examples is the launch of the Advanced Research Projects Agency for Health, also known as ARPA-H, which aims to drive transformative biomedical and health breakthroughs with a focus on equity. “The mission is about making sure there are cures for diseases, better diagnostic platforms and better technologies out there, but also making sure those are actually available to all Americans.” Don’t miss this inside look at new efforts to strengthen the biotechnology workforce, broaden access to clinical genetic sequencing, advance development of treatments for rare diseases, and much more as a new era in health innovation gets underway.  Mentioned in this episode: https://www.whitehouse.gov/ostp/ If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

“It's still early days in the application of all this technology relative to its long-term potential, but even so, it's already producing some big wins for patients,” says Dr. Matthew Might, whose impactful career in computer science and medicine has been shaped by the rare disease odyssey of one of his children. His son, Bertrand, was the first person in the world diagnosed with a particular form of NGLY1 deficiency, a neurogenic degenerative condition that causes developmental delays, seizures and frequent infections. Unfortunately, Bertrand succumbed to an infection at the age of twelve in 2020 but by that time, Dr. Might's work in precision medicine had led to crucial discoveries for dozens of children with NGLY1 deficiency. Now, as director of the Hugh Kaul Institute of Precision Medicine at the University of Alabama at Birmingham, he uses an AI-based system programmed to connect the dots in extensive databases of medical literature to make inferences about potential therapies for uncommon diseases. Check out this fascinating conversation with host Shiv Gaglani about the promise of this approach, the challenges in repurposing drugs and conducting clinical trials in the rare disease community, the need for more genetic counselors and Dr. Might’s work on President Obama’s Precision Medicine Initiative, which he calls the Rosetta stone of the human genome. Mentioned in this episode: https://www.uab.edu/medicine/pmi/  If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

We welcome one of the nation's most respected health and health policy thought leaders to Raise the Line on this episode.  Susan Dentzer’s remarkable career includes many years of reporting on healthcare for major national news outlets, being a senior policy adviser to the Robert Wood Johnson Foundation and serving as a board leader in medical education and health system delivery, just to name a few of her contributions. Earlier this year, Ms. Dentzer was appointed president and CEO of America's Physician Groups, an organization representing more than 350 physician groups providing coordinated, value-based healthcare for more than ninety million patients nationwide. She's also currently board chair of Research America, which advocates on behalf of biomedical and health-related research and innovation. Tune in to this elucidating discussion with host Shiv Gaglani that delves into what the pandemic revealed about value-based care and virtual care; what is helping to lessen clinician burnout; surprising views among medical students on the use of tech in healthcare; what is at the root of the public’s mistrust of science, and much more. “The reality of healthcare is very complicated. What I would make a plea for is that we all try to engage in developing a greater understanding of the issues, as opposed to seeing them through a narrow lens.”Mentioned in this episode: https://www.apg.org/https://www.researchamerica.org/ If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

“There's a reservoir of hope, energy and optimism many of us have that we may not know about until we're really tested,” says attorney and author Scott Reich. The heavy test he and his wife Ilissa have endured for the past three years started when their infant son Eli was diagnosed with a rare brain disorder called FOXG1 Syndrome which causes severe seizures and impedes normal movement, speech and sleep, among other problems. Scott vividly recalls the moment when Eli’s doctor explained there was no hope for treatments or a cure. “I just felt an instantaneous gravitational pull that despite the intense emotion that overtook us in the doctor's office, we were going to do something about it.”  That “something” includes starting the nonprofit foundation Believe in a Cure which is currently funding over fifty research and development projects worldwide focused on this pernicious disorder. Join us for this enlightening conversation with host Shiv Gaglani to learn about the multi-pronged strategy scientists are pursuing to overcome the mutation in this so-called master gene, the promising results emerging from preclinical programs and the supportive global community Believe in a Cure has helped create for the hundreds of other families fighting the same battle.  Mentioned in this episode: https://www.webelieveinacure.org If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

Dr. Chris Gibson, Co-Founder and CEO of Recursion, shares their mission to accelerate drug discovery by leveraging advances in genetics, AI, and more. They discuss their departure from the hypothesis-based approach, their success in animal testing, and the reengineering of drug discovery. Topics also include the merging of biology and advanced technology, recent scientific advancements in healthcare, and the concept of iteration and learning in drug discovery.

You might think improving healthcare quality is largely in the hands of the clinicians providing the care, but much of this challenging work is actually done behind the scenes by professionals who lead quality reviews, institute new protocols for safer care and focus on performance improvement, among many other efforts. “We are on a journey to really change the perception of what quality is. We want to make it more prospective and actually solve problems for patients and the healthcare workforce,” says Stephanie Mercado, CEO and Executive Director of the National Association for Healthcare Quality. On this episode of Raise the Line, we get an inside look at this critically important function in healthcare, and at the Healthcare Quality Workforce ReportNAHQ recently released that recognizes the field’s progress, but also outlines places for improvement. “The areas most important for the future of healthcare are things where the workforce is performing at lower ends of the competency spectrum. Those include health data analytics, change management and payment models. So, we have a lot of work to do.” Check out this revealing conversation with host Michael Carrese to learn what the future holds for quality professionals as healthcare challenges mount. Mentioned in this episode: https://nahq.org/ If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

Regular listeners to Raise the Line know so-called "rare diseases" aren't really rare when you consider up to thirty million Americans are affected by them directly. That makes it likely you know someone who is impacted, or you know one of their colleagues, friends or loved ones. That fact has hit home at Osmosis over the last few months as we've started planning a major focus on rare diseases for 2023, which we're calling The Year of the Zebra. Several teammates have come forward to tell us their rare disease stories and we'll be sharing some of those on the podcast in the coming months. First up is our Director of Nursing Education Dr. Maria Pfrommer and her husband, Jack, who join host Shiv Gaglani to offer insights into the diagnosis and treatment journey they’ve been on to deal with Jack’s battle with retroperitoneal fibrosis, an inflammatory condition which can cause obstructions in the urinary tract. While Maria’s vast clinical knowledge and experience in healthcare systems has obviously been helpful, it was still a struggle to get the right diagnosis and treatment due to limited experience among healthcare professionals with rare conditions. “From my perspective, I really think that we need to learn more about rare disorders from every level of care,” she says. Tune in for great real-world advice for healthcare professionals dealing with rare disease patients including the importance of listening to the patient, understanding their whole life picture and focusing on transitions of care. If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

Grace Wilsey was born with a deadly genetic mutation so rare that at the time of her birth, it had never been identified in another person. The disorder, NGLY1 deficiency, causes a wide range of physical and cognitive problems such as muscle weakness, speech deficiencies and seizures. “The NGLY1 gene is in every cell in the body. It's almost like a firefighter that's on call, ready to go when there's a problem. Without it, the cell just kind of overwhelms itself with stress and starts to die,” explains Matt Wilsey, Grace’s father, who joins us on this episode of Raise the Line to talk about the daunting journey he and his family have been on since Grace was born in 2009. That journey involves starting a foundation and biotech company that’s fueling research on NGLY1 deficiency which could have an impact on more common diseases such as cancer, diabetes and Parkinson's. The researchers the Grace Science Foundation supports include several Nobel laureates whose work is providing reason for optimism. In fact, Matt says they hope to start a clinical trial in early 2023 to study a gene therapy that has shown promise in animal testing.  Listen to this fascinating conversation with host Shiv Gaglani to learn about the race with time to unlock the secrets to a gene that is fundamental to human life. Mentioned in this episode: https://gracescience.org/ If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast

“You know, it's easy to say that default answer that everything's okay, but it's really not. She's lost a lot of her vision, she's got hundreds of seizures at night, and she's having difficulty walking,” shares Luke Rosen about his eight-year-old daughter Susannah.  She was born with KIF1A-associated neurological disorder -- or KAND -- a rare, degenerative genetic disease for which there is currently no cure or treatment.  On this episode of Raise the Line, Luke talks about how he and his wife Sally summoned the strength to move beyond their family’s own challenges to create KIF1A.org which is working to rapidly discover a treatment for all patients and families affected by this devastating disorder, but to also create a supportive community.  “Five years later, we have approximately four hundred families around the world that we've identified and there's not one family I know that doesn't play a significant role in what we do.” Thanks to that global community and partnerships with the Chan Zuckerberg Initiative, Columbia University, the n-Lorem Foundation, the Jackson Laboratory and many other organizations, there’s reason to be hopeful, as Luke shares with host Shiv Gaglani. “Susannah has been fortunate enough to just have started an experimental treatment. We really are on the brink of several things for, hopefully, the entire community.” Tune in for a candid and moving look at how families and supportive scientists and healthcare providers are mobilizing to fight back against a rare and pernicious threat to their children.Mentioned in this episode: https://www.kif1a.org/ If you like this podcast, please share it on your social channels. You can also subscribe to the series and check out all of our episodes at www.osmosis.org/podcast