Raise the Line

“When I was in medical school, no one had even heard of mitochondrial disease. Today, every student who graduates here knows what it is and has seen a patient with it,” says Dr. Mary Kay Koenig, director of the Center for the Treatment of Pediatric Neurodegenerative Disease at UTHealth Houston McGovern Medical School. That remarkable change in awareness has been accompanied by advances in genetic sequencing, the development of clinical guidelines, and the emergence of potential treatments in some forms of mitochondrial disease. In fact, Dr. Koenig’s multidisciplinary team at UTHealth’s Mitochondrial Center of Excellence has been a key player in clinical trials that may yield the first FDA-approved treatments for it. As you’ll learn in this Year of the Zebra conversation with host Michael Carrese, her work in neurodegenerative diseases also includes tuberous sclerosis, where advanced therapies have replaced the need for repeated surgeries, and Leigh Syndrome, which has seen improvements in diagnoses and supportive therapies leading to better quality of life for patients.  Tune in as Dr. Koenig reflects on an era of progress in the space, the rewards of balancing research, teaching and patient care, and the need for more clinicians to center listening, humility and honesty in their approach to caring for rare disease patients and their  families.

Mentioned in this episode:

Mitochondrial Center of Excellence

Center for the Treatment of Pediatric Neurodegenerative Disease

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“Giving learners options gives them a better learning experience. It’s more holistic and more comprehensive,” says Sean Moloney, CEO and founder of EmbodyXR, an extended reality platform focused on the use of immersive technologies in medical education. In this eye-opening Raise the Line conversation, Moloney explains how AI-powered extended reality (XR) --which integrates augmented, virtual, and simulation-based environments -- allows learners to interact with patients, explore multiple diagnostic choices, and experience varied outcomes based on their decisions. The result, he notes, is not only stronger engagement in learning, but a measurable improvement in understanding. Despite these gains, Moloney is quick to point out that he sees these technologies as complements to traditional training, not substitutes for it. “We’ll never replace in-person teaching,” he says, “but we can make learners even better.” Beyond training future clinicians, the EmbodyXR platform is also offering new modes of patient and caregiver education, such as augmented reality guidance for using medical devices at home. Join host Lindsey Smith as she explores how EmbodyXR achieves and maintains clinical accuracy, the connectivity it offers between headsets, personal computers and mobile devices, and other capabilities that are shaping the future of how healthcare professionals and patients will learn. 

Mentioned in this episode:
EmbodyXR

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Why has America struggled so much to effectively manage the opioid use crisis? One of the answers, as you’ll learn in this eye-opening episode of Raise the Line, is rooted in laws and attitudes from the early 20th century that removed addiction from the realm of medicine and defined it as a moral failing.  “The federal Harrison Act of 1914 forbade any physician from prescribing opioids to people with addiction, so it became more the purview of law enforcement or behavioral health or religion,” says Dr. Melody Glenn, who regularly confronts the consequences of this history during shifts in the emergency department at Banner-University Medical Center in Tucson, Arizona. And as Glenn explains to host Caleb Furnas, the resulting stigma associated with addiction has extended to the treatments for it as well, especially methadone, despite its effectiveness. Drawing on her dual expertise in emergency and addiction medicine, Glenn dispels misconceptions that medication-assisted treatment merely replaces one addiction with another, and emphasizes that harm reduction is critical to saving lives. Her desire to break prevailing stigmas led her to discover the story of Dr. Marie Nyswander, who pioneered methadone maintenance therapy in the 1960s and is featured in Dr. Glenn’s new book, Mother of Methadone: A Doctor’s Quest, a Forgotten History, and a Modern-Day Crisis. You’ll leave this instructive interview understanding the roots of our flawed approach to addiction treatment, meeting an overlooked pioneer in the field, and admiring a devoted and compassionate physician who is following in her footsteps.  

Mentioned in this episode:

Banner-University Medical Center

Mother of Methadone book

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“We don't view a person with chronic pain as someone who has a chronic illness and the effect of that is we can't follow patients continuously over prolonged periods of time,” says Dr. Jacob Hascalovici, a neurologist and pain specialist based in New York City.  In co-founding Bliss Health, Dr. Jacob, as he is known, has set out to create a continuous care model for chronic pain treatment that matches the approach taken for patients with diabetes or high blood pressure. The Bliss Health formula includes an initial meeting with a physician that produces a care plan; remote therapeutic monitoring on an ongoing basis; and a monthly meeting with a nurse to review data and determine next steps, including additional appointments with physicians as needed.  All of this occurs via a digital platform which provides a welcome option for patients with mobility issues and can fill gaps in access to specialists, especially in rural areas. Dr. Jacob is also hoping to make chronic pain patents feel respected, which is not always the case in their encounters with the healthcare system. “Because pain is not something that can be seen or measured, oftentimes patients feel marginalized, dismissed and disempowered by providers.” Join Raise the Line host Lindsey Smith for a valuable conversation that also touches on policy changes that could strengthen telemedicine, and has details on the first non-opioid based pain medication to receive FDA approval in over 20 years.

Mentioned in this episode:

Bliss Health

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In recent months, public health advocates in the United States have raised concerns about proposed changes to vaccine policy, cuts to food assistance programs, rollbacks of environmental protections and reductions in public health staffing. Chief among them has been Dr. Georges Benjamin who, as executive director of the American Public Health Association (APHA) since 2002, has led national efforts to create a healthier America. Raise the Line host Lindsey Smith recently sat down with Dr. Benjamin to understand more about the current state of public health and explore the path forward, and learned that a top priority for APHA is battling the misinformation that Dr. Benjamin believes is fueling support for many of these changes. “The challenge we have right now is that as a society, we've gone into our little corners and live in our own ecosystems. More people are getting their information from a single source and they're not validating that information to make sure that it's true.” Tune into this thoughtful and timely conversation to hear Dr. Benjamin’s advice for curbing the spread of misinformation, how APHA is trying to help people understand the value of public health initiatives, and what the U.S. can learn from other countries about improving public health. 

Mentioned in this episode:

American Public Health Association

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“Probably the most exciting thing I've seen in gene therapy over the last ten years is we now have a lot of tools for selective delivery, which will hopefully make treatments more safe and a lot more successful,” says Dr. Jessica Duis, a geneticist and pediatrician focused on the management of individuals with complex, rare disorders. Dr. Duis, who has worked on several gene therapies that are now approved or progressing through the accelerated approval pathway, is currently VP of Clinical Development at GondolaBio, a clinical-stage biopharmaceutical company focused on developing therapeutics for genetic diseases. 

As you’ll learn in this Year of the Zebra episode with host Lindsey Smith, Dr. Duis is encouraged by other recent advances in genetic technology as well, and thinks momentum will grow as breakthrough treatments emerge. “I think we're hopefully going to continue to see companies that are working in rare disease be more successful and really drive how regulators think about making decisions in terms of bringing treatments to patients. I think we’re at the tip of the iceberg in terms of the future of truly transformational therapies.”  This wide ranging conversation also explores Dr. Duis’ team approach to patient care, her work on clinical endpoints, the importance of patient communities, and her book series, Rare Siblings Stories.

Mentioned in this episode:

GondolaBio

RareDiseaseDoc

Elsevier Healthcare Hub on Rare Diseases

Rare Sibling Stories

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With nearly one in ten newborns in the US requiring care in a Neonatal Intensive Care Unit, the importance of NICUs has never been more clear. On today's episode of Raise the Line, we're shining a light on the extraordinary world of NICUs with Lindsay Howard, a veteran nurse with over 17 years of experience caring for premature and critically ill infants. She currently works in a Level IV NICU at Children's Memorial Hermann Hospital in Houston, one of the most advanced neonatal units in the country. “We call ourselves ‘the ER of the neonate world’ because we're never full. We have to make space no matter what comes in off the street, and at the biggest medical center in the world, we see all the things,” she explains. In this enlightening conversation with host Lindsey Smith, Howard describes how advances in medicine have made it possible to provide more types of care for younger and smaller babies, creating a need for NICU nurses to develop subspecialties. In her case, Howard is on a dedicated team that handles the placement and maintenance of all central line IVs, and has earned certifications in neonatal and pediatric chemotherapy and biotherapies. “We see babies that we may not have seen before being born with cancerous tumors who need chemotherapy to try and eliminate it, or just give them more time with their family.” This is a revealing look inside the workings of a top tier NICU where you’ll learn about approaches to care that support healthy neurodevelopment, how clinical staff handle the emotional challenges of the job, and how her own experience as a mother with twins needing NICU care impacted her work.  

Mentioned in this episode:
Children's Memorial Hermann Hospital

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“When you think about where we were as a country before Medicare and Medicaid were created and where we are now, it’s an incredible story,” says Chiquita Brooks-LaSure, who until earlier this year was the administrator for the Centers for Medicare and Medicaid Services (CMS). In a recent essay for The Century Foundation, where she is now a senior fellow, Brooks-LaSure used the 60th anniversary of enactment of those foundational insurance programs to help put their impact on individual Americans, the healthcare system and society at large in perspective. One prominent example is the desegregation of hospitals, which was achieved in part by withholding reimbursements for care unless facilities served Blacks as well as whites. Another is making it possible for more people with disabilities to live at home instead of in institutional settings. But as you’ll hear in this probing Raise the Line conversation with host Lindsey Smith, Brooks-LaSure worries that many gains in coverage and other progress made over the years through Medicare, Medicaid and the Child Health Insurance Program (CHIP) are at risk because of a new federal law that calls for a trillion dollar decrease in spending, resulting in potentially millions of people losing their coverage, cuts to clinical staff and medical services, and the closure of hospitals and clinics, especially in rural areas. “Most rural hospitals in this country are incredibly dependent on both Medicare and Medicaid to keep their doors open and there's an estimate that over 300 hospitals will close as a result of this legislation, so that, I think, is a place of incredible nervousness.” Whether you are a patient, provider, policymaker or health system leader, this is a great opportunity to learn from an expert source about the range of potential impacts that will flow from changes to critically important insurance programs that provide coverage to 40% of adults and nearly 50% of children in the U.S. 

Mentioned in this episode:

The Century Foundation

Essay on 60th Anniversary of Medicare & Medicaid

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It seems there are news stories every week about the accelerating pace of innovation in gene therapy, but only about 50 therapies have been approved so far by the US Food and Drug Administration. Our guest today, Dr. Bobby Gaspar, leads a UK-based biotech company, Orchard Therapeutics, that developed one of those treatments using gene-modified stem cells in your blood that self-renew, so a single administration can give you potentially a lifelong effect. “Our approach is about correcting those hematopoietic stem cells and allowing them to give rise to cells that can then correct the disease,” explains Dr. Gaspar.  The therapy in focus is lenmeldy, the first approved treatment for metachromatic leukodystrophy, also known as MLD, a devastating inherited disorder that affects roughly 600 children worldwide. But Dr. Gaspar is optimistic that learnings from Orchard’s work on MLD could be useful in treating much more common disorders including frontotemporal dementia, Crohn’s disease and others. This highly informative conversation with host Lindsey Smith also explores the importance of newborn screening, community collaboration in advancing clinical trials for rare diseases, and a future in which each gene therapy will be used as a tool for specific applications.  “There will be many gene therapies available, some of which will become the standard of care for certain diseases, but it won't be for every disease.”

Mentioned in this episode:
Orchard Therapeutics

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 are in for a dose of inspiration in this episode of Raise the Line as we introduce you to a rare disease patient who was a leading force in establishing the diagnosis for her own condition, who played a key role in launching the first phase three clinical trials for it, and who is now coordinating research into the disease and related disorders at one of the nation's top hospitals. Rebecca Salky, RN, was first afflicted at the age of four with MOGAD, an autoimmune disorder of the central nervous system that can cause paralysis, vision loss and seizures. In this fascinating conversation with host Lindsey Smith, Rebecca describes her long and challenging journey with MOGAD, her work at the Neuroimmunology Clinic and Research Lab at Massachusetts General Hospital, and the importance of finding a MOGAD community in her early twenties. “There's a sense of power and security when you have others on your side. You're not alone in this journey of the rare disease,” she explains. Be sure to stay tuned to learn about Rebecca’s work in patient advocacy, her experience as a nurse, and the three things she thinks are missing in the care of rare disease patients as our Year of the Zebra series continues.

Mentioned in this episode:

The MOG Project

Neuroimmunology Clinic & Research Lab at Mass General

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