Expanding the Gene Therapy Toolbox: Dr. Bobby Gaspar, Co-Founder & CEO of Orchard Therapeutics
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/raisethelinepodcast
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Rare Disease Patients as Changemakers in Medicine: Rebecca Salky, Senior Clinical Research Coordinator for the Neuroimmunology Clinic & Research Lab at Massachusetts General Hospital
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 ProjectNeuroimmunology 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/raisethelinepodcast
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Providing a Framework for Personal and Professional Growth in Medicine: Dr. David Kelly, HOSA-Future Health Professionals Board Chair
“You have to love what you do, especially in healthcare, and the earlier you find that, the better. So that’s why I love to see HOSA helping young people find what it is that they want to do,” says Dr. David Kelly, a fellow in oculofacial surgery at University of California San Francisco and HOSA’s board chair. You can still hear the excitement in Dr. Kelly’s voice describing his earliest experiences with HOSA -- a student led organization with 300,000 plus members that prepares future health professionals to become leaders in international health – even though they happened sixteen years ago when he was a sophomore in highschool. Through hundreds of competitive events and hands-on projects, HOSA creates a framework for developing skills in communication, professionalism and leadership starting in middle school. Programs are offered throughout highschool and college as well, which Dr. Kelly took advantage of before becoming an active alumnus and joining the HOSA board as a way of giving back to an organization that has given so much to him. Since taking the reins as board chair last year, one key focus has been preparing to mark HOSA’s 50th anniversary in 2026. Dr. Kelly sees the occasion as not only an opportunity to celebrate what HOSA has accomplished, but to ensure it is positioned to continue helping the healthcare industry tackle important challenges in the future. Examples include chronic workforce shortages and improving how clinicians communicate with patients and team members. Join host Lindsey Smith on this uplifting Raise the Line episode for an optimistic look at the next generation of healthcare leaders.Mentioned in this episode:HOSAHOSA Alumni Registration
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/raisethelinepodcast
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How AI Is Aiding Earlier Diagnosis of Autism: Dr. Geraldine Dawson, Founding Director of the Duke Center for Autism and Brain Development
“We've been able to show that even by 30 days of age, we can predict with some accuracy if a child is going to have a diagnosis of autism,” says Dr. Geraldine Dawson, sharing one of the recent advancements in early diagnosis being aided by artificial intelligence. Dr. Dawson -- a leading scholar in the field and founding director of the Duke Center for Autism and Brain Development – explains that an AI examination of a child’s pattern of visits to medical specialists in its very early life is an objective diagnostic tool that can supplement the current subjective reports from parents which vary in reliability. Another objective diagnostic tool in development uses a smartphone app developed at Duke that takes video of babies watching images and applies AI-aided Computer Vision Analysis to measure for signs of autism. This enlightening Raise the Line conversation with host Lindsey Smith is loaded with the latest understandings about Autism Spectrum Disorder including advancements in early therapeutic interventions, the interplay of genetic and environmental factors, and the role of the mother’s health and exposures during pregnancy. You’ll learn as well about what Dawson sees as necessary societal shifts in how autism is perceived, the numerous factors contributing to a near tripling of diagnoses over the past two decades, and how early intervention and informed advocacy can make a meaningful difference in the lives of countless families.Mentioned in this episode:Duke Center for Autism and Brain Development
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/raisethelinepodcast
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Addressing the Root of Burnout and Trauma in Healthcare Providers: Dr. Rola Hallam, Founder of CanDo and Trauma and Burnout Life Coach
"Burnout and trauma are not mental illnesses. They live in your physiology. They live in your biology. They live very specifically in your nervous system,” Dr. Rola Hallam says with a conviction rooted in her own successful journey to overcome the effects of chronic stress she accumulated during many years on the frontlines of humanitarian crises in Syria and other conflict zones. Out of concern for the multitudes of health professionals who, like herself, spend years carrying the weight of their traumatic experiences without seeking help, or who pursue ineffective remedies for relieving it, Dr. Rola -- as she’s known – has shifted her focus to being a trauma and burnout coach. Among her offerings is Beyond Burnout, a twelve-week program that includes multimedia content as well as live coaching and teaching about developing nervous system awareness and regulation. “Most wellness initiatives fail because they're not rewiring the nervous system to come out of survival mode and back into what is called the ventral-vagal state, which is our state of social connection and of healing and repair.” She also stresses that healing is not an individual pursuit, especially for providers who work in a relational field, and teaches about the benefits of borrowing from a colleagues’ state of calm and offering them the same. Don’t miss this insightful and giving conversation with host Lindsey Smith that covers Dr. Rola’s wrenching experiences providing care in desperate conditions, the critically important distinction between empathy and compassion, and how empowering frontline workers to heal their trauma can uplift individuals and empower entire communities. Mentioned in this episode:Dr. Rola CoachingBeyond Burnout AssessmentCanDo - Humanitarian Aid
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/raisethelinepodcast
Join host Lindsey Smith and other Osmosis team members for a global conversation about improving health and healthcare with prominent figures in education and healthcare innovation such as Chelsea Clinton, Mark Cuban, Dr. Ashish Jha, Dr. Eric Topol, Dr. Vivian Lee and Sal Khan, as well as senior leaders at organizations such as the CDC, National Institutes of Health, Johns Hopkins University, WHO, Harvard University, NYU Langone and many others.
Ireland