Raise the Line

Regular listeners to Raise the Line know that research into rare diseases should matter to everybody because it has led to treatments for much more common conditions that have improved and saved millions of lives. Statins are usually the prime example of that. Well, on this episode we're going to get into much more detail on this point with someone who literally wrote the book on the subject: Dr. Jules Berman. His 2014 work published by Elsevier, Rare Diseases and Orphan Drugs, Keys to Understanding and Treating the Common Diseases, shows that much of what we now know about common diseases has been achieved by studying rare diseases, and therefore, accelerating progress in the field of rare diseases will lead to yet more advances affecting common conditions. “If you have a rare disease and you think about the phenotype that results from it, you can often find that same phenotype occurring much more commonly in acquired disease, so the treatment for the rare disease can often help people with the acquired disease.” Don’t miss this provocative conversation with host Michael Carrese as Dr. Berman shares why he thinks researching one rare disease at a time is a flawed approach, especially in light of his belief that there are more than 50,000 rare conditions. 

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Lucy Landman is one of only a few children known worldwide to have a genetic disorder called PGAP3, in which a single missing gene can cause seizures and severe physical and cognitive limitations. Luckily for Lucy, her parents Geri and Zach Landman are both physicians whose expertise has been a big help in obtaining a diagnosis and in advocating for her. The Landmans are bringing that know-how and a fervent desire to help all children with single gene disorders to the non-profit they founded, Moonshots for Unicorns, which is already working with Nationwide Children’s Hospital on a promising gene therapy.  “There are so many of these single gene disorders that should be amenable to things like gene therapy and drug repurposing. So, we don't want this just to focus on PGAP3.” Listen to this moving episode with host Michael Carrese to learn what causes PGAP3, how the rare genetic disorder has impacted Lucy’s life and health, and the suspected connection between PGAP3 and Autism. Dr. Landman also addresses the big gaps she sees in newborn screening, medical education and research efforts from a rare disease perspective.

Mentioned in this episode: www.moonshotsforunicorns.org

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Eighty percent of rare diseases are caused by genetic mutations, which is why many of our recent guests have highlighted the importance of pursuing gene therapies as potential treatments and cures. That’s why we’re particularly pleased to have Dr. Gaurav Shah on Raise the Line today. He’s the CEO of Rocket Pharma, a company that’s in hot pursuit of developing curative gene therapies for patients with inherited genetic diseases, and showing remarkable results in some cases. For instance, a gene therapy for a rare and fatal heart condition called Danon disease is moving every parameter in the right direction, a result Dr. Shah is understandably proud of. “When gene therapy works, it really works. We were able to demonstrate the power of gene therapy for heart disease for the first time in our species,” he tells host Michael Carrese. Don’t miss this fascinating look at the art, science and practicalities of developing gene therapies and where Rocket Pharma is seeing progress. You’ll also learn about the many career options in drug development and hear how Dr. Shah’s background as a Grammy-winning artist informs his approach to leadership.

Mentioned in this episode: https://rocketpharma.com/

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“Burnout to me is about losing control, not overwork. It's about being unable to solve problems, and problems beginning to stack up with no end in sight.” Today’s Raise the Line guest Justin Welsh earned that insight the hard way after a demanding corporate job led to a panic attack so severe it prompted a 911 call. Five years later, after founding a one-man business called “The Saturday Solopreneur,” he’s gained full control of his work life and has the number one rated course on LinkedIn which helps more than 10,000 people identify, share and monetize the knowledge they already possess.  Listen in to this enlightening episode with host Shiv Gaglani as Justin shares his journey from successful digital health executive to self-employment and what he’s learned along the way about himself and what really matters to him. He details how he gained such a large following in short order, and offers advice for healthcare workers and digital health entrepreneurs on advancing their careers and preventing burnout. And stay tuned for an insightful take on the impact of AI that should reassure those with real world knowledge and experience, and the wisdom that can come from both. “Try not to look for ways to cut the line in place of real learning. Do the work, make the mistakes, analyze the mistakes, iterate, repeat.”

Mentioned in this episode: linkedin.com/in/justinwelsh

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“The qualities of a provider that were envisioned fifty years ago are completely different from what the world needs for tomorrow. It’s completely different,” insists Dr. Abebe Bekele, who is entrusted with educating this new breed of physician at the University of Global Health Equity in Rwanda. As Bekele explains to host Shiv Gaglani in this special in-person interview on the campus of UGHE in Butaro, Rwanda, COVID-19 has demonstrated that doctors now need to be able to serve as leaders of institutions, manage large projects, raise money and interface with influential public sector players such as policymakers and journalists. The program at UGHE has been designed with that in mind by providing a grounding in liberal arts and humanities along with the necessary medical content. As you’ll learn in this insightful conversation, the relatively young school -- which was established by Partners in Health in 2015 -- is taking a thoughtful approach to meeting healthcare needs in the region through admissions policies and scholarships that are boosting the number of female physicians and incentivizing its graduates to practice medicine in their home communities. Beyond connecting with Dr. Bekele, Shiv’s visit gave him a chance to meet with students and faculty to gain a deeper understanding of the partnership Osmosis has with UGHE which is part of a larger effort to support medical education in Sub-Saharan Africa, including in Namibia where he was born. As you’ll hear, Shiv came away seeing UGHE as a model for health education in an increasingly interconnected world. 

Mentioned in this episode: https://ughe.org/

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“Our rare disease community is looking to solve for many different types of policy barriers because we have a very diverse patient community,” says Annie Kennedy, who was drawn to the rare disease issue due to some personal experience early in her life.  After spending many years as a patient advocate -- including being with patients during provider visits -- she has developed a keen understanding of where the healthcare system can be improved to do justice to rare disease patients and families. That insight informs her work as chief of Policy, Advocacy and Patient Engagement at the EveryLife Foundation where she helps provide patient communities with tools and resources they can use to make their voice heard in the policy arena. In fact, next week, EveryLife is holding its annual Rare Disease Week on Capitol Hill during which members of the rare disease community will meet with lawmakers in Washington to provide meaningful perspectives and encourage their support. “There are more than thirty million Americans living with rare diseases, so this is a real public health priority,” she tells Raise the Line host Michael Carrese. One resource EveryLife has brought to the table is a study on the total cost of living with a rare disease, not just the costs of medical care, which is helping to inform policy discussions. You’ll also learn about some key steps pharma companies, insurers and providers can take to support the rare disease community and where the field is heading in the next few years.  

Mentioned in this episode: https://everylifefoundation.org/

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“In about three weeks, I went from a completely normal thirty-year-old to somebody with a heart transplant. It was crazy,” says Dr. Alin Gragossian, who shares her remarkable experience on this edition of Raise the Line. What makes her tale even more interesting is that at the time of the life-threatening heart episode that necessitated the transplant, she was finishing up a residency in emergency medicine. In fact, Dr. Gragossian is dually trained in emergency medicine and critical care medicine. Since her transplant, she’s been using her platform to share her unique experiences with other health professionals and raise awareness about the importance of organ donation. “I’ve had a lot of amazing lessons from what I call ‘patient school’ that medical school never really taught me,” she tells host Michael Carrese. Listen in to this fascinating episode to hear Dr. Gragossian describe what life is like after an organ transplant and the lessons learned as a transplant patient that she’s applying to her medical career. She gets candid about what she would change about medical school curriculums and what would encourage more people to become organ donors. Then, she talks about her podcast, “Both Sides of the Stethoscope” and emphasizes the power of strong patient communities and support groups.

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There are so many choices to make as an undergrad in med school: selecting which medical field to go into; whether to go down the academic path; and how to use your knowledge and skills to find success and create positive change in the world, to name a few. On today’s episode we’re going to hear from someone who helps students work through all of those questions and also assists faculty colleagues with adjusting to the changing medical education landscape. Dr. Kim Tartaglia does all of this wearing several hats at Ohio State University Wexner College of Medicine including Professor of Medicine, Director of the “IMWell” program for internal medicine residents and Director of Faculty Mentorship. “There are so many different ways to make an impact that there’s not one path to success and there’s not one path to be impactful,” she tells host Michael Carrese. Listen in to this episode as Dr. Tartaglia shares her perspective on how medical education has changed since the pandemic as well as how students and academic leaders are relating to each other differently as they work to improve the med ed system. You’ll also learn how she chose her specialty in med school, what drew her to stay in academics, how she established an enjoyable career in medical academics, and the benefits of attending OSU’s College of Medicine. And stay tuned to the end for an enlightening discussion of the role of lifestyle medicine in treating and reversing disease, and the benefits of coaching and mentorship for med school residents.

Mentioned in this episode: https://medicine.osu.edu/

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A singing guest!  A poetic chat bot! This special episode of Raise the Line features those unusual artistic highpoints along with the substantive and interesting conversation you always get with host Shiv Gaglani. Our guest, Dr. Mark Korson, is a metabolic geneticist and Director of Education and Physician Support Services at VMP Genetics who believes patients have a crucial role to play in the education of both learners and practicing clinicians, especially when dealing with rare diseases. “Patients teach about disease a whole lot better than I do because they tell stories and storytelling is so incredibly powerful as a teaching tool.” Tune in as Korson talks about career opportunities in genetics and metabolic disease, the ways he integrates patient voices into his teaching, and the biggest opportunities and challenges in treating genetic and metabolic diseases and biochemical disorders. Plus, he shares his advice for learners about pursuing a career in the increasingly complex and demanding healthcare field. “You have to protect your personal life and protect it greedily because if you don't do that, at some point it's going to impact how you like your career.”

Mentioned in this episode: https://www.vmpgenetics.com/

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“There's a real diversity of jobs available that folks don’t always think about initially when they think about going into healthcare,” says Marc Cummings, the President and CEO of Life Science Washington, a nonprofit trade association serving the life sciences industry in the state of Washington. Dr. Tina Albertson, the Chief Medical Officer at nearby Lyell Immunopharma, agrees. For instance at her company, which does R&D on cell therapies for solid tumor cancers, there’s a need for specialists in logistics who organize and monitor the movement of patient cells that need to be flown to other locations to be genetically engineered and returned to the bedside for use in treatment. As these industry veterans share with host Michael Carrese, the Seattle region is a well-established hub in the growing biotech sector due to a unique blend of strengths including longstanding non-profit research institutions and powerhouse tech companies such as Microsoft and Amazon. “This region is really well-prepared for innovation from a basic science standpoint and also from the tech side of our community,” says Albertson. Check out this revealing discussion of the challenges and opportunities in life sciences, the critical role AI and machine learning is now playing, and what they wish more people understood about clinical trials and drug development. 

Mentioned in this episode: 
https://lyell.com/

https://lifesciencewa.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