Behind the Breakthroughs

Josh Denny, MD, is a trailblazer in precision medicine whose journey was deeply influenced by his family’s struggles with cystic fibrosis, a disease that claimed the lives of four of his grandparents’ children. This personal connection fueled his pursuit of a career blending medicine, genetics, and technology, leading to groundbreaking work in medical informatics and personalized healthcare.In this episode of Behind the Breakthroughs, we delve into Josh’s leadership of the All of Us Research Program. Since its launch in 2018, this ambitious initiative has enrolled 850,000 participants and collected over 400,000 genomic profiles. Its goal: to harness diverse biological data to advance understanding of health disparities, rare diseases, and common conditions. Discover how Josh’s work is revolutionizing healthcare, from life-saving genetic testing to targeted therapies, and shaping a future where medicine is tailored to every individual’s unique genetic and environmental profile.Stay up to date with the latest episodes of Behind the Breakthroughs by subscribing to the IPM eNewsletter here.Produced by Bill LevineHosted by Jonathan D. GrinsteinMusic provided by Artem HramushkinAudio mixed and mastered by Jason Mack

The sage-like Uli Stilz, PhD, Vice President of Novo Nordisk, provides insight into the inner workings of Novo Nordisk’s Bio Innovation Hub, which he founded in Boston, to tackle complex, multifactorial diseases that impact billions of people, such as diabetes and cardiometabolic diseases.Stay up to date with the latest episodes of Behind the Breakthroughs by subscribing to the IPM eNewsletter here.Produced by Bill LevineHosted by Jonathan D. GrinsteinMusic provided by Artem HramushkinAudio mixed and mastered by Jason Mack

Progress in cancer diagnostics has been slow. While there has been progress in cancer screening, including new tests like Cologuard for colorectal cancer, improved mammography, and HPV testing for cervical cancer, the only new cancer added to screening since the start of my career is lung cancer. Despite these advancements, only one in six cancers in the U.S. is detected through screening, underscoring the need for significant improvement. This is why the current work to revolutionize and expand screening holds such transformative potential.After a decades-long career as a physician-scientist, Tomasz (Tom) Beer, MD, let go of the majority of his practice at Oregon Health & Science University (OHSU) two years ago and joined Exact Sciences, the developer of Cologuard, to move the needle on cancer screening by developing blood-based multicancer early detection (MCED) tests. In this episode of Behind the Breakthroughs, Beer explains the ins and outs of developing MCED tests that can be used to screen people from all walks of life for cancer.Stay up to date with the latest episodes of Behind the Breakthroughs by subscribing to the IPM eNewsletter here.Produced by Bill LevineHosted by Jonathan D. GrinsteinMusic provided by Artem HramushkinAudio mixed and mastered by Jason Mack

At the end of her clinical training as a hemo-oncologist specializing in bone marrow and stem cell transplantation, Katy Rezvani, MD, PhD, realized that she had this love of immunology and looking after patients who undergo stem cell transplantation, especially allogeneic stem cell transplantation—taking a graft from one healthy donor and giving it to another person. She knew that much of that curative effect is driven by the immune system, so she began to ask the question: can you isolate that robust anti-leukemia immune response from all the side effects that come with doing a stem cell transplantation?That question launched Rezvani into a successful career in understanding and developing allogeneic immune cell therapies. In this episode, Rezvani tells me why she’s so captivated by one particular immune cell—NK cells—for cell therapies. We discuss how NK cells compare to T cells and the different ways they are being engineered to treat cancer and other diseases with accessible and affordable cell therapies.Rezvani is a Professor at MD Anderson Cancer Center and, most recently, was dubbed the vice president & head of the Institute for Cell Therapy Discovery & Innovation. Rezvani also holds the inaugural Melvyn N. Klein Family Endowed Directorship for the Institute for Cell Therapy Discovery & Innovation.Stay up to date with the latest episodes of Behind the Breakthroughs by subscribing to the IPM eNewsletter here.Produced by Bill LevineHosted by Jonathan D. Grinstein, PhDMusic provided by Artem HramushkinAudio mixed and mastered by Jason Mack

"Are we close to curing diseases like ALS and Alzheimer's?" In recent years, several academic and industry endeavors have given reason to be hopeful. Toby Ferguson, MD, PhD, is the Chief Medical Officer at Voyager Therapeutics, which develops genetic medicines and delivery tools to cross the blood-brain barrier (BBB). In this episode, Ferguson explains the current state of therapeutics for neurological and neurodegenerative diseases, challenges in delivering genetic medicines and other therapeutics to the nervous system, and Voyager's approach to overcoming the BBB. Stay up to date with the latest episodes of Behind the Breakthroughs by subscribing to the IPM eNewsletter here.Produced by Bill LevineHosted by Jonathan D. GrinsteinMusic provided by Artem HramushkinAudio mixed and mastered by Jason Mack

For the first episode of 2025, I spoke with Tomi Pastinen, MD, PhD, Director of the Center for Pediatric Genomic Medicine at Children's Mercy Kansas City (CMKC), one of the best pediatric hospitals in the United States. Dr. Pastinen has pioneered genomics for mass diagnosis and treatment of pediatric patients at large medical centers, a fundamental capability necessary for precision medicine. With Dr. Pastinen at the helm, CMKC became the first pediatric hospital in Missouri to offer in-house pharmacogenetic testing with a test called Kiddose and the first in the country to use a genomic sequencing approach that reads not 4 but 5 different nucleotides in the clinical setting. In addition to discussing the clinical upside of incorporating genomic testing into a pediatric hospital setting, we also discuss some challenges that arise from a genetically characterized diagnosis to actual treatment—such as what happens when a child is diagnosed by identifying a disease-causing variant for which no existing treatment exists—as well as the world beyond well-defined inherited diseases to more complex disorders, including autism.Stay up to date with the latest episodes of Behind the Breakthroughs by subscribing to the IPM eNewsletter here.Produced by Bill LevineHosted by Jonathan D. GrinsteinMusic provided by Artem HramushkinAudio mixed and mastered by Jason Mack

One of the most remarkable patient stories of the 21st century has been that of Emily Whitehead—the first pediatric patient to receive chimeric antigen receptor T-cell (CAR T) therapy. Emily's parents have been along her side every step of the way, which is not something everyone can say. However, in the process, her father, Tom Whitehead, became incredibly moved by the experience with Emily and everything he saw. He decided to devote much of his time to helping families and patients who find themselves in Emily's shoes. Today, Tom is doing that through the Emily Whitehead Foundation.In this candid and emotional episode of Behind the Breakthroughs, Tom Whitehead shares intimate details of his journey by Emily’s side up to and through her treatment with CAR T, which occurred through a combination of serendipity and persistence. Tom also discusses in depth the challenges that patients and families face in dire medical situations, especially when it involves research-as-care. This is the 2024 finale for Behind the Breakthroughs, and we will release our next episode on January 8, 2025.Stay up to date with the latest episodes of Behind the Breakthroughs by subscribing to the IPM eNewsletter here.Produced by Bill LevineHosted by Jonathan D. GrinsteinMusic provided by Artem HramushkinAudio mixed and mastered by Jason Mack

Michael Severino, MD, discusses the approaches for developing "genome writers" at Tessera Therapeutics, where he joined as CEO in 2022, and the advantages of performing in vivo genome editing over ex vivo approaches. Severino explains where the company is in utilizing its genome writing platform to perform edits in vivo on its journey toward the clinic.Stay up to date with the latest episodes of Behind the Breakthroughs by subscribing to the IPM eNewsletter here.Produced by Bill LevineHosted by Jonathan D. Grinstein, PhDMusic provided by Artem HramushkinAudio mixed and mastered by Jason Mack