Vital Science (Charles River)
Explore every episode of Vital Science
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| 19 Oct 2021 | Can MILs Strike a Killer Blow to Cancer? | 00:28:46 | |
From a young age, Kim Noonan knew what she wanted in life — a career in science and a desire to help. When her mother succumbed to gastric cancer, it drove her even more to help people, especially cancer patients. Today, she’s at the forefront of a potentially game-changing development in cancer therapy — using bone marrow-infiltrating lymphocytes (MILs) designed to target and kill cancerous tumors of all shapes and sizes. Find out how her early work with myeloma patients started her road to discovering MILs, the origin of WindMIL Therapeutics, challenges associated with manufacturing MIL-based therapies, and what the future holds for this revolutionary treatment. Learn more about WindMIL Therapeutics and the science behind their work with bone marrow immunology. SHOW NOTES
Hosted by: Gina Mullane | |||
| 17 Nov 2020 | Beyond the Headlines: Understanding Accelerated Vaccines | 00:23:11 | |
“Accelerating” a vaccine for COVID-19 has piqued the interest of many, but is it really possible to develop one quickly and safely? Join distinguished scientist Dr. Lauren Black as she dives into the typical mechanisms behind a vaccine and why it takes several years to deliver one safely to the general public. Acknowledgements: Hosted by: Chris Garcia Narrated by: Gina Mullane
Special thanks to: Dr. Lauren Black | |||
| 16 Apr 2024 | Mission Therapeutics: Inhibiting DUBs to Halt Diseases | 00:17:22 | |
At Mission Therapeutics, Sarah Almond serves as their Head of Pharmacology, but she wears many hats to help develop potential treatments for neurodegenerative conditions.
Their main area of focus centers around research into deubiquitinating enzymes (DUBs), which may impact neurodegeneration. By studying DUBs, Sarah and her team have been able to generate highly targeted and potent molecules that contribute to developing safe and effective therapeutics. In fact, one of these compounds (MTX325) is currently enrolled in a clinical trial to test its safety and pharmacokinetics ahead of effectiveness in treating Parkinson’s disease, a neurodegenerative condition impacting the central nervous system. With the first patient dosing scheduled for later this year, they are hopeful it could be a gamechanger for treating these debilitating conditions.
Join Sarah as we discuss Mission Therapeutics, the science behind DUBs, how collaboration with a CRO advanced their research, and what her thoughts are on the future of drug discovery and development for neurodegenerative conditions, among others. Show Notes
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| 16 Mar 2021 | 3D Cell Models: A Blueprint for Drug Development | 00:28:58 | |
Three-dimensional cell models are primed to play a vital role in the future of drug development. By recreating tissue that mimics human systems, scientists can monitor disease progression and evaluate the effects of drug candidates. Join Drs. Elizabeth Anderson and Ian Waddell as they explain the evolution of biologic modeling and how 3D cell models are being used to develop effective therapeutics to patients. Acknowledgements: Hosted by: Gina Mullane Narrated by: Chris Garcia
Special thanks to: Dr. Elizabeth Anderson | |||
| 21 Mar 2023 | Evie's Story Revisited | 00:29:16 | |
Imagine being diagnosed with an incurable rare disease before ever being born. This is the reality that Evie faced when she was diagnosed with hypophosphatasia, a condition impacting bone growth, breathing, and mobility, and often gives patients just 18 months to live.
Luckily, her story doesn’t end there. At three months old, she enrolled in a clinical trial for a first-of-its-kind medication that her mom Lindsey claims “probably saved her life”. Today, Evie is 13 years old and continues to defy the odds, giving hope to other young children suffering from a rare disease that they too can find the strength to overcome anything life throws at them.
Join Evie and Lindsey for this emotional look at their journey, how the power of collaboration has helped them along the way, and Evie’s goals for the future. Show Notes | |||
| 16 Jun 2020 | New Lease on Life | 00:34:38 | |
Dr. Guy Leclerc plays the role of both a researcher and a clinician, changing lives with his work on medical devices. Learn how his work directly improved the quality of life for a very special patient. Learn more about Isabelle’s story, and support what drives Dr. Leclerc by donating to the CHUM foundation. ACKNOWLEDGMENTS | |||
| 14 Nov 2023 | TREND: The Online Community Making a Difference | 00:44:40 | |
When Maria Picone’s daughter was diagnosed with Prader-Willi Syndrome (PWS), she turned to the internet for answers.
What she found was an online community of caregivers sharing their experiences with PWS, giving her comfort and direction with what to do about her daughter’s rare disease. It also spurred her and her husband to create TREND Community, an online platform for caregivers of rare diseases to not only share their experiences, but also make positive contributions to scientific literature.
Join us as we discuss the philosophy of TREND Community, how it transforms general discussions into real-world clinical research, how collaboration drives their progress, what’s next for TREND, and what you can do to help advance their mission.
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| 21 Sep 2021 | A Synergistic Approach to Treating Huntington’s Disease | 00:32:23 | |
Huntington’s disease may only affect one in every 10,000 people in the United States, but its symptoms can drastically debilitate a person’s quality of life. For Ignacio Muñoz-Sanjuan, he witnessed first-hand the devastating effects this disease can have. He’s visited parts of the world that have the highest prevalence of Huntington’s disease on the planet, including Taiwan and South America. Today, he’s involved with two different nonprofit organizations seeking to accelerate the development of treatments to help those afflicted by, or at risk for, this fatal condition. Join us as we explore Ignacio Muñoz-Sanjuan’s journey in raising awareness about Huntington’s disease, how Charles River contributed to developing a framework for potential treatments, and what he believes the future holds for drug development in this disease area. Discover how the CHDI Foundation and Factor-H are working to help those suffering from Huntington’s disease and how you can get involved.
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| 20 Feb 2024 | Cure Rare Disease: Continuing a Legacy | 00:35:57 | |
When he founded Cure Rare Disease in 2018, Rich Horgan was primarily focused on finding a treatment for his younger brother Terry, who had an ultra-rare form of Duchenne Muscular Dystrophy (DMD).
Suffering from a disease that has no cure, Terry served as a “selfless, brave, and courageous” figure that Rich continues to draw inspiration from when seeking new, innovative solutions to treat this progressive, neurodegenerative condition.
Join us for this interview as Rich discusses the origins of Cure Rare Disease, Terry’s ongoing legacy, the collaborative effort it takes to advance the drug development journey, the important role of the patient voice when developing treatments, and the future of rare disease research. Show Notes
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| 18 Jan 2022 | From Humble Beginnings to Potential Treatment for ALS | 00:33:19 | |
In March 2020, we invited Josh Cohen and Justin Klee on Vital Science to discuss Amylyx Pharmaceuticals, a company that had one simple mission – to improve the quality of life for those battling neurodegenerative diseases.
See what Amylyx Pharmaceuticals is currently working on and get updates on clinical trials. | |||
| 17 Mar 2020 | Humble Beginnings | 00:36:14 | |
College can take you many places. In the case of two neuroscience students, they didn’t know where their journey would take them. Their perseverance and fighting spirit built a company that aims to help those battling neurodegenerative diseases. | |||
| 17 Dec 2019 | A Toxigenic Situation | 00:41:09 | |
Donnie Patterson is not just a pharmaceutical microbiologist. He’s also a victim of toxigenic mold, a growing problem in South Carolina schools. Hear how his story exposed an issue that led to his ongoing fight for tougher mold testing regulations. Visit the Global Indoor Health Network website for more information on toxic mold. | |||
| 17 Jan 2023 | AviadoBio: Bold and Courageous | 00:31:09 | |
Recent advancements in research has led to the development of effective therapeutics to combat neurodegenerative diseases, giving patients a renewed sense of hope there will ultimately be a more effective treatment. CEO Lisa Deschamps and her team at AviadoBio are among those conducting research in the space, but they’re doing things a little differently. They’ve been given a unique drug designation by the FDA to develop a treatment for frontotemporal dementia (FTD), a crippling neurodegenerative disorder that affects neuron production in people under 65. It may be a “bold and courageous” undertaking, but the benefits could affect future generations to come. Join us as we take a look at the motivation behind the work AviadoBio is doing, what makes their therapy unique, and Lisa’s vision for their future. Show Notes | |||
| 16 Feb 2021 | Driving Drug Development with CRISPR/Cas9 | 00:26:37 | |
Within the past decade, CRISPR/Cas9 gene editing of mammalian cells has become common practice, but does it hold the key to unlocking future of therapeutic development? Join Dr. David Fischer and our panel for a detailed look at the advantages and applications of this Nobel Prize-winning technology. Acknowledgements: Hosted by: Gina Mullane Narrated by: Chris Garcia
Special thanks to: Dr. David Fischer | |||
| 15 Feb 2020 | An Angel’s Determination | 00:52:46 | |
There are certain moments when life takes on a whole new meaning. For Allyson Berent, her daughter’s diagnosis with a rare genetic disorder gave her a purpose – to embark on the quest for a cure. Hear her remarkable journey from a veterinarian to the COO of GeneTx Biotherapeutics, and how partnering with Ultragenyx pharmaceutical fuels her determination to cure Angelman Syndrome. Learn more about FAST (Foundation for Angelman Syndrome Therapeutics). | |||
| 15 Nov 2022 | A Father’s Race Against Time | 00:29:40 | |
Spastic Paraplegia 50 (SPG50) is a neurodegenerative and neurodevelopmental disorder that is known to affect only 80 people around the world, including just one person in Canada.
Unfortunately for Terry Pirovolakis, it happens to be his son, Michael. As a result, he’s teamed up with gene therapy experts, CROs, and nonprofits to research, manufacture, and deliver a therapy to help his son and others suffering from this ultra-rare disease, but the clock is ticking.
Join us as Terry discusses his grassroots efforts to raise money and found the Cure SPG50 Foundation, how SPG50 affects the body, the future of research and development for this disease, and what you can do to help Terry’s efforts. Show Notes:
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| 16 Jan 2024 | H-Guard: Improving Physical and Mental Health | 00:28:06 | |
For the 850 million people around the world suffering from chronic kidney disease, the grind of dialysis treatment can wear them down in more ways than one.
Dr. Andy Herbert, co-founder of Invizius, is out to change that. This five-year-old company started from humble beginnings and is currently developing H-Guard® Priming Solution, which increases the effectiveness of dialysis while negating, or eliminating, its potentially dangerous side effects. It will be used in an upcoming first-in-human clinical trial and has the potential to improve patients’ quality of life – both physically and mentally.
Join Dr. Herbert as we discuss the origins of Invizius, how H-Guard works, why dialysis can take a toll on mental health, and how working with a strategic partner has helped Invizius progress this treatment. Show Notes Renal Disorders | Charles River | |||
| 21 Feb 2023 | Neurogenesis: Regenerating Hope | 00:27:31 | |
Could it be possible to regenerate neurons in the human body?
It’s a question that has driven Dr. Wise Young for over 40 years. He’s dedicated his career to researching spinal cord injuries and how to develop a treatment for them. Along the way, he’s discovered neurogenesis, a way to regenerate neurons in not just the spinal cord, but also the brain. Thanks to his work, what everybody told him was once impossible, is now close to becoming a reality.
Join us as Dr. Young discusses his founding of, and work at, the W.M. Keck Center for Collaborative Neuroscience, how the components of stem cells and umbilical cord blood can help regenerate neurons, and what his vision is for the future of research in this growing field. Show Notes
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| 21 Dec 2021 | Donors: The Unsung Heroes Advancing Science | 00:15:29 | |
The role of human cells and tissues is key in the development of next-generation therapies. With just a few samples, donors contribute to scientific advancements in medicine. This is especially true with cell and gene therapy, a field that is projected to grow over the next several years, and with it comes the growing need to find reliable and recallable donors willing to help those in need. In our latest episode of Vital Science, learn more about the critical role these unsung heroes play in advancing the future of therapeutic development. Show Notes
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| 19 Dec 2023 | Checking Vitals: The Disruptors | 00:10:17 | |
Over the past 12 months, we have shared some remarkable stories from the scientific community, but three of the most impactful have come from “Disruptors” in their respective fields. Relive these stories with our hosts on the year-end edition of Vital Science. Show Notes
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| 16 Jul 2024 | FOXG1: A Mom’s Mission | 00:31:40 | |
Being the parent of a child with an ultra-rare disease can be a daunting challenge. Nasha Fitter is no different. Her daughter Amara is nonverbal and suffers from epileptic seizures, two of the common symptoms associated with her eventual diagnosis of an ultra-rare neurodevelopmental disorder known as FOXG1 Syndrome. Motivated by her desire to learn more about this condition, she co-founded the FOXG1 Research Foundation (FRF) with her fellow peers. Through all the challenges they’ve faced, Nasha and FRF are set to conduct critical research in the hopes of developing a treatment for not just this condition, but other ultra-rare diseases primarily affecting children to eventually give them “the life they deserve.” Join us as we conclude season five where Nasha discusses the origins of FRF, how Amara’s life has been shaped by her diagnosis, how collaboration has helped advance the Foundation’s mission, and what you can do to support their ultimate goals. Show Notes | |||
| 13 Dec 2022 | Checking Vitals | 00:15:51 | |
As we close in on the end of 2022, we’ve had the privilege to interview some amazing people who are doing great work with drug discovery. Join Gina Mullane and Todd Poley as we reflect on some of the memorable moments and guests from this season, including an update on Amylyx Pharmaceuticals’ new ALS treatment, Chris Claussen’s discussion on the positive impacts of psychedelic drugs, and the work of Project ALS to develop a treatment for this crippling disease. On behalf of our entire team, thank you for listening this year. We hope you’ll join us for an exciting lineup of remarkable stories set to premiere in 2023. Show Notes:
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| 15 Jun 2021 | Standing on the Shoulders of Giants | 00:30:44 | |
What if it was possible to speed up drug development without having to reinvent the wheel? By repurposing existing drugs, drug hunters are taking a giant step forward towards changing the future of drug discovery. Join our panel of drug hunters to hear how they reinvent previously approved drugs to get a head start towards treating patients. Acknowledgements: Hosted by: Chris Garcia Narrated by: Gina Mullane and Chris Garcia
Special thanks to: Ian Waddell | |||
| 21 Jun 2022 | Retrogenix: The Screen Door of Drug Development | 00:19:44 | |
Drug developers face long odds and complex regulatory challenges when it comes to crafting a suitable candidate. In fact, only one of every 10 drug candidates gain regulatory approval, and when they do, it takes an average of 14 years and costs well over $2 billion.
So what makes for a successful candidate? It starts with a suitable screening platform that identifies targets and the molecules that bind to them to create a therapeutic response. Retrogenix is one such platform that the top 20 drug developers in the world currently use to increase their chances of success thanks to its unique technology that doesn’t require animal models or human tissue to evaluate effectiveness.
Join us to find out how Retrogenix works to identify targets, accelerate drug development, get vulnerable patients the therapies they need more quickly, and adapt to meet the growing demands of regulatory agencies. Show Notes
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| 15 Mar 2022 | Are we One Shot Away? | 00:21:51 | |
Imagine a gene therapy treatment that gives drug-resistant focal epilepsy patients more control over their seizures and ultimately improve their quality of life? We sat down with Karin Agerman to discuss this possibility and discovered that her work at Combigene is at the forefront of a novel gene therapy called CG01. Find out how this single-injection therapy is giving renewed hope to this patient population and learn more about CombiGene’s research into CG01. SHOW NOTES
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| 18 Apr 2023 | Free for Life: The Story of n-Lorem Foundation | 00:30:41 | |
When patients face the diagnosis of a rare disease, they face a costly, uphill battle to develop a treatment for their condition… that is, if they ever do.
Dr. Stanley Crooke is aiming to change that. As the CEO of n-Lorem Foundation, his mission is to provide personalized treatments to ultra-rare disease patients for free as long as they live, no questions asked. Thanks to his humility, coupled with the generosity of other individuals and companies, ultra-rare disease patients have a renewed hope to get the treatments others may not be able to provide.
Join Dr. Crooke as he reflects on his humble beginnings at Ionis, the approach n-Lorem takes to developing drugs for ultra-rare diseases, and what the future holds for research in this area. Show Notes
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| 15 May 2020 | Leading the Way | 00:30:14 | |
Jake Glanville, founding partner and CEO of Distributed Bio, has been featured in the Netflix series "Pandemic." Now, he and his team are on the front lines of the fight against COVID-19, working tirelessly to find a cure. Learn how his roots in Guatemala have shaped his beliefs on healthcare, how a family business has developed his skills as a leader, and how it all came together in his leadership of a talented, accomplished team of scientists. Read more about antibody discovery technologies and advancements. ACKNOWLEDGMENTS Hosted by: Gina Mullane Special thanks to: Jacob Glanville, PhD | |||
| 19 Nov 2019 | The Pathway | 00:48:34 | |
Mila’s groundbreaking treatment, the first created and approved for a single patient, has given hope to thousands of rare-disease patients around the world. Hear Julia Vitarello, Mila’s Mom, and Dr. Timothy Yu discuss their plans to help other children with rare disease. Watch the documentary and learn more about Mila’s story at: www.criver.com/everystep In partnership with Mila’s Miracle Foundation, we are on a mission to bring this new treatment path to children fighting rare diseases. An anonymous donor will match up to $150,000 in donations to help with this fight, and Charles River has already donated $50,000 to get the campaign started. ACKNOWLEDGMENTS Hosted by: Gina Mullane and Chris Garcia Special thanks to: Julia Vitarello, Dr. Timothy Yu, and Boston Children’s Hospital. | |||
| 19 Jul 2022 | Logica– Drug Discovery Made Smarter | 00:35:11 | |
What happens when you combine an industry-altering, AI-powered platform with a leading CRO's preclinical expertise?
That’s what Valo Health and Charles River are answering with the introduction of Logica™. This revolutionary platform aims to accelerate drug discovery and development by finding the most viable molecules faster, helping biotechs and pharma focus their resources only on the ones that will evolve into therapies that patients will receive more quickly.
Join Guido Lanza (Valo) and Julie Frearson (Charles River) as they discuss the components of Logica™, what makes it unique from other platforms, and how it will transform the future of drug discovery. Show Notes
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| 21 Jan 2020 | Turning Point | 00:28:56 | |
What we experience can leave a lasting impact on our lives. In Carina’s case, her father’s diagnosis changed her career forever. Hear the emotional story of her ongoing fight to honor his memory and help others battling this rare neurodegenerative disease. | |||
| 20 Apr 2021 | The Life (Saving) Blood of Horseshoe Crabs | 00:43:41 | |
The discovery of limulus amebocyte lysate (LAL) in the blood of horseshoe crabs has forever changed bacterial endotoxin testing. Today, researchers continue to look for any way they can protect these seafaring creatures while refining testing methods to decrease the need for animals. Join our panel for an in-depth look at how they’re working with legislators to preserve the horseshoe crab population and how LAL testing is the safest in vitro method to detect harmful bacteria. Acknowledgements: Hosted by: Gina Mullane Narrated by: Chris Garcia
Special thanks to: Dr. Norman Wainwright | |||
| 21 Apr 2020 | Uncommon Bond | 00:46:19 | |
Twins share many unique bonds, and for Jaci and Alex, fighting the same rare disease is one of them. Hear this emotional tale through the perspective of their mom, Lori, and their uncle, Mike, about how they have battled their illness, knowing the risks, and helped to develop a new treatment for this rare form of ALS. To learn more about Jaci’s story, please visit Cowgirl Up for Jaci: Roping in a CURE for ALS. For more information about ALS, please visit Project ALS. ACKNOWLEDGMENTS Hosted by: Chris Garcia Special thanks to: Lori Hermstad, and Mike Hadden | |||
| 19 Jan 2021 | Get the Series Recap: Vaccines | 00:03:20 | |
Get your four-part dose of vaccine…information, that is. In this brief recap, Vital Science hosts Gina Mullane and Chris Garcia chat about the content of our first several Season 2 episodes – a miniseries covering infectious disease and immunity, origins of vaccination, therapeutic development and regulation, and the scientific community’s recent accomplishments in the fight against COVID. Download individual episodes: See what you might have missed in the vaccine series or download and save individual episodes on our Season 2 tab.
Beyond the podcast: Season 1 of Vital Science™ shared numerous compelling stories, like that of the Hermstad twins and their battle with ALS (Episode 7, An Uncommon Bond). Sadly, Jaci passed away shortly after her podcast aired, but the fight continues as clinicians, advocates, and drug developers continue to work in her honor. As Chris mentions in the recap, if you’re interested in the next chapter of her story, you won’t want to miss a free virtual event with our special guests Ben Stiller and Project ALS:
The Hermstad Legacy: Advances in Treatment for ALS A Rare Disease Trailblazer Event Thursday, February 4 | 11 a.m. – 4:00 p.m. ET Acknowledgements: Hosted by: Gina Mullane and Chris Garcia | |||
| 22 Oct 2019 | Being the First | 00:43:56 | |
Mila is a young girl fighting Batten Disease, a rare, fatal disease with no cure. Her Mom, Julia Vitarello sits down with Gina to tell the story of Mila’s diagnosis and her incredible journey since; from diagnosis to Mila’s experimental genetic treatment, created just for her. Watch the documentary and learn more about Mila’s story at www.criver.com/everystep. Hosted by: Gina Mullane and Chris Garcia | |||
| 18 Jul 2023 | Modernizing Drug Development | 00:25:58 | |
For decades, researchers have utilized animal models to help predict the effects of a drug compound in humans.
With recent advances in technology and the passage of the FDA Modernization Act 2.0, researchers are increasingly looking at new methods to refine, reduce, and replace in vivo testing to advance drug candidates. However, one important question remains – how are all these modern advancements and laws driving progress in drug discovery?
Find out as Michael Templin, a member of Charles River’s Scientific Advisory Services team, joins us for a look at the impacts of this legislation on regulatory submission for biosimilars, how improving animal welfare will make drug development better, and what knowledge a CRO can bring to optimize projects. Show Notes
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| 20 Oct 2020 | A Formidable Defense Against Infectious Diseases | 00:46:44 | |
When the immune system is compromised, people rely on vaccines to eliminate and inactivate the threat. However, the process with which this happens is anything but simple. Dr. Christina Satterwhite joins us to explain how the immune system works and how vaccines target harmful pathogens in the second installment of our series on vaccines. Acknowledgements: Hosted by: Chris Garcia Narrated by: Gina Mullane
Special thanks to: Dr. Christina Satterwhite | |||
| 20 Sep 2022 | Project ALS: Fueled by Love | 00:35:17 | |
For Valerie Estess, finding a cure for ALS is personal. Her sister, Jenifer, was diagnosed with this crippling disease in 1998, which led to the founding of Project ALS, a nonprofit designed to raise awareness and eventually find a cure. Nearly 25 years later, they have raised over $100 million and helped develop Jacifusen, the first therapy designed to help treat ALS. However, Valerie believes their work is far from finished. Join us for an intimate discussion on the origins of Project ALS, their collaborative efforts with leading scientists, academic institutions, and contract research organizations to further their knowledge of the disease, and what lies ahead for Project ALS’ mission and research. SHOW NOTES | |||
| 22 Sep 2020 | Infectious Disease and The Rise of Vaccines | 00:19:40 | |
Since 1796, vaccines have helped humans combat smallpox, the flu, and other infectious diseases. The process of vaccine development has changed drastically since then, and is now front and center in the wake of COVID-19. In the first episode of our series on vaccines, join us as we talk with Dr. Sarah Gould about the history of vaccines and infectious disease. Acknowledgements: Hosted by: Chris Garcia Narrated by: Gina Mullane
Special thanks to: Dr. Sarah Gould | |||
| 21 May 2024 | Clear Scientific: Combatting Drug Overdose from Methamphetamine, Fentanyl, and Polydrug | 00:26:53 | |
Stimulant use in the United States has exploded into a nationwide crisis, as deaths and emergency room visits resulting from methamphetamine and cocaine use are rising at an alarming rate. The overall substance abuse disorder crisis is further exacerbated by the high level of fentanyl use, which has long been referred to as an opioid epidemic, and the increasing use of more than substance at once. One fundamental limitation on the ability to respond to the crisis is that, to date, there is no FDA-approved therapeutic to treat overdose from stimulants like methamphetamine and cocaine, or polydrug. Users who want to get clean and rid themselves of methamphetamine, cocaine, and/or fentanyl often find it difficult, essentially “chaining” themselves into a cycle of use that’s mostly impossible to break. First responders and emergency department physicians lack sufficient tools to treat overdose. But what if there was a treatment that could eliminate toxins and restore normal bodily function within minutes? That’s the aim of Clear Scientific, a small biotech in Cambridge, MA that’s currently testing a treatment (CS-1103) using small molecules to “eat” toxins and deactivate their harmful effects almost instantly. With Phase I of a first-in-human (FIH) clinical trial ongoing, this revolutionary therapeutic is offering hope to those suffering from drug misuse and addiction a “chance to get better.” Join us as Mitch Zakin, Co-founder and Executive Vice President of Innovation, and Winston Henderson, Co-founder and General Counsel, discuss their therapeutic approach, offer an update on their progress with CS-1103, how collaboration has enhanced their development, and how they hope it will eventually impact this epidemic. Show Notes World Congress: Drug Modality Game Changers Clear Scientific to Start Phase 1 Trial for Stimulant Antidote | |||
| 20 Jun 2023 | SCA3: A Family Affair | 00:23:49 | |
Spinocerebellar ataxia type 3 (SCA3) is a neuromuscular disease affecting just 50,000 people in the United States, including three generations of the Klassen family.
Greg Klassen, his father, and his son have all been diagnosed with this rare condition that impacts the central nervous system, affecting balance, coordination, and mood. Motivated by the 50/50 chance his son Jeff will develop its unforgiving symptoms, Greg joined the board of the Cure Rare Disease foundation and is currently in touch with the efforts being made to develop a treatment that will improve his quality of life and others affected by SCA3.
Join us for a conversation on how Greg copes with SCA3 in his own life, what the rare disease community is doing to raise awareness, how antisense oligonucleotides (ASOs) could lead to a potential treatment, and what it would mean to him if a cure was developed. Show Notes | |||
| 15 Dec 2020 | COVID-19 Vaccines – Behind the Scenes | 00:20:29 | |
Recent developments in the race to develop a COVID vaccine show promise, but the question remains – when will it become available to the general public? Dr. Sarah Gould returns for the fourth and final installment of our vaccine miniseries to examine where we stand with the development of COVID-19 vaccines and some lingering challenges that may hamper their final approval. Acknowledgements: Hosted by: Chris Garcia Narrated by: Gina Mullane
Special thanks to: Dr. Sarah Gould | |||
| 19 Apr 2022 | (Re)programming Stem Cells for Drug Development | 00:27:51 | |
A medic by trade, Mark Kotter has always held a particular interest in neuroscience and stem cell research. As founder and CEO of bit.bio, he's overseen the development of a unique approach to "reprogram" stem cells and change them into consistent and scalable disease-relevant cells, which can then be screened for potential therapeutics to help treat such diseases as Huntington's Alzheimer's, and ALS. Hear about this revolutionary technology and how it works, what benefits it can provide researchers, and how it could very well shape the future of drug development. Show Notes
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| 17 May 2022 | Open Science: The Parallel Universe | 00:28:05 | |
Aled Edwards’ vision of science is set to shake the foundation of drug discovery and development as we know it.
The Director of M4K Pharma and CEO of the Structural Genomics Consortium is leading the charge for a more open scientific approach. It’s a parallel universe where scientists can work together and get therapies designed to treat rare diseases in the hands of patients even faster – one that isn’t bound by patents, siloes, and profits.
Join Aled as he discusses the “open science” approach, the current state of the pharmaceutical industry, and what the future holds for genetic biology. Show Notes
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| 17 Oct 2023 | Strength in Numbers | 00:54:06 | |
Imagine a world where your child has a rare disease that’s so rare, it doesn’t have a name and only a handful of people around the world have a similar condition. It can leave you feeling helpless and alone knowing the odds are stacked against you.
This is the situation Bo Bigelow experienced with his daughter, Tess. She suffers from a USP7, a neurodevelopmental disorder that impacts her motor function, speech, and bone development. Tess’ condition left Bo looking for help… and he found a lot of other patients and families with other ultra-rare diseases that wanted to share their stories too. Today, Bo’s initiatives have given hope to other people and help them find strength in knowing they’re not alone.
Join us as we discuss the ways Bo documented his daughter’s journey, learn more about USP7-related diseases and their impact, how Bo created the Disorder Channel as a way for other rare disease patients to tell their stories, what progress is being made to learn more about Tess’ condition through the Foundation for USP7-Related Diseases, and how you can help advance their research.
Foundation for USP-7 Related Diseases
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| 18 May 2021 | Can a Repurposed Drug Be the Answer? | 00:46:07 | |
What if the cure for your untreatable disease was just waiting on the shelf at your neighborhood pharmacy? Join physician, patient, and researcher Dr. David Fajgenbaum as he shares how his unexpected battle with Castleman disease set him on a journey to find his cure and develop new ways of using existing drugs. Acknowledgements: Hosted by: Chris Garcia Narrated by: Gina Mullane
Special thanks to: Dr. David Fajgenbaum | |||
| 26 Sep 2019 | This is Vital Science™ | 00:01:12 | |
Meet our hosts Gina Mullane and Chris Garcia as they introduce Vital Science™, a brand new podcast that shares the personal stories of those impacted by life science. Subscribe so you don't miss us, starting October 2019. ACKNOWLEDGMENTS Hosted by: Gina Mullane and Chris Garcia | |||
| 19 Sep 2023 | Emily Whitehead: Pioneer and Hero | 00:33:29 | |
18-year-old Emily Whitehead aspires to be a writer, but nothing could have prepared her for the plot twist that struck her as a child.
In the space of just 24 hours, five-year-old Emily suffered from various symptoms that culminated in a diagnosis of acute lymphoblastic leukemia (ALL). After almost two years of failed chemotherapy treatments, her family turned towards an experimental CAR-T therapy that had been successful with adults but never been used on a child. Three weeks later, Emily became the first child in the world to beat her cancer by “training” her immune system to fight these malicious cells.
Join Emily and her father, Tom, as they relive the chain of events that led to her diagnosis, what helped her stay calm as she underwent this experimental treatment, and how the Emily Whitehead Foundation aims to help others who are going through pediatric cancer treatment. Show Notes
Vital Science S3 E02: Can MILs Strike a Killer Blow to Cancer? World Congress Event Page | Charles River Cell and Gene Therapy | Charles River Oncology CRO Services | Charles River | |||
| 20 Jul 2021 | Opening Treatments | 00:38:14 | |
Sanath Ramesh was determined to track down the resources needed to repurpose a drug for his son Raghav’s ultra-rare disease. This determination led him to apply his experiences and empower the rare disease community with his collaborative platform, the OpenTreatments Foundation. Find out how his repurposed drug platform is now giving hope to other rare disease patients. OTHER RESOURCES: | |||
| 16 May 2023 | Moving FAST to Treat Angelman Syndrome | 00:30:42 | |
The effects of Angelman Syndrome (AS) on young children are debilitating. From an early age, they face an uphill battle with this neurogenetic disease’s unrelenting symptoms, including seizures, cognitive impairments, and loss of motor function. It can also be tough on caretakers – just ask Dr. Allyson Berent and Jennifer Panagoulias. They both have personal connections to this disease, which in Dr. Berent’s case led to her creating the Foundation for Angelman Syndrome Therapeutics (FAST) in 2008. Today, Panagoulias, whose niece has AS, serves as their head of Regulatory and Policy, tasked with the responsibility to find a path that will develop treatments to improve the quality of life for both patients and caregivers. Join Jennifer as we discuss the origins of FAST, the science behind Angelman Syndrome, the crucial role outside partnerships have played in research, and what’s in the pipeline for potential therapeutics. Show Notes
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| 18 Jun 2024 | Cure EB: Healing the Wounds | 00:41:24 | |
Imagine giving birth to a baby girl and noticing pieces of skin missing from her body almost immediately. For Sharmila Nikapota, this was the case with her firstborn, Sohana. Genetic testing revealed that she was one of the over 500,000 people globally suffering from Epidermolysis Bullosa (EB), a “constantly painful and debilitating” skin condition where minimal contact can lead to blisters, wounds, tissue damage, eating difficulties, and eye injuries, among other symptoms. After seeking answers for her daughter’s prognosis and not getting any, Sharmila started Cure EB to educate people and eventually discover ways to manage this painful condition. With the UK approving a first-of-its-kind topical treatment for EB, she hopes it’s the first of many that can give patients like her daughter a better quality of life. Join Sharmila as she discusses how Sohana’s experience with EB has shaped her personality, the origin and mission of Cure EB, the challenges of managing Sohana’s condition, and what you can do to further Cure EB’s ultimate goals. Show Notes Cure EB - Accelerating Research to End Painful Skin | |||
| 21 Jul 2020 | Two in the Same | 00:41:32 | |
Cate and Laura Cheevers are sisters who were both born with cystic fibrosis. Together, they’ve been through multiple clinical trials for genotype-specific treatments and continue to defy the odds. Hear their unique tales of growing up with this rare genetic disorder and how it’s shaped their lives today. Learn more about how the Cystic Fibrosis Foundation and Vertex Pharmaceuticals are working on treatments to aid those living with CF, and support Great Strides on their quest to find a cure for CF. Acknowledgements: Hosted by: Chris Garcia Produced and Edited by: Meaghan Root and Ryan Stevenson Sound and Music by: Ryan Stevenson
Special thanks to: Kim, Cate, and Laura Cheevers | |||