Wellness Exchange: Health Discussions
Breakthrough: CRISPR-Edited Cells Conquer Autoimmune Diseases in Trial
(upbeat music) - Welcome to "Listen To." This is Ted. The news was published on Monday, October 7th. Today we're joined by Eric and Kate to discuss a fascinating new medical breakthrough. Folks, it's great to have you here. - Thanks, Ted, happy to be here. - Glad to be on the show. - Today we're discussing a groundbreaking study on CRISPR modified cell therapy for autoimmune diseases. Can you explain what this therapy involves and why it's significant? - Sure thing, Ted. So this new therapy called BRL301 is like a superhero makeover for regular old T-cells. We're talking about taking donor T-cells and giving them a CRISPR facelift. It's a real game changer because it's an off-the-shelf treatment. Unlike the traditional car T-cell therapies where we have to use the patient's own cells, which is a real pain in the neck, time-wise and money-wise, this new approach is more like grabbing a can of soup off-the-shelf, quick, easy and ready to go. - While it sounds promising, we shouldn't get ahead of ourselves using donor- - I hear your concern, Kate, but let me jump in here. The researchers weren't born yesterday. They've actually addressed that issue head-on. They used CRISPR to knock out five specific genes that are involved in immune rejection. It's like giving these donor cells an invisibility cloak. So the patient's immune system doesn't freak out and attack them. Pretty clever, right? - But gene editing itself can have unintended consequences. We're not just painting-- - I get where you're coming from, Kate, but let's not throw the baby out with the bath water. CRISPR technology has come a long way and the researchers are being incredibly careful. They're not just willy-nilly cutting and pasting genes. This is precision work. - Interesting points from both of you. Can you both elaborate on how this therapy specifically targets autoimmune diseases? - Absolutely, Ted. So picture this. These souped up T-cells are like tiny superheroes equipped with special goggles, we call them cars, that can spot a specific bad guy called CD-19. Now CD-19 is a receptor found on B-cells, which are often the troublemakers in autoimmune disorders. By targeting these B-cells, the therapy can potentially hit the reset button on the immune system. It's like rebooting your computer when it's acting up. - That's oversimplifying it. Autoimmune diseases are complex and we-- - I get that it might seem oversimplified, Kate, but sometimes simple solutions can tackle complex problems. Look, the study shows some pretty exciting results. Three patients achieved complete B-cell depletion, that's like wiping the slate clean, and they went into deep remission without any serious side effects, that's huge. - Three patients is an extremely small sample size. We need much larger trials to truly assess the safety and efficacy of this treatment. It's like judging a restaurant based on one bite of an appetizer. We need to see the full menu and try multiple dishes before we can really say if it's any good. - Let's discuss the potential impact. How might this therapy change the landscape of autoimmune disease treatment? - Ted, if this pans out, we're looking at a potential revolution in treating autoimmune diseases. Imagine offering a one-time treatment for severe cases that don't respond to conventional therapies. We're not just talking about managing symptoms here. This could be a bona fide cure. It's like the difference between taking painkillers for a broken leg versus actually setting and healing the bone. - That's a big if, Eric. We need to pump the brakes a bit here. What about the long-term effects? What if patients relapse? And let's not even get started on the cost. This kind of cutting-edge treatment is likely to cost an arm and a leg. We could be creating a cure that only the super rich can afford. - Let's put this in historical context. Can you think of a similar breakthrough in medical history that faced similar promise and challenges? - Great question, Ted. This reminds me a lot of the development of monoclonal antibodies back in the '70s. When they first came on the scene, people were freaking out about safety and whether they'd actually work. Fast forward to today and they're a cornerstone of modern medicine, especially for treating autoimmune diseases. It's like when people were skeptical about the first automobiles and now we can't imagine life without cars. - That's not really comparable. Monoclonal antibodies don't permanently alter a patient's cells or immune system. - I see where you're going with that, Kate, but let's not forget that gene therapy has come a long way since those early days. The first gene therapy got the green light in 2012 and now we've got several treatments that are knocking it out of the park. This CRISPR therapy is standing on the shoulders of giants, so to speak. - But those advancements took decades, Eric, and there were serious casualties along the way. Are we rushing into this new frontier without fully understanding the risks? It's like we're explorers charging into uncharted territory without proper maps or supplies. - Interesting comparisons. How do you think the regulatory landscape today might affect the development of this therapy compared to those historical examples? - Well, Ted, the regulatory landscape today is like Fort Knox compared to the Wild West of yesteryear. We've got way better safeguards and monitoring systems in place now. This therapy didn't just appear out of thin air. It's already been through the ringer with rigorous preclinical testing before even touching a human patient. It's like how we now have airbags, seatbelts, and crumple zones in cars compared to the early days of automobile safety. - For regulations struggle to keep pace with rapidly advancing technologies like CRISPR. We're in uncharted territory. - I hear you, Kate. But that's precisely why these initial trials are so small and closely monitored. It's not a headlong rush. It's a cautious step-by-step approach. We're dipping our toes in the water before diving in, making sure it's safe while still pushing the boundaries of science. - Small trials can miss rare but serious side effects, Eric. We need to be prepared for the possibility of unexpected long-term consequences. It's like testing a new airplane design with only a few flights. You might miss a critical flaw that only shows up after thousands of hours in the air. - Both valid points. How do you think public perception and ethical considerations might influence the development of this therapy? - I think there's going to be a ton of excitement about this TED. We're talking about a potential cure for debilitating autoimmune diseases that ruin lives. That kind of hope can be a powerful driving force. It could really light a fire under investors and researchers, potentially fast-tracking the development process. It's like the space race, but for medicine. - But we can't ignore the ethical implications, Eric. We're talking about altering human cells and potentially passing those changes to future generations. This isn't just about curing diseases. It's about fundamentally changing what it means to be human. We need careful public discourse and oversight, not just a mad dash to the finish line. - Looking to the future, how do you see this technology developing? What are the best and worst-case scenarios? - In the best-case scenario, Ted, this therapy could be the key that unlocks treatments for a whole host of autoimmune diseases. Imagine a world where conditions like lupus, rheumatoid arthritis, and multiple sclerosis can be cured with a single treatment. It's like we're on the cusp of a medical revolution, similar to the discovery of antibiotics. - That's overly optimistic. Even if it works, it's likely to be extremely expensive and inaccessible to most patients. - I get your concern about cost, Kate, but let's not forget that as with any new technology, prices tend to come down over time. Remember when cell phones were a luxury item? Now, almost everyone has one. Plus, we need to consider the long-term savings from curing chronic diseases. It could actually save healthcare systems a fortune in the long run. - That assumes it works as well as you hope, Eric. A worst-case scenario could involve patients developing new autoimmune conditions or cancers years after treatment due to unforeseen effects of the gene editing. We could be opening a Pandora's box here. - Interesting perspectives. How might this technology be applied beyond autoimmune diseases? - The potential is mind-blowing, Ted. We could be looking at similar approaches to tackle genetic disorders, certain types of cancer, or even supercharge the human immune system to fight off infections better. It's like we're unlocking cheat codes for the human body. - That's veering into dangerous territory. Are we talking about designer babies and human enhancement? There are serious ethical issues. - Whoa, hold your horses, Kate. We're not talking about designer babies here. This is about curing diseases and reducing human suffering. We shouldn't let fear hold back potentially life-saving treatments. It's like refusing to use electricity because you're afraid of getting shocked. - But we need to proceed with extreme caution, Eric. Once we start down this road of altering human genetics, it's hard to go back. We could be opening doors we can't close. - Both of you raise important points. How do you think society should balance the potential benefits with the risks and ethical concerns? - Great question, Ted. I think we need robust but flexible regulatory frameworks that allow for scientific progress while ensuring patient safety. It's like having traffic laws that keep people safe, but still allow us to get where we need to go. And public education about these technologies is crucial. We need everyone to understand what's at stake. - I agree that education is key, but we also need diverse voices at the table when making decisions about these technologies. Scientists and biotech companies shouldn't be the only ones steering this ship. We need ethicists, patient advocates, and representatives from different communities to weigh in. It's too important to leave to any one group. - Well, folks, this has been an incredibly insightful discussion. We've covered a lot of ground today from the exciting potential of CRISPR-modified cell therapy to the crucial ethical considerations we need to keep in mind. Thanks to both Eric and Kate for sharing your perspectives. It's clear that as we move forward in this field, we'll need to balance innovation with caution and hope with realistic expectations. That's all for today's show. Thanks for tuning in to Listen 2.