Menin Inhibition Shows Potential for Precision Therapy in AML with Dr. Mollie Leoni Kura Oncology
Release Date: 06/25/2024
Novel Precision Gene Therapy for Friedreich’s Ataxia with Dr. Gabriel Brooks Solid Biosciences TRANSCRIPT
Empowered Patient Podcast
Dr. Gabriel Brooks is Chief Medical Officer at Solid Biosciences, a precision molecular genetic medicines company focused on rare cardiovascular and neuromuscular diseases, including Friedreich's ataxia. Currently, there are very limited treatments for this rare, progressive neurologic disease caused by a genetic deficiency. Solid Biowsciences' novel gene therapy uses dual-route administration to deliver directly to the heart and brain and to replace the missing frataxin gene, which is critical for energy production. Dr. Brooks explains, "Our flagship program is our DMD program, where we...
info_outline
Novel Precision Gene Therapy for Friedreich’s Ataxia with Dr. Gabriel Brooks Solid Biosciences
Empowered Patient Podcast
Dr. Gabriel Brooks is Chief Medical Officer at Solid Biosciences, a precision molecular genetic medicines company focused on rare cardiovascular and neuromuscular diseases, including Friedreich's ataxia. Currently, there are very limited treatments for this rare, progressive neurologic disease caused by a genetic deficiency. Solid Biowsciences' novel gene therapy uses dual-route administration to deliver directly to the heart and brain and to replace the missing frataxin gene, which is critical for energy production. Dr. Brooks explains, "Our flagship program is our DMD program, where we...
info_outline
Omnichannel Healthcare Model Providing Care When Needed with Dr. Payam Zamani MY DR NOW TRANSCRIPT
Empowered Patient Podcast
Dr. Payam Zamani, Founder and CEO of MY DR NOW, is directly solving the challenge of access to healthcare by creating a hybrid model that includes convenient walk-in clinics with extended hours, house calls, and virtual visits. This approach is consistent with modern on-demand expectations and attracting patients in dense urban areas as well as rural communities. Patient acquisition is driven by word-of-mouth and relationships with payers and hospital systems, to improve outcomes and reduce hospital readmissions. The model is also attractive to physicians by minimizing administrative...
info_outline
Omnichannel Healthcare Model Providing Care When Needed with Dr. Payam Zamani MY DR NOW
Empowered Patient Podcast
Dr. Payam Zamani, Founder and CEO of MY DR NOW, is directly solving the challenge of access to healthcare by creating a hybrid model that includes convenient walk-in clinics with extended hours, house calls, and virtual visits. This approach is consistent with modern on-demand expectations and attracting patients in dense urban areas as well as rural communities. Patient acquisition is driven by word-of-mouth and relationships with payers and hospital systems, to improve outcomes and reduce hospital readmissions. The model is also attractive to physicians by minimizing administrative...
info_outline
ACOs Using Value-Based Care to Improve Outcomes and Reduce Hospital Readmissions with Dr. Tom Kim Sound Long-Term Care Management TRANSCRIPT
Empowered Patient Podcast
Tom Kim, Chief Medical Officer at Sound Long-Term Care Management, discusses the role of accountable care organizations, ACOs, in improving healthcare for the long-term care population. The unique complexities and challenges of these patients include multiple medical conditions, frequent hospitalizations, and fragmented care. By using predictive analytics, telemedicine, and value-based care principles to deliver more coordinated care, patients are avoiding hospitalizations, receiving higher-quality care, and experiencing reduced costs. Tom explains, "So, in essence, an accountable care...
info_outline
ACOs Using Value-Based Care to Improve Outcomes and Reduce Hospital Readmissions with Dr. Tom Kim Sound Long-Term Care Management
Empowered Patient Podcast
Tom Kim, Chief Medical Officer at Sound Long-Term Care Management, discusses the role of accountable care organizations, ACOs, in improving healthcare for the long-term care population. The unique complexities and challenges of these patients include multiple medical conditions, frequent hospitalizations, and fragmented care. By using predictive analytics, telemedicine, and value-based care principles to deliver more coordinated care, patients are avoiding hospitalizations, receiving higher-quality care, and experiencing reduced costs. Tom explains, "So, in essence, an accountable care...
info_outline
Healthcare Supply Chain Management Goes Digital With Lisa Israelovitch AssistIQ TRANSCRIPT
Empowered Patient Podcast
Lisa Israelovitch, Co-Founder and CEO of AssistIQ, is addressing the challenges in healthcare supply chain management caused by a lack of accurate data on supplies used across the care facility, which leads to operational inefficiencies and financial losses. The previous methods of tracking have often been manual and unreliable, involving product stickers, barcode readers, and manual data entry. Using computer vision and AI to automatically see, track, and document every product used is improving data accuracy, saving clinicians' time, enabling inventory tracking and product expiration...
info_outline
Healthcare Supply Chain Management Goes Digital With Lisa Israelovitch AssistIQ
Empowered Patient Podcast
Lisa Israelovitch, Co-Founder and CEO of AssistIQ, is addressing the challenges in healthcare supply chain management caused by a lack of accurate data on supplies used across the care facility, which leads to operational inefficiencies and financial losses. The previous methods of tracking have often been manual and unreliable, involving product stickers, barcode readers, and manual data entry. Using computer vision and AI to automatically see, track, and document every product used is improving data accuracy, saving clinicians' time, enabling inventory tracking and product expiration...
info_outline
Shape-Shifting Device Extends Stroke Treatment to Smaller Brain Vessels with Ronen Eckhouse Rapid Medical TRANSCRIPT
Empowered Patient Podcast
Ronen Eckhouse, Co-Founder and CEO of Rapid Medical, identifies the limitations of current stroke treatments, which are primarily effective for large vessel occlusions. The Rapid Medical TIGERTRIEVER device is designed to safely and effectively treat strokes and remove clots in smaller vessels deeper in the brain. The unique adjustability of the device allows physicians to control the size and force during a procedure, allowing for precise and safer clot removal in complex anatomies. Ronen explains, "So the quicker or the less time there is between the stroke and the ability to...
info_outline
Shape-Shifting Device Extends Stroke Treatment to Smaller Brain Vessels with Ronen Eckhouse Rapid Medical
Empowered Patient Podcast
Ronen Eckhouse, Co-Founder and CEO of Rapid Medical, identifies the limitations of current stroke treatments, which are primarily effective for large vessel occlusions. The Rapid Medical TIGERTRIEVER device is designed to safely and effectively treat strokes and remove clots in smaller vessels deeper in the brain. The unique adjustability of the device allows physicians to control the size and force during a procedure, allowing for precise and safer clot removal in complex anatomies. Ronen explains, "So the quicker or the less time there is between the stroke and the ability to...
info_outlineDr. Mollie Leoni, Executive Vice President for Clinical Development of Kura Oncology, discusses the company's ziftomenib program, a menin inhibitor for acute myeloid leukemia (AML). The company initially allowed all AML patients to participate in the clinical study, but later found that specific subtypes, such as those with NPM1 mutation or KMT2A rearrangement, were more likely to respond to the menin inhibitor. They also discovered other subtypes that were responsive, expanding the potential patient population. Understanding the role of menin and menin inhibitors in addressing abnormal gene expression and promoting healthy cell development opens the door to potential combination therapy, where menin inhibitors could be layered onto existing treatments for various cancers related to menin independence.
Mollie explains, "In oncology, we have gotten good at treating the end state, but cancer has many, many causes with a common final endpoint to the way the cells look. So, it's many, many different diseases all at once. We've learned and are learning that with better technology and a better understanding of cancer in general we can identify not just how to stop the end state but how to stop it from starting."
"That's where ziftomenib comes in. Ziftomenib addresses key mutations that when they occur, are what cause the development of cancer. So you're able to shut down the development and force the cells to develop normally rather than waiting until they're already diseased and killing them with chemotherapy or some other cytotoxic agent."
"There is a protein complex that we refer to as the menin MLL complex, which goes rogue when something unusual happens in the cell. For example, that could be an NPM1 mutation. That could be a KMT2A rearrangement. Those are two things that are well-known to happen within AML diseases. It could also be a SETD2 RUNX1 mutation. So many types of mutations could happen that could cause this machinery to go rogue within the cell. And that complex causes genes to become active at levels and at times that are abnormal."
#KuraOncology #Ziftomenib #MeninInhibitors #PrecisionMedicine #BTD #AML
