Menin Inhibition Shows Potential for Precision Therapy in AML with Dr. Mollie Leoni Kura Oncology
Release Date: 06/25/2024
Empowered Patient Podcast
Steve Brown, Founder and CEO and Lisa Booth, Vice President of Operations of CureWise, are both cancer survivors, which led them to develop an AI-powered tumor board platform to advance cancer diagnoses and treatment. This approach to precision medicine provides patients with the opportunity to better understand their specific condition, educating them about possible courses of action that may be more appropriate than the standard of care. The platform also helps patients find relevant clinical trials and manage the side effects of their treatments. Steve explains, "So the mission...
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Steve Brown, Founder and CEO and Lisa Booth, Vice President of Operations of CureWise, are both cancer survivors, which led them to develop an AI-powered tumor board platform to advance cancer diagnoses and treatment. This approach to precision medicine provides patients with the opportunity to better understand their specific condition, educating them about possible courses of action that may be more appropriate than the standard of care. The platform also helps patients find relevant clinical trials and manage the side effects of their treatments. Steve explains, "So the mission...
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Nayla Chaijale, Medical Strategy Lead for Rare Syndromes at UCB, describes Dravet syndrome, a rare, medication-resistant epileptic encephalopathy that involves seizures and significant co-morbidities like cognitive and developmental delays. Their approved drug FINTEPLA has a unique mechanism of action that modulates pathways in the brain and has demonstrated a significant reduction in the frequency of convulsive seizures in those with this condition. Nayla notes that the good news is that most patients with Dravet syndrome have a mutation in the SCN1A gene, a critical biomarker that...
info_outlineEmpowered Patient Podcast
Nayla Chaijale, Medical Strategy Lead for Rare Syndromes at UCB, describes Dravet syndrome, a rare, medication-resistant epileptic encephalopathy that involves seizures and significant co-morbidities like cognitive and developmental delays. Their approved drug FINTEPLA has a unique mechanism of action that modulates pathways in the brain and has demonstrated a significant reduction in the frequency of convulsive seizures in those with this condition. Nayla notes that the good news is that most patients with Dravet syndrome have a mutation in the SCN1A gene, a critical biomarker that...
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Vanessa Lemarié, Chief Operations Officer at myTomorrows, which is a platform for connecting patients, physicians, and clinical trial sponsors using a Trial Search AI tool designed to improve the accuracy of matching patients with suitable trials. The technology aims to speed up trial recruitment, increase diversity by reaching underserved populations, and ultimately shorten the drug development timeline. There is a particular focus on patients with no treatment options or who have not responded to current therapies. Vanessa explains, "We are all about patients. First and foremost, we want...
info_outlineEmpowered Patient Podcast
Vanessa Lemarié, Chief Operations Officer at myTomorrows, which is a platform for connecting patients, physicians, and clinical trial sponsors using a Trial Search AI tool designed to improve the accuracy of matching patients with suitable trials. The technology aims to speed up trial recruitment, increase diversity by reaching underserved populations, and ultimately shorten the drug development timeline. There is a particular focus on patients with no treatment options or who have not responded to current therapies. Vanessa explains, "We are all about patients. First and foremost, we want...
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Paul Pruitt, Chief Growth Officer at SHARx, which is a company that partners with employers to provide access to drugs not covered by insurance, such as specialty medications. The SHARx model acts as a complementary service to existing Pharmacy Benefit Managers and allows employers to opt out of covering the most expensive drugs. Paul's personal experience managing his sons' rare condition and navigating the administrative burden and frustration of the insurance system gives him deep insights into the factors driving up the cost of medications. Paul explains, "When we started SHARx eight...
info_outlineEmpowered Patient Podcast
Paul Pruitt, Chief Growth Officer at SHARx, which is a company that partners with employers to provide access to drugs not covered by insurance, such as specialty medications. The SHARx model acts as a complementary service to existing Pharmacy Benefit Managers and allows employers to opt out of covering the most expensive drugs. Paul's personal experience managing his sons' rare condition and navigating the administrative burden and frustration of the insurance system gives him deep insights into the factors driving up the cost of medications. Paul explains, "When we started SHARx eight...
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Dr. Marc Hedrick, President and CEO of Plus Therapeutics Inc., has expanded their focus from glioblastoma to leptomeningeal metastasis, a central nervous system cancer that is a growing challenge due to increased survival rates from primary cancers. Their highly sensitive, advanced diagnostic test, CNSide, can detect cancer cells in cerebrospinal fluid, addressing the shortcomings of imaging and outdated standard-of-care practices. Using AI and advanced data analytics, their lead drug candidate is uniquely suited for treating CNS cancers because its safety at high doses enables the...
info_outlineEmpowered Patient Podcast
Dr. Marc Hedrick, President and CEO of Plus Therapeutics Inc., has expanded their focus from glioblastoma to leptomeningeal metastasis, a central nervous system cancer that is a growing challenge due to increased survival rates from primary cancers. Their highly sensitive, advanced diagnostic test, CNSide, can detect cancer cells in cerebrospinal fluid, addressing the shortcomings of imaging and outdated standard-of-care practices. Using AI and advanced data analytics, their lead drug candidate is uniquely suited for treating CNS cancers because its safety at high doses enables the...
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
