Samuel B Barone

#PRIMR’s 78 IRB-approved pieces empower patients with simplified trial info. The RadiCaL Study for kidney cancer patients with bone metastases examines Ra-223 with cabozantinib to see if it improves outcomes. PI: Rana McKay UC San Diego School of Medicine Alliance for Clinical Trials in Oncology See IRB-approved video below 👇 https://lnkd.in/d97fj9dj #kidneycancer #kidneycancerawareness #cabozantinib #radium223 #radiopharmaceuticals #clinicaltrials #cancer #clinicalresearch #DEI #digital #health #immunotherapy #kidneycancer #men #menshealth #metastatickidneycancer #oncology #patienteducation #video #women #womenshealth #digital

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CMG publishes new ACMG Therapeutics Bulletin: “Trofinetide approved for children and adults with Rett syndrome (RTT)” in GIM Open, the College’s gold open access journal: https://bit.ly/3zOe6iI #Therapeutics #medicalgenetics #GIMOpen #RettSyndrome #MedicalEducation Rett syndrome (RTT) affects 1 in 10,000 to 23,000 females globally, making it one of the most common genetic causes of intellectual and developmental impairment in females. Trofinetide improved neuronal and synaptic function in study participants with RTT. Participants showed improvement in various behavioral, visual & communication aspects. Clinicians should plan for the most frequent side effects of trofinetide (diarrhea & vomiting) should they occur.

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🚨 Webinar Announcement: Improving Outcomes and Access to Precision Medicine🚨- https://lnkd.in/eSvkPZPG Despite groundbreaking advances in precision medicine, a critical gap remains: the lack of diverse representation in genomic data, leading to unequal healthcare outcomes—especially for people of color. 🔬 What is the impact? Genomic testing is a cornerstone of modern medicine, yet current databases are largely based on data from individuals of European descent. This leaves underrepresented populations at a disadvantage, with less effective treatments and limited access to life-saving innovations. 💡What you’ll learn: 1️⃣ Understanding the genomic testing gap and the structural barriers in healthcare 2️⃣ How to advance precision medicine for all, ensuring inclusivity 3️⃣ Practical strategies for improving access, including policy changes, community engagement, and innovative healthcare models 📅 Date: Thursday, 21st November, 2024 ⏰ Time: 12:00pm - 1:00pm ET 📍 Location: Zoom (Register Here https://lnkd.in/eSvkPZPG) Let’s work together to create a healthcare system that serves everyone. #PrecisionMedicine #GenomicTesting #HealthEquity

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If you missed our poster presentation at #ASCO24 titled "Feasibility and Utilization of Electronic Patient-Reported Outcome Measures for Lung Cancer in Routine Clinical Care," in collaboration with our partners at the University of North Carolina and Takeda Pharmaceuticals, below are the key findings.   This study explored the feasibility of standardizing PRO measures among patients with lung cancer in US clinical practice. Using the ePRO and alerts data collected from Carevive RSM, we explored patient engagement, measured by survey compliance and data completeness, as well as clinical team engagement measured by the platform utilization time, time to alert response, and clinical action taken when patients reported moderate/severe symptoms (Figures 2 and 4). ✅ Results support the feasibility of integrating a standard set of multi-domain ePRO measures, comparable in breadth and scope to the Health Outcomes Observatory (H2O) Core Outcomes Set, into routine lung cancer care. ✅ Patient engagement was high, as indicated by survey compliance and completeness, particularly in the first 5 weeks of enrollment. The median survey follow-up was 8 weeks, with 85% median compliance over that time. ✅ The clinical team's engagement was high, as indicated by frequent usage of the platform and timely response to symptom alerts, with a median of 20 hours and 69.3% addressed in less than 2 days. ✅ Findings support the value of remote symptom alerts, when integrated into clinical workflow, for enhancing the timeliness of patient and care team communications, and timely addressing of symptoms to optimize care. Read more of the presentation here: https://lnkd.in/esFXwb69 Aaron Galaznik Emelly Rusli Keri Collette Mordecai Kramer Bruno Lempernesse #carevive #digitaloncology #cancer #cancercare #oncology #patientreportedoutcomes #oncologydata #clinicalinnovation #drugcommercialization #RWD #RWE #smartdata

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CytoAgents is an innovative, clinical-stage biotech company focused on bringing to market novel treatments for Cytokine Release Syndrome (CRS), an overreaction of the immune system. CytoAgents' solution targets cytokines specifically active in CRS, allowing treatment without disrupting normal immune system mechanisms. Learn more about CytoAgents and CRS here: https://cytoagents.com/ #cytoinereleasesyndrome #crs #cytokines Teresa Whalen, RPh

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Congratulations to Amy Alabaster and Yanina (Yana) Natanzon on their manuscript collaboration with Friends of Cancer Research among other partners in the JCO Clinical Cancer Informatics, American Society of Clinical Oncology (ASCO). This publication demonstrates the feasibility of aligning disparate real-world (rw) data sources to evaluate rw-response to treatment using clinician-documented response in patients with metastatic non-small cell lung cancer (mNSCLC). Alignment of methodologies analyzing real-world data (RWD) is critical for supporting evidence generation using these data RWD to inform oncology drug development and regulatory decision-making. These findings are from the latest pilot project in Friends’ Real-World Evidence Portfolio which establishes methodologies for using real-world data (RWD) to evaluate safety and efficacy of therapies for patients. Read "Evaluation of real-world tumor response derived from electronic health record data sources: A feasibility analysis in mNSCLC patients treated with chemotherapy” here: https://hubs.ly/Q02LJDYb0 #RWEFriends #publication #patients #RWD #EHR

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🗞️👓 New on the Catalyst Clinical Research Blog: Highlights from hubXchange Immunotherapies Maryland 2024! 📖 Catalyst Clinical Research was excited to share insights from this year’s hubXchange Immunotherapies Maryland 2024, where their Executive Director, Meredith M., joined industry experts to address the latest advancements and challenges in immunotherapy trials. This exclusive event covered everything from optimizing dosing strategies to leveraging cutting-edge biomarkers for improved patient safety and efficacy. In Meredith's keynote presentation and “Project Optimus” roundtable, Meredith led discussions on the FDA’s dose optimization guidance, emphasizing the shift toward minimally and maximally effective doses for early-phase trials. Catalyst also explored how PK-PD modeling is reshaping dose-response understanding, while advanced biomarkers are offering new ways to monitor immunotherapy impact and safety. Visit the blog to see how Catalyst is helping shape the future of immuno-oncology through tailored solutions that drive safety and efficacy in clinical trials. 👀 Read the full article: https://lnkd.in/eJtQB4jP 🌐 https://lnkd.in/e8pDqkji #Immunotherapy #PatientSafety #ClinicalTrials #CatalystOncology #HubXchange

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Innovative Study Led by Dr. Raid Aljumaily Aims to Revolutionize Lung Cancer Treatment and Enhance Real-World Clinical Outcomes FDA Daniel Morgensztern Sheila Prindiville University of Oklahoma https://lnkd.in/ejbmYBfR #OncoDaily #Oncology #Cancer #Health #Medicine #LungCancer #LungCancerTreatment #NSCLC

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Dana-Farber Launches Center for RAS Therapeutics Focused on RAS-Driven Cancers The new center will foster collaboration between Dana-Farber Cancer Institute scientists and clinicians with industry partners and academic centers to speed up the development of targeted therapies for cancers driven by RAS mutations.

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Check out this excellent tool from NCODA: the PQI In Action for Minimal Residual Disease Testing in Multiple Myeloma. Created with Florida Cancer Specialists & Research Institute and Rocky Mountain Cancer Centers, this easy-to-use clinical resource guide breaks down how medical teams can use clonoSEQ to optimize patient outcomes. https://lnkd.in/eNRhfyHN FDA-cleared to measure MRD in MM, CLL, & ALL; CLIA-validated in other lymphoid cancers. Review the FDA-cleared uses of clonoSEQ: https://rb.gy/8tju9y

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PTC was excited to learn, share and collaborate with other leading RNA experts at the recent 7th Annual RNA-Targeted Drug Discovery & Development Summit in Boston, MA, and the RNA at the Bench and Bedside IV conference in La Jolla, CA. Anu Bhattacharyya, Kerstin Effenberger, and Marla Weetall from our research team presented on ways PTC is leveraging its leading splicing platform to identify promising therapies for rare diseases with high unmet need. Learn more about our splicing platform here: https://bit.ly/4fi4w6L

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In 2021, over 16.7 MILLION prescriptions for clopidogrel (Plavix) were filled in the U.S.* This means that over 5 million of those prescriptions were filled for patients who likely lack enough enzymatic activity in CYP2C19 to sufficiently activate this prodrug to its active form. Those patients are essentially taking a placebo. This is scary. Patients can be taking clopidogrel with a false sense of security in its efficacy. But in reality, if they are part of those unfortunate 1/3 of patients with certain genetic features, they’re at increased risk of having a second #HeartAttack or #stroke because of their DNA! 🧬 Can this issue be prevented? YES How??? 🤔 #Pharmacogenomics testing allows us to check for genetic features that affect drug therapies. A simple cheek swab can give us a wealth of knowledge about how your body uniquely processes over 150 medications. And the test is good for a lifetime of future drug therapy decisions! In this case, if we identify a CYP2C19 deficiency, alternatives to clopidogrel, such as prasugrel or ticagrelor, could be prescribed instead. * Kane SP. Clopidogrel, ClinCalc DrugStats Database, Version 2024.01. ClinCalc: https://lnkd.in/eUeCPPJz. Updated January 1, 2024. Accessed May 6, 2024. #PersonalizedMedicine #technology

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InnaRx to Present Positive Phase 2 Data on MY008211A in Poster Presentation at the 66th ASH Annual Meeting & Exposition Gaithersburg, M.D., Oct 25, 2024— InnaRx, a clinical-stage biotechnology company specializing in oral modulators targeting innate immunity pathways, today announced an upcoming poster presentation at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, being held December 7–10, 2024, in San Diego, CA. This poster presentation will feature findings from a Phase 2 dose-finding study of MY008211A, an investigational oral complement factor B inhibitor, in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) experiencing active hemolysis. A Phase 2 Dose-Finding Trial of MY008211A in Complement Inhibitor–Naive Patients with Paroxysmal Nocturnal Hemoglobinuria and Signs of Active Hemolysis (Abstract #2701) Session Name: 508. Bone Marrow Failure: Acquired: Poster II Session Date: Sunday, December 8, 2024 Presentation Time: 6:00 PM - 8:00 PM PT Location: San Diego Convention Center, Halls G-H This Phase 2, multicenter, open-label study evaluated three dosing regimens of MY008211A in complement inhibitor–naive patients with PNH, a life-threatening blood disorder characterized by red blood cell destruction, blood clots, and bone marrow failure. Over the 12-week study period, all 34 patients achieved the primary endpoint—an increase in hemoglobin (Hb) concentration of ≥20 g/L from baseline—while also demonstrating significant control over hemolysis and improvements in patient-reported fatigue levels. MY008211A was well tolerated across all dosing regimens, with no treatment-related serious adverse events and only mild to moderate side effects, supporting its potential as a new treatment option for PNH. "We are excited to share these promising results with the hematology community at ASH," said Wilson Wang, VP of corporate development. "The success in our proof-of-concept study in PNH demonstrated MY008’s broad potential as a proven innate immunity modulator for complement-mediated autoimmune diseases.” InnaRx invites attendees at the ASH Annual Meeting to visit the poster session to discuss these findings and learn more about MY008’s development. About InnaRx InnaRx is a clinical-stage biotechnology company developing best-in-class oral modulators targeting innate immunity pathways for immunological and inflammatory (I&I) diseases. InnaRx’s innovative approach to immune modulation offers new options for managing complex and rare immunological conditions, with proof-of-concept demonstrated in clinical studies.

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