THE JOHNS HOPKINS UNIVERSITY
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Private
- Established
- 1876-02-22
- Employees
- 10K
- Market Cap
- -
- Website
- http://www.jhu.edu
Immune Checkpoint Inhibitors Equally Effective in Older and Younger Cancer Patients Despite Immune Phenotype Differences
• New research published in Nature Communications reveals that immune checkpoint inhibitors (ICIs) demonstrate comparable efficacy in cancer patients regardless of age, with some older patients showing even better outcomes.
• The study identified significant age-related differences in immune phenotypes, including variations in cytokine dynamics, circulating immune composition, and immune cell phenotype dynamics at baseline between patients over and under 65 years.
• Researchers from Johns Hopkins University suggest these findings could lead to more personalized immunotherapy approaches tailored to age-specific immune characteristics, potentially improving treatment outcomes across age groups.
Physical Fitness Linked to Better Cognitive Health in Older Adults, Studies Show
• Recent research reveals that higher cardiorespiratory fitness is associated with better cognitive performance across multiple domains in older adults, regardless of genetic risk factors for Alzheimer's disease.
• A separate study found that older adults with smaller skeletal muscles are approximately 60% more likely to develop dementia, suggesting muscle maintenance may be a protective factor against cognitive decline.
• These findings highlight the importance of both aerobic exercise and strength training as potential therapeutic targets for preserving brain function in late adulthood.
Novel BCMA-Directed CAR T-Cell Therapy Shows Promising Efficacy in Relapsed/Refractory Multiple Myeloma and AL Amyloidosis
• A second-generation BCMA-directed CAR T-cell therapy, MDC-CAR-BCMA001, demonstrated remarkable efficacy in patients with relapsed/refractory multiple myeloma and AL amyloidosis, achieving a 5/6 overall response rate with 4 complete responses.
• The novel therapy showed a favorable safety profile with manageable toxicity, including low incidence of severe cytokine release syndrome and no neurotoxicity, even in patients with significant organ dysfunction.
• These promising results have prompted further investigation through ongoing clinical trials, including CARLOTTA001 (NCT05836896) and the CLEAR AL trial, potentially offering new hope for difficult-to-treat hematologic malignancies.
Phantom Neuro's Neural Interface Platform Receives Dual FDA Fast-Track Designations for Prosthetic Control
• Phantom Neuro's minimally invasive Phantom X platform has secured both FDA Breakthrough Device and TAP Designations, accelerating its path to market for advanced prosthetic control technology.
• The neural interface system demonstrated 94% accuracy in decoding 11 essential hand and wrist movements in the recent ASCENT study, showing promise for intuitive prosthetic control.
• The platform's outpatient procedure design makes it accessible to over 70,000 qualified surgeons nationwide, potentially transforming care for amputees and those with mobility challenges.
Breakthrough Bone Marrow Transplant Shows 88% Cure Rate for Sickle Cell Disease
• A novel reduced-intensity haploidentical bone marrow transplant procedure developed at Johns Hopkins demonstrates a 95% two-year survival rate and 88% cure rate for sickle cell disease patients.
• The treatment accepts half-matched donors and costs significantly less than gene therapy at $467,747 versus $2-3 million, while requiring shorter hospital stays and fewer blood transfusions.
• Clinical trial results from 42 patients show the procedure is safe and effective, with minimal serious side effects, making it a viable alternative to recently approved gene therapy treatments.
THC Shows Promise in Managing Agitation in Alzheimer's Disease, CTAD Trial Reports
A clinical trial presented at CTAD 2024 demonstrates potential benefits of dronabinol in treating agitation symptoms in Alzheimer's disease patients. The randomized, double-blind study evaluated twice-daily THC administration in elderly patients, offering a possible alternative to traditional antipsychotics which carry significant risks.
NIH Collaboratory Launches ARBOR-Telehealth Trial to Transform Rural Back Pain Care
• Johns Hopkins researchers are leading ARBOR-Telehealth, an innovative NIH Collaboratory trial investigating telehealth physical therapy solutions for rural patients with low back pain.
• The study aims to evaluate the effectiveness of remote physical therapy delivery and implement a novel risk-stratification approach in primary care settings.
• Principal investigators Dr. Richard Skolasky and Dr. Kevin McLaughlin will present early progress and lessons learned from the trial on January 31, 2025.
FORE Biotherapeutics Advances Plixorafenib in BRAF-Driven Tumors, Eyes Potential FDA Submission
• FORE Biotherapeutics is advancing plixorafenib in clinical trials targeting BRAF V600-mutated recurrent CNS tumors, rare solid tumors, and solid tumors with BRAF fusions.
• Interim efficacy analyses for plixorafenib in these monotherapy indications are expected throughout 2025, potentially leading to an FDA submission under the accelerated approval pathway.
• Plixorafenib's unique mechanism targets both BRAFV600 monomers and dimeric BRAF mutants, differentiating it from current BRAF inhibitors and offering a potential new standard of care.
• Nonclinical data presented at AACR 2024 showed plixorafenib combined with binimetinib is more potent than other BRAF inhibitors in combination, demonstrating synergistic activity.
RSV Vaccines Show Weaker Immune Response in Immunocompromised Older Adults
• A recent study reveals that older, immunocompromised adults exhibit a weaker antibody response to RSV vaccines compared to healthy individuals.
• The research indicates that approximately 40% of immunocompromised participants did not achieve seroconversion or meet neutralization thresholds post-RSV vaccination.
• An adjuvanted vaccine (Arexvy) showed a tendency to induce higher levels of RSV-neutralizing antibodies compared to a non-adjuvanted vaccine (Abrysvo) in this population.
• Further research is needed to optimize RSV vaccine strategies, including timing and selection, for immunocompromised individuals to enhance protection.
NCCN Updates 2024: Major Guideline Changes Across Multiple Cancer Types
• The NCCN has issued significant updates across 20 different cancer types in 2024, including new tumor-agnostic approvals for repotrectinib and trastuzumab deruxtecan for solid tumors with specific genetic markers.
• Multiple myeloma guidelines now recommend quadruplet regimens as standard of care for newly diagnosed patients, while CLL guidelines have shifted away from chemotherapy in favor of targeted therapies like BTK inhibitors.
• Major changes in breast cancer include the addition of ribociclib for HR+/HER2- disease, while lung cancer updates feature new recommendations for osimertinib after chemoradiation in EGFR-mutated NSCLC.
Clearmind Medicine's CMND-100 Receives IRB Approval for AUD Clinical Trial at Yale
• Clearmind Medicine received IRB approval for its Phase I/IIa clinical trial of CMND-100, a novel therapeutic targeting alcohol use disorder (AUD).
• The trial, to be conducted at Yale School of Medicine, will assess the safety, tolerability, and efficacy of CMND-100 in reducing alcohol cravings.
• CMND-100 aims to address the critical gap in AUD treatment, where current options have limited efficacy (less than 30%) and patient compliance.
• Alcohol consumption causes 2.6 million deaths annually worldwide, highlighting the urgent need for effective AUD treatments.
CVAC System's SURE Procedure Shows Noninferior Stone-Free Rate Compared to Standard Ureteroscopy in ASPIRE Trial
• The ASPIRE trial demonstrated that steerable ureteroscopic renal evacuation (SURE) using the CVAC system is noninferior to standard ureteroscopy (URS) for nephrolithiasis, achieving comparable stone-free rates.
• The SURE procedure resulted in significantly better stone clearance and reduced residual stone volume compared to URS, indicating a more complete removal of kidney stones.
• Unlike URS, the effectiveness of the SURE procedure did not decline with larger baseline stone volumes, showcasing its potential for treating a broader range of stone sizes.
• Safety profiles were comparable between the SURE procedure and standard URS, supporting the CVAC system as a safe and effective alternative for kidney stone treatment.
Pfizer's Braftovi Combo Shows Survival Benefit in BRAF-Mutated Metastatic Colorectal Cancer
• Pfizer's Braftovi, combined with cetuximab and mFOLFOX6, significantly improved progression-free survival in metastatic colorectal cancer patients with BRAF V600E mutation.
• The BREAKWATER trial demonstrated a clinically meaningful improvement in overall survival with the Braftovi regimen compared to chemotherapy.
• The FDA granted accelerated approval to the Braftovi combination in December 2024, marking it as a first-line targeted therapy option.
• Pfizer plans to share the BREAKWATER data with regulatory authorities to support full approval and broader use of the Braftovi combination.
Risk-Adapted Therapy Shows Promise in Muscle-Invasive Bladder Cancer
• A phase II trial evaluated risk-adapted therapy after neoadjuvant chemotherapy (NAC) in muscle-invasive bladder cancer (MIBC).
• The trial used biomarker selection and clinical staging to identify patients for cystectomy-sparing active surveillance (AS).
• The 2-year metastasis-free survival (MFS) rate was 72.9% for all patients, with 17% avoiding cystectomy without metastatic disease.
• Active surveillance showed a 76% 2-year MFS rate, with 48% of patients metastasis-free with an intact bladder.
Emerging Intravesical and Systemic Therapies Revolutionize Bladder Cancer Treatment
• Intravesical therapies like cretostimogene and TAR-200 show promising complete response rates in BCG-unresponsive NMIBC, but duration of response remains a key factor.
• Perioperative systemic therapies, including neoadjuvant chemo-immunotherapy with durvalumab, aim to improve event-free and overall survival in muscle-invasive urothelial carcinoma.
• Antibody-drug conjugates (ADCs) targeting Nectin-4 and HER2, such as enfortumab vedotin and disitamab vedotin, demonstrate significant potential in metastatic urothelial cancer.
• Novel strategies, including dual-targeting antibodies and combinations of ADCs with immunotherapies, are being explored to further enhance treatment efficacy and overcome resistance.
ANGPTL4 Overexpression May Limit Anti-VEGF Therapy Efficacy in Wet AMD
• Researchers have found that anti-VEGF treatments for wet age-related macular degeneration (AMD) may paradoxically increase levels of ANGPTL4, a protein that promotes abnormal blood vessel growth.
• The study showed that patients receiving anti-VEGF injections experienced a decrease in VEGF levels but a corresponding increase in ANGPTL4 levels in their eye fluid.
• An experimental drug targeting HIF-4, when combined with anti-VEGF medication, effectively reduced both VEGF and ANGPTL4 levels in mice with wet AMD, limiting blood vessel overgrowth.
• These findings suggest that targeting ANGPTL4 in conjunction with VEGF could improve treatment outcomes for wet AMD patients who do not respond well to current therapies.
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