JOHNS HOPKINS BLOOMBERG SCHOOL OF PUBLIC HEALTH
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Private, Subsidiary
- Established
- 2014-01-01
- Employees
- 11
- Market Cap
- -
- Website
- http://www.jhsph.edu
CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder
• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder.
• The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein.
• This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.
FDA Requests Additional Trial for Novavax COVID-19 Vaccine Before Full Approval
• The FDA has asked Novavax to conduct an additional randomized, controlled study of its COVID-19 vaccine NVX-CoV2601 before considering full approval, despite the vaccine having emergency use authorization since 2022.
• Interim results from a phase 2/3 study showed Novavax's updated XBB.1.5 variant vaccine generated 5.8 times higher neutralizing antibody levels compared to the original formulation, with a favorable safety profile.
• The FDA's request represents a significant setback for Novavax, which had expected approval based on prior conversations with regulators and had already passed its April 1 PDUFA date.
GLP-1 Drug Use Surges Among Type 1 Diabetes Patients Despite Safety Concerns
• GLP-1 receptor agonist use has increased dramatically among type 1 diabetes patients, with prescriptions rising from 4% to 33% in severely obese adults over the past decade.
• Obesity rates have climbed significantly in the type 1 diabetes population, increasing from 18% to 26% in children and from 30% to 38% in adults between 2008 and 2023.
• Researchers from Johns Hopkins emphasize the urgent need for clinical trial data on GLP-1 drugs in type 1 diabetes patients, as these medications are not FDA-approved for this population and may pose hypoglycemia risks.
FDA to Phase Out Animal Testing Requirement for Monoclonal Antibodies and Drugs
• The FDA announced it will phase out requirements for animal testing of monoclonal antibodies and other drugs, citing the availability of "more effective, human-relevant methods" for safety evaluation.
• The initiative aims to improve drug safety, accelerate evaluation processes, reduce R&D costs, and ultimately lower drug prices by implementing AI-based computational models, humanoid models, and real-world human data.
• This regulatory shift, enabled by the bipartisan FDA Modernization Act 2.0 of 2022, represents what FDA Commissioner Marty Makary calls "a paradigm shift in drug evaluation" that could expedite development of new treatments.
Johns Hopkins CIDID Pioneers Next Generation of Rapid Diagnostic Tests for Global Health Impact
• Johns Hopkins University's Center for Innovative Diagnostics for Infectious Diseases (CIDID) is spearheading the development of rapid diagnostic tests for various infectious diseases, building on COVID-19 testing technology.
• Under Dr. Yuka Manabe's leadership, CIDID has achieved significant breakthroughs, including the FDA approval of the first point-of-care molecular diagnostic test for hepatitis C in June 2024.
• The center is advancing affordable diagnostic solutions, including a $2 tuberculosis test, while evaluating and supporting the development of rapid tests for STIs, respiratory infections, and other conditions.
Antibiotic Use Not Linked to Increased Risk of Cognitive Decline in Older Adults
• A new study of over 13,500 older adults found no increased risk of cognitive decline or dementia associated with antibiotic use.
• The research tracked participants for an average of five years, assessing cognitive function through regular testing.
• Findings offer reassurance for seniors needing antibiotics, though further research on dosage and specific antibiotic types is recommended.
• The study, published in Neurology, challenges previous concerns about antibiotics disrupting gut health and impacting brain function.
Clinical Trial Opportunity for Erectile Dysfunction Treatment
A clinical trial is seeking participants to evaluate the safety and effectiveness of the VERTICA device, a non-invasive treatment for erectile dysfunction using low-intensity radiofrequency energy.
Toripalimab Plus Chemotherapy Improves Survival in Lung Cancer
• A phase III trial showed that adding toripalimab to chemotherapy significantly improved progression-free survival in extensive-stage small cell lung cancer.
• The combination of toripalimab and chemotherapy also led to a statistically significant improvement in overall survival compared to chemotherapy alone.
• Genomic analysis identified potential biomarkers, such as mutations in the FA-PI3K-Akt and IL-7 signaling pathways, associated with improved outcomes with toripalimab.
• The addition of toripalimab to chemotherapy showed an acceptable safety profile, supporting its use as a new treatment option.
Trump's Healthcare Agency Nominations Signal Major Shake-up in US Pharma Landscape
• President-elect Trump's controversial healthcare nominations, including Dr. Marty Makary for FDA and Dr. Dave Weldon for CDC, have sparked mixed reactions from industry experts, ranging from panic to excitement.
• The nominees' track records suggest a strong focus on clinical trial data transparency, vaccine safety research, and radical reforms in healthcare administration, though Senate confirmation remains uncertain.
• With Republicans holding a seven-seat majority in the Senate, the administration can only afford to lose three Republican votes for confirmations, making the upcoming hearings crucial for the future of US healthcare policy.
LENZ Therapeutics' LNZ100 NDA Accepted by FDA for Presbyopia Treatment
• The FDA has accepted the New Drug Application (NDA) for LENZ Therapeutics' LNZ100, a potential treatment for presbyopia.
• The NDA is supported by Phase 3 CLARITY study data, which demonstrated significant near vision improvement in participants.
• Participants in the CLARITY study achieved at least a 3-line improvement in Best Corrected Distance Visual Acuity at near without losing distance visual acuity.
Incorrect Arm Position During Blood Pressure Readings Leads to Overestimation, Study Finds
• A recent study reveals that incorrect arm positioning during blood pressure (BP) measurement significantly overestimates BP readings.
• Supporting the arm on the lap can overestimate systolic BP by 3.9 mm Hg and diastolic BP by 4.0 mm Hg.
• An unsupported arm at the side leads to even greater overestimation, with systolic BP increasing by 6.5 mm Hg and diastolic BP by 4.4 mm Hg.
• Standardized arm positioning is crucial for accurate BP measurement, potentially preventing misdiagnosis and overestimation of hypertension.
Convalescent Plasma Could Have Saved Thousands More Lives During COVID-19 Pandemic
• A new study estimates that broader use of convalescent plasma could have saved thousands of lives during the first year of the COVID-19 pandemic.
• Researchers suggest that early use of high-titer convalescent plasma in both hospitalized and high-risk outpatients could have significantly reduced mortality.
• The study highlights the potential of convalescent plasma as a readily available and relatively inexpensive therapy for infectious disease emergencies.
• The authors recommend incorporating convalescent plasma collection and delivery into public health preparedness plans for future outbreaks.
Assessing the Clinical Impact of Drug-Drug Interactions: A Focus on Colchicine and COVID-19
• A recent study in JAMA Network Open evaluated drug-drug interactions (DDIs) involving colchicine in COVID-19 patients, utilizing data from the COLCORONA trial.
• The analysis found no significant increase in adverse events or hospitalization/death risk due to potential DDIs with colchicine in COVID-19 patients.
• The study highlights the challenge of translating theoretical DDIs into real-world clinical harms, emphasizing the need for robust screening and testing methods.
• Conservative prescribing practices and vigilance regarding potential adverse effects remain crucial in managing DDIs, considering over-the-counter medicines and supplements.
FDA Approves Moderna's mResvia, Third RSV Vaccine for Older Adults
• The FDA has approved Moderna's mResvia vaccine for the prevention of RSV in adults aged 60 and older, making it the third approved RSV vaccine.
• mResvia, like Moderna's COVID-19 vaccine, utilizes mRNA technology to target the prefusion F protein of RSV, offering a prefilled syringe for easier administration.
• Moderna anticipates the RSV vaccine market to reach $6-8 billion in annual sales, with mResvia potentially gaining market share based on efficacy and ease of use.
• Clinical trials showed mResvia to be approximately 84% effective against RSV-related disease with two or more symptoms, with effectiveness waning to 63% after 8.6 months.
© Copyright 2025. All Rights Reserved by MedPath