UNIVERSITY OF CINCINNATI
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- -
- Established
- 1819-01-01
- Employees
- 5.9K
- Market Cap
- -
- Website
- https://www.uc.edu/
FDA Approves Genentech's TNKase for Acute Ischemic Stroke with Simplified Administration
• The FDA has approved Genentech's TNKase, a clot-dissolving agent, for treating acute ischemic stroke in adults, marking the company's second stroke treatment approval.
• TNKase offers a streamlined five-second intravenous bolus administration, presenting a significant advantage over the current standard treatment Activase's 60-minute infusion protocol.
• The approval is supported by a multi-center non-inferiority study across 22 Canadian stroke centers, demonstrating comparable safety and efficacy to Activase in patients with disabling neurological deficits.
Endometriosis Clinical Trial Pipeline Shows Promise with 20+ Therapies in Development
• The endometriosis clinical trial landscape is robust, featuring over 15 companies developing more than 20 potential therapies.
• Recent clinical trial activity includes positive Phase II results for Hope Medicine's HMI-115 and TiumBio's Merigolix, alongside Lisata Therapeutics' preclinical research.
• Key companies like Kissei Pharmaceutical, Mithra Pharmaceuticals, and Organon are advancing novel treatments, including Linzagolix and Estelle, to address unmet needs.
• These emerging therapies aim to improve pain management and overall outcomes for the estimated 190 million women affected by endometriosis worldwide.
Aerpio Initiates Phase 2 Trial of Razuprotafib for COVID-19 Blood Vessel Damage
Aerpio Pharma has commenced a phase 2 trial for razuprotafib, a drug aimed at treating blood vessel damage in COVID-19 patients, focusing on reducing inflammation, preventing blood clots, and mitigating acute respiratory distress syndrome.
Lomond Therapeutics' Lonitoclax Shows Promising Safety Profile in Phase 1 Trial
• Lomond Therapeutics announced positive Phase 1 clinical trial results for lonitoclax, an oral BCL-2 inhibitor, demonstrating a favorable safety profile in healthy volunteers.
• The study showed robust BCL-2 inhibition without significant safety signals, suggesting potential advantages over venetoclax in tolerability and outpatient treatment feasibility.
• Lonitoclax exhibited minimal immunosuppressive activity and reduced P4503A4 inhibition, potentially mitigating tumor lysis syndrome and drug accumulation risks.
• The company is advancing lonitoclax into further clinical studies for CLL, AML, and other oncology indications, building on its differentiated profile.
Lungpacer's AeroPace System Receives FDA Approval for Ventilator Weaning
• The FDA has granted premarket approval to Lungpacer Medical's AeroPace System, a neurostimulation therapy designed to improve weaning outcomes for adults on mechanical ventilation.
• AeroPace stimulates the phrenic nerves to strengthen the diaphragm, leading to improved breathing and reduced dependence on mechanical ventilation for patients ventilated for at least 96 hours.
• Clinical trials demonstrated that AeroPace increased weaning success, reduced ventilator days by nearly three days, and decreased the risk of reintubation by up to 60%.
• The AeroPace System represents a significant advancement in respiratory care, offering a new treatment option for critical care patients struggling to wean from mechanical ventilation.
Mission Bio Showcases Single-Cell Multiomics Advancements at ASH 2024
• Mission Bio presented new data at ASH 2024 demonstrating the Tapestri platform's utility in integrating genomic, immunophenotypic, and clonotypic assessment for multiple myeloma.
• A Heidelberg University Hospital study highlighted Tapestri's potential to refine minimal residual disease (MRD) assessment in acute myeloid leukemia (AML) with greater precision.
• Researchers from the University of Cincinnati introduced the first data integrating DNA and fusion profiling at the single-cell level for pediatric leukemia.
• Stanford University presented findings on single-cell DNA sequencing to uncover genomic insights in CAR T-cell therapy, potentially leading to safer treatments.
Personalized mRNA Vaccine Shows Promise in Preventing Lung Cancer Recurrence
• Researchers at the University of Cincinnati are trialing personalized mRNA vaccines to prevent lung cancer recurrence after surgery.
• The vaccine is created from the patient's own tumor cells, aiming to train the immune system to target remaining cancer cells.
• The clinical trial combines the personalized vaccine with immunotherapy to enhance effectiveness.
• Early research suggests potential for identifying common tumor antigens for broader preventative vaccine development.
Newer Epilepsy Medications During Pregnancy Show No Impact on Children's Neurodevelopment at Age 6
• A recent study indicates that newer anti-seizure medications, like lamotrigine and levetiracetam, pose no significant risk to children's language development when taken during pregnancy.
• The research followed 387 children up to age 6, assessing their verbal abilities and finding no difference between those exposed to the drugs in utero and those who were not.
• Findings suggest that controlling seizures with newer medications during pregnancy is a safe approach, contrasting with older medications like valproate, which have known risks.
• The study also highlights the potential benefits of folate supplementation during early pregnancy for improved cognitive and behavioral outcomes in children.
Stroke Trial Consent Methods Explored for Ethical, Efficient Research
• A recent Neurology editorial explores alternatives to traditional informed consent in acute stroke trials, where rapid treatment is crucial.
• Exception From Informed Consent (EFIC) protocols, like those used in the FASTEST trial, balance patient autonomy with research advancement.
• Community consultation and public disclosure, unique to EFIC, can be costly and time-consuming, prompting efforts to improve efficiency.
• The authors emphasize the need to honor patient autonomy while advancing stroke research to deliver better treatments.
Medical Cannabis Use in Children Under 18: Balancing Promise and Research Gaps
• Ohio and Kentucky laws permit doctors to recommend medical cannabis for patients under 18, raising questions about its safety and efficacy.
• Epidiolex, an FDA-approved CBD medication for epilepsy, is a notable exception with established dosing and efficacy for both adults and children.
• Research on THC-based treatments for children is hindered by the Schedule 1 classification, limiting clinical trials and data on safety and efficacy.
• Ongoing trials are exploring the potential of CBD-based formulations like Epidiolex to treat other conditions in pediatric populations.
STRIPE Initiative Advances Pharmacogenetic Testing Standards with Focus on DPYD-Guided Chemotherapy
• Leading experts at the STRIPE Annual Meeting addressed the need for unified guidelines across FDA, EMA, CPIC, and NCCN for DPYD genotype-guided chemotherapy dosing in cancer treatments.
• The innovative MetaCensus platform was introduced as a collaborative tool for evidence sharing and consensus building in pharmacogenetic testing, promoting transparent evaluation processes.
• Pharmacists are playing a crucial role in advancing pharmacogenomics through clinical consultations and research, while emphasizing the importance of multi-disciplinary collaboration in precision medicine implementation.
Ganaxolone Shows Promise in Phase III RAISE Trial for Refractory Status Epilepticus
• Marinus Pharmaceuticals' RAISE trial evaluated IV ganaxolone for refractory status epilepticus (RSE), meeting one co-primary endpoint.
• Ganaxolone significantly improved seizure cessation within 30 minutes compared to placebo (80% vs. 13%, p<0.0001) in RSE patients.
• Secondary endpoints showed ganaxolone-treated patients had a median time to seizure cessation of 4.2 minutes versus 307.2 minutes for placebo.
• 45% of ganaxolone patients experienced no treatment escalation within 24 hours, compared to 19% for placebo, indicating potential for rapid seizure control.
DHODH Inhibitor HOSU-53, Born from Undergraduate Research, Enters Phase I/II Trials for Cancer
• A novel dihydroorotate dehydrogenase (DHODH) inhibitor, HOSU-53, is entering Phase I/II clinical trials, aiming to selectively target and kill cancer cells.
• The drug's origins trace back to a collaboration between Dr. John C. Byrd and Dr. Thomas E. Goodwin, with initial discovery work involving undergraduate students at Hendrix College.
• HOSU-53 is derived from an earlier molecule, Hendrix-OSU-3 (HOSU-3), created by undergraduate students, showcasing the impact of undergraduate research in drug discovery.
• The single-site clinical trial is set to commence at The Ohio State University Comprehensive Cancer Center – Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC – James) in winter 2024.
AM-101 Enhances Radiation Therapy Efficacy in Lung Cancer with Brain Metastasis
• Researchers have discovered that AM-101, a synthetic benzodiazepine analog, can enhance the efficacy of radiation therapy for lung cancer that has metastasized to the brain.
• AM-101 activates GABA(A) receptors in NSCLC cells, promoting autophagy and increasing the sensitivity of cancer cells to radiation treatment.
• Animal models demonstrated that AM-101, combined with radiation, significantly improves survival rates and slows the growth of both primary NSCLC and brain metastases.
• Phase 1 clinical trials are planned to test the combination of AM-101 and radiation in lung cancer patients with and without brain metastases.
Progesterone Drug Provera Linked to Vision Loss in Meningioma Patients
• A recent study identifies progesterone, specifically Provera, as a significant risk factor for vision loss related to meningiomas in premenopausal women.
• Researchers are now conducting tests on patient samples to determine the prevalence of progesterone receptor expression in meningiomas.
• The study emphasizes the importance of age-appropriate counseling and consistent assessment of hormone receptor status in meningiomas.
• Clinicians have begun informing patients about the risks associated with Provera, leading many to discontinue its use.
FDA's Approval of Kisunla Faces Scrutiny Over Advisory Committee Conflicts of Interest
• An investigation reveals that a significant number of FDA advisory committee members who recommended Kisunla's approval had financial ties to Eli Lilly and other companies developing anti-amyloid drugs.
• Concerns are raised about the potential influence of these conflicts on the drug approval process and the public's trust in the FDA's decisions, especially after the controversy surrounding Aduhelm.
• Experts question whether the benefits of Kisunla outweigh the risks of brain swelling and bleeding, despite the advisory committee's unanimous recommendation for approval.
• The FDA's practice of granting waivers for committee members with financial conflicts is under scrutiny, with calls for stricter guidelines to bolster public confidence in the agency.
Donanemab Approval Under Scrutiny: Concerns Raised Over Safety, Efficacy, and Conflicts of Interest
• An investigation raises concerns about the safety and efficacy of donanemab, a newly approved Alzheimer's drug, citing patient deaths and adverse events during trials.
• Financial ties between FDA advisory committee members and the drug's manufacturer, Eli Lilly, and other related companies are under scrutiny for potential conflicts of interest.
• The FDA's initial rejection of donanemab due to missing data and safety concerns, followed by its approval after additional data collection, raises questions about the drug's evaluation process.
• Claims of donanemab's effectiveness in slowing Alzheimer's progression are being challenged, with experts pointing to misleading use of relative differences in cognitive decline.
Beacon Therapeutics' AGTC-501 Shows Sustained Efficacy in XLRP Phase 2 Trial
• Beacon Therapeutics announced positive 24-month data from the Phase 2 SKYLINE trial of AGTC-501 for X-linked retinitis pigmentosa (XLRP).
• The high-dose cohort of AGTC-501 demonstrated a 57% response rate in retinal sensitivity improvement, the trial's primary endpoint.
• AGTC-501 was generally safe and well-tolerated, with no clinically significant safety events associated with the treatment.
• Pivotal Phase 2/3 VISTA and open-label Phase 2 DAWN trials are ongoing, supporting further development of AGTC-501 for XLRP.
Alzheimer's Theory Challenged: Brain Protein Levels, Not Plaques, Linked to Cognitive Decline
• A new study challenges the prevailing theory that amyloid-beta plaque buildup causes Alzheimer's, suggesting that decreasing levels of the Aβ42 protein are linked to cognitive decline.
• Researchers analyzed data from 26,000 participants in clinical trials for monoclonal antibody treatments, finding that higher Aβ42 levels correlated with slower cognitive impairment.
• The study proposes that future Alzheimer's medications should focus on directly increasing Aβ42 levels, rather than solely targeting amyloid plaque reduction.
• Experts emphasize the importance of addressing modifiable risk factors and exploring alternative mechanisms beyond amyloid for Alzheimer's disease.
Recombinant Factor 7A Shows Promise in Halting Brain Bleeds After Stroke
• A global study is underway to evaluate Recombinant Factor 7A's efficacy in stopping brain bleeds following a stroke, potentially improving patient outcomes.
• The trial focuses on administering the drug within the first few hours of stroke onset, when it is believed to have the greatest impact.
• Mobile stroke units are being utilized to expedite treatment, aiming to administer the drug within a critical two-hour window.
• The FDA has granted permission for "exception from informed consent" to facilitate rapid treatment in eligible patients.
© Copyright 2025. All Rights Reserved by MedPath