UNIVERSITY OF LIVERPOOL

Liverpool Researchers Trial Oral Treatment for Deadly Leishmaniasis Disease

a month ago

• A groundbreaking clinical trial for visceral leishmaniasis is underway in Liverpool, testing an oral drug (DNDi-6899) that could replace current painful injection treatments requiring hospitalization. • The collaborative effort involves NHS University Hospitals of Liverpool Group, University of Liverpool, and Drugs for Neglected Diseases initiative (DNDi) to combat a disease that affects 50,000-90,000 people annually. • Researchers aim to develop a treatment that can be easily stored, transported, and administered in low-income regions, potentially saving thousands of lives affected by this neglected tropical disease.

Auxora Shows 63% Mortality Reduction in COVID-19 Patients with Acute Kidney Injury

3 months ago

• Post-hoc analysis of CARDEA Phase 2 trial reveals Auxora reduced mortality by 62.7% in COVID-19 patients with acute kidney injury compared to placebo, with benefits persisting through 60 days. • CalciMedica's CRAC channel inhibitor demonstrates promising tissue-protective and immunomodulatory effects in preclinical studies, suggesting potential therapeutic mechanism for AKI treatment. • Results support ongoing Phase 2 KOURAGE trial investigating Auxora in patients with Stage 2 or 3 AKI and acute hypoxemic respiratory failure, with data expected in 2025.

UK Clinical Trial Regulations Undergo Major Overhaul to Streamline Approvals and Boost Transparency

5 months ago

• The UK's Medicines and Healthcare products Regulatory Agency (MHRA) is implementing significant changes to clinical trial regulations, the most substantial in 20 years, to enhance the UK's appeal as a global research hub. • Key regulatory changes include a streamlined approval process with combined MHRA and ethics reviews, aiming for decisions within 30 days, and a new notification scheme for lower-risk trials. • To bolster research transparency, the updated regulations mandate trial registration on a WHO-recognized public registry and require sponsors to publish lay-friendly summaries of results within 12 months of trial completion. • The amendments, expected to take effect in early 2026, also address good clinical practice, manufacturing standards for non-investigational medicinal products, and safety reporting to align with international standards.

Keytruda/Lenvima Combo Shows Mixed Results in Gastroesophageal Adenocarcinoma Trial

5 months ago

• The Phase 3 LEAP-015 trial evaluated Keytruda plus Lenvima with chemotherapy for HER2-negative gastroesophageal adenocarcinoma. • The combination significantly improved progression-free survival and objective response rate compared to chemotherapy alone. • However, the trial failed to meet its other primary endpoint of overall survival in the final analysis. • The safety profile of the Keytruda plus Lenvima regimen was consistent with prior studies.

HER2 and αVβ6 Integrin Crosstalk Drives Breast Cancer Invasion and Trastuzumab Resistance

6 months ago

• A University of Liverpool study reveals a novel 'crosstalk' mechanism between HER2 and αVβ6 integrin, driving invasion in HER2-positive breast cancer. • The study identifies that disruption of the αVβ6-HER2 connection in trastuzumab-resistant cells leads to alternative, more invasive pathways. • Higher GDI2 levels correlate with improved survival, while αVβ6 expression predicts increased relapse risk post-trastuzumab, suggesting potential biomarkers. • Targeting the RAB5/RAB7A/GDI2 module or restoring its function may prevent or delay trastuzumab resistance in HER2-positive breast cancers.

NHS DigiTrials to Enhance Children's Surgical Outcome Reporting

6 months ago

• NHS DigiTrials will aid the Children’s Surgery Outcome Reporting (CSOR) program in identifying care differences for children needing complex surgery shortly after birth. • The CSOR program aims to improve care and health outcomes for children with conditions like Hirschsprung’s disease and congenital diaphragmatic hernia. • By contacting parents through NHS DigiTrials, researchers aim to gather quality of life data linked with hospital information to identify areas for improvement. • The initiative targets collecting data on approximately 550 children annually, with plans to expand as more hospitals join the program.

Lenacapavir Shows Promise as HIV Prophylactic but Affordability Remains a Key Hurdle

7 months ago

• Lenacapavir, an antiretroviral drug, has demonstrated near 100% effectiveness in preventing HIV infections with twice-yearly injections, offering a more discreet and convenient option compared to daily oral medications. • Clinical trial PURPOSE 1 showed lenacapavir was 100% effective in preventing HIV infections among cisgender women, outperforming daily oral regimens with low adherence rates. • Despite its potential, the high cost of lenacapavir (approximately $40,000 per year) raises concerns about accessibility, particularly in low-income countries with high HIV rates. • Gilead is negotiating licenses with generic drug manufacturers to produce and sell lenacapavir more cheaply in low-income countries, but some regions may still face limited access.

John Gorman's Anti-D Breakthrough Eradicates Rh Disease in Developed World

7 months ago

• John Gorman's pioneering work in the 1960s led to the development of RhoGAM, effectively eliminating Rh disease, a once-lethal condition for newborns. • Australia was the first country to implement a national anti-D program, ensuring widespread access to the treatment for Rh-negative pregnant women. • Despite its eradication in developed countries, Rh disease remains a significant issue in the developing world, causing an estimated 160,000 fetal and neonatal deaths annually. • Gorman is now focused on expanding access to anti-D treatment globally through the World Initiative for Rh disease Eradication (WIRhE).

Zegocractin Shows Promise in Reducing Acute Pancreatitis Symptoms in Phase 2 Trial

7 months ago

• Zegocractin, a CRAC channel inhibitor, significantly reduced the time to solid food tolerance in acute pancreatitis patients with systemic inflammatory response syndrome (SIRS). • Higher doses of zegocractin (1.0 and 2.0 mg/kg) showed no new cases of severe respiratory failure compared to placebo in the CARPO phase 2b trial. • The study suggests that zegocractin has the potential to decrease the risk of respiratory failure and necrotizing pancreatitis in acute pancreatitis patients. • The phase 2b trial results support further late-stage assessment for zegocractin as a potential treatment for acute pancreatitis and acute kidney injury.

Real-World Analysis of Nirmatrelvir/Ritonavir Drug-Drug Interactions Shows Low Reporting Rate

7 months ago

• A recent analysis of Pfizer's global safety database reveals a low reporting rate (0.005%) of drug-drug interactions (DDIs) associated with nirmatrelvir/ritonavir (NMV/r). • Simvastatin was the most frequently reported contraindicated drug, while tacrolimus was the most common drug used with caution in NMV/r-related DDI cases. • The majority (66.8%) of reported DDI cases were nonserious, with common adverse events including altered taste and gastrointestinal issues, aligning with NMV/r's known safety profile. • The study underscores the importance of careful patient selection and medication management to mitigate DDI risks, supporting the safe use of NMV/r in treating COVID-19.

UK Government Invests in AI and MedTech to Revolutionize Cancer Detection and Treatment

8 months ago

• The UK government is investing in new technologies, including AI, to improve cancer detection and treatment, aiming to boost the life sciences industry. • New partnerships between the NHS, pharmaceutical companies, and universities will trial innovative approaches for faster cancer diagnoses and better treatments. • Funding of £118 million will establish five new health technology hubs across the UK, fostering collaboration between academia and the private sector. • The investments also focus on personalized treatments and utilizing patient data to train AI models for quicker and more accurate cancer diagnoses.

Infex Therapeutics Doses First Patient in Phase IIa RESP-X Trial for NCFB

8 months ago

• Infex Therapeutics has dosed the first patient in its Phase IIa clinical trial of RESP-X for non-cystic fibrosis bronchiectasis (NCFB). • RESP-X is a first-in-class anti-virulence antibody targeting chronic Pseudomonas aeruginosa (Pa) infections in respiratory diseases. • The Phase IIa trial is a double-blinded, placebo-controlled study assessing the safety, efficacy, and optimal dosing of RESP-X in 12 NCFB patients. • RESP-X, in-licensed from Shionogi, aims to reduce the frequency of debilitating exacerbations in NCFB patients colonized with Pa.

Twice-Yearly Lenacapavir Injections Show 100% Efficacy in Preventing HIV in Women

a year ago

• A clinical trial in South Africa and Uganda demonstrated that lenacapavir, administered via injection every six months, provided complete protection against HIV infection in young women. • The Purpose 1 trial compared lenacapavir to daily oral PrEP medications, Truvada (F/TDF) and Descovy (F/TAF), with lenacapavir showing significantly higher efficacy. • The trial's independent data safety monitoring board recommended halting the blinded phase due to the clear benefit of lenacapavir, offering all participants a choice of PrEP. • Gilead Sciences, the drug developer, plans to submit the trial results to regulatory bodies and the WHO, with hopes of incorporating lenacapavir into HIV prevention guidelines.

Rare Disease Research Faces Critical Funding Gap Despite Scientific Value

12 years ago

• Major UK medical research funders allocate less than 1% of their budgets to rare disease research, despite these conditions consuming 10% of the NHS specialized services budget. • Research into rare diseases like alkaptonuria (AKU) has proven valuable for understanding common conditions such as osteoarthritis, demonstrating their broader scientific importance. • Public awareness initiatives like the French Telethon, which raised €94 million in 2011, show the potential for increased rare disease funding through effective advocacy campaigns.