UNIVERSITY COLLEGE LONDON

Novel Blood Test Could Guide Precision Treatment for Resistant Lupus with Rituximab-Belimumab Combination

13 days ago

• A £1.7 million clinical trial called "Stratify Lupus" will test if a blood biomarker (IgA2 anti-dsDNA) can identify lupus patients who would respond to a novel combination of rituximab and belimumab. • Previous research shows patients with this biomarker are 17 times more likely to respond to the drug combination, potentially offering new hope for treatment-resistant cases. • The UK-wide trial beginning in spring 2026 could establish a personalized medicine approach for lupus, particularly benefiting women of African and Caribbean descent who are disproportionately affected.

AviadoBio Completes Second Cohort in Phase 1/2 Trial for Frontotemporal Dementia Gene Therapy

21 days ago

• AviadoBio has completed dosing the second cohort in its ASPIRE-FTD clinical trial evaluating AVB-101, a gene therapy for frontotemporal dementia with GRN mutations, with plans to initiate a third cohort in Q3 2025. • The company reported no clinically significant safety findings in the first cohort through 52 weeks of follow-up, with no immunosuppression required, marking an important milestone in the development pathway. • AVB-101 uses a novel delivery approach, administering the gene therapy directly to the thalamus via neurosurgical procedure to bypass the blood-brain barrier and target affected brain regions more precisely.

NHS Launches £10 Million Clinical Trial on Puberty Blockers Following Cass Review Recommendations

2 months ago

• The NHS has approved a £10.7 million clinical trial to investigate the potential benefits of puberty blockers for young people with gender dysphoria, following recommendations from Baroness Cass's comprehensive review. • The King's College London-led research aims to address significant evidence gaps by monitoring participants' physical, social, and emotional wellbeing over a two-year period, with strict ethical oversight and regulatory approval. • The trial comes amid ongoing controversy, with the UK government maintaining an "indefinite ban" on routine prescription of puberty blockers to under-18s until 2027, while medical experts remain divided on their clinical efficacy and safety.

Breakthrough Gene Therapy Restores Vision in Children with Rare Genetic Blindness

3 months ago

• A groundbreaking gene therapy developed by Moorfields Eye Hospital and MeiraGTx has successfully restored vision in children born with LCA-AIPL1, a severe inherited eye disorder affecting 2-3 in 10 million newborns. • Clinical trials demonstrated significant vision improvement in all eleven treated children, with some achieving 20/80 vision, enabling them to identify pictures, safely navigate, and engage in visual activities for the first time. • The treatment must be administered before age four due to irreversible retinal degeneration, with regulatory approval expected within 1-2 years following successful safety and efficacy demonstrations.

Personalized mRNA Vaccines Show Promise in Early-Phase Pancreatic Cancer Trial

3 months ago

• A Phase 1 clinical trial published in Nature demonstrates encouraging results for personalized mRNA vaccines in treating pancreatic cancer, offering hope for one of the deadliest cancer types. • The development addresses a critical medical need, as pancreatic cancer has a devastating five-year survival rate of less than 13% and is typically diagnosed at advanced stages in 90% of cases. • The trial represents a significant advancement in applying mRNA technology beyond infectious diseases, potentially opening new avenues for personalized cancer immunotherapy.

Novel Gene-Silencing Therapy Shows Promise for Long-Term Cholesterol Control with Single Dose

3 months ago

• Groundbreaking epigenetic editing technique demonstrates over 98% reduction in PCSK9 levels and 70% decrease in LDL cholesterol through a single treatment in preclinical studies. • The novel PCSK9-epigenetic editor (PCSK9-EE) therapy maintains its effectiveness for over a year without permanently altering DNA, offering a potential alternative to daily cholesterol medications. • Research in non-human primates shows promising safety profile with only transient liver enzyme elevations, while maintaining the ability to reverse the treatment if needed.

GLP-1 Drug Exenatide Shows No Benefit in Large-Scale Parkinson's Disease Trial

4 months ago

• A rigorous 96-week Phase 3 trial involving 194 Parkinson's patients across six UK hospitals found that exenatide, a GLP-1 drug related to Ozempic, showed no benefit in treating or slowing disease progression. • The double-blind study, led by UCL researchers, revealed no improvements in patient movements, symptoms, or brain imaging results, despite earlier smaller studies suggesting potential benefits. • The disappointing results represent a setback for the half million Americans with Parkinson's disease, who currently have treatments for symptoms but no options to slow disease progression.

Clinical Trial Industry Faces Complex Challenges in 2025: Adaptive Designs, Political Shifts, and Diversity Requirements

4 months ago

• Clinical trials are becoming increasingly complex and expensive as they target smaller patient populations and face stricter regulations, driving the need for smarter and more efficient trial designs. • Political changes, including Trump's presidency, could lead to significant regulatory shifts in the clinical trial landscape, potentially affecting approval pathways and oversight mechanisms. • New legislation like the Inflation Reduction Act is expected to impact trial initiations and drug development, while diversity guidelines from WHO, FDA, and EMA present both opportunities and challenges for patient recruitment.

Most Young People with Long COVID Recover Within Two Years, Study Finds

6 months ago

• A new study reveals that approximately 70% of children and teenagers diagnosed with Long COVID recover within 24 months of their initial COVID-19 infection. • The research analyzed data from 233 young individuals in England, identifying that 165 of them had recovered from Long COVID after two years. • The study indicated that girls were nearly twice as likely as boys to still experience Long COVID after two years, with older teenagers also less likely to recover. • Researchers plan further investigation to understand why the remaining 30% of teenagers had not recovered, aiming to identify factors influencing prolonged symptoms.

FDA Approves Aucatzyl (obecabtagene autoleucel) for Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia

6 months ago

• The FDA has approved Aucatzyl (obecabtagene autoleucel) for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL). • Aucatzyl demonstrated a 63% overall complete remission rate in efficacy-evaluable patients, with a median duration of remission of 14.1 months in the FELIX clinical trial. • The therapy showed manageable safety profile with low rates of severe Cytokine Release Syndrome (CRS) and Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS). • Aucatzyl, a CD19-directed CAR T-cell therapy, offers a new treatment option for adult r/r B-ALL patients with high unmet medical needs.

AI and Automation Set to Transform Pharmaceutical R&D: Industry Faces Cultural and Technical Hurdles

4 years ago

• Pharmaceutical R&D teams are adopting Computer-Aided Biology (CAB) approaches to tackle increasing complexity in drug development, combining AI and automation to enhance scientific workflows. • McKinsey reports that digital transformation in pharma advanced more in the first ten months of COVID-19 than in the previous decade, with over 60% of life sciences companies investing heavily in AI initiatives. • Industry faces dual challenges of technical integration and cultural resistance, with lack of skilled workforce ranked as the top barrier to digital transformation in pharmaceuticals.