CAMBRIDGE UNIVERSITY HOSPITALS NHS FOUNDATION TRUST

Novel Drug Combination Shows Promise for B-Cell Acute Lymphoblastic Leukemia with Reduced Toxicity

2 days ago

• Cambridge researchers have developed a promising combination therapy using venetoclax and inobrodib that effectively targets B-cell acute lymphoblastic leukemia (B-ALL), potentially reducing the need for toxic chemotherapy. • The drug combination works by exploiting cancer cell metabolism, triggering ferroptotic cell death even in cells resistant to venetoclax alone, offering new hope for both pediatric and adult patients. • Unlike current treatments that require over two years of chemotherapy or risky procedures like bone marrow transplants, this oral therapy could provide a less toxic alternative while maintaining effectiveness.

Olaparib Before Surgery Shows 100% Survival Rate in BRCA-Mutated Breast Cancer Patients

9 days ago

• A groundbreaking clinical trial led by Addenbrooke's Hospital demonstrates 100% three-year survival rate for patients with BRCA-mutated breast cancer when treated with olaparib plus chemotherapy before surgery. • The innovative "Partner" trial revealed that administering olaparib 48 hours after chemotherapy creates a therapeutic window that maximizes treatment efficacy while allowing bone marrow recovery. • This treatment approach could potentially transform care for approximately 1,200 UK patients annually with inherited BRCA mutations and may extend to other BRCA-related cancers including ovarian, prostate, and pancreatic.

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.

Diabetes Drug Metformin Shows Promise in Preventing Acute Myeloid Leukemia

a month ago

• Cambridge researchers discover that metformin, a widely prescribed diabetes medication, may help prevent acute myeloid leukemia (AML) in high-risk individuals by targeting mutations in the DNMT3A gene. • The drug appears to disrupt cancer development by interfering with pre-cancerous cell energy production and reversing damage caused by genetic mutations, offering a potential preventive strategy for this aggressive blood cancer. • With metformin's established safety profile and low cost (about 35p per pill), researchers are preparing clinical trials to confirm its efficacy as a repurposed cancer prevention therapy, potentially accelerating treatment availability.

Archer Platform Launches at Cambridge University Hospitals to Revolutionize Clinical Trial Data Management

2 months ago

• IgniteData, AstraZeneca, and Cambridge University Hospitals have launched the Archer platform in a Phase 3 clinical trial, automating data transfer between electronic health records and clinical trial databases. • The technology eliminates manual data entry by securely transferring hundreds of data points per patient from electronic health records to electronic data capture systems, significantly improving research efficiency. • Following successful pilots at University College London Hospitals and Memorial Sloan Kettering Cancer Center, this implementation represents a major advancement in clinical research methodology with potential for global expansion.

Morning Asthma Testing Yields More Reliable Results, Study Reveals Significant Seasonal Variations

2 months ago

• Research analyzing 1,600 patient records shows asthma spirometry tests are 8% more reliable for each hour closer to 8:30 AM, highlighting the impact of circadian rhythms on diagnostic accuracy. • Winter testing produces significantly more accurate results than autumn testing, with patients 33% less likely to show a positive response to salbutamol during autumn months. • Findings from the Victor Phillip Dahdaleh Heart and Lung Research Institute suggest clinical guidelines may need revision to account for time-of-day and seasonal factors in asthma diagnosis.

Groundbreaking UK Study Advances Treatment Monitoring for Pediatric Traumatic Brain Injuries

3 months ago

• A pioneering multi-center study across 10 UK Pediatric Intensive Care Units, led by Dr. Shruti Agrawal from Addenbrooke's Hospital, has validated new monitoring techniques for children with traumatic brain injuries. • The STARSHIP study, involving 135 children under 16, demonstrates the effectiveness of pressure reactivity index (PRx) monitoring for personalizing treatment targets in severe pediatric head trauma. • The research establishes critical PRx thresholds for patient outcomes and creates a valuable database for future investigations, potentially transforming how traumatic brain injuries in children are managed.

Novel Minimally Invasive 'Triple T' Therapy Shows Promise in Treating Primary Aldosteronism

3 months ago

• UK researchers have developed Triple T (Targeted Thermal Therapy), a groundbreaking 20-minute minimally invasive procedure that treats primary aldosteronism without removing the adrenal gland. • The FABULAS trial demonstrated Triple T's safety and effectiveness in 28 patients, with most achieving normal hormone levels after six months and many discontinuing blood pressure medications. • This innovative treatment could potentially help millions worldwide, as primary aldosteronism affects 1 in 20 people with high blood pressure but is currently underdiagnosed and undertreated.

Amicus Therapeutics to Present Extensive Research on Fabry and Pompe Diseases at WORLDSymposium 2025

4 months ago

• Amicus Therapeutics will showcase 16 presentations at WORLDSymposium 2025, featuring comprehensive research on migalastat for Fabry disease and cipaglucosidase alfa plus miglustat for Pompe disease. • Key studies include long-term patient outcomes from the FollowME Fabry Pathfinders registry and real-world evidence of treatment effectiveness in both rare diseases. • Notable research highlights treatment satisfaction, quality of life improvements, and clinical outcomes in patients switching from existing therapies to Amicus' novel treatment approaches.

Pivotal BEST4 Trial Assesses 'Pill-on-a-Thread' for Oesophageal Cancer Screening

6 months ago

• The BEST4 Screening trial is evaluating Medtronic's Cytosponge-TFF3 for Barrett's oesophagus screening, a precursor to oesophageal cancer. • The trial aims to recruit 120,000 participants taking heartburn medication, utilizing mobile screening vans across England for convenient testing. • The capsule sponge test offers a faster, cheaper, and less invasive alternative to endoscopies, with results available in just ten minutes. • The BEST4 trial is backed by £6.4 million in funding from Cancer Research UK and the NIHR, building on promising results from the BEST3 study.

Key Advances in Inflammatory Bowel Disease: New Therapies, Treatment Strategies, and Long-Term Data

6 months ago

• Early, intensive 'top-down' therapy with TNF blockers plus immunomodulators shows superior outcomes compared to the traditional 'step-up' approach for newly diagnosed Crohn's disease. • Risankizumab (Skyrizi) and guselkumab (Tremfya) receive FDA approval for ulcerative colitis, offering new options for patients who have not responded well to conventional treatments. • Research indicates intensified infliximab dosing does not improve outcomes in steroid-refractory acute severe ulcerative colitis, suggesting a need for alternative strategies. • Long-term data reveals tulisokibart maintains clinical and endoscopic improvements in IBD patients, supporting its advancement to phase III trials.

Adaptive Trial Platform Launches to Accelerate Glioblastoma Treatment Development

7 months ago

• A new adaptive clinical trial, the 5G platform trial, will test multiple treatments for glioblastoma, an aggressive brain cancer, in the UK. • The trial employs whole-genome sequencing to personalize treatment based on the genetic makeup of each patient's tumor. • Cancer Research UK and Minderoo Foundation are jointly funding the trial with £3.36 million to expedite the development of effective therapies. • The adaptive design allows for real-time adjustments to treatment regimens, potentially improving outcomes for patients with this unmet need.

CRISPR Gene Editing Shows Promise as Single-Dose Treatment for Hereditary Angioedema

7 months ago

• A single dose of CRISPR-Cas9 gene editing therapy, NTLA-2002, significantly reduces angioedema attacks in patients with hereditary angioedema (HAE). • The Phase 2 study demonstrated a substantial and sustained reduction in kallikrein levels, a key mediator of swelling in HAE patients. • The international trial involved multiple national excellence centers and showed promising results, potentially offering a functional cure for HAE. • NTLA-2002 is advancing to Phase 3 clinical trials, building on the positive outcomes from Phase 1 and 2 studies.

Personalized Brain Cancer Trial Aims to Revolutionize Glioblastoma Treatment

7 months ago

• A new clinical trial, the 5G platform trial, will use individual patient DNA to tailor treatments for glioblastoma, an aggressive brain cancer. • The trial, backed by Cancer Research UK and the Minderoo Foundation, seeks to address the lack of breakthroughs in glioblastoma treatment over the past two decades. • Researchers will sequence each patient's genome to target treatments with greater precision, potentially testing drugs developed for other cancers. • The study, sponsored by The Institute of Cancer Research, London, aims to accelerate the development of new treatments and improve patient outcomes.

CRUK Receives $225 Million Investment; New CAR-T Cell Facility; Promising Gene Therapy for Parkinson's

7 months ago

• Cancer Research UK (CRUK) has received a US$225 million investment to enhance cancer research and treatment, focusing on personalized medicine and early detection. • The University of Cambridge is opening a new facility to manufacture CAR-T cell therapies, expanding treatment options for patients across the East of England. • MeiraGTx Holdings plc announced positive data from its MGT-GAD-025 study, assessing AAV-GAD gene therapy for Parkinson’s disease, demonstrating motor improvements.

UK Boy Enrolls in Global Trial for Novel Low-Grade Glioma Drug

9 months ago

• A six-year-old boy from Peterborough is the first UK participant in a global clinical trial for a novel drug targeting low-grade gliomas. • The trial evaluates the efficacy of a new drug against standard chemotherapy in newly diagnosed pediatric low-grade gliomas with RAF alterations. • The investigational drug, developed by a US biotech, aims to stabilize tumors, induce shrinkage, and reduce long-term side effects compared to traditional treatments. • The trial includes a crossover option, allowing patients initially assigned to chemotherapy to receive the investigational drug if the initial treatment proves ineffective.