CARDIFF UNIVERSITY

Novel Fecal Microbiota Pill Enters Clinical Trial for Advanced Pancreatic Cancer Treatment

3 months ago

• Canadian researchers launch Phase 1 trial of LND101, an oral fecal transplant therapy, targeting pancreatic cancer's dismal 10% five-year survival rate. • The innovative "poop pill" aims to modify tumor microbiome composition, potentially enhancing chemotherapy effectiveness in advanced pancreatic cancer patients. • Following success in melanoma, lung cancer, and renal cell carcinoma, the two-year trial will evaluate gut bacteria changes and immune responses in 20 patients.

MediWound Initiates Phase III Trial of EscharEx for Venous Leg Ulcers

5 months ago

• MediWound has commenced a Phase III clinical trial (VALUE) to evaluate EscharEx for the treatment of venous leg ulcers (VLUs). • The VALUE trial is a randomized, double-blind, placebo-controlled study across 40 sites in the U.S. and Europe, enrolling 216 patients. • EscharEx has shown promise in Phase II trials, demonstrating effective and rapid debridement of chronic wounds, potentially redefining VLU treatment. • An interim analysis of the VALUE trial is expected in mid-2026, with strategic collaborations in place to ensure consistent wound management.

European Commission Approves Emcitate (Tiratricol) as First Treatment for MCT8 Deficiency

5 months ago

• The European Commission has granted marketing authorization for Emcitate (tiratricol), making it the first approved treatment for monocarboxylate transporter 8 (MCT8) deficiency in the EU. • The approval is based on data from Triac Trial I, the Erasmus University Medical Center Cohort Study, and preliminary results from Triac Trial II, demonstrating significant reductions in serum T3 concentrations. • Emcitate is indicated for treating peripheral thyrotoxicosis in MCT8 deficiency patients from birth, addressing symptoms like increased heart rate and muscle weakness. • Egetis Therapeutics anticipates initiating pricing and reimbursement discussions in Europe, with the first launch expected in the second quarter of 2025.

Donanemab Approved by MHRA but Rejected by NICE for NHS Use Due to Cost Concerns

7 months ago

• Donanemab, a new Alzheimer's treatment by Eli Lilly, has been approved by the MHRA but rejected by NICE for use in the NHS due to high costs. • NICE determined that donanemab does not offer sufficient value for NHS use, despite clinical evidence showing it could slow disease progression by 4-7 months. • Concerns over the drug's safety profile, including amyloid-related imaging abnormalities (ARIA) in 33% of participants, and the need for monthly infusions contributed to the rejection. • Alzheimer's Society expressed mixed emotions, acknowledging the hope offered by disease-modifying therapies but respecting NICE's decision and advocating for improved affordability.

Leqembi Faces Rejection in Australia Over Efficacy and Safety Concerns

7 months ago

• Australia's Therapeutic Goods Administration (TGA) has declined to approve Leqembi, citing that its efficacy does not outweigh the risks of brain swelling and bleeds. • The TGA's decision aligns with the European Medicines Agency's earlier recommendation against Leqembi, raising questions about its clinical benefit. • Eisai Australia intends to request a reconsideration of the decision within 90 days, while patient advocacy groups express disappointment over limited treatment options. • Leqembi is currently approved in the US, Japan, China, South Korea, Hong Kong, Israel, the UAE, and GB, but faces access challenges in the UK due to cost-effectiveness concerns.

Psoriasis Drug Ustekinumab Shows Promise in Preserving Insulin Production in Young Type 1 Diabetes Patients

10 months ago

• A clinical trial found that ustekinumab, a drug currently used for psoriasis, preserved insulin production in young type 1 diabetes patients, with C-peptide levels 49% higher after one year compared to placebo. • Researchers discovered ustekinumab targets a specific subset of immune cells called Th17.1 cells, which make up only 0.1% of blood immune cells but play a key role in destroying insulin-producing cells. • The treatment could potentially reduce or eliminate the need for insulin injections if administered early enough, with researchers suggesting future trials combining screening and early intervention.