Latest News

• Trogenix announces publication of breakthrough pre-clinical data in Nature demonstrating complete tumour eradication and durable protection in aggressive brain cancer model        

  A recent publication in Springer Nature reports preclinical results for an experimental gene therapy developed by the Scottish company Trogenix for glioblastoma (GBM). The therapy uses engineered DNA elements called Synthetic Super-Enhancers (SSEs), delivered via adeno-associated virus (AAV) vectors. The SSEs are designed to activate specifically in GBM stem-like cells by exploiting SOX2-driven transcriptional programs. Once activated, they deliver two payloads: HSV-TK for targeted tumor cell killing via a prodrug mechanism, and IL-12 to stimulate an anti-tumor immune response.

  The approach was first validated in patient-derived GBM stem cell cultures, where SSE activity was seen in tumor cells but not in non-tumor neural cells. It was then tested in orthotopic, immunocompetent mouse models of GBM. In these models, a single dose led to rapid tumor regression within 1-2 weeks, with complete tumor clearance in 83% of treated animals. No tumor recurrence or significant toxicity was observed over 11 months, and re-challenged mice did not develop new tumors, suggesting a durable immune response.

  A first-in-human Phase I/II GBM trial is planned for 2026.

  
  
• Wearable Technology's Growing Role in Monitoring Brain Health        

  This article highlights growing interest in wearable devices to monitor brain health, including seizure detection. Current wearables may be helpful for brain tumor patients who experience tonic-clonic seizures, as they can alert a caregiver remotely during an event. However, they do not reliably detect focal seizures, which limits their usefulness for many patients with brain tumor-related epilepsy.

  
  
• Mayo Clinic experimental dual-drug nanotherapy crosses the blood-brain barrier and improved survival in preclinical glioblastoma models        

  A new study from Mayo Clinic published in Nature Communications Medicine reports preclinical results for a nanotherapy designed to improve drug delivery in glioblastoma (GBM). The approach uses liposomal nanoparticles engineered to cross the blood-brain barrier and co-deliver vinorelbine with either everolimus or rapamycin directly to tumor cells. The combination is designed to inhibit tumor growth signaling pathways and impair DNA repair, thereby increasing sensitivity to radiation.

  The therapy was tested in patient-derived glioblastoma models, including orthotopic mouse models. When combined with radiation, the treatment more than doubled survival in mice compared with untreated controls, with the vinorelbine-rapamycin arm showing the strongest overall survival benefit.

  While this therapy is still in early development, liposome-based drug delivery systems are an active area of GBM research and we hope to see this work translated to human trials in the future.

  
  
• GCAR and Purdue Pharma L.P. Announce Initiation of Tinostamustine in GBM AGILE Trial        

  GBM AGILE is a large, international Phase 2/3 trial for newly diagnosed and recurrent glioblastoma. It has an innovative adaptive design that allows multiple experimental therapies to be tested at the same time against a shared control arm to more efficiently identify promising treatments. We have a Musella Foundation recorded webinar about the trial available HERE.

  GBM AGILE is now adding a new study arm for Tinostamustine, an investigational drug that combines DNA-alkylating activity (to damage tumor DNA and trigger cell death) with HDAC inhibition (an epigenetic mechanism that may increase tumor sensitivity and immune recognition). Earlier phase 1 studies showed the drug has a manageable safety profile.