Brain Tumour Research:
This week, we were proud to bring together more than 80 brain tumour experts from across our network of four Brain Tumour Research Centres of Excellence and funded initiatives, as well as from other institutions and organisations, for our annual Researcher Workshop which took place in our home city of Milton Keynes.
Our varied programme had an overarching theme: Translation and how we can deliver impact for patients, and every presentation was as inspiring as it was informative. Read more about the event here.
FDA Gives Priority Review to Tovorafenib in Paediatric Low-Grade Glioma. The FDA has granted priority review to a new drug application (NDA) for single-agent Tovorafenib as a treatment for those with relapsed or progressive paediatric, low-grade glioma, according to a press release from Day One Biopharmaceuticals. The supporting data for the NDA came from the phase 2 FIREFLY-1 trial (NCT04775485) which highlighted an overall response rate of 67%—including a complete response rate of 17% and a partial response rate of 49%—and a clinical benefit rate of 93% among 69 evaluable patients.
Intrathecal delivery of nanoparticle PARP inhibitor to the cerebrospinal fluid for the treatment of metastatic medulloblastoma. The morbidity associated with paediatric medulloblastoma, in particular in patients who develop leptomeningeal metastases, remains high in the absence of effective therapies. Administration of substances directly into the cerebrospinal fluid (CSF) is one approach to circumvent the blood-brain barrier and focus delivery of drugs to the site of tumour. However, high rates of CSF turnover prevent adequate drug accumulation and lead to rapid systemic clearance and toxicity. To overcome this, Khang et al. have optimised delivery of PARP inhibitor therapy through injection into the cerebrospinal fluid (CSF). This intrathecal injection of PARP inhibitor nanoparticles alone, or in combination with Temozolomide, was highly effective in a mouse model of MB and represents a promising strategy that requires further study in patients.
For straightforward information on the blood-brain barrier and why it is a challenge for brain tumour treatment, read our blog here.
Lateral flow test to detect brain cancer recurrence. Scientists in Nottingham are developing a lateral flow test to detect recurring, aggressive brain tumours. The new test would work via a simple finger prick, and it is hoped it could improve the lives of tens of thousands of people across the world. The work is being led by Nottingham Trent University (NTU), funded by the Medical Research Council, working with scientists at the University of Sheffield.
A fully automated deep-learning model for predicting the molecular subtypes of posterior fossa ependymomas using T2-weighted images. Researchers in this study have developed and validated a deep learning (DL) model which automatically segments posterior fossa ependymoma (PF-EPN) and predicts its molecular subtypes (Group A and Group B) from preoperative MR images, which could help in clinical decision-making. Published in AACR Journals, behind a paywall.
Study shows DNA organisation can influence the growth of deadly brain tumours in response to neuronal signals. A study at Umeå University, Sweden, has unveiled that the 3D organisation of DNA can influence the progression of glioblastoma. Having identified the factors that glioblastoma uses to respond to neurons by growing and spreading, this discovery paves the way for further research into new treatments for brain tumours. Published in Nature Communications.