Successful drug development for people with cancers of the CNS has been challenging. There are multiple barriers to successful drug development including biological factors, rarity of the disease, and ineffective use of clinical trials. Based upon a series of presentations at the First Central Nervous System Clinical Trials Conference hosted by the American Society of Clinical Oncology and the Society for Neuro-Oncology, we provide an overview on drug development and novel trial designs in neuro-oncology. This Review discusses the challenges of therapeutic development in neuro-oncology and proposes strategies to improve the drug discovery process by enriching the pipeline of promising therapies, optimising trial design, incorporating biomarkers, using external data, and maximising efficacy and reproducibility of clinical trials.
Download full-text PDF |
Source |
---|---|
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC10401610 | PMC |
http://dx.doi.org/10.1016/S1470-2045(23)00005-0 | DOI Listing |
Elife
January 2025
Department of Molecular and Cell Biology, Berkeley, United States.
Type II nuclear receptors (T2NRs) require heterodimerization with a common partner, the retinoid X receptor (RXR), to bind cognate DNA recognition sites in chromatin. Based on previous biochemical and overexpression studies, binding of T2NRs to chromatin is proposed to be regulated by competition for a limiting pool of the core RXR subunit. However, this mechanism has not yet been tested for endogenous proteins in live cells.
View Article and Find Full Text PDFNeuro Oncol
January 2025
Childhood Cancer & Cell Death team (C3 team), Consortium South-ROCK, LabEx DEVweCAN, Institut Convergence Plascan, Centre Léon Bérard, Centre de Recherche en Cancérologie de Lyon (CRCL), Université Claude Bernard Lyon 1, INSERM 1052, CNRS 5286, 69008 Lyon, France.
Background: Brain tumors are the deadliest solid tumors in children and adolescents. Most of these tumors are glial in origin and exhibit strong heterogeneity, hampering the development of effective therapeutic strategies. In the past decades, patient-derived tumor organoids (PDT-O) have emerged as powerful tools for modeling tumoral cell diversity and dynamics, and they could then help defining new therapeutic options for pediatric brain tumors.
View Article and Find Full Text PDFCNS Drugs
January 2025
School of Medicine and Dentistry, Gold Coast Campus, Griffith University, Southport, QLD, 4222, Australia.
Background: Epstein-Barr virus (EBV) is implicated as a necessary factor in the development of multiple sclerosis (MS) and may also be a driver of disease activity. Although it is not clear whether ongoing viral replication is the driver for MS pathology, MS researchers have considered the prospect of using drugs with potential efficacy against EBV in the treatment of MS. We have undertaken scientific and lived experience expert panel reviews to shortlist existing licensed therapies that could be used in later-stage clinical trials in MS.
View Article and Find Full Text PDFAppl Biochem Biotechnol
January 2025
Department of Life Science and Biochemical Engineering, Graduate School, SunMoon University, Asan, 31460, Republic of Korea.
Antarctic organisms are known for producing unique secondary metabolites, and this study specifically focuses on the less-explored metabolites of the moss Warnstorfia fontinaliopsis. To evaluate their potential bioactivity, we extracted secondary metabolites using four different solvents and identified significant lipase inhibitory activity in the methanol extract. Non-targeted metabolomic analysis using liquid chromatography-tandem mass spectrometry (LC-MS/MS) on this extract predicted the presence of 12 compounds, including several not previously reported in mosses.
View Article and Find Full Text PDFDrug Saf
January 2025
Pfizer (Worldwide Medical & Safety), New York, NY, USA.
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!