This week in drug discovery (3-7 October)

News round-up for 3-7 October by DDW Digital Content Editor Diana Spencer

This week has brought new trial results and regulatory approvals shedding light and bringing hope into therapy areas of unmet need. The University of Birmingham and King’s College London have furthered our understanding of difficult to treat cancers, while Janssen has revealed encouraging results in gene therapies for X-linked retinitis pigmentosa. 

We have also seen the power of social media to make a difference in the FDA approval of a new treatment for ALS and a new hope for patients with mild Alzheimer’s disease. 

This week’s top stories: 

Study highlights importance of biomarkers in tumour progression 

The University of Birmingham has published the results of a study undertaken in head and neck squamous cell carcinoma (HNSCC) by liquid biopsy company ANGLE, which characterised the epithelial-mesenchymal transition (EMT) status of circulating tumour cells (CTCs).   

‘Ice Bucket Challenge’ funds FDA-approved ALS treatment 

The US Food and Drug Administration (FDA) has granted approval to AMX0035, a new treatment for people living with amyotrophic lateral sclerosis (ALS). 

Janssen shares ‘encouraging’ gene therapy study results 

Results from Phase I/II MGT009 study into investigational gene therapy botaretigene sparoparvovec suggest sustained vision improvement in patients with X-linked retinitis pigmentosa (XLRP). 

New Alzheimer’s treatment reduces decline in Phase III trial 

A new Alzheimer’s disease (AD) treatment has been shown to reduce clinical decline in mild cognitive impairment (MCI) due to AD and mild AD with confirmed presence of amyloid pathology in the brain. 

Immune targets identified for chemo-resistant breast cancers 

Researchers from King’s College London and The Institute of Cancer Research, London, have performed a deep dive into the different immune markers within tumour tissue and blood samples of early breast cancer patients whose cancer failed to respond to chemotherapy given to them prior to surgery. 

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