Rare cancers

The AACR Annual Meeting’s second clinical trials plenary session, ‘Hope for Rare Cancers: Novel Targeted and Immunotherapy Agents’, highlighted promising studies that suggest new treatment approaches for rare cancers. First Hippo-YAP pathway inhibitor shows success in advanced solid tumours Timothy A Yap reported on a study that provides the first clinical proof-of-concept for effectively drugging […]

The addition of pembrolizumab (Keytruda) to gemcitabine and cisplatin improved overall survival in patients with untreated metastatic or unresectable biliary tract cancer, according to results from the Phase III KEYNOTE-966 clinical trial. The results were presented at the AACR Annual Meeting 2023, held April 14-19 and published in The Lancet. Biliary tract cancers are rare […]

DDW Editor Reece Armstrong speaks to Nautilus Co-Founder and Chief Scientist Parag Mallick, about the company’s recent collaboration which aims to uncover the proteins that could be behind a rare and fatal childhood cancer. At the beginning of 2023, proteomics company Nautilus Biotechnology announced a partnership with the Translational Genomics Research Institute (TGen), to study […]

This paid-for advertorial by IRBM appeared in DDW Volume 24 – Issue 2, Spring 2023 The landscape for treating rare cancers is changing with advances in molecular genetics, novel drug modalities and patient-derived models. Dr Alessandro Carugo, IRBM, explains how building unique drug discovery strategies and expert alliances is reshaping the rare cancer drug ecosystem. Why […]

Rare diseases are often overlooked for funding and research, but with various incentives and priority review programmes now in place, many companies are beginning to explore these unusual but often devastating conditions. This Rare Disease Day we provide an overview of the progress that has been made in a range of rare diseases so far […]

Cancer Research UK, The University of Manchester and Roche have opened a multi-drug, precision medicine trial for people with rare cancers.  The trial is set up to recruit both paediatric and adult patients with any rare cancer type and is one of very few precision medicine platform trials in the world targeting these populations.  The […]