Our Success Stories

We were instrumental in the development of ovarian cancer treatment, Rubraca®. From initial drug discovery through to use with patients, the drug's development can be traced right through our University.

• Ovarian cancer patients get access to life-extending drug
• Newcastle University to invest cancer drug IP earning into research and teaching
• New ovarian cancer drug now available on the NHS

Our Drug Discovery Unit pursued the idea of developing a selective FGFR tyrosine kinase inhibitor and established a single-project collaboration with Astex Pharmaceuticals for this purpose, leading to the discovery of compounds that were shown to have very significant antitumour activity in FGF/FGFR-dependent tumour models. The inhibitors formed the basis of a further collaboration between Astex and Janssen, resulting in the clinical development candidate, erdafitinib (JNJ-42756493).

In April 2019, erdafitinib became the first FGFR tyrosine kinase inhibitor to receive an FDA (USA) approval in patients with locally advanced and unresectable or metastatic urothelial cancer that harboured aberrant forms of FGFR-2 or -3, and who had previously progressed on at least one line of platinum-containing chemotherapy. The compound is now marketed by Janssen as Balversa^TM.

• Urothelial cancer treatment breakthrough

Since 1992, the Leukaemia Research Cytogenetics Group has developed and leveraged a unique research infrastructure. It links:

• clinicians
• clinical trials
• NHS laboratories
• researchers

Using this network, we integrate diagnostic cytogenetics with state-of-art genomics to discovery and evaluate biomarkers in acute leukemia.

In 2003, we discovered a complex abnormality of chromosome 21 (iAMP21) affecting children with acute lymphoblastic leukaemia and driving a very high risk of relapse. Prospective clinical trials showed patients with iAMP21 benefited from high intensive chemotherapy. iAMP21 is now part of the WHO classification and patients worldwide are treated with high intensity chemotherapy.

More recently, our research integrating chromosomal abnormalities, copy number alterations and treatment response data led to the development of a novel risk stratification algorithm being used by the ALLTogether consortium in 14 European countries.

Our 2005 and 2006 discoveries of the WNT molecular subgroup of medulloblastomas, and its association with almost 100% survival, led to their incorporation into the World Health Organisation (WHO) classification of CNS tumours in 2016.

These discoveries now form the basis of routine medulloblastoma diagnostics and clinical trials of personalised medicine around the world, including pioneering reduced intensity therapies for WNT patients.

In 2017, we discovered further novel clinically-significant biological subtypes of medulloblastoma using large-scale genomic and epigenomic investigations. Working with our international partners, our subsequent analysis in 2019 reached consensus on the number and nature of these subtypes, and they will form part of the 2021 WHO classification.

Our childhood cancer research groups founded and host national diagnostics reference services for medulloblastoma, neuroblastoma and childhood leukaemia, on behalf of the UK clinical coordinating groups (CCLG, NCRI).

We further lead on diagnostics implementation and biological research studies within international clinical trials. These reference services were initially developed out of our research programmes in these diseases, and now deliver advanced diagnostics in conjunction with our NHS partners at the Newcastle Hospitals Trust. We develop bespoke tests and are continually updating our testing repertoire to reflect new discoveries from ourselves and others, through our ongoing R&D activities within our University and NHS laboratories.

Our translational research on the anticancer drug carboplatin, has established us as the national centre for Therapeutic Drug Monitoring (TDM) in a childhood cancer setting.

The award winning service, initially developed out of our Cancer Research UK funding programme of work and more recently through dedicated NIHR funding, is implemented in national treatment guidelines approved by the Childhood Cancer and Leukaemia Group (CCLG) for a wide range of tumour types including retinoblastoma, neuroblastoma and childhood brain tumours. The group now deliver real-time TDM to support the treatment of some of the most challenging childhood cancer patient groups (including neonates and infants) at paediatric oncology primary treatment centres across the UK.