Potential new drug for metastatic urothelial bladder cancer (UBC)
The results of a groundbreaking ECMC clinical trial lead by Professor Thomas Powles at the Barts Cancer Institute has major significance in finding new treatment for metastatic urothelial bladder cancer (UBC).
The phase I trial tested the effectiveness of the drug MPDL3280A (produced by Roche) in patients with advanced bladder cancer.
The trial had an adaptative design which allows researchers to assess the frequency and relevance of biomarkers as well as the efficacy in specific tumour types. Of the 68 patients with UBC who were given the drug, more than half showed signs of effectiveness whilst toxicity remained low.
The positive response among patients to this drug has had a significant impact in the field of immunotherapy. MPDL3280A is an antibody that targets programmed death-ligand 1 (PD-L1), a protein used by cancer cells to hide from immune surveillance. The binding of this drug to PD-L1 exposes cancer cells to the immune system, which facilitates their destruction.
Prof. Powles emphasised the importance of his findings to clinical practice. No major advances have occurred in UBC for the last 30 years and chemotherapy, the main treatment option, is poorly tolerated by the majority of patients.
As a result, outcomes for patients with advanced bladder cancer (the seventh most common form of cancer in the UK) remains poor. Thus, the promising outcomes from this ECMC trial granted MPDL3280A a breakthrough designation status by the US Food and Drug Administration (FDA) in June 2014.
Find the paper, published in Nature, here.