A new collaboration based at the University of Cambridge will aim to discover and develop new medicines to treat liver disease.
The partnership, between the University and global pharmaceutical company GlaxoSmithKline (GSK), will build upon the work of researchers who have identified a molecular mechanism that plays a major role in the life-threatening liver disease that develops in a population of patients with a particular mutant gene.
Alpha-1 antitrypsin (A1AT) deficiency is one of the most common genetic disorders in the UK, affecting approximately one in 2,000 people. The A1AT protein is produced mainly in the liver and circulates to the lungs, where it serves a protective function against enzymes which can break down lung tissue.
In patients with A1AT deficiency, the protein cannot circulate freely and accumulates in the liver, leading to potentially life-threatening liver conditions including neonatal hepatitis, cirrhosis and hepatocellular carcinoma. Additionally, without A1AT circulating to the lungs, lung tissue can break down, predisposing patients to early onset emphysema. Currently, the only available treatments are liver transplantation for cirrhosis and protein replacement therapy for emphysema.
The mechanism that underlies protein accumulation in the livers of individuals with A1AT deficiency has been defined by the team led by Professor David Lomas in the University's Cambridge Institute for Medical Research (CIMR). The collaboration with GSK will combine Professor Lomas' long-term research with GSK's expertise in drug discovery and development in order to develop new therapeutics. Work on the project will be carried out both at GSK and at the CIMR.
"Currently, the only option for patients with liver disease as a result of alpha-1 antitrypsin deficiency is transplantation," said Professor Lomas. "This partnership brings together our collective expertise in target biology and drug discovery to tackle an unmet medical need and by teaming up with GSK, we have a great opportunity to turn our research into effective treatments."