New collaboration to develop treatments for liver disease

September 7, 2012

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 that plays a major role in the life-threatening liver disease that develops in a population of patients with a particular .

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 .

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, 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 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 as a result of alpha-1 antitrypsin deficiency is transplantation," said Professor Lomas. "This partnership brings together our collective expertise in 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."

This alliance is part of GSK's Discovery Partnerships with Academia (DPAc) initiative, designed to bring together the complementary skill sets of GSK and individual academic groups in the search for . Dr Pearl Huang, who leads GSK's Global DPAc, said, "Through this collaboration, we'll be able to combine the substantial knowledge and insight of the Cambridge University scientists working in this field with GSK's drug discovery expertise. By combining our strengths in this way and creating an integrated partnership we will have in place a strong framework for discovering new medicines for patients."

Dr Emma Barker of Cambridge Enterprise, the University's commercialisation group said: "This is a great example of translating academic research. We are delighted to be involved with GlaxoSmithKline's DPAc alliance with the goal of developing & commercialising medicines to treat a clear unmet medical need."

Under the terms of the agreement, the University and Cambridge Enterprise will receive success-based financial support from GSK linked to reaching agreed milestones, as well as an undisclosed upfront payment and royalties on sales from any product that is successfully commercialised out of the collaboration.

Explore further: 'Bird brains' are smart on Alzheimer's

Related Stories

'Bird brains' are smart on Alzheimer's

April 18, 2011
(PhysOrg.com) -- Tel Aviv University research says our feathered friends may hold the key to a treatment for brain-related diseases

British drugs firm GSK settles US drug disputes for $3.0 bln

November 3, 2011
GlaxoSmithKline said on Thursday it had agreed to pay $3.0 billion (2.2 billion euros) to settle long-running disputes with the US government over how the British pharmaceuticals group marketed and developed drugs.

GlaxoSmithKline reports return to profit in second quarter

July 26, 2011
British drugmaker GlaxoSmithKline posted net profit of more than £1.1 billion in the second quarter on Tuesday following a loss during the equivalent three-month period in 2010.

New gene therapy methods accurately correct mutation in patient's stem cells

October 12, 2011
For the first time, scientists have cleanly corrected a human gene mutation in a patient's stem cells. The result, reported in Nature on Wednesday 12 October, brings the possibility of patient-specific therapies closer to ...

Recommended for you

Two Group A Streptococcus genes linked to 'flesh-eating' bacterial infections

September 22, 2017
Group A Streptococcus bacteria cause a variety of illnesses that range from mild nuisances like strep throat to life-threatening conditions including pneumonia, toxic shock syndrome and the flesh-eating disease formally known ...

Ecosystem approach makes urinary tract infection more treatable

September 22, 2017
The biological term 'ecosystem' is not usually associated with urinary tract infections, but this should change according to Wageningen scientists.

Residents: Frontline defenders against antibiotic resistance?

September 22, 2017
Antibiotic resistance continues to grow around the world, with sometimes disastrous results. Some strains of bacteria no longer respond to any currently available antibiotic, making death by infections that were once easily ...

Individualized diets for irritable bowel syndrome better than placebo

September 21, 2017
Patients with irritable bowel syndrome who follow individualized diets based on food sensitivity testing experience fewer symptoms, say Yale researchers. Their study is among the first to provide scientific evidence for this ...

Superbug's spread to Vietnam threatens malaria control

September 21, 2017
A highly drug resistant malaria 'superbug' from western Cambodia is now present in southern Vietnam, leading to alarming failure rates for dihydroartemisinin (DHA)-piperaquine—Vietnam's national first-line malaria treatment, ...

A dose of 'wait-and-see' reduces unnecessary antibiotic use

September 21, 2017
Asking patients to take a 'wait-and-see' approach before having their antibiotic prescriptions filled significantly reduces unnecessary use, a University of Queensland study has shown.

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.