McGill University and GlaxoSmithKline plc (GSK) have signed a collaboration agreement to develop a potential breakthrough approach to treat cystic fibrosis, a fatal genetic disease. The trans-Atlantic effort between researchers from McGill’s Faculty of Medicine and their GSK collaborators in the UK, will focus on developing molecules that could treat the disease by correcting the dysfunction caused by the mutated gene. This revolutionary approach will be a departure from current treatments, which only manage the symptoms or complications of the disorder. The collaborative research is co-sponsored by the Canadian Institutes of Health Research (CIHR).
Cystic fibrosis (CF) is one of the most common fatal genetic diseases. It is a multi-organ disease but primarily affects the lungs and digestive system, causing mucous to thicken and leaving patients at higher risk of infections. CF is caused by a mutation in the gene for the protein cystic fibrosis transmembrane conductance regulator (CFTR). About 90 per cent of those living with cystic fibrosis have a particular mutant form of this gene, which makes a protein that could still work but has a defect that prevents it getting to the right place in the cell to function correctly.
Dr. David Thomas, Chair of McGill’s Department of Biochemistry and Canada Research Chair in Molecular Genetics, is an expert in protein quality control. Dr. John Hanrahan is Professor in McGill’s Department of Physiology and an expert in chloride transport and epithelial biology. They are uniquely qualified as lead investigators on McGill’s side, having conducted extensive research on the CFTR protein and have identified a number of molecules that look to restore normal CFTR function.
“If we can restore normal function to the mutant cystic fibrosis protein, we can radically transform the lives of millions of children. It will move us from treatment of symptoms to prevention of the disease and would ultimately save lives,” said Dr. Thomas. “We will now be looking at enhancing these small molecules to improve their ability to “rescue” or “correct” the mutant protein, allowing it to reach its proper location and be more active,” added Dr. Hanrahan.
“GSK is excited to be applying our expertise in areas including medicinal chemistry to this innovative area of research,” said Dr. Roberto Solari, head of respiratory biology at GSK. “We hope at the end of this two year collaboration we will have a compound that is be ready to be investigated as a potential new medicine. Collaborations such as these – where we share knowledge, expertise and resource – provide a highly effective way of progressing cutting edge research.”
“This strong collaboration between an industry partner, government granting agencies and the University is a superb example of partners building on mutual strengths and collaborating where our research interests converge,” said Dr. Rose Goldstein, Vice-Principal (Research and International Relations). “We are grateful for the support provided by GSK and CIHR, facilitating our work together to give people with cystic fibrosis a healthy and full life expectancy.” ”Health research successes in the 21st century will be very much dependent on the ability of organizations from the public, academic and private sector to combine efforts and pool resources,” said Dr. Marc Ouellette, Scientific Director for The Canadian Institutes of Health Research’s Institute of Infection and Immunity. “In particular, this grant will increase our understanding of the cellular mechanisms causing cystic fibrosis and provide novel ways of targeting genetic based diseases. Research done through the support of this partnership will add to groundbreaking work done by Canada’s strong Cystic Fibrosis research community, including those funded under the Canadian led Human Microbiome Initiative.”
This research program has funding from both GSK and from an R&D operating grant from CIHR. It builds on previous work carried out with support from the Canadian and US CF Foundations, Génome Québec, CIHR and the Canadian Foundation for Innovation (CFI). It is the flagship project of a new cystic fibrosis translational research CFTR centre at McGill that has been established with support from the CFI.
On the web:
Dr. David Thomas: http://www.mcgill.ca/biochemistry/department/faculty/thomas/
Dr. John Hanrahan’s Lab: www.medicine.mcgill.ca/physio/hanrahanlab/default.htm
About Cystic Fibrosis
Cystic Fibrosis is caused by a single faulty gene that controls the movement of salt in the body. In people with Cystic Fibrosis, the internal organs become clogged with thick, sticky mucus resulting in infections and inflammation making it hard to breathe and digest food. There is no cure for the disease. About one in every 3,600 children born in Canada has CF. Treatment is aimed at controlling symptoms and infections. All disease-causing mutations in the gene for CFTR prevent the chloride channel from functioning properly, blocking movement of salt and water into and out of cells. As a result, cells that line the passageways of the lungs, pancreas, and other organs produce abnormally thick, sticky mucus. This viscous mucus cannot be easily cleared by the body, leading to obstruction of airways and glands and trapping bacteria that give rise to chronic infections.
About McGill University
Founded in Montreal, Que., in 1821, is Canada’s leading post-secondary institution. It has two campuses, 11 faculties, 10 professional schools, 300 programs of study and more than 36,000 students. McGill attracts students from some 150 countries around the world. Almost half of McGill students claim a first language other than English – including over 6,000 francophones – with more than 7,200 international students making up almost 20 per cent of the student body.
GlaxoSmithKline (GSK) is a leading research-based pharmaceutical company with a challenging and inspiring mission: to improve the quality of human life by enabling people to do more, feel better, and live longer. This mission gives GSK the purpose to develop innovative medicines, vaccines and healthcare solutions that help millions of people.
In Canada, GSK Inc is consistently recognized as one of the 50 best employers and is a top 15 investor in Canadian research and development, contributing more than $144 million in 2009 alone. With a proud tradition of charitable and community support, GSK is designated a Caring Company by Imagine Canada. Discover more at www.GSK.ca.
About The Canadian Institutes of Health Research
The Canadian Institutes of Health Research (CIHR) is the Government of Canada’s agency for health research. CIHR’s mission is to create new scientific knowledge and to enable its translation into improved health, more effective health services and products, and a strengthened Canadian health-care system. Composed of 13 Institutes, CIHR provides leadership and support to nearly 12,000 health researchers and trainees across Canada.