Navigation Links
Breakthrough on cystic fibrosis 1 step closer as new research alliance formed
Date:1/27/2011

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, Gnome Qubec, 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.


'/>"/>

Contact: Allison Flynn
allison.j.flynn@mcgill.ca
514-398-7698
McGill University
Source:Eurekalert

Related biology news :

1. Biomedical breakthrough: Blood vessels for lab-grown tissues
2. 7 CAMH scientists win Young Investigator awards focused on breakthrough research
3. Shellfish safer to eat thanks to breakthrough by Queens scientists
4. IVF breakthrough to hit the world market
5. An important breakthrough by IRCM researchers in hematopoiesis and the development of B cells
6. Damon Runyon Cancer Research Foundation awards breakthrough scientists
7. Breakthrough in worm research has implications for human disease studies
8. VCU Massey discovery could lead to breakthrough for non-small cell lung cancer
9. Longevity breakthrough: The metabolic state of mitochondria controls life span
10. Argonne scientists awarded supercomputing time to enable scientific breakthroughs
11. $1.6 million to take forward breakthrough research in heart disease
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:6/16/2016)... June 16, 2016 The ... expected to reach USD 1.83 billion by 2024, ... Research, Inc. Technological proliferation and increasing demand in ... expected to drive the market growth. ... The development of advanced multimodal techniques for ...
(Date:6/9/2016)... , June 9, 2016  Perkotek an innovation leader in attendance control systems ... seamlessly log work hours, for employers to make sure the right employees are actually ... http://photos.prnewswire.com/prnh/20160609/377486LOGO ... ... ...
(Date:6/7/2016)... , June 7, 2016  Syngrafii Inc. ... a business relationship that includes integrating Syngrafii,s patented ... branch project. This collaboration will result in greater ... the credit union, while maintaining existing document workflow ... http://photos.prnewswire.com/prnh/20160606/375871LOGO ...
Breaking Biology News(10 mins):
(Date:6/23/2016)... Mass. , June 23, 2016   ... development of novel compounds designed to target cancer ... napabucasin, has been granted Orphan Drug Designation from ... the treatment of gastric cancer, including gastroesophageal junction ... stemness inhibitor designed to inhibit cancer stemness pathways ...
(Date:6/23/2016)... 23, 2016 A person commits a crime, and ... to track the criminal down. An outbreak of ... Drug Administration (FDA) uses DNA evidence to track down the ... Sound far-fetched? It,s not. The FDA has increasingly used a ... of foodborne illnesses. Put as simply as possible, whole genome ...
(Date:6/23/2016)... , ... June 23, 2016 , ... ... YM (Yeast and Mold) microbial test has received AOAC Research Institute approval 061601. ... microbial tests introduced last year,” stated Bob Salter, Vice President of Regulatory and ...
(Date:6/23/2016)... , June, 23, 2016  The Biodesign Challenge ... envision new ways to harness living systems and biotechnology, ... Art (MoMA) in New York City ... 130 participating students, showcased projects at MoMA,s Celeste Bartos ... Paola Antonelli , MoMA,s senior curator of architecture and ...
Breaking Biology Technology: