Facebook Pixel Code
About Sickkids
About SickKids

August 26, 2012

SickKids first to grow lung cells using stem cell technology

Novel research may advance individualized medicine for cystic fibrosis patients

New stem cell research paves the way towards individualized medicine for patients with cystic fibrosis and other lung diseases. The study, led by The Hospital for Sick Children (SickKids), is the first to successfully use stem cells to produce mature lung cells that could potentially be used to study the disease and test drugs. The study is published in the August 26 advance online edition of Nature Biotechnology.

Researchers were able to induce human embryonic stem cells to become mature lung cells, that contained a gene, called CFTR that when mutated is responsible for cystic fibrosis (CFTR gene was discovered at SickKids in 1989). They then took the experiment a step further, by using induced pluripotent stem cells derived from the skin of patients with cystic fibrosis. They prompted these stem cells to become lung cells, which contain mutations specific to the patients involved. (Induced pluripotent stem cells are adult cells genetically induced to function like embryonic stem cells.)

Once researchers found that they could create lung cells derived from individual patients they then used a compound that resembles an investigational drug that is currently being tested for cystic fibrosis to see if it would rescue the CFTR gene mutation.

This study shows the major impact stem cell research can have on the field of individualized medicine,” says Dr. Janet Rossant, Principal Investigator of the study and Chief of Research at SickKids. “It is a promising move toward targeted therapy for patients with cystic fibrosis.”

According to Rossant, if we can generate lung cells derived from a particular patient, then we can test to see if a specific drug will work in that individual patient’s cells. If the drug is effective in vitro, then the next step would be to see if it works on the patient.

Prior to this year, the only therapies available for patients with cystic fibrosis have targeted the symptoms (like infection and digestive disorders) rather than the CFTR gene mutation. “More recently there has been a paradigm shift and now drugs are being developed to target the mutant CFTR specifically,” says Christine Bear, a co-investigator of the study, Co-Director of the SickKids CF Centre and Senior Scientist in Molecular Structure & Function at SickKids. “However, every patient is unique, so one drug isn’t necessarily going to work on all patients with the same disease,” says Bear. “Take cancer as an example, each individual responds differently to each treatment. For some, a certain drug works, and for others it doesn’t. This tells us that we need to be prepared to find the best option for that individual patient.”

In this particular study, the compound used did not work in all of the derived cell lines and according to Bear, who is also Professor in Physiology at the University of Toronto, this finding further emphasizes the need for individualized medicine.

Researchers say the next step is to perfect the method of generating epithelial lung cells, so that the process is more efficient and can be used to investigate other genetic diseases.

This research was funded by an Emerging Team grant from the Canadian Institutes of Health Research, an Ontario Ministry of Economic Development and Innovation (MEDI) grant and SickKids Foundation.

Dr. Janet Rossant is also University Professor in the Departments of Molecular Genetics, Obstetrics and Gynaecology at the University of Toronto.

About The Hospital for Sick Children
The Hospital for Sick Children (SickKids) is recognized as one of the world’s foremost paediatric health-care institutions and is Canada’s leading centre dedicated to advancing children’s health through the integration of patient care, research and education. Founded in 1875 and affiliated with the University of Toronto, SickKids is one of Canada’s most research-intensive hospitals and has generated discoveries that have helped children globally.  Its mission is to provide the best in complex and specialized family-centred care; pioneer scientific and clinical advancements; share expertise; foster an academic environment that nurtures health-care professionals; and champion an accessible, comprehensive and sustainable child health system.  SickKids is proud of its vision of Healthier Children. A Better World.™ For more information, please visit www.sickkids.ca. 

About Peter Gilgan Centre for Research and Learning 
The Peter Gilgan Centre for Research and Learning will bring together researchers from different scientific disciplines and a variety of clinical perspectives, to accelerate discoveries, new knowledge and their application to child health — a different concept from traditional research building designs. The facility will physically connect SickKids science, discovery and learning activities to its clinical operations. Designed by award-winning architects Diamond Schmitt Architects Inc. and HDR Inc. with a goal to achieve LEED® Gold Certification for sustainable design, the Gilgan Centre will create an architectural landmark as the eastern gateway to Toronto’s Discovery District. The Peter Gilgan Centre for Research and Learning is funded by a grant from the Canada Foundation for Innovation, the Government of Ontario, philanthropist Peter Gilgan and community support for the ongoing fundraising campaign. For more information, please visit www.sickkidsfoundation.com/bepartofit.

For more information, please contact:

Matet Nebres
The Hospital for Sick Children
Phone: 416-813-6380
email: matet.nebres@sickkids.ca

Caitlin McNamee-Lamb
The Hospital for Sick Children
Phone: 416-813-7654 ext. 1436
email: caitlin.mcnamee-lamb@sickkids.ca