Cystic Fibrosis and Stem Cells
For a child to be born with cystic fibrosis, both parents must be carriers of the faulty gene that causes it. When both parents carry the faulty gene, each child they have has a 25% chance of having the condition, a 25% chance of being healthy, and a 50% chance of being a carrier.[2]
There is no known cure for cystic fibrosis, and current treatments are symptomatic. Treatments can include antibiotics for frequent lung infections, physiotherapy to help clear the airways of mucus, and drugs to expand the airways. In some cases, a lung transplant may also be necessary.[3]
Cystic fibrosis is one of nine illnesses tested for on the newborn screening programme through the blood spot heel prick test.[5] Early diagnosis and treatment are critical to improve quality of life and life expectancy. A report in 2012 found that cystic fibrosis is progressively moving from being a childhood killer to an adult disease. Thanks to improvements in the care of patients, the average life expectancy for sufferers in the UK is now 43.5 years. [6]
Cystic Fibrosis Facts
- It is estimated that 46.8 million people have dementia worldwide [4]
- CF is most common in people of Northern European descent [3]
- CF is the most common inherited disease in white people [4]
- 9,000 people are living with CF in the UK[3]
- It is estimated that 1 in 2,500 babies are born with CF [3]
- The average life expectancy in the UK for CF sufferers is 43.5 years of age [6]
- There are currently 2 clinical trials investigating the application of stem cells for treatment [7]
Cystic Fibrosis and Stem Cells
While standard medical protocols focus on symptom management and life extension, early-stage regenerative research is exploring potential future therapies. Research at the University of Melbourne Faculty of Medicine aims to prove that cord blood could potentially be used to treat cystic fibrosis. The study suggests that umbilical cord blood may be used to restore lung function. While the research is in preliminary stages, the researchers hope to use sibling cord blood to treat the condition.[8] Because CF is a genetic condition, a child cannot use their own cord blood to correct it, making the preservation of a healthy sibling’s cord blood a potentially valuable resource for these emerging investigational treatments.
The Cystic Fibrosis Trust has also funded a project to correct two rare mutations of the cystic fibrosis gene. The project aims to use induced pluripotent stem cells made from white blood cells, gene repair, and editing technology to repair the two rare “stop codon” mutations.
Once the researchers have fixed the mutation, they will generate lung epithelial cells to confirm that normal function has been restored. It is hoped these cells could then be used to replace the cells in the lungs of patients with cystic fibrosis in the future. [9] While these cutting-edge laboratory techniques remain experimental and have not yet been approved as standard clinical cures, they do offer immense hope for a healthier future for sufferers.
References
- http://www.nhs.uk/conditions/Cystic-fibrosis/Pages/Introduction.aspx
- http://www.geneticalliance.org.uk/docs/translations/english/8-cf-t.pdf
- http://www.nhs.uk/conditions/Cystic-fibrosis/Pages/Introduction.aspx
- http://patient.info/doctor/cystic-fibrosis-pro
- http://www.nhs.uk/Conditions/pregnancy-and-baby/Pages/newborn-blood-spot-test.aspx
- http://cysticfibrosis.org.uk/news/registry-report-2012
- https://clinicaltrials.gov/ct2/results?term=cystic+fibrosis+stem+cells&Search=Search
- http://www.nzherald.co.nz/bay-of-plenty-times/news/article.cfm?c_id=1503343&objectid=11033787
- http://www.cysticfibrosis.org.uk/news/stem-cell-project
The information contained in this article is for information purposes only and is not intended to replace the advice of a medical expert. If you have any concerns about your health, we urge you to discuss them with your doctor.
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