Stem cells for CF

Something we don't hear enough about in the CF community, notably from the CFF is stem cell therapy.

This abstract points out that only a tiny amount we only need to get a tiny amount 6-10 % of CF protein to express as normal (wild-type) in order to restore chloride transportation.  This is huge.  Here's hoping that the CFF will spend some of their newly acquired $3.3 billion to helping the stem cell effort.  I used to work in biotech and know how long virus-enable gene therapies take to implement.  For people with severe lung damage, regeneration of the damaged tissue may be the only answer.

And to say that CF patients with advanced lung disease fail to have a "satisfactory quality of life" is an understatement of vast proportions.  I love it when the CFF web site talks about "keeping you healthy."  Ahem, if the CFF were keeping us healthy, we wouldn't be so sick!

Also - to all the religious nuts out there - this does not involve embryonic stem cells, so you can keep on hitting each other on the head with your bibles and keep getting stupider.

Adv Exp Med Biol. 2014 Sep 24. [Epub ahead of print]

The Potential of Wharton's Jelly Derived Mesenchymal Stem Cells in Treating Patients with Cystic Fibrosis.

Boruczkowski D1, Gładysz D, Demkow U, Pawelec K.

Author information

Abstract

Cystic fibrosis (CF) is a life-threatening autosomal recessive multi-organ disorder with the mean incidence of 0.737 per 10,000 people worldwide. Despite many advances in therapy, patients fail to have a satisfactory quality of life. The end-stage lung disease still accounts for significant mortality and puts patients in the need of lung transplantation. Even though the disease is monogenic, the trials of topical gene transfer into airway epithelial cells have so far been disappointing. It is proven that stem cells can be differentiated into type II alveolar epithelial cells. Wharton's jelly-derived mesenchymal stem cells (MSC) from non-CF carrier third-party donors could be an effective alternative to bone marrow or embryonic stem cells. The harvesting process is an easy and ethically uncontroversial procedure. The MSC cell should be applied through repetitive infusions due to rapid lung epithelial cell turnover. However, the low stemcell incorporation remains a problem. Pre-clinical studies imply that even 6-10 % of the wild-type cystic fibrosis transmembrane conductance regulator (CFTR) expression could be enough to restore chloride secretion. The route of administration, the optimal dose, as well as the intervals between infusions have yet to be determined. This review discusses the clinical potential of mesenchymal stemcell in CF patients.

PMID:

 

25248343

 

[PubMed - as supplied by publisher]

http://www.ncbi.nlm.nih.gov/pubmed/25248343