25
Mar2015

By Kathy Banks, BSc, MSc, continuing review coordinator, animal ethics, University of British Columbia 

PRIM&R is pleased to share a post from Kathy Banks, a member of the PRIM&R Blog Squad for the 2015 Institutional Animal Care and Use Committee (IACUC) Conference. The PRIM&R Blog Squad is composed of PRIM&R members who blog here, on Ampersand, to give our readers an inside peek of what happened at the conference in Boston, MA.

Gene therapy! We can cure ALL the diseases! More than 20 years ago, this was (and still is, honestly) a fanciful notion, but targeted therapeutics was one of the newest advances in genetics. At the time, it was a field of study that found me (instead of me finding it), and I was smitten. The first gene therapy treatment was done in 1990, and it was HUGE news. When I started graduate school in 1996 (at University of British Columbia, in the Department of Medical Genetics), I was immediately plunged into this "new field" of research. One of my graduate classes was about gene mutations that result in diseases with classic genetic inheritance patterns, including cystic fibrosis (CF) caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
When I read that David K. Meyerholz, DVM, PhD, was one of the keynote speakers at the 2015 IACUC Conference, I knew his talk was something I could not miss. He studies lung diseases (asthma runs in my family), including CF. CF is a disease which has become much more personal for me as the daughter of a friend has it. With two scientists as parents, they know her prognosis, but they also know how science can change things. Dr. Meyerholz's keynote and the preceding interview with Avery Avrakotos, PRIM&R's education and policy manager, solidified my decision to attend.
Dr. Meyerholz showed us data from his research on CF and his insights from these studies. One of the main things he and his team have studied is a "chicken or the egg" question: what comes first in CF, inflammation or infection? This was a key insight that had been studied by many for a long time, but there were no means to answer the question as the existing genetic models were insufficient. By creating a workable and human-like equivalent model to study CF as it develops, they have shown that newborn CF lungs lack inflammatory cells, but are already prone to infection by bacteria. They found that CF neonates are born with airway defects that can enhance contamination of the lungs.They identified abnormal mucus in CF airways that gets "stuck", preventing cilia from sweeping the mucus and debris out of the lungs. Furthermore, they have shown that multiple "hits" to the defenses of the CF lung make the lung prone to infection to propagate the "cycle of destruction" in CF lungs. These breakthroughs came about because the science evolved, which created the possibility of the model, which in turn allowed the studies to take place.
In 1980, the life expectancy for a CF patient was 14. Thirty-five years later it has almost tripled to 37 years. Who knows where we will be in another ten or fifteen years? Based on the advances being made in understanding the disease and on my scientific background, my expectations are that the life expectancy will be extended even longer. So, maybe the thought that with gene therapy, we CAN cure all the diseases isn't so fanciful after all?

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