The Million Dollar Drug!
Canada is a pretty cool country, we have maple syrup, beavertails, we’re amazing at hockey, and we dominate in the Winter Olympics. What you might not know, though, is that researchers at the University of British Columbia invented Glybera. What is that? It’s one of Canada’s, if not the world’s, greatest scientific achievements of late. Glybera is a drug that has been approved to fix a faulty gene with a single dose. This faulty gene is the cause of lipoprotein lipase disorder (LPLD) - a potentially fatal genetic disorder, which causes the body not to produce an important protein which processes dietary fat.
People with this disorder must follow an extremely strict diet including cutting out any food which contains fat or any alcohol. It can also increase the risk of miscarriage for women who have it by a significant amount. The disorder is more common in the Saguenay region of northeastern Quebec than anywhere else in the world. Glybera does work, and it is safe- it was tested in a clinical trial, but only 31 patients have experienced its benefits, and it is no longer available anywhere in the world. Why? The exorbitant price tag.
Glybera essentially works by using a harmless virus designed to carry a human gene. The virus then inserts itself into a human cell and makes copies of the new gene, which is capable of producing the missing protein. However, the incredible price of Glybera was its downfall. When it went on sale in Europe for the first time in 2015, it carried a price tag of ~ 1 million US dollars - a price based on a business calculation due to the rarity of LPLD. In April 2017, the company that sold Glybera abandoned it. Unfortunately, Glybera was defeated by the reality of the drug industry - it simply was not profitable enough.
Source: https://newsinteractives.cbc.ca/longform/glybera