The Million-Dollar Drug and Big Pharma's Only Concern - Profit

The million-dollar drug

How a Canadian medical breakthrough that was 30 years in the making became

the world’s most expensive drug — and then quickly disappeared

By Kelly Crowe, CBC

I.

It is one of this country's great scientific achievements.

The first drug ever approved that can fix a faulty gene.

It's called Glybera, and it can treat a painful and potentially deadly genetic disorder with a single dose — a genuine made-in-Canada medical breakthrough.

But most Canadians have never heard of it.

A team of researchers at the University of British Columbia spent decades developing the treatment for people born with a genetic mutation that causes lipoprotein lipase disorder (LPLD).

LPLD affects communities in the Saguenay region of northeastern Quebec at a higher rate than anywhere else in the world.

Montreal psychologist Cynthia Turcotte, 42, was born with LPLD, but wasn't diagnosed until it almost killed her when she was eight months old.

As a result of the gene mutation, her body is missing an essential protein that processes dietary fat. Her blood becomes thick and white with fat particles that can destroy her pancreas.

All her life, Turcotte has had to follow a strict diet. She can't eat cheese or chocolate or any food that contains fat. And she can't drink even a drop of alcohol.

All of that made it difficult to have a normal social life when she was younger.

But the worst part was learning that she could never have children. Women with the disease are warned to avoid pregnancy because there is a high risk of miscarriage.

Cynthia Turcotte received Glybera as part of a clinical trial in Chicoutimi, Que.

She says it changed her life. (Craig Chivers/CBC)

Despite accepting all of that and following a very strict diet, Turcotte experienced the most dangerous symptom of LPLD a decade ago — an agonizing pancreatitis attack.

"It's like digesting yourself," she said of the pain.

"Pancreatitis is like acid going through your belly and your abdomen. At that time, I was screaming. I was hospitalized for 10 days."

Frightened for her future, Turcotte learned about a clinical trial in Chicoutimi, Que., where doctors were testing an experimental treatment for LPLD. She immediately volunteered.

A series of injections in her leg muscles during a single visit changed Turcotte's life, ending the pancreatitis attacks and giving her the chance to start the family she thought she’d never have.

"It was a turning point in my life," said Turcotte, who is the proud mother of two young children — her "two miracles," she calls them.

But Turcotte would be one of only 31 people with LPLD to ever experience the benefits of Glybera.

The drug works. It is safe. But it's no longer available anywhere in the world.

People with lipoprotein lipase disorder (LPLD) are missing a protein that processes dietary fat.

This can cause their blood to turn white. (Craig Chivers/CBC)

Glybera was never sold in North America and was available in Europe for just two years, beginning in 2015. During that time, only one patient received the drug. Then it was abandoned by the company that held its European licensing rights.

The problem was the price.

The world's first gene therapy, a remarkable discovery by a dedicated team of scientists who came together in a Vancouver lab, had earned a second, more dubious distinction:

The world's most expensive drug.

The rest of this fascinating and frustrating story can be read here.

There is a certain amount of insanity in the fact that this brilliant research cannot be made useful to those suffering from LPLD. If those who own the license cannot move the product, they should lose that license and non-licensed pharmaceutical companies should be allowed to produce it and sell it at a reasonable price. 

It seems that so many Big Pharma companies are predatorial by nature and have this need to make enormous profits and make them immediately. If this is how Free-Enterprise is supposed to work, then there is something seriously wrong with the system. Big Pharma - the companies that make the drugs for sick people - have no attitude of responsibility toward sick people!