Big Pharma forges ahead with risky ‘bubble boy’ treatments

Jolee Mohr was just 36 years old, raising a family and working full time.

And you can bet she never imagined a knee arthritis treatment would kill her.

It wasn’t some risky surgery or powerful painkiller that ended Jolee’s life.

She was a victim of another gene therapy treatment gone horribly wrong.

We’ve been told that drugs to alter or repair our genes will keep us young and conquer everything from hair loss to cancer. And gene therapy isn’t some science fiction or medicine of the future – it’s being offered experimentally in hospitals and clinics across America right now.

But with dozens of new medications in the pipeline… and billions of dollars at stake… there’s a side to gene therapy that drug companies are working hard to sweep under the rug.

And stories of cancer, organ failure, internal bleeding, and sudden death that they hoped you’d never hear.

A shot in the dark

GlaxoSmithKline is the first major drug company to bet big on gene therapy – and the entire industry is watching.

GSK is asking European regulators to approve a gene therapy shot to treat ADA-SCID, a rare immune system disorder often called “Bubble Boy” disease.

And while GSK floats this trial balloon to see if it can get a gene therapy medicine approved, dozens of drug companies like Bristol-Myers Squibb are waiting in the wings. They’re ready to flood America and Europe with applications for gene therapy drugs to treat everything from hemophilia to cancer.

This gene therapy gold rush is what GSK likes to call a “new era in medicine.”

After all, that’s how Big Pharma prefers to talk about gene therapy – like it has a future, but no past.

But when you look at gene therapy’s ugly history, you realize that it’s not the next great chapter in medical care.

It’s more like a sequel to Frankenstein.

Gene therapy uses viruses – often called vectors – to deliver corrected genes to our cells. Scientists can’t guarantee that the genes will ever arrive at the right destination or function correctly – and when they don’t, the consequences can be disastrous.

Researchers had previously tested gene therapy in children suffering from that same ADA-SCID immune disorder. Five of the kids developed leukemia after the inserted genes accidentally attached themselves to a gene that regulates cell division.

Even when the alien genes injected into your body don’t harm you, the virus – or vector – that delivers them can. That’s what happened to 18-year-old Jesse Gelsinger, who had a metabolic disorder called OTC that was well-maintained with drugs and a careful diet.

He was even described as “not being sick” before his death. But soon after his shot, Jesse’s organs basically shut down. It was what his doctors called at the time an “immune-system revolt.”

And then there’s the case of Jolee Mohr. She signed up for a clinical trial to see if gene therapy could help her chronic knee pain.

After just two injections, Jolee died from internal bleeding and kidney failure. Her husband says it’s clear the gene therapy industry is just “trying to make millions of dollars on people before they can truly understand what’s going on.”

The media and Big Pharma like to trumpet every successful use of gene therapy – but they’re a lot less talkative when things go badly, as in Jolee’s case.

You may remember a couple years back when stories popped up all over the world about an experiment at the University of Pennsylvania that claimed to use gene therapy to “cure” blindness. When patients began losing their sight again after a year, there was barely a whimper out of the press – or the drug companies.

And that’s the real risk of gene therapy – that its hype machine is offering false hope and even putting people directly in harm’s way.

Dr. James M. Wilson still talks glowingly about the “simplicity of the concept.”

“You just put the gene in,” he said.

He was the head of the Penn Institute for Human Gene Therapy where Jesse Gelsinger got his gene therapy injection.

The one that killed him.

Sources:

“GSK readies ‘bubble boy’ drug as big pharma bets on gene therapy” Ben Hirschler, April 27, 2015, Reuters, reuters.com

GlaxoSmithKline files for gene therapy OK as a case of the jitters sets in” John Carroll, May 5, 2015, FierceBiotech, fiercebiotech.com