One-Shot Gene Therapy Cure $665K

GlaxoSmithKline is offering a one-shot cure, called Strimveli, for a bone marrow disease called ADA-SCID, a sometimes fatal inability to fight infections. The cost: $665,000, making it the most expensive one-time treatment sold by any drug firm. (But it comes with a guarantee.)

The idea behind gene therapy is that a one-time correction to a patient’s DNA will lead to a lifelong cure. Strimvelis is the first treatment to be commercialized that lives up to the promise. But potential cures for hemophilia, a rare eye disease, and a fatal brain illness could reach the market next, and they could be similarly expensive.

“We do not expect to recover all of the costs of building a platform to deliver gene and cell therapy from Strimvelis alone,” says Anna Padula, a spokesperson for GSK’s rare-diseases group. “We hope that Strimvelis will be the first of a number of innovative gene-therapy medicines that we will bring to patients.” The company recognizes, she adds, “that the industry will need to adapt the way in which medicines are priced and funded.”

In reading about this, I was reminded of the cure offered to Case, the protagonist of William Gibson's 1984 novel Neuromancer. It's not a very close match, but the idea of a single treatment offering a complete cure is striking. Case had been given a mycotoxin that burned out subtle areas of his nervous system and his ability to jack into cyberspace was lost.

"What would you say if I told you we could correct the damage, Case?" Ermitage suddenly looked to Case as if he were carved from a block of metal; inert, enormously heavy...

"It'll work, Case. You got no idea, the kind of stuff Armitage has. Like he's gonna pay these nerve boys for fixing you with th program he's giving them to tell them how to do it...

Cold steel odor. Ice caressed his spine.

Then black fire found the branching tributaries of the nerves...

He woke... "My back hurts."

That's where they replaced your fluid... some new stuff patched into your liver. The nerve stuff, I dunno. Lot of injections. They didn't have to open anything up for the main show..."

Read about other gene therapies at Gene Therapy Could Restore Hearing To The Deaf and Gene Therapy Builds Muscles Fast. Via Technology Review.

Scroll down for more stories in the same category. (Story submitted 8/4/2016)

Follow this kind of news @Technovelgy.

| Email | RSS | Blog It | Stumble | del.icio.us | Digg | Reddit |

Would you like to contribute a story tip? It's easy:
Get the URL of the story, and the related sf author, and add it here.

Comment/Join discussion ( 0 )

Related News Stories - (" Medical ")

Sleeep PRO Earplug For Maximum Rest
'Merton... placed the electrodes of the sleep-inducer on his forehead.' - Arthur C. Clarke, 1963.

You'll Regrow That Limb, One Day
'... forcing the energy transfer which allowed him to regrow his lost fingers.' - Frank Herbert, 1972.

First 3D Printed Human Corneas From Stem Cells
Just what we need! Lots of spare parts.

Nanorobots Roam Your Bloodstream, Cleaning It
Too bad they won't have lasers, though...

 

Google
  Web TechNovelgy.com   

Technovelgy (that's tech-novel-gee!) is devoted to the creative science inventions and ideas of sf authors. Look for the Invention Category that interests you, the Glossary, the Invention Timeline, or see what's New.

 

 

 

 

 

Current News

SWEEPER Robot Peter Piper Picking Peppers
'... little machines, that went from plant to plant, apparently on caterpillar tracks, cutting off the ripe fruit.'

Oil from Algae - Can It Be Done?
'We dump everything that's waste into the tanks, pump the oil off the top.'

Moving Whole Planets, Revisited
There was a lot of work done on this idea over the years.

Disney Keeps Backups Of Star Wars Franchise Actors
'She is a personality-construct, a congeries of software agents...'

Farming In Space Starts With Mycorrhiza
'The inner leaves were beginning to curl faster than the outer leaves.'

Jaguar I-Pace Audible Vehicle Alert System For EVs
'Of course not a vehicle moved by means of internal explosions of a derivative of rock oil...'

Autonomous 'Fiberbots' Weave Large Structures
'It extrudes material like a spider.'

Birds Aren't Real - Wake Up, California! (With Bird Watching Guide)
'When he had first built them, they had been crude indeed, flying mechanisms with little more than a reflex-response unit.'

Self-Healing Material Pulls Carbon Out Of The Air
'... could seal the punctures.'

IRL Glasses Block Screens, Limit Vision To Real Life
'If you couldn't see the ads, how would you know what was fashionable?'

Testing The Single-Person Spacecraft
'...the lower part of the suit was simply a rigid cylinder.'

Shapeshifting Materials Transform By Light
'Its lines wavered, flowed, and then painfully reformed.'

Fully Automated Farm Iron Ox Hydroponics
'Had these machines in some incredible fashion been provided with brains?'

BrainNet Social Network Of Brains
'I used my implant to tell MILLIE what we wanted and she took care of it'

Phil Nuyttnn's City Under The Sea
'Under the lower roof there was no water, but a clear and luminous atmosphere...'

IONITY Opens First 10 Fast-Charging Stations
'Recharge the batteries... in almost every town and village...'

More SF in the News Stories

More Beyond Technovelgy science news stories

Home | Glossary | Invention Timeline | Category | New | Contact Us | FAQ | Advertise |
Technovelgy.com - where science meets fiction™

Copyright© Technovelgy LLC; all rights reserved.