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 ")

Purdue Pharma Ready To Profit From OxyContin Use Or Addiction Recovery
'It may be organic damage. It may be permanent. Time'll tell, and only after you are off Substance D for a long while.' - Philip K. Dick, 1977.

Organaut! Russians 3D Print Living Tissue In Space
'For a while your colonists will have to come up [to orbit] to the Hospital...' - Larry Niven, 1968.

MIT Scientists Create 'Peek-a-Boo Prober' From Jetsons
Well, George, it's the latest thing.

Wound Healing With Wearable Nanogenerators
'... forcing the energy transfer which allowed him to ... erase the other internal-external damage.' - Frank Herbert, 1972.

 

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

North Focals Smart Glasses Provide Augmented Reality In Style
'The world ... is drenched in unfamiliar information all the way to the horizon.'

Tesla Driver Caught Napping Behind The Wheel
'Mary Risling settled back for a little nap...'

Hayabusa 2 To Begin Asteroid Mining
'We must dig down, and then doubtless we shall find the metal.'

Ionocraft Drone Powered By Electrohydrodynamic Thrust
'He saw one hiss by him as he rounded the corner, trailing a short whip antenna...'

Purdue Pharma Ready To Profit From OxyContin Use Or Addiction Recovery
'It may be organic damage. It may be permanent. Time'll tell, and only after you are off Substance D for a long while.'

BloxVox Mutes Cellphone Convos
It's the polite thing to do, and has been the polite thing to do for about four generations.

Superfast Replicator: Volumetric Additive Manufacturing
I can't wait. Bring it on.

DNA May Contain Malware
'You were told to embed the logical pathogen.'

I Can't Resist Worm Robots
'Seen close it was not completely flexible...'

Rplate Digital License Plates Now Legal In Michigan
'Gragg's digital ink license plates ...'

Can Musk Starship Astronauts Use Magnetic Boots?
'Walking awkwardly in the magnetic boots that held him to the black mass of meteoric iron...'

Giant Dolphin Spotted On Jupiter!
'Now at last he could appreciate its real size and complexity...'

Musk's Starship An SF Fan's Dream Come True
Perfect for testing, perfect for fans!

TinyMobileRobots Are Sewer Sentinels
Every movie monster gets its start someplace.

Fishy Facial Recognition Now Possible
'Palenkis can identify random line patterns better than any other species in the universe.'

Spicy Tomatoes Created With Genetic Engineering
How about mashed potatoes and brown gravy?

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.