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 ")

Skin Electronics Can Show Electrocardiogram
'... the young men in the streets who applied polyimde OLED body film to their bared shoulders.' - Chen Qiufan, 2019.

Neurodevices For Consumers? Neuroethicists (And Philip K Dick) Say 'Caveat Emptor'
'They tried to use it today and it wouldn't work. No colors and no ceph patterns, neither one...' - Philip K. Dick, 1977.

BrainEx Restores Some Activity To Severed Pig Head
'... they placed the brain in a special solution, having all the properties of Nursing the brain cells.' - Edmond Hamilton, 1929.

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.

 

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

China Deploys Robot Traffic Police
'The robot came up smooth and fast as a rocket...'

Better Than Dune Chromoplastic? This Guy Might Have Done It
'But when Old Father Sun departs, the chromoplastic reverts to transparency in the dark.'

Gather, An AI Warehouse Inventory Drone Startup
'It extended three of its tiny arms sideways to lock onto the registration pins...'

China's Artificial Intelligence-Enhanced Education
'The grey gas not only cut off his vision, but also his other senses...'

Orbital Manufacturer 'Made in Space' Gets $73 Million NASA Contract
'Mass-produced in the orbiting factories...'

Soli Gesture Tech Will Be In Google Pixel 4
'I enjoy watching this way, but - He waved his hand and the circuit switched abruptly.'

Uber Eats Pairs Cars With Drones
Fresh grub? Let's hope they aren't delivering grubs.

Space-Based Solar Power Roundup
SF writers popularized and elaborated on this idea a generation before the first patents were filed.

Lost Language Meanings Found By Machine Learning
'The autopilot would need data before it could begin a translation...'

'Aerogel' Sheets For Martian Gardens
'Sealed to the ground along all the sides, Honey, he growled...'

France's 'Red Team' Of Science Fiction Authors
'They're the only experts we have.'

Dim The Sun With Stratospheric Controlled Perturbation Experiment
'Those twin volcanoes; d'ye see them, Mr. Renner?'

Mashambas Skyscraper Farm Design Wins
'...a towering eighty-story structure like the office In-and Out baskets stacked up to the sky.'

Self-Driving Tractors From China Plan Ahead
'Machines that seemingly with full consciousness walked out into the fields to do their daily work.'

Jet-Powered Hoverboard Works!
L'audace, l'audace, toujours l'audace!

Nobe 3-Wheel Electric Vehicle Parking Like I, Robot
Spidercar, Spidercar, does whatever a spidercar does.

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.