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.

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