Friday, March 17, 2023

CRISPR Therapy for Sickle-Cell Disease: Progress Report

The first patients with the dreaded sickle-cell gene were treated with gene editing therapy back in 2019. After three years, all 31 seem to be doing ok. (Articles on the therapy: here, here)

So what now? The treatment is very complex. It involves removing bone marrow from the patient, editing the DNA of the hemocytoblast cells that produce red blood cells, and putting billions of those cells back into the body. No information on the cost has yet been made public but it is surely in the millions of dollars. There are about 100,000 people in the US who have the condition, most of them black, many poor. That's a big burden for Medicaid and the rest of the insurance system.

And globally, most of the 20 million people with the condition live in Sub Saharan Africa. What happens when they start migrating to the US and Europe and applying for asylum on the basis that without this treatment they will die?

Other people question the treatment because nobody knows how long it will last. Hemocytoblasts don't live forever, and presumably the new ones the body makes will have the sickle cell gene. Can we afford to give people this treatment every decade?

CRISPR technology is advancing rapidly, so presumably the cost of the gene editing and cell cloning steps will come down. But bone marrow surgery is a mature technology and it is likely to remain very expensive. I suppose in the long run the solution will be to use gene editing to remove the faulty gene from the population, but then again I suppose that will have to wait on an effective vaccine for malaria. 

This is going to create a lot of ugly fights in years to come.

A thought comes to mind: the reason we don't have flying cars, as people like to ask, is that we are spending our resources helping people live longer, healthier lives.

3 comments:

Anonymous said...

Personally I don't want to live longer if we don't have flying cars

G. Verloren said...

We already -have- flying cars. They're called personal aircraft.

They're only good for luxury recreation rather than everyday transportation and they're the most dangerous way to fly by a LOT, but they absolutely exist.

If you want to insist on the popular imagination version that somehow can hover and float around, then you're going to have a lot trouble with convincing elemental physics to get on board with your plan. And even then, all you'll be doing is creating a variation on personal aircraft that is somehow even more dangerous.

You are never realistically going to fly from your driveway to the grocery store, because it's absurdly hard to do technically, because it'd actually be extremely wasteful even, and because the average person is a moron who can barely handle driving a simple car without crashing it.

The number of total aircraft in America is currently a bit over 200,000.
The number of private planes in America is currently a bit over 15,000.
The number of cars in America is approaching 300,000,000.

If "flying cars" somehow became an everyday reality supplanting ordinary cars, it'd be a catastrophe - it's already hard enough to manage aviation with the planes we have, it would become effectively impossible with FIFTEEN THOUSAND times as many aircraft in the skies.

More likely, if they could be made to actually work, they'd just become the new 'personal aircraft', and remain a niche luxury item owned and used by only 0.0005% of the population.

G. Verloren said...

Typo, second to last paragraph - should read "Fifteen Hundred".