Live data from Hacker News

Macular degeneration: 'I've been given my sight back'

bbc.com

1–10 of 106 posts

Re: Macular degeneration: 'I've been given my sight back'

#4
post #2

> Cells from a human embryo Are they sure? They can now harvest stem cells from regular tissue and blood, as I understand it

Yea but they probably need to be young versions of your stem cells.

iPS stem cells from old cells will still be fraught with DNA damage and can be cancerous.

Re: Macular degeneration: 'I've been given my sight back'

#6

“He was one of two patients given pioneering stem" I find the tiny test groups in medical ‘research’ so strange.

It's a test to see if they should bother moving forward. You don't want to gear up a big study just to find out that the treatment gives everyone cancer (which is a concern mentioned in the article) or that it doesn't work at all. You do a pilot study to see if there is potential before you waste a lot of work and maybe hurt people.

Anyway if the effect size is big enough, you don't need a really large study to get statistical significance. I mean, what are the odds of macular degeneration reversing by chance?

Re: Macular degeneration: 'I've been given my sight back'

#7

“He was one of two patients given pioneering stem" I find the tiny test groups in medical ‘research’ so strange.

unfortunately that is all we have. There is nobody on earth with your exact genetic makeup (even identical twins, though then the random mutation is small enough to ignore: consider it an exception). As a result nothing exactly will behave correctly for you. A drug that causes death in 99% of those it is tried on might be the one you need, but because it is so deadly trials will ensure it never makes it to market.

Re: Macular degeneration: 'I've been given my sight back'

#8
post #2

> Cells from a human embryo Are they sure? They can now harvest stem cells from regular tissue and blood, as I understand it

It's the BBC. They make mistakes, but it is entirely reasonable to start from the assumption that they have not made such a big mistake.

Anyway, here's a publication about the study that makes it pretty clear that they got it right (abstract only, paywall):

https://www.nature.com/articles/nbt.4114

(I found the study by assuming a page like http://www.thelondonproject.org/ would exist and then following the link provided)

Re: Macular degeneration: 'I've been given my sight back'

#9
post #2

> Cells from a human embryo Are they sure? They can now harvest stem cells from regular tissue and blood, as I understand it

> Are they sure? They can now harvest stem cells from regular tissue and blood, as I understand it

There are adult stem cells which are differentiated or partly differentiated to particular systems, eyes, skin, blood etc, and there are induced pluripotent stem cells, where chemical or viral factors have been used to 'wind back the clock' for adult, differentiated cells back to completely non-differentiated (pluripotent) stem cells, equivalent to embryonic stem cells.

In the long run, it seems that embryonic stem cells won't be needed, but for the moment induced pluripotent cells are not 100% understood, everywhere apart from Japan clinical researchers are wary of using them in human trials, because they are not completely certain a particular preparation doesn't have abnormalities. Embryonic stem cells for the moment are the gold standard, and some work is needed to iron out the technology to standardize and check the process for inducing pluripotency.

But in principle any cure proven with embryonic stem cells could be 'ported' over to induced pluripotent stem cells once the iPSC process is standardized, it's just a question of whether you want to pursue these two research paths in parallel, or wait for the iPSC problems to be ironed out before even starting the process of running human trials.

Post reply on HN