bentlimerick t1_j0x1z9d wrote on December 20, 2022 at 1:27 AM

Those are amazing results. Those confidence intervals for complete wound healing are awesome.

It's a shame that clinical trial results aren't given in a standard report card style that always shows the complete response rates and confidence intervals.

Some of these "butterfly" patients might even be able to get a decent hug thanks to these treatments.

There's a line at the end of the paper "Longer and larger trials are warranted to determine the durability and side effects of B-VEC for this disease." that kind of misses the point.

Trials just don't last that long. Fifteen years is about as long as they come.

The number of pediatric patients and the fact that they need this to work for their whole life and the fact that this could dramatically increase their life expectancy really does highlight how we need a way to follow them for another 50 years or more.

It's crazy that we have modern medical technology like gene therapy but we're still doing clinical trial tracking as if the internet and databases weren't invented and everything is being kept in manilla folders.

Kids have social security numbers. Kids with this kind of condition often get submitted for disability through social security. All of these patients were registered to a clinical trial that is tracked by the NIH. It really shouldn't be that hard whether with an opt in or an opt out system to set things up so that trial arm information can be checked against disability information on an annual basis.

DerBassSpieler t1_j0xwjc7 wrote on December 20, 2022 at 5:42 AM

There are countries where such databases exist for citizens though, like in the scandinavian countries. Trials conducted there would have the long-term data that you're wishing for.

threeshadows t1_j0yx02y wrote on December 20, 2022 at 1:19 PM

https://clinicaltrials.gov/

The issue is that worldwide about 8000 new study results are published each day. So it’s hard to get people to all use the same system

anotherone121 t1_j0zwenj wrote on December 20, 2022 at 5:32 PM

>There's a line at the end of the paper "Longer and larger trials are warranted to determine the durability and side effects of B-VEC for this disease." that kind of misses the point.
>
>Trials just don't last that long. Fifteen years is about as long as they come.

I suspect far shorter is fine. The concern around durability likely has to do with the vector used and the turnover of the tissue, transfected. Is the transgene being inserted as an epizome (doesn't multiply during cell replication... so gets diluted out over time) or is it integrating into the chromosome (--> safety concerns)? If it integrates, into what cell types, with what efficiency, and in what tissue layer, and how quickly do these turnover? In short, the clinical effect may be temporary.... quite temporary, depending on the technical details.