
A Tale of Do-It-Yourself Gene Therapy - Turing_Machine
http://www.technologyreview.com/news/542371/a-tale-of-do-it-yourself-gene-therapy/
======
Turing_Machine
Regardless of whether this particular instance turns out to be legit or fake,
I think we're at the beginning of the "garage startup" era for this stuff,
with all that implies.

------
reasonattlm
Disruption at work. The cost of gene therapy is plummeting, and the existing
establishment wants you to do things the slow expensive way when it is
becoming cheap and fast. When people try the cheap and fast way, much pearl-
clutching results.

Here is a Reddit AMA with Liz Parrish that provides details on the gene
therapies used:

[https://www.reddit.com/r/Futurology/comments/3ocsbi/ama_my_n...](https://www.reddit.com/r/Futurology/comments/3ocsbi/ama_my_name_is_liz_parrish_ceo_of_bioviva_the/)

Some of my earlier comments on the BioViva announcement:

\----

BioViva is one of the small groups interested in bringing telomerase therapies
to humans sooner rather than later. It seems they have started in on their
small long-term trial of human gene therapy for telomerase activation, and
have treated the first volunteer.

I should say that at any given time there is a fairly large gap between what
can be done in human medicine, the technology that actually exists and works,
and what is being done in trials. Most of this gap is due to regulation, and
the rest of it because development groups want to have a reasonable certain
that what they are doing actually works, does more good than harm, and so
forth. The regulatory process might last a decade, while the actually useful
part of that testing (does it basically work, and is the risk profile
sufficiently defined and acceptable to patients) is only a few years. As the
cost of research and development in the life sciences falls, it will become
increasingly untenable that a huge ball and chain slows progress thanks to
regulatory risk aversion, and a growing number of initiatives will forge ahead
and build anyway. Some years ago I proposed the Vegas Group fable on the
growth of DIYbio into action based on the falling cost of that action,
something that I think will happen in the fullness of time: alternative roads
that bypass official regulation in favor of faster progress, an inevitability
in an environment of low-cost research. Also, I think, a necessity.

What about the science here? I've never been a big fan of telomere lengthening
approaches, as average telomere length as it is measured today in immune cells
looks very much like a marker of the progress of aging, an end stage
consequence far removed from root causes. Telomeres shorten with cell division
and new long-telomere cells are delivered into tissues by stem cell
populations. Thus average telomere length in immune cells reflects some
combination of immune health and stem cell activity, both of which are known
to decline with age. You can't argue with the fact that telomerase gene
therapy has been shown to extend life in mice, however, though you can
certainly note that the size of the effect has been getting smaller as the
research groups have refined their data and approaches.

How does this work to slow aging in mice? At this point I lump enhanced
telomerase activity into the general category of approaches that either
probably work or intend to work by boosting the activation of old stem cell
populations, resulting in increased repair and tissue maintenance and thus a
slower decline into frailty and organ failure. More telomerase doesn't seem to
raise cancer risk in mice, but mice have very different telomere dynamics and
cancer risk profiles than we humans. The fastest way to figure out what is
going to happen in humans is of course to try it, and kudos to anyone
volunteering at this stage, but I'd be waiting for a few more years of testing
first in animal or tissue models closer to human telomere dynamics. In part
that decision would be driven by the fact that I don't think that this is the
best approach to move ahead with practical applications, to push ahead and get
things done. I absolutely agree that pushing ahead to get things done needs to
happen, but I'd rather see this sort of boldness for SENS treatments like
senescent cell clearance.

