
F.D.A. Approves Second Gene-Altering Treatment for Cancer - sethbannon
https://www.nytimes.com/2017/10/18/health/immunotherapy-cancer-kite.html
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jfarlow
And congratulations once again. This kind of therapy is a big deal because of
just how different it is from standard 'drugs'. This is a precise and
intentional genetic change to a patient's own immune cells to provide it a
_new_ capability. This new capability permits one's own immune system to
detect a cancer with precision, clearing it from the body without harming
other non-cancerous cells. The same protein doing the work here can be easily
tuned to become more and more precise, and the scaffold of the technique can
be ported to other targets relatively easily - again very much unlike most
small molecule drugs. In this way it is nothing like most any drug of the 20th
century. And there are many more to come.

There are >100 in the FDA pipeline right now for various types of cancer [1].
And the concepts used here to introduce DNA encoding proteins that confer NEW
functions can be used to introduce any number of new functions.

So in less than 2 months, the FDA has approved 3 gene therapies:

1) Cure for Leukemia (Novartis' CAR)

2) Cure for Blindness (Spark's hRpe65)

3) Cure for Lymphoma (Gilead's (nee Kite) CAR)

Here's our description of the designed protein in case you want to learn what
it does at a molecular level, or want to design your own:

[https://serotiny.bio/notes/proteins/car19/](https://serotiny.bio/notes/proteins/car19/)

[1] (paywalled CAR trails list):
[https://www.ncbi.nlm.nih.gov/pubmed/27613725](https://www.ncbi.nlm.nih.gov/pubmed/27613725)

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nonbel
This is the full text of the study?
[http://onlinelibrary.wiley.com/doi/10.1002/hon.2437_7/full](http://onlinelibrary.wiley.com/doi/10.1002/hon.2437_7/full)

Is that really it? Shouldn't they have to publish something substantive before
approval? It doesn't have to be in a journal, but somewhere on the FDA site,
etc.

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jfarlow
There are a LOT of papers out there about CARs [1], how they work, and what
their effects are. The DNA sequence used here has been effectively frozen for
quite some time as it runs through all sorts of examinations.

Further, your link is to the description of an Oral Presentation, not even a
paper. It's not supposed to have data associated with it.

This is the paper from the Phase I results of the same therapy (I very much
appreciate that the paper is open-access):

[http://www.cell.com/molecular-therapy-family/molecular-
thera...](http://www.cell.com/molecular-therapy-family/molecular-
therapy/fulltext/S1525-0016%2816%2945375-X)

[1]
[https://www.ncbi.nlm.nih.gov/pubmed/?term=chimeric+antigen+r...](https://www.ncbi.nlm.nih.gov/pubmed/?term=chimeric+antigen+receptor)

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nonbel
Thanks, here is an example of the type of questions I would expect a doctor to
ask (which is impossible if no study is published).

Looking at your paper I can't really tell what they did to determine CR. Did
they only do PET-CT? The paper they cite[1] mentions some biopsies (for some
cases), etc but they only show any results for that assay.

Assuming it was only PET-CT for now, they say[1]:

"Typically FDG-avid lymphoma: in patients with no pretreatment PET scan or
when the PET scan was positive before therapy, a post-treatment residual mass
of any size is permitted as long as it is PET negative."

So positive/negative is apparently based completely on relative metabolic
activity. Just looking at figure 1B in your paper, it appears that baseline vs
30 days is done perhaps using different exposures. Or for some reason the
intestines were much more active at 30 days (patients ate more recently?).

1) How do they ensure there isn't some response to the drug/environment that
has increased general metabolic activity, thus decreasing the relative signal
from the tumor?

2) Were the people processing and examining these scans blinded to the fact
they were part of a drug trial? There seems to be ample room to play with
exposures, etc here.

[1]
[http://ascopubs.org/doi/10.1200/JCO.2006.09.2403](http://ascopubs.org/doi/10.1200/JCO.2006.09.2403)

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lawlessone
375K, would insurance cover that?

~~~
RcouF1uZ4gsC
The cost is actually inline with what would be expected from a cancer cure.
The NHS uses 20000 - 30000 pounds/quality adjusted life year
[https://www.nice.org.uk/advice/lgb10/chapter/judging-the-
cos...](https://www.nice.org.uk/advice/lgb10/chapter/judging-the-cost-
effectiveness-of-public-health-activities) which would be about
$40,000/quality adjusted life year.

If this drug provides on average 10 quality years of life, then this would
fall under the guidelines as cost effective.

