
New leukemia treatment "exceeded our wildest expectations" - xd
http://www.msnbc.msn.com/id/44090512/
======
iqster
From the article:

"Both the National Cancer Institute and several pharmaceutical companies
declined to pay for the research. Neither applicants nor funders discuss the
reasons an application is turned down. But good guesses are the general
shortage of funds and the concept tried in this experiment was too novel and,
thus, too risky for consideration."

The keywords are "experiment was too novel, and thus, too risky for
consideration". Unfortunately, this seems to have become the rule rather than
the exception in scientific research. All the young folks (pre-tenure) are on
a treadmill to get funding and publish _something_ in the top
journals/conferences. I wish there was some accountability in the system ...
the geniuses who rejected funding this research will go about completely
unaffected.

~~~
mortenjorck
The article doesn't touch on one aspect of the research likely to have been
particularly unpalatable to pharmaceutical companies: Potential cures are bad
business.

Of course, there is no conspiracy here. It's just the human tendency to
rationalize when money is involved. Imagine you run a pharmaceutical company:
Your products save lives, right? You want to be able to continue saving lives,
so you have to stay profitable. And to stay profitable, you have to make good
investments. Products that are used in long-term treatments are good
investments. One-time cures aren't.

~~~
JunkDNA
I spent 8 years in pharmaceutical industry R&D and this is so ill-informed I
feel compelled to refute it every time someone says it. I know it sounds like
it makes sense on the surface, but it's just outright false.

I worked in cancer research with some really, really smart people. Every
single one of them was sincerely hopeful that the drug they were working on
would be a total cure. I know it's hard to believe, but pharma companies are
composed of real people who have seen patients, friends, and relatives die of
cancer or similar diseases. Researchers want so badly to find a cure for the
disease they are working on. The existence of vaccines are proof that when
pharma companies can cure something, they do it. I have been in conversations
where business factors for a drug were discussed and I can tell you the one
thing that was never said was, "Hey guys, make sure it's not an actual cure,
or we'll need to kill the project".

~~~
mortenjorck
Firstly: You're right; I am ill-informed on this. I haven't any doubt that you
or your colleagues had and continue to have strong humanitarian convictions in
your work, and I was out of line to speak so broadly.

I recognize that the argument I made earlier is something of an old saw. But
you and I both know that businesses on the scale of most pharma companies are
not run only by researchers and engineers, and that the explicit reasons for
business decisions anywhere often stem from any number of unspoken ones. I
don't imagine even the coldest of bean counters ever says "we need to make
sure this research doesn't lead to a cure," and I doubt such a black-and-white
assessment of a complex R&D process even occurs to them. But incentives are
incentives, and humans are masters of rationalization.

I'm heartened to learn that your teams never felt pressure to put business
interests ahead of patient interests. I sincerely hope your experience is
common.

~~~
JunkDNA
Thanks for your comments. The pharma industry has a lousy public perception,
some of which is quite justified, so it can be hard to separate the reality
from the perception.

There is also a practical reason why I can assure you that people are usually
swinging for the fences, especially in cancer: most of the time you don't even
know if a drug will work, let alone work well. There is no way to predict
(with the obvious exception of vaccines which owing to biology are destined to
be cures from the beginning) what your drug will _really_ do until it hits the
clinic. So there isn't even a mechanism to offer people an incentive to
develop a drug someone has to take over and over vs. a cure, especially in
cancer. Certainly, there are chronic conditions like diabetes where there is
no real "cure" on the horizon because of the biology of the situation. But in
the case of cancer, you almost have to try for a total success because you
know you're likely to get far, far less than that when you hit the clinic.
Because of this problem, one of the bigger issues in pharma is that flawed
projects are allowed to linger long after they should have just been scrapped
because powerful scientists _believed_ they would work and convinced
management to keep funding them despite some of their issues.

------
testcock1
One thing I was left wondering about and maybe someone here can shed some more
light on this, what happens to the supposedly enormous amount of "serial
killer" white blood cells after they're done with the cancer cells? Do they
function normally, until a new "infection" comes, basically serving as a
permanent leukemia antidote?

(I hope no one is offended by my quite flimsy grasp of medicine and the human
body)

~~~
camtarn
According to this article: [http://www.geek.com/articles/geek-pick/hiv-virus-
used-to-tur...](http://www.geek.com/articles/geek-pick/hiv-virus-used-to-turn-
white-blood-cells-into-cancer-serial-killers-20110811/)

"More good news stems from the fact that the modified cells remain in the body
and have been seen to reactivate and kill new cancer cells as long as 12
months after they were first injected."

Incredible stuff :)

~~~
orblivion
It almost sounds like a cancer vaccine.

------
ComputerGuru
Original New England Journal of Medicine paper on this subject:
<http://www.nejm.org/doi/full/10.1056/NEJMoa1103849>

------
curt
Came up with an idea (edit: wrote in generalization, took to much credit,
sorry, didn't want to bore everyone with details) 10 years ago, everybody
thought I was crazy for wanting to inject HIV into people. The idea I had was
to treat the human immune system like a computers anti-virus software and
upload treatments using the HIV virus. Didn't think the FDA would even allow
something like this for at least another decade. Really believe HIV is the key
to ending disease in humans, even possibly aging.

Wonder how they handled its affinity for mutation? That was the big hurtle.

EDIT: xd corrected me. They did it in vitro. Which explains how they got
around the FDA.

~~~
burgerbrain
<https://secure.wikimedia.org/wikipedia/en/wiki/Viral_vector>

This idea has been around a hell of a lot longer than 10 years. Looks like you
were late to the party. There also isn't a particular need to use HIV to do
this in the general case either, the purpose of virii in general is to inject
DNA into existing organisms.

~~~
curt
Completely right. Didn't want to go into detail meant the anti-virus theory
and why. I'll try to explain. You want to use HIV because of a few unique
properties specifically the fact that you can split the gene material used in
replication from the infection machinery. In addition it actual has most of
the genes in place to do all the work you need since it's so well mapped and
studied modifying the structure becomes much easier. My idea was to limit the
insertion to only the original material (like a seedless watermelon) to remove
the danger. And have virus upload the sequence needed to produce the
antibodies or receptors required to target the foreign object. Then have the
body kill it itself.

------
justinph
As someone with a genetic disorder, this type of gene therapy is really
amazing to read about.

~~~
ChuckMcM
What is masked by some of the hoopla around cancer is exactly this, gene
therapy is advancing, and it can rewrite your DNA in-situ. This meets pretty
much every definition of the term 'game changing' from atheletes who want the
Sherpa gene for making more red blood cells to a host of genetic disorders
that lead to a myriad of complications in the patients life. Some resarch has
been marred by some very serious negative outcomes [1] but if it can be
mastered, the world will not be the same, for any of us.

[1] <http://www.medpagetoday.com/Genetics/GeneralGenetics/6275>

~~~
cal5k
Here's what I don't get about your hypothesis... very rarely is there a single
gene for specific trait in humans, right? Usually it's a combination of genes,
and often the trait produced is the effect of their impact on embryology.

So wouldn't it be impossible (and possibly extremely dangerous) to attempt to
change many traits by performing in-situ DNA rewrites?

~~~
ChuckMcM
Yes, it would be extremely dangerous.

As I understand the current research, its focussed on 'fixing' problems where
one link of the chain is broken. So when a specific set of enzymes are missing
(think lactose intolerance) or immunodeficiencies.

Wholesale rewriting chunks of DNA would, as far as I can tell, likely kill the
patient. That being said, the potential gain for some to have larger changes
to their DNA will no doubt compel someone to try it. A biologist that gave a
tech talk at Google on the technology said he had received calls from althetic
trainers asking about how his work might be applied to other areas. He was
convinced that there was already some efforts at using gene therapy to
'improve' some athletes capabilities.

------
jcfrei
has this method of cancer fighting been only applied to leukemia patients or
could it work for other types of cancer as well? since T-cells reach almost
every part of the body, couldn't they be reprogrammed to kill some other cells
apart from carcinogenic white blood cells?

------
Vadoff
I love how they didn't get any funding. If the government stopped wasting it's
money by funding a needless war, and actually put that money to good use in
research and education... maybe America could be a leader in innovation once
more.

------
yxhuvud
© 2010 msnbc.com Reprints

Ok, so what has happened after the article was printed the first time?

EDIT: ok, looks like it was just an error by msnbc. It does seem fresh:
[http://hosted.ap.org/dynamic/stories/U/US_MED_GENE_THERAPY_L...](http://hosted.ap.org/dynamic/stories/U/US_MED_GENE_THERAPY_LEUKEMIA?SITE=NYWNE&SECTION=HOME&TEMPLATE=DEFAULT)

------
kingkawn
Great outcomes for those patients, but it has to go >3 patients, and survival
>5 years before people get worked up. This is a difficult disease and people
will get hyped up quickly.

------
rokhayakebe
_In each of the patients as much as five pounds of cancerous tissue completely
melted away_

I always thought the entire cancerous tissue would be no more than 1/8 pound.

------
spottiness
I voted up your posting, but please use the original title "New leukemia
treatment exceeds 'wildest expectations'". The HIV part is pretty irrelevant.

~~~
ryanisinallofus
To me the use of a harmless, modified HIV was the most interesting part.

~~~
MatthewPhillips
Me too, I'd like to know whether a consensus exists that this HIV is harmless
or whether it's just these researchers' opinions.

~~~
xd
The HIV is used to modify white blood cells .. so I'd wager harmless as they
are not putting the HIV into the patient.

This is why I changed the title after a little nudge, to avoid people getting
the wrong end of the stick.

------
badhairday
Wasn't UPenn banned from receiving federal funding for gene therapy research
after the whole Jesse Gelsinger controversy in the late 90s? How did they
receive funding for this?

Also, 3 patients isn't statistically significant. Having this in the national
media is going to cause mass confusion in the general public. There needs to
be many more studies on this therapy before it's actually used as a treatment.

~~~
ellyagg
Actually, statistical significance has a definition. Not to be snide, but you
should look it up before commenting.

~~~
badhairday
For this particular study the numbers are fantastic, but there is no
indication that this therapy will actually cure any other CLL patients. Just
last year the NCI estimated there would be 15,000 CLL new patients. A
favorable result with just 3 patients isn't a big enough percentage to call
this new treatment anything but experimental.

However, that wasn't my original point. This therapy is obviously at a very
early stage. Headlines like this in the lay media are extremely misleading to
the general public, because a majority of people will not take the time to
read or understand the entirety of the article.

------
gojomo
I am hopeful but also reminded of the opening of 'I am Legend':

<http://www.youtube.com/watch?v=8aouT6EMJh8#t=00m21s>

(The distracting leading quote and music has been added by the uploader... but
I can't find an alternate clip of this set-up scene that also includes the
important cut to 'three years later' at the end.)

------
yaix
>> ...several pharmaceutical companies declined to pay for the research.

Great! No greedy corporation to patent it and sell off the method at
overpriced rates.

------
sweetchar
This is definitely promising!

------
earl
Wow, that's amazing. I hope the results hold up.

My father died of type 2 non hodkins lymphoma in 1997 when I was 17; he was
sick for 4 years from two rounds of lymphoma, plus a marrow transplant. Every
time I read about advances like this I feel bad about wasting my life building
better advertising instead of doing biomed. I just wish biomed paid better
wages -- I much more than doubled my income by leaving a wetlab and doing
advertising instead. It's a lesson in what our society really values. And now
I feel like Steve Yegge.

~~~
bdr
I don't think your conclusion about society's values necessarily follows. In a
society that valued biomed more, you'd have to pay the same candidate a higher
salary to get them to work in advertising.

~~~
zasz
Uh, typically when society "values" an industry more, they show their
appreciation of this value through a higher salary.

------
ck2
So this means it will be $10k a shot in the USA and $500 everywhere else until
India clones it?

~~~
oxtopus
$10K? A bargain if you ask me. Especially considering the current cost of
Leukemia treatment.

~~~
ck2
So if you cannot afford the $10k you should just die quietly?

