
Scientist who cured type I Diabetes in mice was denied funding for human trials - bpick
http://www.columbiaspectator.com/2009/01/23/scientist-revives-research
======
ai09
I submit that there's more to this story than "pharmaceutical companies
weren’t interested in developing the therapy".

The Juvenile Diabetes Research Foundation <http://www.jdrf.org/> funded $100
million dollars of research last year alone into research for juvenile
diabetes (type I). Link:
[http://www.jdrf.org/index.cfm?fuseaction=home.viewPage&p...](http://www.jdrf.org/index.cfm?fuseaction=home.viewPage&page_id=0B36CA86-9128-4C49-B7D8F55955507931)
Funding is predominantly from individuals and families searching for a cure
for diabetes. That includes people like myself and my family. We could care
less what mechanism or what business model cures diabetes.

Of that research, there is significant research into stem cell therapies and
cures Link: <http://onlineapps.jdfcure.org/AbstractSearchEngine.cfm> Select
"stem cell therapy" from the drop down.

Lastly, the head of JDRF is a tech star.
<http://www.jdrf.org/index.cfm?page_id=113971> He founded Citysearch and
Overture (the originator of keyword marketing. Bought by Yahoo).

I'm a big JDRF supporter since my sister has Type 1 diabetes. For any of you
that are passionate about curing Type 1 diabetes, look into JDRF. Link:
<http://www.jdrf.org/>

------
jgoewert
I always read things like this with a hefty grain of salt.

To say that all "pharmaceutical companies" blocked him on this because
treatment is more profitable than cure seems rather absurd. You would think
that with all the non profits out there already involved in solving this,
there would be quite a few with interest in the research.

Also, as most startup people know, a short term large profit is awesome! Any
company that truly finds a cure would make buku bucks in the short term and be
able to bail from the market when everyone that can be cured is. Think about
polio and Salk. The guy became a legend, and though it isn't talked about,
decently rich.

------
carbocation
Hacker News reminds me of my first year of med school. Everyone is smart and
fundamentally clueless about human pathophysiology. It's a wonderful thing,
learning about the fruits of devilishly challenging science. But it's also a
conspiratorial time. I suspect the reason is that first year med students
suddenly feel empowered by the small amounts of knowledge that they have
gained. HNers probably feel empowered for a different reason: that they are
extremely good at rapidly acquiring new ideas and implementing them.

This is normally a great thing, but it opens up two risks: the blind leading
the blind, and conspiratorial thinking.

The blind leading the blind is what happens when someone has an idea about a
disease or treatment, finds one article in pubmed or one book by one author to
support that idea, and proceeds to remain ignorant about the entire rest of
the body of work on the topic. It's almost like a race: if the good
information gets there first, people believe it; so too for the complete
hogwash in Medical Hypotheses.

The second issue that plagues first year med students and HNers is
conspiratorial thinking. This is largely a consequence of having little
knowledge, and this finally brings me on topic.

This current article discusses how someone was denied funding for human trials
of stem cell therapy despite the fact that it cured T1D in animal models. When
I see this, I think, "Of course nobody would have funded that!" Here's why I
think that:

 _Ten years ago, stem cell biology was far more limited than it is now. And
I'm talking strictly about biology, not ethics or politics._

 _The first gene therapy trials resulted in the death of Jesse Geisinger;
though stem cells are a different beast entirely from the viral vector used in
that trial, this still cast a pall over the use of active biologics._

 _Using stem cells that do not come from the recipient may require
immunosuppressive drugs. This is such a high burden to pay that it seems
inconceivable that someone would take this risk. I'd rather inject insulin all
day long than be on immunosuppressive therapy. Induced pluripotent stem cells
did not become a possibility until Yamanaka discovered his factors._

 _There is the risk that stem cells will lead to tumor-like conditions. A
woman died in 2009 from an unlicensed stem cell therapy that caused just that.
Animals are the model in which we should fully understand these risks,
subjecting humans to them only after we understand what the risks are, and why
they might occur. Especially for T1D, which has very good, lifesaving
therapeutics already. The human risk-benefit has to be there._

 _People often work on animal models for years, even a decade, before going to
human trials. This fuy had a successful mouse model. So far, so good. But
let's see primate work; let's see replication in other labs._

At the end if the day, this just doesn't seem like a conspiracy to me. Some
small drug company would love nothing more than to completely disrupt the
market for diabetes therapeutics. Sure, it might transform a $100 billion
market into a $10 billion one - but they currently have 0% of the 100 billion
market, and would have 100% of the 10 billion one.

Tl;dr - Stem cell biology is novel and poorly understood, especially 10 years
ago. It is unsurprising that nobody wanted to fund a highly risky human trial
for a disease that already has lifesaving therapeutics. I find conspiratorial
thinking to be a trait shared by those early in their medical training and by
HNers, and it can be frustrating to see great minds turn to those rarely-
correct conspiratorial thoughts.

~~~
detst
While I can't really disagree with you, I don't think it's crazy to think that
there are many influential people and organizations that have a strong
interest in seeing that we avoid potential cures for common diseases. As such,
I don't think this type of reaction is necessarily based largely in ignorance.

Then again, I'm largely ignorant on this subject so I'm certainly not making
any accusations in this case.

~~~
karsa
One such case is of Barry Marshall and H.Pylori, he was awarded the nobel
price in 2005 for discovering the cure to ulcers, (antibiotics) but he first
discovered the cure in 1983 and after about 10 years of 'trying' to convince
the medical community of which (Glaxo who was dominant in that market) was
making 90% of their profits from Zantac (an anti acid treatment) - (known
inside as the 'Zantac years'), they were accused of thwarting his discovery.
Tired of the lack of interest, in about 1992 he did a live experiment on
himself by swallowing a wapping dose of H.pylori and induced an ulcer, then
proceeded to cure this with a course of antibiotics. He did this in the glare
of the media and thereby changed the course of treatment worldwide for
ulcers... How many would do that?

However, I agree with the point though that too many HNers often seem to be
naive about the larger picture which I guess we could all be accused of in
some way.

~~~
rsheridan6
Barry Marshall did not have to convince Glaxo Kline Smith. He had to convince
other doctors and scientists. I don't believe that companies can often keep
genies in bottles, but dogma and groupthink is another matter.

~~~
karsa
He was also ignored totally by all the main stream publications, it was later
alleged they had close ties to Glaxo and the editors were fully aware of the
significance the impact would have had on Glaxo.

<http://www.orc.ru/~yur77/pylori.htm>

~~~
rsheridan6
The article claims, without evidence, that Nature and Science didn't publish
Marshall because of ties with Glaxo. Even if that's true, there are a lot of
other journals out there. Are they ALL in Glaxos pocket?

I doubt it. But they're all reviewed by his peers, who made a living by giving
endoscopies and prescriptions to patients every year or so.

~~~
alextp
Peer review is usually by scientist peers, and not professional peers. I doubt
that most academic researchers in medicine were prescribing anything, really.

~~~
rsheridan6
MDs do research all the time. Some MDs are also phDs. Marshall himself was a
doctor/researcher.

------
apinstein
I call bunk.

There are many thousands of biotech companies, the vast majority of which do
_not_ sell drugs that treat chronic diabetes.

Any one of them would love to sell a diabetes drug that cures diabetes.

Either this researcher only courted a few companies that already sell diabetes
treatments, who unsurprisingly didn't want to fund him (does Comcast fund
startups that help you avoid buying cable? NO!) or there is something else
about his research that made people not want to continue with it.

------
hnote
A very similar story can be told about Denise Faustman's ongoing research into
the cure.

She has also cured Type I in mice, although using other means.

And she also got her share of first-class controversy. Cf.
[http://www.diabeteshealth.com/read/2005/05/01/4126/why-
did-t...](http://www.diabeteshealth.com/read/2005/05/01/4126/why-did-the-jdrf-
try-to-discredit-cure-research/) and the preceding New York Times article "I
BEG TO DIFFER; A Diabetes Researcher Forges Her Own Path to a Cure"
<http://bit.ly/bzUkWs> . The JDRF doens't look good in this case at all.

More information about her approach at <http://www.faustmanlab.org/>

A good overview of other immune-based therapeutic approaches from 2006 can be
found at <http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1868847/>

It's interesting to follow the scientific debate. The 2006 review sponsored by
JDRF claims that the methods used by Faustman offer little promise, and can
even be harmful. She offers a completely opposite view, claiming in a 2008
paper

    
    
      Because our findings showing potential benefits of TNF 
      or TNF agonism for treating AI, it seems paradoxical 
      that anti-TNF therapies are a major therapeutic class 
      of drugs currently marketed for AI. TNF antagonists 
      have provided clinical benefit to about half of AI   patients,
      those with rheumatoid arthritis and Crohn's disease. Yet an 
      expanding body of research in animal models on spontaneous 
      autoimmunity suggests the opposite strategy may be 
      warranted. Furthermore, in humans, several clinical 
      observations deserve mention. First, many Crohn's and 
      rheumatoid arthritis patients never respond to TNF 
      antagonists. Second, long-term treatment with anti-TNF drugs 
      can be accompanied by onset of new or aggravated forms of 
      autoimmunity, sometimes new autoantibodies, suggesting that, 
      for some AIs, anti-TNF therapy may not be the drug of choice .

(<http://www.pnas.org/content/105/36/13644.full>)

To me, the JDRF arguments sound more fishy than hers. It's a tricky business
of solving puzzles like that, especially as an outsider. But it's fun to see
how far one can go following the debate.

------
corruption
He should put the project on kickstarter and let the people speak :) I'd throw
in some money, as would every type-1 I know.

~~~
dmpayton
As the husband of a Type I, I would gladly donate a portion of my income if it
would go directly to finding a cure.

~~~
JoeAltmaier
BUt how do we know this is "a cure", and not just the favorite project of
bitter researcher who's funding was cut off?

The stringing along of desperate people is WHY the FDA exists.

------
hsmyers
Normally it's the diabetics who make such claims about the industry--- it's a
surprise (a pleasant one) to hear such come from a possible solution source!
As a long time type-2 diabetic, I'm still happy for ANYTHING that may someday
help my fellow type-1s...

~~~
mkramlich
Yep, diabetes is one of the Big Three health problems in the US, along with
cancer and heart disease. There are people in my family that have it as well.
And supposedly it's one of the fastest growing health problems in recent
decades.

------
Ripster
I have type I diabetes.

"Weissman implied that the pharmaceutical companies had put profit over
principle, preferring to keep diabetes sufferers dependent on costly insulin
than to cure them once and for all."

This is not fun.

I wonder when are we going to agree that you cant make business out of
anything.

------
digamber_kamat
Can we call it fault-lines of capitalism ?

~~~
Ripster
That is exactly what it is. Voted.

------
ck2
The great thing about the internet is now everyone can read about such
scandals.

The bad thing about the internet is the noise level is so high, few will read
about such scandals.

I hope in 100 years, advanced hobbyists can do sophisticated medical R&D in
their garage and create small quantities of medicine for stuff that big pharma
doesn't find profitable (the problem is the government will probably shut that
down).

------
FraaJad
Is America the only country in the world where he could get his drugs trial
tested and put into market? There are umpteen number of pharma companies in
the developing countries which are ready to take a working product to _their_
market. Why not use them?

~~~
narrator
Good point. I think the French are the only ones that develop any decent
pharmaceuticals outside the U.S. One of the most successful French
pharmaceuticals is Tianeptine.

<http://en.wikipedia.org/wiki/Tianeptine>

It's sort of a French Prozac but with a number of unique benefits that Prozac
doesn't have. It's approved in several European countries but NOT in the U.K,
and is only available in the U.S on the imported pharmaceutical grey market.
Once it goes out of patent, and if it has not been approved for use in the
U.S, it will NEVER be approved, ever, for any use in the U.S. That's because
nobody would pay the 100s of millions of dollars to approve a drug that a
competitor can produce as a generic.

------
dgregd
If someone from here knows Bill Gates please forward this article to him.

------
known
Hope they've at-least patented it.

------
mkramlich
The article touches on one of the risks to the health care industry today, at
least here in the US:

In many cases, the pharmaceutical companies are making billions by selling
drugs which merely reduce undesired symptoms. But do not CURE the underlying
problem. And the symptom reduction only lasts while the user continues to buy
and consume the drug. Therefore, in many cases, these companies have a strong
financial incentive to NOT cure a disease, instead, to prolong it and only
sell palliatives. Government funding and government directed research should
be one of the ways we ensure that we have people actively trying to CURE
diseases. It's a classic example of an area where government can do something
better than business, because there does not have to be a profit motive. Just
a collective desire to reduce human suffering.

This also touches on why it's important to vote carefully in US presidential
and congressional elections. Because certain political groups cater to the Big
Pharma companies. A vote for them is almost certainly a vote towards a world
where there are more palliatives than cures. Where even new forms of ill
health can just be considered new "markets" or new ways to increase profits.

~~~
dennisgorelik
That's a classic example of an area where government can NOT do better than
business, because government sucks at innovation. There are plenty of
companies that are not selling insulin, so they would be interested to be able
to sell diabetes cure. Assuming the chances of developing the cure are
reasonably good.

~~~
donw
Wow, massive strawman. The government sucks at innovation? Who do you think
funded and developed ARPANet? You know, the network we're using to communicate
right now. Upon which countless businesses have been built.

Nuclear power? Government. First electronic computer? Government.

Furthermore, when it comes to drug discovery, that 'evil government' has one
advantage over businesses, because they have no profit motive. Businesses
exist to make money. Period.

No business is going to destroy its market for the sake of the common good. If
you're in the business of creating drugs, you aren't going to wipe out a
disease with a one-time cure, because drugs are expensive to produce, and
palliative treatments are way more profitable.

This is exactly an area where governments do better than businesses, because
the reward (improving quality of life) means more votes and support for those
in office.

~~~
dennisgorelik
Could anyone please point me to government scientific or technology
accomplishments in the last 30 years? Something that noticeably improved our
quality of life. Something on par with achievements by IBM (Business PC),
Microsoft (OS, Office, and other software), Google (search), Apple
(smartphones, music, ...), Intel (microprocessors), Pfizer (drugs).

It was surprising to see down-voting just on the basis that you disagree with
the idea. Welcome to groupthink.

~~~
sgift
WWW (1989): Developed at CERN, the European Organization for Nuclear Research,
a multinational, government-funded research organization.

edit: I've found your post <http://news.ycombinator.com/item?id=1466419> in
which you exclude CERN. Could you give an example for a valid organization?

------
hackermom
To state the obvious: there's more money in keeping people ill and in need of
temporary amelioration than there is in curing them, and such is the
foundation of american pharmaceutical industry.

~~~
MichaelSalib
Is this really true in this case? I mean, insulin is fairly cheap, right?

~~~
jmcarlin
Cheap/expensive is relative. More importantly, insulin is only a tiny cost of
the care of Type 1 diabetes. Diabetics need to check blood sugar levels
anywhere from 3-5 times a day, sometimes more if ill or for other
circumstances. Test strips can cost a patient as much as $1 per strip.

Also, insulin requires a subcutaneous injection for delivery. That means a new
syringe 3-4 times per day depending on the therapy. Or, you could get an
insulin pump at about $5k and pay for insets, tubing, reservoirs and other
miscellaneous supplies. Those need to be swapped out every three days.

Want to add continuous glucose monitoring to your therapy? Even more supplies.
Add it all up and treating T1 diabetes is nothing close to cheap no matter how
deep your pockets are.

