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Scientist who cured type I Diabetes in mice was denied funding for human trials

134 points| bpick | 15 years ago |columbiaspectator.com

122 comments

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ai09|15 years ago

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... 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|15 years ago

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|15 years ago

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.

marciovm123|15 years ago

Well said. Another failing arises from just how complicated biology is, and how little we understand of it. Unlike in computer programming, in biology reliable layers of abstraction do not yet exist. The equivalent of changing one line of code in a cell's program (through a drug, gene, whatever) in a sense has unknown side-effects on every other line of code. A programmer's approach to working with or trying to understand such a system quickly leads to maddening (and unsupported) conclusions.

detst|15 years ago

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.

mkramlich|15 years ago

Suspicion or expectation of conspiracy can also be due to past exposure to evidence of actual conspiracy. Men colluding against others has been going on for thousands of years if history is to be believed. And yes one common motive to conspire is to gain large amounts of money to the detriment of another group, often involving lies and force. Are all imagined conspiracies real? Probably not. Do some exist? Of course.

crocowhile|15 years ago

>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

Weissman is not "someone". He is one of the founding fathers of the stem cell field and has done amazing thing. He is a leader in the field and got really amazing results with animals. Sorry to say that but you sound like the first year student here.

MikeGale|15 years ago

There's one area where variations on Conspiratorial Thinking happens regularly and much of the population has their minds bathed in the result every day.

These ideas, conceived in the medical domain, apply, in some way, to the wider world of media reports.

Ever notice that when you really know an area, news reports about it nearly always get it wrong. Completely misunderstand what's going on. Often interpret it as one of a few stock, basely motivated, scenarios.

(You can debate whether the authors are all smart. I think that some of them are, but those still do it.)

So we have a society where the blind lead the blind down blind alleys as a matter of course.

Thank goodness that we have resources like Hacker News, to sometimes give a peek at more thorough thinking. (Chaotic though it may be!)

grandalf|15 years ago

Would it follow from your argument that Weissman is either too foolish, disinterested, or arrogant to pursue the next step of animal trials that would have made human trials more likely?

stretchwithme|15 years ago

Nobody would have funded many bad ideas. That's when you turn to government to do it.

apinstein|15 years ago

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|15 years ago

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... 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|15 years ago

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|15 years ago

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.

hsmyers|15 years ago

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|15 years ago

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|15 years ago

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|15 years ago

Can we call it fault-lines of capitalism ?

Ripster|15 years ago

That is exactly what it is. Voted.

ck2|15 years ago

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|15 years ago

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|15 years ago

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|15 years ago

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

known|15 years ago

Hope they've at-least patented it.

mkramlich|15 years ago

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.

HeyLaughingBoy|15 years ago

I hear this argument all the time and it ignores one fundamental fact: there is more than one large pharmaceutical company and they don't all make $disease_x_treatment.

That one company makes billions selling a treatment for a disease has no bearing on a competing company making a cure for that disease and cornering the market. If anything, it's the very definition of a market-driven solution to a problem.

Now, it's true that there are diseases which are so rare that it's not commercially worth developing a cure, and perhaps government funding is useful in that case but that's a completely different topic.

rsheridan6|15 years ago

The big companies that already have treatments for diabetes (for example) definitely have an interest in not curing it, but a small startup would be happy to make millions destroying a multibillion dollar industry.

stretchwithme|15 years ago

a lot of truth to that.

I also think that if consumers were actually paying the full direct cost of treatments, they would be a lot less willing to pay for a chronic drug habit instead of a cure.

Selling cures can be profitable too.

Consumers are perfectly able to see and pay for the benefits of a one time solution as opposed to a cheaper chronic solution. That's why they buy cars and houses instead of renting them. The market will deliver what the consumer wants if the consumer were making the spending decisions.

Government can't be somehow better than business at deciding what to fund. Its manipulated by business! The current pattern has evolved in a government funded research environment. Only ending the interference in the normal process of making decisions at the consumer level can fix this problem.

dennisgorelik|15 years ago

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.

hackermom|15 years ago

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|15 years ago

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