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

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

#61

Earlier quoted context omitted.

My sense is that he followed the basic science, working deeply in stem cell biology and adding significantly to our knowledge. This is another important route to convincing people that they should pursue something like stem cells - if you know how they do what they do, then you can make educated guesses about how to prevent side effects, too. So, to the contrary, I think he's done many things over the past 10 years t…

Do you think his research will result in type I diabetes being cured in humans in our lifetimes?

Stem cell therapy is not likely to be curative for Type I DM, although this is simply my opinion and I have no evidence to support it. Part of this is definitional. A true cure for Type I would involve stopping the autoimmune attack of the pancreatic islets.

A therapy that's as good as a cure (such as one that is stem cell based, or otherwise) does seem likely in the next 15 years to me. I've worked on this problem briefly; my role was miniscule, but the basic idea is that you can encapsulate beta cells in an artificially immunologically privileged space, such as an alginate capsule. If done just right, this will let you sense glucose, secrete insulin, and avoid immune attack.

At any rate, yes, I think that we're getting closer to meaningful "post-insulin" therapeutics. Whether or not that will be due to stem cell biology, I don't really know; to be honest, I don't really mind what technique is used so long as it is safe, effective, and maximizes the patients' quality of life.

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

#62

Earlier quoted context omitted.

My sense is that he followed the basic science, working deeply in stem cell biology and adding significantly to our knowledge. This is another important route to convincing people that they should pursue something like stem cells - if you know how they do what they do, then you can make educated guesses about how to prevent side effects, too. So, to the contrary, I think he's done many things over the past 10 years t…

Do you think his research will result in type I diabetes being cured in humans in our lifetimes?

For some reason I can't reply to carbocation, but in reply to his objection that stem cells wouldn't help due to the autoimmune issue, there's a lab that claims to have solved that problem in mice. Apparently, when you get rid of the autoimmune cells, the insulin-producing beta cells grow back. The lab claims to have had a successful phase I (human) trial. If they're right, stem cells are irrelevant. I don't know enough to really evaluate this, but these are definitely serious people.

Here's the lab: http://www.faustmanlab.org/index.html

There's research going on for MS, another autoimmune disease, which also involves getting rid of the autoimmune cells. Here's one approach: http://en.wikipedia.org/wiki/Tovaxin

If they can get this kind of thing right, we'll live to see many autoimmune diseases cured.

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

#63
post #54
post #48

Earlier quoted context omitted.

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.

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 ac…

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.

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

#64

Earlier quoted context omitted.

Do you think his research will result in type I diabetes being cured in humans in our lifetimes?

For some reason I can't reply to carbocation, but in reply to his objection that stem cells wouldn't help due to the autoimmune issue, there's a lab that claims to have solved that problem in mice. Apparently, when you get rid of the autoimmune cells, the insulin-producing beta cells grow back. The lab claims to have had a successful phase I (human) trial. If they're right, stem cells are irrelevant. I don't know eno…

Excellent! This is the type of thing that gets me more excited, because it's treatment targeted at the cause of the disorder. (Stem cells will be so for certain diseases, too.) I hope they succeed.

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

#65

Earlier quoted context omitted.

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.

People upvoting this are dreamers as that industry costs a phenomenal amount of money to put something through trials. Millions is a trivial amount on what you could throw down the drain and end up with no actual product. It's not vulnerable to a startup as a startup can't practically enter that market. You'd need to be producing something else first. And small? Not gonna cut it. Something that works on mice and even…

I believe the story of Lipitor involved Pfizer not being interested, followed by the creation of a startup around it's further development with the company eventually being bought by Pfizer. It then became the top-selling branded pharmaceutical in the world [1]. I guess the world just needs more dreamers like these people.

[1] http://en.wikipedia.org/wiki/Atorvastatin (yes, a wikipedia reference)

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

#66

Earlier quoted context omitted.

People upvoting this are dreamers as that industry costs a phenomenal amount of money to put something through trials. Millions is a trivial amount on what you could throw down the drain and end up with no actual product. It's not vulnerable to a startup as a startup can't practically enter that market. You'd need to be producing something else first. And small? Not gonna cut it. Something that works on mice and even…

There are lots of pharmaceutical startups. And, yeah, drug development is high risk. In other news, the sky is still blue. Type 1 diabetes is still a pretty big market even if you had a one shot cure. There are hundreds of thousands of type 1 diabetics. If they pay a thousand dollars each (a steal at that price), that's hundreds of millions of dollars. For comparison, there are a few thousand people with CML, but tha…

A steal at a thousand dollars ? I have Type 1 and would consider an actual cure a great deal even if it cost everything I own plus my entire earnings for 10 years.

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

#67

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…

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.

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

#68
post #48

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…

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.

> 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.

I do.

How about some evidence show that this has happened more than a handful of times in the last 20 years?

Note a company refusing to fund a competitor is NOT evidence of "avoid potential cure". Heck - a company refusing to fund something for eany reason is not evidence.

The act that you need to document must be someone going out of their way to block something, not merely refusing to help.

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

#69
post #42

Earlier quoted context omitted.

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.

The small startup doesn't have the 10s to 100s of millions of dollars that it takes to get a drug approved in the US. So they would be happy to do it but are incapable.

more like 1000s of millions. It's incredibly expensive to demonstrate safety and efficacy, and many promising candidates fail at the later stages (after 100s of millions already spent) when the response in humans is different from that of animal models.

http://en.wikipedia.org/wiki/Drug_development

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

#70

Earlier quoted context omitted.

If you have "no presence at all" then you either have no money, in which case you can't really afford entering it (in this case, anyway), or you have the money, but then the above rules still apply. Effectively it would be better to market your mediocre product to physicians than to kill the cash-cow. Also, large risks are actually typical for this type of research. It's expensive and risky, so it has to give really…

Google had no presence at all in the spreadsheet market in 2005. I guess they really couldn't afford to enter the spreadsheet market. Too bad, because if google had money, and the above rules didn't apply, then google might create a free spreadsheet web service that competes with MS Excel even at the risk of destroying a gigantic cash cow owned by somebody else.

Spreadsheets are not drugs. Mostly.

Developing and deploying a spreadsheet is cheaper and less risky than developing and deploying a completely new drug.

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