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

There's a whole sub-industry based on diabetes! You have goods providers shuttling out boatloads of sharps, insulin testers, strips, etc. A lot of companies profit beyond just insulin, which isn't terribly expensive unless you have to use a high dosage.

"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." But it's actually more efficient to develop a treatment for the second company.

It's better to share an extremely big market with a competitor than to corner it while making it much smaller. The goal is not to destroy all competition, it's to make more money.

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.

Yes, but what if you have no presence at all in that extremely big market?

In addition to the big ones, there are dozens of smaller companies with the cash and the experience in drug development that would jump at the opportunity to cure type 1 diabetes. Unless the risks vastly outweigh the rewards, which seems to have been the case here.

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 large rewards. Research for completely new cures is even more risky and expensive, and the rewards are smaller.