December 23, 2012
Still Waiting For A Biotech Revolution

The first attempts at human gene therapy were made in 1990. The excitement around gene therapy was high in the 1990s. Yet not till 22 years later in 2012 the first gene therapy drug, Glybera for a rare genetic disease called lipoprotein lipase deficiency, was approved for clinical use in Europe. That same treatment might become the first gene therapy to get regulatory approval in the US in 2013. The great promise of gene therapy remains just that, a promise.

Use of cell therapies has expanded more rapidly. However, the only stem cell therapy in use is for bone marrow transplant in cancer treatments. Cell therapy usage still remains rare as compared to the number of diseases and disorders potentially treatable with cell therapies. In a nutshell, the biotech revolution so far has been a bust for curing diseases.

A look at the orders of magnitude drops in DNA sequencing costs shows at least some biotechnologies racing ahead faster than Moore's Law advances in computer processing power. But a much higher critical mass of biotechnologies is needed to easily make lots of stem cell therapies and gene therapies. We also need many more biotechnologies to be able to do the tissue engineering needed to grow replacements for most types of internal organs.

On the bright side the rate at which new drugs have been getting thru regulatory pipelines has increased. But the drugs have less financial impact. That suggests these drugs are aimed at narrower niches with smaller benefits.

In total, the world's 12 top pharmaceutical companies had 41 new drugs approved, with combined forecast revenues of $211 billion, while the year-earlier tally was 32 products with expected revenues of $309 billion.

My suspicion: most of the remaining problems that chemical drugs haven't cured or slowed down are problems that chemical drugs can't fix. For most of what goes wrong as we age we need gene therapies, cell therapies, and other techniques that are powerful enough to repair or replace aged tissue. Chemical drugs are just too simple in structure and in potential effects. They can't do much tissue repair. Though chemical drugs might at least be able to kill off a lot more damaged and dangerous cells. That leads us to a hopeful report.

An article in the New York Times about the attempts by Merck, Roche and Sanofi to make wide spectrum anti-cancer drugs against proteins made by mutated genes p53 and MDM2 suggests a big step toward a real biotech revolution might be within reach using only conventional chemical drugs. Imagine a drug that can wipe out half of all cancers.

For the first time ever, three pharmaceutical companies are poised to test whether new drugs can work against a wide range of cancers independently of where they originated — breast, prostate, liver, lung. The drugs go after an aberration involving a cancer gene fundamental to tumor growth. Many scientists see this as the beginning of a new genetic age in cancer research.

Read the whole thing. Very exciting. Imagine a small number of chemical compounds killing half of all cancers. Might be possible. Though I fear the slow rate at which drugs get tested will make this attempt take many more years.

Roche was the first to start testing a p53 drug in patients. The company began, as required, with an attempt to establish a dose strong enough to be effective but not too toxic. It took a surprisingly long time — three years — because Roche was cautious, starting with a tiny dose and gradually escalating it.

The slow rate of cancer drug testing and the regulatory environment that causes the snail's pace is tragic. In America alone about 600 thousand people per year die of cancer. So while Roche was doing toxicity studies almost 2 million people died of cancer in the US and millions more elsewhere.

If I was dying of cancer and had months to live I'd volunteer to take a large dose of an experimental drug to find out its toxicity. If I was really lucky I'd be cured. If I was only moderately lucky the drug would kill me quickly so I wouldn't have to spend months in pain slowly dying. I bet if dying cancer patients were given the choice of whether to risk a fast death due to aggressive testing of new drugs enough would say yes that drug testing could be sped up substantially.

The regulators who create this slow drug development environment also place high hurdles in the way of trying stem cell therapies and gene therapies for fatal illnesses. The US FDA has won a court case that gives it broad power to regulate stem cell therapies. Well, FDA regulation basically means "spend hundreds of millions of dollars and spend a decade getting approval". Got a disease that'll kill you 5 or even 10 years from now? Time to get a passport if you don't already have one. When the biotech revolution finally starts arriving with great clinical treatments you'll have to go abroad to get the latest treatments.

We need a faster rate of progress in biotechnology.

Update: It says something about how much faster cancer research could advance that when researcher Ralph Steinman was diagnosed with pancreatic cancer he experimented on himself and extended his life.

In the long struggle that was to come, Steinman would try anything and everything that might extend his life, but he placed his greatest hope in a field he helped create, one based on discoveries for which he would earn his Nobel Prize. He hoped to reprogram his immune cells to defeat his cancer — to concoct a set of treatments from his body’s own ingredients, which could take over from his chemotherapy and form a customized, dynamic treatment for his disease. These would be as far from off-the-shelf as medicines can get: vaccines designed for the tumor in his gut, made from the products of his plasma, that could only ever work for him.

He did things to himself much faster than he could have gotten permission to experiment on others who had similar very short life expectancies. Why not let people with very short life expectancies more easily get access to experimental treatments?

Share |      Randall Parker, 2012 December 23 03:52 PM 

Brett Bellmore said at December 24, 2012 6:33 AM:

I think there are two issues slowing this down, in addition to the regulatory drag:

First, human biology is fiendishly complex. And we're not really going to get anywhere big until we understand it. There's no end of what we can do, once we've disassembled our source code. But that's going to be the work of decades yet.

Second, not enough garage experimenters yet. I hope to do something about that, in some small part, in the next few years.

Randall Parker said at December 24, 2012 9:58 AM:


I agreed on the complexity. The human body is a really really hard problem.

Garage experimenters: We need faster looping by the professional experimenters. I don't think the microfluidics devices are far enough along yet to allow much home lab experimentation. However, smart software developers and people with mathematical chops could start doing analyses on large publicly available data sets.

bmack500 said at December 25, 2012 4:39 AM:

Can't some billionaire just buy an nice tropical island somewhere, build schools and the best research facilities to attract the best & brightest scientists, out of U.S. jurisdiction? The freedom to experiment!

We could grow clones there without higher brain function and have a ready supply of body parts. (no, not like the move "The Island")

Brett Bellmore said at December 25, 2012 5:16 AM:

I don't think the US actually admits there's any place out of it's jurisdiction, these days. And growing clones is so five minutes ago; You'd likely use large format tissue printers.

Ronald Brak said at December 29, 2012 3:25 AM:

In agriculture there hasn't been a biotech revolution, but there has been steady biotech progress. While crop yields more or less peaked a generation ago, in the time since important advances have been made in reducing the need for inputs, whether they be fertilizer, herbicide, insecticide, arthopodcide, fungicide, etc. This has helped to keep down the price of food and also helped reduce impact on the environment.

Brett Bellmore said at December 30, 2012 6:53 AM:

My birthday gift from my wife, Mitochodria, by Scheffler, has shipped. First step in figuring out what I want to do in that home lab I'm starting as soon as I have a home to set it up in. Was quite surprised the wife, instead of complaining of the price, commented that she'd like to read it, too. Maybe there won't be much push back on my plans to unleash my inner mad scientist...

Phillep Harding said at January 2, 2013 2:12 PM:

bmack, as I recall, someone did set up an independent nation for business purposes that the US fedgov did not like. "Someone" gave the ruler of a nearby island nation some weapons and boats, and that new and independent nation is no more.

While your idea has (IMO) great merit, it does need to be done cautiously.

Ronald Brak said at January 2, 2013 7:53 PM:

Remember, scientists can do pretty much anything they want to wheat in a lab, and while important advances have been made, they haven't quite produced any miracles as of yet. And in practice pretty anything that might in some way help humans at some point can be done to a rat, so when we can reliably regenerate rat limbs but not human ones it's time to start passing waivers around. Personally, rather than regulation, I think the biggest road block is a lack of funds for research. Even in Australia we like to drip feed funds to our scientists.

Randall Parker said at January 3, 2013 7:33 PM:


It is already possible to set up research labs in more lenient countries. The problem comes with delivery of the result. People will have to travel to get the drugs. I expect they will eventually.

Matthew Wolfinbarger said at January 3, 2013 9:53 PM:

I would love to be a guinea pig for biotech research--and I mean the kind that to do in the U.S., you'd better have some damn good lawyers (due to the risk for subjects--I don't care, my risk level is so incredibly high I'd do something that has a 10% or more risk of death). I'm thinking most likely the research would be primarily run by Asians and/or other high IQ minorities so they can cry racism if they ever get investigated. Any ideas?

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