Wednesday, August 5, 2009

Why isn't my disease cured yet?
And then there's the cost.

the long therapy "pipeline" starts with broad basic research

I want to get back to this question of curing diseases. In a way, Palin/Chavez/Begley are right: the academic community (like most sectors of our society) could do more relevant work. It's not that fruit flies and worms are useless - after all, breakthroughs like RNA interference, which cures cancer in animal models, emerged from such model systems. But research without grounding can turn into an intellectual "random walk," mental masturbation that rewards scientists for playing it safe. For biology to be relevant, we must avoid such distractions and focus on the big questions, using systems great and small.

Curing disease is a noble goal, but the fiscal and legal barriers are formidable. People are not lab rats, and thus experimental medicine requires an M.D. and carries serious legal/professional/ethical considerations. Remember Jesse Gelsinger's death, and the lawsuit that followed? That killed the field of gene therapy. And then there's the cost. Funding a biotech through a clinical trial costs ~500 million dollars - enough money to fund 1,000 academic research labs. (The FDA requires the doctors to take extremely detailed notes on each patient's progress, which takes up hundreds of billing-hours.) Most of these trials fail, and even successful drugs are often recalled when side effects are identified.

As a country, we are smart enough and rich enough to overcome these barriers if we want to. Health-related research funding is less than 5% of what we spend on the military, so each F-22 we cut is another clinical trial we can sponsor. But those trials shouldn't come at the cost of basic research that helps us understand how biology works. The problem is not the 1% of people doing basic research - it's the 99% of people that just don't care. As science journalist Henry Fountain recently put it, Our mass culture is just not that interested in hearing about it. But if we want better therapies, we need to encourage biologists to make those big discoveries, chase those big unknowns.

UPDATE 8/6: Jim Watson just weighed in on this issue from the perspective of the war on cancer. For once, he doesn't sound crazy.

Next: Cutting the cost of new cures.

3 comments:

Anonymous said...

Yes, good idea for post. But really- Do you really think that 'random walk' research is such a waste of time. Read Fire et al's early stuff on RNAi and you'll think that they were totally wasting their time.

Palin et al don't have a good point. The transformation of a single cell into a billion or trillion cell organism counts as a 'big question'. I personally think that this question deserves orders of magnitude more funding and time than those allocated for studying the rare disease research, like ALS, for an example.

The lessons learned from basic research will aid in preventing/treating/curing diseases in unforeseen ways.

Samurai Scientist said...

@Anon, thx for your comments.
Read Fire et al's early stuff on RNAi and you'll think that they were totally wasting their time.

Well, yes, but on the other hand more directed research might have revealed RNAi a decade earlier :)

The transformation of a single cell into a billion or trillion cell organism counts as a 'big question'.

Agreed... although we've also learned that development differs quite a bit from organism to organism.

The lessons learned from basic research will aid in preventing/treating/curing diseases in unforeseen ways.

I'd add to this that human disease is hardly the world's biggest problem. Nor are biologists in the business of saving lives - if we were, we'd all be handing out condoms in Africa :)

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