Proof-of-principle

I work in a research lab at Oregon Health & Science University. There, I study the cellular mechanisms underlying iron homeostasis, but OHSU is best known as the home of Gleevec.

Brain Druker, an oncologist-researcher at OHSU, discovered Gleevec. It’s a drug that completely cures a type of leukemia (white blood cell cancer) with few side effects. This is a stark contrast to other cancers, that are attacked by surgery, radiation, and chemotherapy that kill healthy cells and have deadly side effects.

Cancer is scary. It can be activated by external factors like smoking, but it is a disease caused by your own body. Tumors are caused by your own cells that grow uncontrollably. Cancer differs from say, diseases of poverty, or diseases of gender, because it is caused by the cellular mechanisms that are common to every human being.

As a result, cancer does not bias itself, and it has a tendency to pick the most devastating targets. Katie’s friend from home was recently diagnosed with a malignant brain tumor. Said friend is only thirty, has a wife, two kids, and another on the way.

For years, scientists that study the cellular mechanisms responsible for cancer had been convinced that you could target cancer in ways other than blindly killing cells, by just targeting the mechanisms that cause cancer growth. Insert Gleevec, which targets a specific mechanism of leukemia cells, and only kills cancerous white blood cells.

Gleevec is a miracle of a cancer drug, and it will probably win Druker a Nobel Prize one day, but it’s greatest strength is as a proof-of-principle experiment. I recently came across the idea of proof-of-principle, and I’ve been intrigued by it.

Here’s a sidenote to explain proof-of-principle:

Before Pixar started its series of computer animated movies like Toy Story and Finding Nemo, it first launched a mini-movie to test out the principle of animating human facial expressions. It was a success, and Pixar went ahead and produced larger projects like Toy Story. The mini-movie wasn’t the ultimate goal, but it was necessary to open the door for a whole genre/type of movies.

The type of leukemia that Gleevec cures is a relatively rare cancer, but it has single-handedly proven that it’s possible to hone in and eliminate cancerous cells without harming healthy cells. It is, simply, the proof to all scientists, doctors, and thirty year old brain tumor patients, that cancer can be cured.

As a scientist, I’m interested in Gleevec as a proof-of-principle experiment in cancer research.

But my real interest in science is bigger.

Before Gleevec, everyone was satisfied with the marginal gains of traditional chemotherapy or surgery. After Gleevec, the paradigm has completely shifted, and there is an entirely new option for cancer therapy. It’s still a little bit hazy in trying to get on that road, but Gleevec has proved to the doubters that the road exists and that cancer can be cured.

Cancer researchers everywhere are hot on the trail for the next Gleevec, and I see them as the scientists trying to get on the Gleevec road. But I also see other scientists who are looking for the roads that lead to the Gleevec road.

And I think that’s what I’m interested in.

Are there shortcuts?

Are all roads leading there uphill?

Can you wander around with no maps and end up there?

As a scientist, I want to know what leads people to insights like Gleevec. What environments, what K through PhD educational programs, what sorts of communities foster these scientists?

Is it dumb luck?

Is it once in an eon creative geniuses given a touch and vision that cannot be taught?

I’m a scientist, so I think everything can be figured out. So I guess I am one to believe that it can’t be dumb luck, or an unteachable magic touch. So I want to know. I want to know the system that creates these great scientists and then set them loose.

from ken

Feel free to comment! I would love to hear your thoughts.