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Today the NY Times printed the third part of Amy Harmon’s excellent feature on the ups and downs and promise of some clinical trials for cancer. The focus is on a new drug, PLX4032, some people with melanoma who chose to try this experimental agent, and the oncologists who prescribed it to them.

What I like about this story is that, besides offering some insight on the drug itself, it balances the patients’ and doctors’ perspectives; it explains why some people might elect to take a new medication in an early-stage clinical trial and why some physicians push for these protocols because they think it’s best for their patients.

And it provides a window into the world of academic medicine, where doctors’ collaborate among themselves and sometimes with corporations.

Here’s some of what I learned:

PLX4032 is a targeted therapy, a drug that’s designed to interfere with a specific, disease-causing molecular abnormality. It’s a small compound, taken by mouth, manufactured by Plexxikon that alters BRAF activity.

BRAF is a cellular enzyme, or kinase, that normally regulates how cells grow and divide. It’s encoded by an oncogene, a segment of DNA that can cause cancer when overly-expressed.

In most but not nearly all cases of melanoma, and in some other cancer clones, the malignant cells bear a mutated BRAF gene. This change can lead to a perpetually “turned on” state in the cells’ signaling machinery by which they proliferate without control. It’s thought that when PLX4032 works, it does so by blocking BRAF-mediated signaling and growth activity.

Harmon’s piece is long but easy to get through. She covers the human side of the story realistically. Some of the patients she describes with advanced tumors are desperate. The oncologists are, for the most part, hard-working idealists who work tirelessly for their patients.

There are real issues here, as in the setting of most clinical trials. I recommend this series to anyone who contemplates enrolling in a new drug study.

A remarkable point, as reported, is that the patients who ultimately succumbed to melanoma after a long period weren’t angry. As described, they didn’t feel “used” by their doctors or otherwise. Rather, they expressed appreciation. If these reported feelings are representative, that’s a testament to the quality of the care they received on study and, perhaps even more so, to effective communication between the patients and their physicians.

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