Top Docs 2011

From face transplants to computerized glasses that help blind people “see” to a groundbreaking development in the fight against Lou Gehrig’s disease, here are 14 stunning medical breakthroughs.

It’s All in the Genes

Illustration by Adam McCauley

Illustration by Adam McCauley

Patients have typically had two options for attacking cancer: surgery or chemotherapy. But now, thanks in part to the work of a team of researchers at Dana-Farber Cancer Institute, we may be at the dawn of a new era of treatment. The researchers have come up with “smart medicines,” or molecular-targeted therapies, that attack certain genetic defects in patients that can cause cancerous tumors.

These particular cancers are fed by proteins gone haywire. The smart meds turn off those proteins, cutting off the tumor’s food supply and causing it to die. Success rates in clinical trials have been startlingly high. In one instance, a woman named Beverly was diagnosed with late-stage lung cancer and given six months to live. Then Pasi Jänne, associate professor of medicine at Dana-Farber, discovered that Beverly’s lung cells contained an alteration in a protein called anaplastic lymphoma kinase (ALK), a mutation that produces rapidly growing tumors in 5 percent of non-small-cell lung cancer patients. Jänne enrolled her in a clinical trial of a new drug, crizotinib, that essentially acts as a kill switch for ALK.

Within six weeks, Beverly’s tumors had shrunk by more than half. Where chemo typically has an efficacy rate of 30 to 40 percent for cancers like this, with smart medicines it’s more like 70 to 80 percent. “When they work, they usually work in days,” Jänne says, “not in weeks or months that we see with chemotherapy.” The FDA approved crizotinib in August, and other smart medicines are in the works for certain types of melanoma and leukemia.  —  Casey Lyons