Boston’s Top Docs: 14 Medical Breakthroughs
It’s All in the Genes
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
Closing the Door on the Ebola Virus
IN AUGUST, two groups of researchers from Brigham and Women’s and the Whitehead Institute identified a cholesterol-transporting protein in our bodies that turns out to be the door through which the Ebola virus enters our cells. “We tested six different strains, and all of them were completely dependent on the protein,” says Brigham and Women’s researcher James Cunningham. Block that door in mice and cell cultures, and there is no infection, no death. It’s very early, but should discovery pan out into therapeutics, it could be the key to stopping a human Ebola infection dead in its tracks. — S.F.