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.
The city’s biotech community community cranked out a number of promising new treatments this year. Here’s a look at groundbreaking medications for four serious ailments.
Company: Vertex, Cambridge
Incivek represents a less-complicated, more-effective treatment plan for the four million Americans with hepatitis C. The drug works as a protease inhibitor, blocking the proteins that the virus needs to copy itself in the body. Taken three times a day for 12 weeks, it produces recovery times that are twice as fast as previous treatments. It’s estimated that Incivek, which was approved in June, will do $750 million in sales this year.
Company: Biogen Idec, Cambridge
BG-12 is one of the first oral medications for multiple sclerosis, an affliction of the nervous system that affects more than 350,000 Americans. The drug reduces brain inflammation and protects neurons that have been attacked by the disease. In the third stage of clinical trials, it’s been found to reduce the rate of annual relapse in patients by 53 percent. Biogen Idec hopes to submit BG-12 to the FDA for approval next year.
Homozygous Familial Hypercholesterolemia
Company: Genzyme, Cambridge
Those who suffer from this extremely rare genetic disorder are born without the low-density lipoprotein receptors that pull bad cholesterol out of the blood, which can lead to heart attacks by their teens or twenties. Mipomersen blocks production of the protein that bad cholesterol adheres to in the blood, reducing atherosclerosis. In Phase 3 trials, the drug — submitted to the FDA this year — cut patients’ LDL counts by 25 percent.
Advanced Basal Cell Carcinoma
Company: Curis, Lexington
Vismodegib is the first orally administered drug to help treat advanced basal cell carcinoma, the most common skin cancer in the U.S. It works by blocking a common protein that, in rare cases, can mutate and cause the disease. In a second round of trials, it was found to substantially shrink tumors and heal lesions in 43 percent of patients.— J.N.
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.