Pharmaceutical find

Physicians can offer little help for the sufferers of lymphedema, a painful, disfiguring condition affecting hundreds of millions worldwide. The condition results from a damaged lymphatic system and leads to swelling in various parts of the body — often limbs. The only available treatments are wearing compression garments or massaging the affected area. But School of Medicine scientists have discovered what triggers the ailment, along with a drug that may inhibit that process.

“We figured out that the biology behind what has been historically deemed the irreversible process of lymphedema is, in fact, reversible if you can turn the molecular machinery around,” says Stanley Rockson, MD, professor of cardiovascular medicine. The study was published May 10 in Science Translational Medicine; Rockson and Mark Nicolls, MD, professor of pulmonary and critical care medicine, are its senior authors.

The researchers found that the buildup of lymph fluid is an inflammatory response, not a “plumbing” problem, as previously thought: An inflammatory substance known as LTB4 is elevated in lymphedema, causing inflammation and impaired lymphatic function. This discovery led researchers to investigate drugs that target LTB4 for their ability to trigger lymphatic repair. One in particular offered promise: betastin, which is used in Japan to treat cancer. It’s now being tested for use in the United States as a lymphedema treatment in a trial sponsored by Eiger BioPharmaceuticals. Rockson and Nicolls are scientific advisers to the company.