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Study finds first possible drug treatment for lymphedema

Collaboration between two Stanford labs has resulted in the discovery of a molecular cause for lymphedema and the first possible drug treatment for it.

Tracey Campbell has lived for seven years with lymphedema, a chronic condition that causes unsightly swelling in her left leg.

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The disease, which stems from a damaged lymphatic system, can lead to infections, disfigurement, debilitating pain and disability. There is no cure. The only available treatment is to wear compression garments or use massage to suppress the swelling, which can occur throughout the body in some cases. Campbell — who had two quarts of excess water in her left leg by the time she was diagnosed — has for years worn restrictive garments 24 hours a day and has spent an hour each night massaging the lymph fluid out of her leg.

Lymphedema is uncomfortable, exhausting and dangerous if left uncontrolled. As many as 10 million Americans and hundreds of millions of people worldwide suffer from the condition, many from the after-effects of cancer therapy treatments.

“There’s this extra layer of emotional burden,” said Campbell, who added that she has to be constantly vigilant to protect against infection. “All you want to be is normal.”

Now there’s new hope for a possible pharmaceutical treatment for patients like Campbell. A study led by scientists at the Stanford University School of Medicine has uncovered for the first time the molecular mechanism responsible for triggering lymphedema, as well as a drug with the potential for inhibiting that process.

The study was published May 10 in Science Translational Medicine.

“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,” said Stanley Rockson, MD, professor of cardiovascular medicine and the Allan and Tina Neill Professor of Lymphatic Research and Medicine at Stanford. Rockson shares senior authorship of the study with Mark Nicolls, MD, professor of pulmonary and critical care medicine. Stanford research scientists Wen “Amy” Tian, PhD, and Xinguo Jiang, MD, PhD, share lead authorship of the study and are also affiliated with the Veterans Affairs Palo Alto Health Care System.

‘Fundamental new discovery’

“This is a fundamental new discovery,” said Nicolls, who is also a researcher at the VA Palo Alto.

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