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Stanford research uncovers possible drug treatment for lymphedema

For decades, Stanley Rockson, MD, a Stanford professor of  medicine, has been researching possible causes and cures for lymphedema, which affects millions around the world. Damage to the lymphatic system — often from cancer treatment — leads to unsightly and often painful swelling in the limbs.

Currently, treatments for the disorder, which can lead to infections, disfigurement and disability, have been limited to restrictive garments, physical therapy, massage — all mechanical methods of controlling the lymph fluid.

Now, new research, conducted by Rockson and colleagues at Stanford, 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 research was published today in Science Translational Medicine.

“I’m still in awe,” Rockson told me in an interview for our news release, adding that he’s hopeful there may be new drug treatments available for patients within five years. “There are few situations where you take a problem at the bedside, and go into the lab, and then take discoveries back to the bedside. It’s amazingly gratifying.”

The study was unusual for two reasons. First, it was a collaboration between two different labs studying two completely different diseases, and second it resulted in a clinical trial that started in May 2016, almost a year before the research was published. As Mark Nicolls, MD, professor of pulmonary and critical care medicine, the co-principal investigator on the study, explained:

The cool thing about this story — which you almost never see — is that a clinical trial testing the therapy has already started before the basic research was even published... This is the first pharmaceutical company-sponsored trial for a medical treatment of lymphedema ...

When the collaboration began four years ago, the Nicolls and Rockson labs had become aware that they were possibly studying similar inflammatory pathways in vascular tissue — but in different diseases — Nicolls in pulmonary hypertension and Rockson in lymphedema.

After working in mouse models, and with patients with lymphedema, the researchers discovered that lymphedema — as was already proven in pulmonary hypertension —involved the same molecular pathway, and by blocking one of the branches of this pathway, they were able to reverse symptoms of the disease. Eiger BioPharmaceuticals is now conducting a trial studying a drug called bestatin as a possible cure for lymphedema. Rockson is the principal investigator of the study. And Tracey Campbell, one of Rockson’s patients who is shown above, is a participant in the trial. She described her experience in the release:

When all of the sudden one of your limbs begins to swell, you want to understand what the heck is going on... It’s a tough condition that few people seem to care about, even though millions and millions suffer with it. We’re hoping for something that gives some relief.

Previously: Limb compression device reduces skin infections caused by lymphedema, Home health care treatments for lymphedema patients cut costs and improve care and New blood test could bring lymphedema detection (and treatment) into the 21st century
Photo by Mark Williams

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