Bestatin is not approved in the United States, but it has been used for years in Japan as a cancer treatment.
The advantage of the drug, according to Rockson, is that it has more “selective” action against LTB4 — and fewer side effects — than ketoprofen.
Based on the lab findings, a clinical trial testing bestatin against lymphedema is already underway, Rockson said.
Eiger BioPharmaceuticals, based in Palo Alto, Calif., is funding that trial (and acquiring the drug from its Japanese manufacturer). Rockson and a colleague on the study are consultants to the company.
“I think patients should feel very encouraged that work is being done,” said Dr. Theresa Gillis, chief of the rehabilitation service at Memorial Sloan Kettering Cancer Center in New York City.
Still, the role of bestatin — if any — won’t be clear until the clinical trial results are in, according to Gillis, who wasn’t involved in the research.
The “mouse model” of lymphedema, she noted, doesn’t exactly replicate what happens in cancer patients who undergo radiation therapy or surgical removal of the lymph nodes. And, treatments that work well in animals don’t always pan out in people.
Gillis agreed that new lymphedema treatments are badly needed.
“Millions of dollars are spent each year in the U.S. on treatment of lymphedema,” she said. “And those millions are often costs carried by the patients themselves.”
Beyond costs, Gillis added, the current therapies are burdensome.
“Patients with severe lymphedema wear specialty compression garments daily, and may need to use compression bandaging every night — or a second specialty night garment,” she said. “Even with optimal efforts by the patient, edema can become progressively worse over time.”
The new findings offer a clearer understanding of the underlying process in lymphedema, according to Gillis. Hopefully, the new understanding will “lead us toward eventually preventing the condition altogether,” she said.
The bestatin trial results are a few years away. But Rockson said he thinks patients can take heart in the fact that the work is happening.
“Historically,” he said, “lymphedema patients have been largely ignored, and that’s led to a certain sense of hopelessness. But now I think we can say answers are on the horizon.”