The following article was published in the Arkansas Democrat Gazette May 11, 2014
This article was published May 11, 2014 at 5:30 a.m.
The U.S. Food and Drug Administration has approved the first-ever treatment for a rare, debilitating and often deadly blood disorder after a Little Rock doctor and researcher spent more than 10 years leading a worldwide team in laboratory studies and patient trials on the disease.
Before the April 23 FDA approval of the drug siltuximab, those afflicted with Castleman’s disease had only chemotherapy and steroid regimens as treatment options for the disorder that mimics lymphoma, a cancer of the lymph nodes. Those options offered very limited long-term success.
The years-long study was led by Dr. Frits van Rhee, a University of Arkansas for Medical Sciences professor of medicine and director of clinical research for the Myeloma Institute, in conjunction with Janssen Biotech Inc. in Horsham, Pa.
The study included hundreds of patients from all over the nation and the world who traveled to UAMS for treatment and testing.
The breakthrough is nothing short of miraculous, said Carl Guenther, a Castleman’s patient who has made the trek from his Wilmington, Ohio, home to UAMS every three weeks for the past nine years to get the drug through an infusion pump.
Guenther was diagnosed in 2004 after experiencing lymph nodes the “size of golf balls,” constant fever, weakness, night sweats, nausea and weight loss. He had thought the extreme symptoms were brought on by the grief he was enduring over the death of his wife, Susan, from ovarian cancer.
“We were high school sweethearts, married for 24 years. I was her caregiver for four years while she was sick, then I had my two teenagers to take care of after she passed away,” Guenther said. “I just thought I had worn myself out.”
Guenther went to the same doctor who had treated his wife, and biopsies were ordered that same day. The medical team at the small community hospital was baffled when the lymph nodes came back benign.
Castleman’s is so rare that there are only 4,500 people in the United States known to have the disease. It is difficult to diagnose and is often confused for other conditions.
“I lucked out, although I’m a Christian and don’t believe in luck,” Guenther said, explaining that a pathologist fresh out of medical school soon recognized the disease from his textbooks.
The prognosis, however, was gut-wrenching. His children, who had just lost their mother months earlier, were told that doctors had given their father only two years to live at the most.
Patients with Castleman’s overproduce lymphocytes, leading to enlarged lymph nodes. The disease can also cause the liver, spleen or other organs to enlarge, possibly leading to multisystem organ failure and death. Though the tumors that can result from the disease are usually benign, Castleman’s can also progress to malignant lymphoma.
After a brutal regimen of high-dose steroids with extreme and painful side effects, Guenther reached out to van Rhee — who was listed as the world’s leading Castleman’s disease expert on many of the websites Guenther visited.
“I emailed him at 10:30 that night. By 10:45, he called me, and we talked for a full hour,” Guenther said.
Nine years and 148 treatments with siltuximab later, Guenther is in full remission with no symptoms or side effects. His life today is filled with days spent with his second wife, Debra, and their four children; frequent hunting and fishing trips; performing piano concerts for the American Cancer Society; and helping a friend farm “almost full time.”
“I now know what normal is,” Guenther said.
In his office last week, van Rhee flipped through graphics on his computer screen, pointing out treatment comparisons, patient outcomes and other related data. The recent FDA approval of siltuximab is a milestone, he said, but his work is far from over.
There is still no cure for Castleman’s disease. And if patients stop the newly approved treatment, the disease comes back full force, van Rhee said.
Because the drug was only recently approved, Guenther will have to continue making trips every three weeks to UAMS for treatment.
The new drug for the disease was approved only for use in multicentric Castleman’s patients — those who have multiple lymph nodes affected — who are HIV and human herpes virus-8 negative.
And the underlying cause for Castleman’s has still not been identified.
“We have a very effective therapy now, but Castleman’s disease is not well understood. We’re still trying to understand what causes it and to develop a treatment that is not ongoing. Right now, Castleman’s patients have to be in treatment for the rest of their lives,” van Rhee said. “We’ve got a lot more to do.”