UAMS Drug Trial Leads to First FDA-Approved Treatment for Blood Disorder Called Castleman’s Disease
| LITTLE ROCK – The first-ever treatment for a rare blood disorder called multicentric Castleman’s disease was approved recently by the Food and Drug Administration after drug trials headed by a researcher at the University of Arkansas for Medical Sciences (UAMS).
Frits van Rhee, M.D., Ph.D., the nation’s leading expert in Castleman’s disease, led the years-long study of siltuximab in conjunction with Horsham, Pa.-based Janssen Biotech Inc. The drug was approved April 23 under the trade name Sylvant for patients who are HIV negative and human herpes virus-8 negative.
“The approval of Sylvant gives physicians a long-awaited treatment option for a group of patients who have been suffering with this chronic, serious and debilitating disease,” said van Rhee, a UAMS College of Medicine professor and director of developmental and translational medicine at UAMS. He also holds the Charles & Clydene Scharlau Chair for Hematological Malignancies Research.
Castleman’s disease is a rare blood disorder that acts very much like lymphoma, a cancer of the lymph nodes. With Castleman’s, white blood cells called lymphocytes are overproduced, leading to enlarged lymph nodes. It also can affect lymphoid tissue of internal organs, causing the liver, spleen, or other organs to enlarge. Infections, multisystem organ failure, and malignancies, including malignant lymphoma, are common causes of death in patients with the disease.
“Castleman’s is a complex disease, and up until this point, physicians have tried to reduce lymph node masses and put the disease in remission through a combination of treatments, but it often returns,” said van Rhee. “There has been a serious need for treatment options for patients with multicentric Castleman’s disease.”
Common symptoms include enlarged lymph nodes, which appear as lumps under the skin, fever, weakness, fatigue, night sweats, weight loss, loss of appetite, nausea, vomiting and nerve damage that leads to numbness and weakness.
The disease is so rare that tracking the number of cases is difficult, but a 2012 study estimated 4,500 people in the United States have Castleman’s disease.
Since 2005 as part of the clinical trial of the drug at UAMS, Van Rhee has treated Carl Guenther. An Ohio resident, Guenther was the third patient in the clinical trial. His symptoms before treatment included fatigue, night sweats, weight loss and severely swollen lymph nodes.
“Not only have those symptoms not returned, I’m better than before,” Guenther said. “I never get a cold or flu. I had symptoms for years before diagnosis, and I don’t have those anymore. I’m doing so well that most of the guys who come to Arkansas with me can’t keep up with me. I attribute that to the treatments. I haven’t felt this good for a long time.”
Sylvant is administered through an intravenous infusion once every three weeks. Guenther has made the trip from Ohio every three weeks for almost nine years. Like other Castleman’s patients, he expects to have to continue to receive treatments the rest of his life.
“Everyone in the community of patients with Castleman’s is excited about the FDA approval,” Guenther said. “We’ll be able to get it wherever there’s oncology treatment. It’s a mix of emotions for me. I enjoy coming down here, but it’ll be nice to not have to drive.”
Janssen Biotech Inc. is one of the Janssen Pharmaceutical Companies of Johnson & Johnson.
UAMS is the state’s only health sciences university, with colleges of Medicine, Nursing, Pharmacy, Health Professions and Public Health; a graduate school; a hospital; a main campus in Little Rock; a Northwest Arkansas regional campus in Fayetteville; a statewide network of regional campuses; and eight institutes: the Winthrop P. Rockefeller Cancer Institute, Jackson T. Stephens Spine & Neurosciences Institute, Harvey & Bernice Jones Eye Institute, Psychiatric Research Institute, Donald W. Reynolds Institute on Aging, Translational Research Institute, Institute for Digital Health & Innovation and the Institute for Community Health Innovation. UAMS includes UAMS Health, a statewide health system that encompasses all of UAMS’ clinical enterprise. UAMS is the only adult Level 1 trauma center in the state. UAMS has 3,485 students, 915 medical residents and fellows, and seven dental residents. It is the state’s largest public employer with more than 11,000 employees, including 1,200 physicians who provide care to patients at UAMS, its regional campuses, Arkansas Children’s, the VA Medical Center and Baptist Health. Visit www.uams.edu or uamshealth.com. Find us on Facebook, X (formerly Twitter), YouTube or Instagram.###