SuperGen Inc announced that a clinical study suggests that its anticancer agent Nipent (pentostatin for injection) is active in treating children diagnosed with chronic graft-versus-host disease (cGVHD) refractory to standard therapy. Nipent is approved as a single-agent treatment for patients with hairy cell leukemia. Nipent is not approved in children, in any combination regimens or as a treatment for any form of GVHD.

Principal Investigator Georgia B. Vogelsang at Johns Hopkins Hospital conducted the clinical study in five patients ages 22 months to 12 years that were diagnosed with either active or progressive cGVHD that was refractory to standard therapy. The primary endpoint was response to therapy, defined as improvement of symptoms. All patients were also observed for toxicity.

Study researchers reported the following results: 'considerable symptom response' in each patient (every patient demonstrated a significant improvement in skin and oral symptoms); an increased incidence of infection secondary to Nipent was not observed; and, no patient was permanently discontinued from Nipent subsequent to side effects.

"Our early experience with treating children with pentostatin has been encouraging, with significant improvement of the most serious symptoms in every patient," said Dr. Vogelsang. "If additional studies confirm these results in a small number of patients, a trial of pentostatin as front-line therapy may be indicated."

No patients experienced significant infections that were attributed to immunosuppression resulting from Nipent therapy. Mild nausea/vomiting, responsive to antiemetics, and fatigue affected most patients at various stages of treatment. Gastrointestinal symptoms prompted discontinuation of the drug in only one patient, who suffered unrelated gastrointestinal symptoms that were possibly complicated by nausea secondary to Nipent.

"These findings, along with previous clinical data in adults, reinforce our belief that Nipent is an active compound in treating graft-versus-host disease," said Charlene Holt, Medical Director, Commercial Operations at SuperGen. "Looking ahead, we hope to initiate an expanded clinical study in this indication."

GVHD, perhaps the most serious complication arising from bone marrow transplant, is a syndrome wherein immune cells from the transplant donor reject the recipient's normal tissue following an allogeneic (from a donor rather than one's self) bone marrow transplant (BMT). Chronic GVHD develops in 35 to 50 percent of all patients after transplant and is a significant cause of morbidity and mortality. It is currently treated unsuccessfully with various immunosuppressive therapies. While the incidence of cGVHD in children has been reported to be lower than the incidence in the adult population, the morbidity is even more significant in children, as the effects may remain with the child lifelong.