A potential new therapeutic approach for blastic plasmacytoid dendritic cell neoplasm (BPDCN), a very rare form of blood cancer with no standard treatment, continues to show promise as it moves through early clinical trials.
The Leukemia & Lymphoma Society (LLS) has a long history of supporting the development of SL-401, a targeted therapy that has shown significant anti-tumor effects. The current trials by Stemline Therapeutics are being sponsored in part by over $3 million in LLS funding through the Therapy Acceleration Program (TAP). TAP is a strategic initiative to partner with biotechnology companies and speed the development of new therapies.
BPDCN is a rare aggressive disease of the bone marrow and blood cells that has few effective treatment options. The disease is frequently misdiagnosed and under-reported and its overall incidence is extremely low, accounting for 0.44% of all hematologic malignancies.
Because there is no widely accepted protocol for BPDCN, patients are often treated as leukemia or lymphoma patients. Those who relapse after chemotherapy have a particularly poor prognosis. LLS considers its support critical to the development of this therapy.
Stemline announced encouraging data, including high response rates, from the early stages of a SL-401 trial at the recent American Society of Hematology (ASH) Annual Meeting. Results showed that multiple consecutive cycles of SL-401 produced an 86 percent overall response rate, with a 100 percent response in patients for whom this was the initial treatment and a 60 percent response in patients who didn’t respond to previous treatment or had their disease come back. There were four complete responses and four “clinical” complete responses (where there may still be some minimal residual skin abnormality).
Of nine responders treated at 12 ug/kg/day, five patients (including two relapsed/refractory patients) remain in remission and two additional patients were successfully bridged to stem cell transplant, which generally hasn’t been possible with existing therapies. The results demonstrate an acceptable safety profile with no cumulative side effects.
Stemline is hoping to advance the agent as an FDA-registered therapy for BPDCN as well as expand its potential for use with other diseases.
Newly diagnosed patients as well as those with relapsed/refractory disease are currently being enrolled in the expansion stage of the trial, and clinical studies are also open in additional malignancies including acute lymphoblastic leukemia (AML) and several high-risk myeloproliferative neoplasms (MPN).
To learn more about these or any other clinical trials, please contact LLS’s Information Resource Center at (800) 955-4572.