Results from research into an experimental therapy called SL-401 for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm have been shared at the Congress of the European Haematology Association. The oral presentation’s full abstract, which this article is based on, is available here.
Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
BPDCN, previously known as natural killer cell leukaemia or lymphoma, is a type of acute myeloid leukaemia. According to the Leukaemia and Lymphoma Society, not much is currently known about BPDCN, and there is not an established method of treatment. Furthermore, the condition is often misdiagnosed as another form of cancer, such as non-Hodgkin lymphoma or melanoma.
The average age for BPDCN to be diagnosed is between 60 to 70 years old, and, in about 80% of cases, it affects the skin. However, as the condition develops it is likely to also involve the bone marrow.
The Leukaemia and Lymphoma Society says that current treatment options for patients often involve chemotherapy, although there is not currently an agreed upon standard first treatment. However, relapses are common and new treatment options for patients are urgently needed.
About the Study of SL-401
SL-401 is a targeted therapy that is designed to affect the interleukin-3 receptor-α and work as a treatment for BPDCN. It has been awarded Breakthrough Therapy Designation for this purpose.
A pivotal Phase 2 clinical trial of SL-401 was carried out in a total of 45 patients with BPDCN to investigate how safe the experimental therapy is and its efficacy. It took part at seven sites and was comprised of three stages.
The study met its main goal with seven out of the thirteen participants in stage three achieving a complete response or a clinical complete response (defined as the absence of gross disease with minimal residual skin abnormality). Over all three stages, the side effects of SL-401 was consistent, and a BLA submission is currently planned.