Prof Pemmaraju talks to ecancertv at EHA 2016 about SL401, a novel agent to treat blastic plasmacytoid dendritic cell neoplasms (BPDCN).
He details the history of the disease and unusual constellation of symptoms, before introducing SL401 - a truncated diphtheria toxin attached to IL3, corresponding to over-expression of IL3R on BPDCN cell surface.
In phase I and dose escalation trials, Dr Pemmeraju reports a significant response, with further trials now enrolling patients, and discusses the growing availability of immunopathogens.
He also highlights the role of public engagement and social media in reaching healthcare providers and those affected by the disease, who may not otherwise know of this rare disease.
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