Advancing Blood Disorder Treatments Through Precision Therapeutics

In this week's episode, Blood editor Dr. Laura Michaelis interviews authors Drs. Terri Parker and Peter Lenting on their latest papers published in Blood Journal. Dr. Lenting discusses his work on introducing a new therapeutic approach to von Willebrand disease with the development of a novel bispecific antibody (KB-V13A12) that links endogenous mouse VWF to albumin, extending VWF half-life twofold with cessation of provoked bleeding. Dr Parker shares the results of a 43-patient phase 2 study that evaluates the single agent isatuximab, a CD38 monoclonal antibody, in patients with relapsed/refractory AL amyloidosis. With a hematological response rate of 77%, organ response rates between 50 and 57%, and an excellent safety profile, the current study lays the foundation for future use of isatuximab across treatment settings and combination strategies.

Featured Articles

• Isatuximab for Relapsed and/or Refractory AL Amyloidosis: Results of a Prospective Phase 2 Trial (SWOG S1702)
• A bispecific nanobody for the treatment of von Willebrand disease type 1