Blood Podcast Advancing Blood Disorder Treatments Through Precision Therapeutics
Nov 20, 2025
Terri Parker is a hematologist and clinical researcher focusing on AL amyloidosis, while Peter Lenting specializes in bleeding disorders. They discuss groundbreaking advancements in treatments for these conditions. Parker reveals impressive results from a phase 2 trial using isatuximab in AL amyloidosis, showcasing a 77% hematologic response. Meanwhile, Lenting shares his innovative work on a bispecific nanobody designed to extend the half-life of von Willebrand factor, potentially transforming care for patients with type 1 VWD.
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Isatuximab Shows Strong Hematologic And Organ Activity
- Isatuximab monotherapy produced a 77% overall hematologic response in relapsed/refractory AL amyloidosis patients in this phase 2 trial.
- VGPR-or-better occurred in 57% and translated into cardiac responses of 57% and renal responses of 50%.
Run Rare-Disease Trials Via Cooperative Groups
- Use multicenter cooperative-group trials to study rare diseases like AL amyloidosis rather than single-center efforts.
- Enroll relatively fit patients (ECOG 0–1, early Mayo stage) to improve tolerability and completion rates in early-phase studies.
Data Too Limited On Prior Daratumumab Effect
- Only two patients had prior daratumumab exposure, limiting conclusions about cross-resistance between daratumumab and isatuximab.
- Further trials are needed to define isatuximab's role after frontline daratumumab becomes standard.