VIDEO: SUSTAIN trial brings potential ‘disease-modifying’ treatment to sickle cell

SAN DIEGO — Results of the randomized phase 2 SUSTAIN trial could provide patients with sickle cell disease a much-needed treatment option, according to Nigel Key, MB, ChB, FRCP, chief of the section of hematology in the division of hematology/oncology at the UNC School of Medicine.

“With sickle cell disease, there’s only one licensed therapy for sickle cell disease – which is hydroxyurea – and the rest of our treatment modalities are really transfusion and sometimes bone marrow transplantation,” Key told HemOnc Today.

In this video, Key discusses the phase 2 results of the SUSTAIN trial which demonstrated the anti–P-selectin antibody crizanlizumab (SEG101, Novartis) significantly reduced frequency of pain crises compared with placebo in adolescents and adults with sickle cell disease.

Key acknowledges that although there is tremendous excitement around the results, physicians must make sure that the phase 3 study further validates the results.

“This is potentially a disease-modifying agent, or at least in the short-term gets at the concept of adhesion of cells which appears to be dependent on the P-selectin ligand and this is an inhibitor given by infusion that prevents cellular adhesion which is central to the pathogenesis of sickle cell disease,” Key said. “We will have to see how that develops.”

Reference:

Ataga KI, et al. Abstract 1. Presented at: ASH Annual Meeting and Exposition; Dec. 3-6, 2016; San Diego.

Disclosure: Key reports no relevant financial disclosures.