Novel drug prevents AML cells from hiding

It’s generally accepted that leukaemic stem cells can lodge in the protective bone marrow niche, thereby escaping chemotherapy and with time potentially initiate a relapse.

GMI-1271 is a novel E-selectin (adhesion molecule) antagonist, designed to block E-selectin from binding to AML cells in the bone marrow. GMI-1271 disrupts the protective bone marrow microenvironment so that AML cells can't 'hide', therefore inducing sensitivity to standard chemotherapy.

The ongoing clinical trial has so far shown GMI-1271 to be well-tolerated with a high remission rate in combination with induction chemotherapy (cytarabine and idarubicin) in patients with relapsed/refractory AML.

The first newly diagnosed AML patient has now been enrolled in the phase 2 portion of the study, opening up for new treatment possibilities for patients with otherwise very limited options.

The importance of combination therapies in the treatment of haematological cancers

This key study has highlighted the importance of combining therapies, in this case a target specific E-selectin antagonist with already established chemotherapy. At Epistem we can offer several orthotopic AML animal models, where cytarabine is validated as standard of care, allowing for a combination therapy approach to drug development. In addition, we can add in vivo imaging, to monitor treatment effects in the same animal over time and to detect treatment potentiation and disease regression after introduction of additional treatments.

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