Trials Begin on New Sickle Cell Disease Drug

27 June 2010 (Last Updated June 27th, 2010 18:30)

A Phase II trial of GMI-1070 as a treatment for vaso-occlusive crisis of sickle cell disease has begun, after the US Food and Drug Administration granted the drug a fast-track designation. Vaso-occlusive crisis is a life-threatening condition where sickle cells get stuck within a blood v

A Phase II trial of GMI-1070 as a treatment for vaso-occlusive crisis of sickle cell disease has begun, after the US Food and Drug Administration granted the drug a fast-track designation.

Vaso-occlusive crisis is a life-threatening condition where sickle cells get stuck within a blood vessel, impeding blood flow.

The study is designed to measure the effects of GMI-1070, a pan-selectin inhibitor, in blocking the inflammatory process underlying cell adhesion, thereby reducing the duration and intensity of vaso-occlusive episodes.

It will also measure the efficacy, safety and pharmacokinetics of GMI-1070 in hospitalised sickle cell disease patients experiencing vaso-occlusive crisis.

The drug is developed by GlycoMimetics, a clinical-stage biotechnology company manufacturing.