Global Blood Therapeutics, a biotechnology company, is developing GBT440, an experimental drug which has been shown in laboratory studies to stop polymerization of sickle hemoglobin and the sickling of red blood cells. Sickle hemoglobin polymerization is the key event that leads to the clinical effects and organ damage in sickle cell disease.

Global Blood Therapeutics recently announced the initiation of a clinical trial of GBT440, an experimental drug, taken by mouth once a day, being investigated for the treatment of sickle cell disease (SCD). This experimental drug has shown potent anti-sickling activity by blocking polymerization of sickle hemoglobin in laboratory experiments with human sickle cell blood and in a mouse model of sickle cell disease. The study is being conducted at Quintiles drug research unit at Guy’s Hospital in London and several London area haematologists will be referring their patients to participate in the study, including Dr. Jo Howard, consultant haematologist at Guy’s Hospital and Dr. Paul Telfer, consultant haematologist at Royal London Hospital.

‘’Despite comprehensive care and the increasing use of hydroxycarbamide (hydroxyurea), sickle cell patients continue to experience intermittent episodes of severe pain and also develop other acute and chronic complications of sickle cell disease” said Dr Jo Howard. “We are in desperate need of new treatments which will modify the disease and improve our patients’ quality of life.”
“Through this study, we will learn whether GBT440 could help patients with SCD by addressing the fundamental problem of the disease- sickle hemoglobin polymerization and the red blood cell sickling process, which is hoped to translate to clinical benefits,” said Dr. Paul Telfer.

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