For sickle cell disease, two therapies have been developed for treating the disease. These are gene editing methods that have been proven effective. Few people could afford these two treatments as the costs are extremely high. Pharmaceutical giant Novartis in the field of medicine and research for novel therapies have decided to find another way to treat this disease. Their work is now published in the Science journal, they have decided to conduct a tiring study that would find out the degrader of the WIZ transcription factor for fetal hemoglobin induction and used animal models as a test study.
They have worked on finding and developing a drug that has potential to ‘switch on’ fetal haemoglobin in adult. Researchers have a belief that this could cure patients by change in the oxygen transportation pathway. Unfortunately, so far, the results have arrived back with the hazardous negative side effects like bone marrow suppression that negatively impacts the stem cell production and this leads to anaemia.
Sickle cell disease occurs when the red blood cells that are crescent-shaped have problems transporting oxygen. Douglas Higgs and Mira Kassouf with the University of Oxford have published a groundbreaking piece on the work done by the research team. The Novartis research team is confident in a compound that could be manufactured in a pill form that can potentially reduce the symptoms of sickle cell disease.
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