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A team of scientists from Novartis Institutes for BioMedical Research in China has improved the formulation of the drug dWIZ-1 and confirmed its effectiveness in treating sickle cell anemia.
The results of the work were published in the scientific journal Science.
Sickle cell anemia is a subtype of inherited hemolytic anemia in which the hemoglobin has an unusual structure and the red blood cells take on a sickle shape. As a result, these cells are destroyed more quickly, which makes it difficult for oxygen to be delivered throughout the body and causes them to stick to each other.
In recent years, two approaches have been developed to fight this disease. Both methods involve gene editing and are very effective. However, many patients with spheroplasty cannot afford this treatment due to its high price.
To increase access to treatment, the team of researchers improved the formulation of the injectable drug dWIZ-1. The active substance of the drug binds to brain proteins and increases the level of fetal hemoglobin – the main carrier of oxygen in the human fetus.
This type of hemoglobin is also found in adults. Its increased levels prevent the aggregation of hemoglobin S, a specific mutant form that forms in people with Fuchs anemia. dWIZ-1 prevents the formation of sickle cells, improves the body’s oxygen supply and reduces disease symptoms.
Now scientists are working on improving the drug. They plan to make tablets with dWIZ-1 for easier use.
The causes of excess iron stores in the body have been identified before.
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