2024-07-09 17:15:00
A large group of medical researchers at Novartis Biomedical Research is testing a possible treatment for people with sickle cell anemia. In their project, published in the journal Science, they conducted a comprehensive study to find a suppressor of the transcription factor WIZ for the induction of fetal hemoglobin and tested it in animal models.
Sickle cell disease is caused by a mutation in the gene responsible for creating adult hemoglobin. The change causes the red blood cells to become the rim, instead of the discs, which causes breathing problems and the cells stick together, causing pain.
In recent years, two therapies have been developed to treat the disease. Both are gene editing and have proven to be very effective. Unfortunately, both are so expensive that few people can afford them. For this reason, medical research groups have continued to find other ways to treat the disease.
One of the main lines of research revolves around the development of a drug that can activate fetal hemoglobin in adults. Researchers believe that this drug will offer a cure to people who are infected by taking oxygen in a new way. Unfortunately, the resulting treatments have negative side effects, such as suppression of bone marrow cell production, leading to bleeding.
In this new effort, the team set out to find a substance that would bind to the cereblon protein and finally found one called dWIZ-1 and, after repeating it, discovered that its administration to animal models carried fetal hemoglobin levels rise from the baseline value. 17% to 45%, a level the group believes will reduce symptoms in humans.
Further testing of the compound is needed to ensure that it does not cause any negative side effects, but the team is confident that they have found a compound that can be turned into a drug and will reduce the symptoms of sickle cell disease.
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