A few weeks ago while doing my usual research on sickle cell disease, I came across an exciting online article on sciencemag.org about a promising new drug called Voxelotor.
This is an investigational drug being developed as an 'oral, once daily therapy for patients with sickle cell disease'. It is believed that Voxelotor will block the sticking together aka polymerization of oxygenated sickle hemoglobin and the resultant sickling of red blood cells.
Because there is an obvious need for new treatments with scd, the FDA has granted approval study use of Voxelotor as a treatment for patients with sickle cell. Previously called GBT440 (Global Blood Therapeutics), Voxelotor aims to restore normal red blood cell function and oxygen delivery. It should also help reduce the risk of of pain crises caused by sickle cells blocking blood vessels. Although this drug is still currently being evaluated for sickle cell treatment, initial results from clinical trials showed an increase in hemoglobin with a profound reduction in hemolysis.
Further demonstrations of the drug's safety and its capacity to modify the course of sickle cell anemia are being investigated in 2 clinical trials. So far, the most common adverse effects associated with the use of Voxelotor are headaches with no further serious effects recorded. It is also being studied as a potential therapy to treat other conditions like hypoxemia.
The sickle cell community will definitely welcome the introduction of this new drug that aims to treat and significantly manage sickle cell and its attendant painful crisis episodes. Hopefully, all ongoing trials to confirm the efficacy and safety of Voxelotor will result in the FDA finally granting its approval for its use in the alleviation of the pain and suffering of sickle cell patients.
For more details on this potential new drug, see HERE
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