According to this press release, a venerable blood research group has made a breakthrough in fighting sickle cell disease:
The research team led by Donald Abraham, Ph.D., of Biological and Medicinal Chemistry, in the Department of Medicinal Chemistry in VCU’s School of Pharmacy, has shown that 5-HMF, a pure compound developed by the team, has a high affinity for sickle cell hemoglobin and holds promise for the treatment of sickle cell disease.
“Our findings suggest that this anti-sickling agent may lead to new drug treatments and may one day help those suffering with sickle cell disease. This molecule, 5-HMF, is the most promising molecule to treat sickle cell anemia to come from our research group in more than 30 years,” said Abraham, who is also the director of the Institute of Structural Biology and Drug Discovery.
…Sickle cell disease is caused by an abnormality in the hemoglobin molecule. Normal red blood cells carrying hemoglobin are smooth, round and flexible and can travel easily throughout blood vessels. However, sickle cells are stiff, abnormally shaped, red blood cells that do not flow freely through blood vessels. The sickle cells also may clot together causing a blockage to form which results in pain and potentially dangerous complications that can compromise a patient’s organs.
According to Abraham, the 5-membered, heterocyclic, anti-sickling agent binds to hemoglobin to increase the oxygen affinity of both normal and sickle hemoglobin. In a patient with sickle cell disease, the binding action of 5-HMF would allow sickle cells to move more smoothly throughout the blood vessels of the body and prevent blockages from forming.
There is apparently very little toxicity to this drug, and the FDA has granted a “notice of allowance” for further study.
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