When David Williams was a newly minted pediatrician 30 years ago, he divided his caseload between sickle cell and hemophilia patients. Though hemophilia treatments are virtually cures, sickle cell patients still experience uncontrollable bleeding. Williams devised an experimental therapy based on the genetics of sickle cells. He argued that by knocking out enough of the gene BCL11A, the disease could be averted. While therapies to prevent the flipping of BCL11A are far from perfect, they are an important step in a cure for sickle cell anemia.
Transfusions cure sickle cell anemia
The use of blood transfusions can be effective in curing sickle cell anemia. These blood transfusions deliver healthy red blood cells to sickle cell disease patients. Because red blood cells cannot eliminate iron themselves, repeated transfusions can cause an accumulation of iron. When this happens, excess iron accumulates in organs and can lead to damage. Fortunately, treatments have emerged to prevent iron overload and cure sickle cell anemia.
Blood transfusions are one of the most common treatments for sickle cell disease. They can be given for acute complications or chronic disease-modifying treatment. Although transfusions are not the best cure for sickle cell anemia, they are extremely helpful in certain situations. For example, blood transfusions can help protect infants from brain damage and may reduce the risk of seizures or a heart attack. While there are many complications associated with blood transfusions, in many cases they can be lifesaving.
In addition to lowering the concentration of sickled red blood cells, blood transfusions can help increase the number of normal red blood cells. Increased red blood cell count helps increase oxygen supply to the body. Patients who have undergone several transfusions are more likely to experience iron overload. This condition can lead to liver and heart failure if left untreated. The amount of iron in the body’s tissues is assessed using a blood ferritin test.
Bone marrow transplant is cure for sickle cell anemia
The only known cure for sickle cell anemia is a bone marrow transplant. In this procedure, healthy stem cells from the bone marrow of a donor are used to replace the sickle cells in the patient’s blood. Healthy stem cells from the donor’s bone marrow grow into normal red blood cells. The transplant can help the patient reverse the effects of sickle cell anemia and lead to a full recovery.
Before undergoing a bone marrow transplant, patients must undergo chemotherapy. This medication destroys cells that produce abnormal blood cells, weakening the immune system so it does not attack the new stem cells. In addition to chemotherapy, a patient may also receive radiation therapy. After the transplant, the new bone marrow should start making healthy blood cells. The care team will monitor the new cells for a month.
The procedure has been effective in curing sickle cell anemia for many people. Patients with sickle cell disease receive stem cells from a healthy sibling. These cells grow new, healthy blood cells, eliminating all symptoms of sickle cell anemia. Because the stem cells are not from the patient’s own bone marrow, patients must take immunosuppressive drugs for a year after the transplant.
Genetic therapies may provide new treatments or cure for sickle cell anemia
Scientists are now testing gene therapies in children and adults with sickle cell anemia. The gene therapy encourages the production of fetal hemoglobin, the form of hemoglobin that young children and fetuses naturally produce. The virus delivers DNA for making microRNA into bone marrow cells, where it becomes permanently inserted into the cell’s genetic blueprint. This interferes with the production of protein, preventing the production of sickle cells. In turn, this inhibits the production of sickle cells and turns on their production of fetal hemoglobin.
A recent study by Harvard Medical School and Boston Children’s Hospital suggests a new treatment for sickle cell anemia. This treatment encourages the body to produce more normal hemoglobin, a molecule necessary for carrying oxygen. The human body’s red blood cells naturally produce normal hemoglobin, but a malfunctioning gene results in sickle cells. The healthy form of hemoglobin is called fetal hemoglobin, and is produced in infants, but it cannot be made by adult blood.
Gene therapy works by replacing damaged genes or turning off genes in a patient’s cells. It has the potential to cure certain blood cancers, including sickle cell anemia. However, despite some concerns and potential benefits, these therapies have had mixed results in human trials. Some failed trials resulted in deadly side effects. While this is a positive sign, further research is needed to determine if gene therapies are safe and effective.
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