Sickle cell anemia is a condition where the body lacks enough healthy red blood cells to oxygenate the body. It is an inherited anemia, and both parents must carry the gene to pass the disease on to their children. The condition causes vision problems, delayed growth, frequent infections, and chronic pain. Treatments continue to advance as researchers seek a cure.
Sickle Cell Statistics
The exact numbers are hard to determine, but approximately 100,000 Americans are known to currently have sickle cell anemia (SCA). About 300,000 babies born in the world each year have the disease. One out of every 365 black infants is born with the condition in the United States. The life expectancy for SCA patients is 42 for males and 48 for females.
Black people are vulnerable to SCA because it developed in regions of the world where many had ancestors. The human body appears to have developed sickle cell to protect itself from the complications of malaria, and those that are carriers are less at risk to malaria. There are consistently higher numbers of SCA in areas of the world where malaria is common.
Sickle Cell Treatments
Patients with sickle cell anemia typically receive pain medication for comfort and antibiotics to treat their infections. Hydroxyurea is prescribed to many patients and has been effective at reducing complications, but it is not known if using the medication during pregnancy is safe or if there are any long-term complications from its use.
An L-glutamine oral powder is another medication for patients who are at least 5 years old. L-glutamine has been effective at reducing the effects of the disease. If used properly, it is also believed to be safe, and no serious side effects have been noted yet.
A bone marrow transplant, a potential cure for SCA, replaces damaged bone marrow with healthy stem cells. The difficulty of locating a matched donor for every patient and the potentially fatal risks associated with transplants make this an option that many cannot undergo or are hesitant to attempt.
A new transplant procedure that has shown promise is giving patients immunosuppressive drugs, along with a small dose of irradiation, before their bone marrow transplant. This method is used in place of the chemotherapy treatments once given to make room for the new cells. A study of 13 patients showed positive results from this method.
Gene Therapy Advancement
In 2008, researchers discovered a gene that causes the production of adult hemoglobin. When they prevented the gene from growing in mice, the blood cells continued to make fetal hemoglobin instead and the mice were cured of sickle cell.
It was reported in 2017 that gene therapy had cured a teenager of SCA after 15 months of treatment. The treatment is one that is useful for people that cannot find a match for a bone marrow transplant. Because the treatment is relatively new, there is no way to determine if it would be effective for all patients or what the long-term consequences may be.
Continued research is needed for any method that is thought to be a potential cure, and more adjustments are required to make the processes safer for the patients. However, the advancement in the last few years have been promising and many people are benefiting from the treatments.
Control and comfort are wonderful when people are suffering, but they are never as desirable as a cure. At Key Biologics (A Cellero Company), we are working to develop cures for diseases like sickle cell disease and malaria so people never have to suffer from one disease to protect themselves from another. Learn more about our efforts and discover how you can contribute to the effort.