First New Sickle Cell Drug in Over 20 Years brings Hope and Anticipated Relief to Many

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By D.L. White

A common concern for individuals living with sickle cell anemia is the onset of an attack, when the pain becomes so excruciating; individuals ball up in fetal positions and attempt to rock themselves until the pain diminishes.

Aspirins, Tylenol, Excedrin, are no relief when a sickle cell attack comes on.

A second concern, for those living with the disease, uninformed medical staff who often (if not aware of the patient) falsely assume the individuals is seeking drugs, thus making a difficult situation even more difficult with a lengthy battery of integration tactics.

Ideally, those days will soon be unpleasant memories from a day far spent. A new medication is on the horizon and will soon be available.

According to a recent report from the U.S. Food and Drug Administration, a new drug to address the effects of the blood disorder known as sickle cell anemia has been approved.

Endari, is the name of the drug that has passed laboratory trials and is approved for individuals five years and older, to reduce the complications associated with sickle cell anemia.

The drug consists of L-glutamine, an amino acid which is usually deficient in individuals with the blood disorder.

Dr. Alexis Thompson, head of the Hematology Section and Director of the Comprehensive Thalassemia Program at the Ann and Robert Lurie Children’s Hospital in Chicago, stated, “I am hoping we are finally seeing channels opening and this will be the first of many new drugs to hit the market to address this disease.”

According to the Sickle Cell Foundation of Georgia, sickle cell disease affects more than 100,000 Americans, with an estimated annual cost in medical care of 1.1 billion dollars.

Sickle cell disease, which predominately affects African-Americans, Latinos and other minority groups, in the past decades have been identified in other groups as well.

The blood disorder is inherited when two individuals with abnormal hemoglobin genes (one from each parent) pass the trait and or disease to the child.

Normally round and disc-shaped red-blood cells are instead cresant or sickle shaped due to abnormal hemoglobin. The red cells do not have enough of the amino acid L-glutamine, which makes the cells more fragile, and prone to clog blood vessels.

Endari works by increasing the levels of L-glutamine in the blood, thus causing less sickling and clogging.

The National Heart, Lung and Blood Institute, report that one in (13) African American babies are born as a genetic carrier of sickle cell (regarded as the trait).

One in every (365) African American children are born with the actual disease, as a result every state and the District of Columbia is required to screen every baby for sickle cell disease and sickle cell trait.

Dr. Tyeese Gaines, another of the researchers in the sickle cell study noted, “Endari was studied in patients age five to 58 years of age. It was confirmed that those patients who received the medication had few hospitalizations and shorter hospital stays than those who only took the placebo.”

“We clearly have much more room for improvement in what we can provide for people with sickle cell disease and while we are clearly very excited to see another drug reach the marketplace, we need more,” affirmed Dr. Thompson.

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