Sickle Cell Disease Drug Trial to Begin

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Dr. Joel Linden, an expert in sickle cell disease and research, will conduct a new clinical study assessing the drug Lexiscan and its effects on combating some aspects of sickle cell disease (otherwise known as sickle cell anemia).

Researchers will combine efforts from the La Jolla Institute for Allergy and Immunology, the Dana-Farber Cancer Institute in Boston, and Washington University in St. Louis to conduct the study. Patient recruitment is underway. In the first year, researchers will work with patients who have sickle cell disease but no pain crises and then later that year will add patients who have pain crises and acute chest syndrome. The second year, if the first goes well, researchers will include children age 14 and older.

Sickle Cell Anemia affects more than 70,000 people in the United States, and several million worldwide. The disease affects those primarily of African and Hispanic descent. Others affected by this disease include those with Middle Eastern, Asian, Indian, Latin American, Native American, and Mediterranean descents, although to a lesser degree.

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Sickle cell disease happens when the cells become “sickled” or stiff, sticky and misshapen. The body is unable to carry these cells through its smaller blood vessels leading to the diseases symptoms which include pain crises, acute chest syndrome and death. Those with sickle cell disease do not often live beyond their mid-40s or 50s. The most common complication resulting in death is the pulmonary complications. This is what Dr. Linden hopes to treat with Lexiscan.

Lexiscan has been used as an alternative to exercise-based stress tests for heart patients who could not withstand the physical activity. Rather than injecting patients with Lexiscan on the same schedule though, Dr. Linden plans to do smaller doses over longer periods of time. There will be two different test doses given over 12 or 24 hour periods to avoid toxicity. It is believed that Lexiscan will combat the inflammation found in patients with sickle cell disease and acute chest syndrome.

Dr. Linden, while working with others, has already discovered the cause of the inflammation, namely invariant natural killer T cells, or iNKT cells for short. In these other studies, Dr. Linden discovered that adenosine (a similar component to Lexiscan’s active ingredient) dramatically reduced the inflammation in mice with sickle cell disease.

Perhaps the most famous person with sickle cell disease is singer and rapper, Tionne Watkins, better known as T-boz from the R&B group, TLC. She became spokeswoman for the Sickle Cell Disease Association of America in after she came out about her battle in 1996. She wanted to bring awareness to the disease and urge further research into the disorder to continue.

For more information: please visit the SCDAA website

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