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New sickle cell anemia therapy advances to Phase II clinical trials
Date:6/12/2013

e print edition of the journal Blood on April 25th, 2013.

David G. Nathan, M.D., president emeritus of the Dana-Farber Cancer Institute, who is the trial's co-lead investigator with Dr. Linden and Joshua Field, M.D., of the BloodCenter of Wisconsin, says he is excited by the prospect of reducing some of the worst symptoms of sickle cell disease, particularly periodic disease exacerbations that lead to severe pain or breathing problems, known as vaso-occlusive crises and acute chest syndrome, respectively. Patients typically do not live beyond their late 40s or early 50s, with pulmonary problems being the most common cause of death.

"Pulmonary complications of sickle cell disease can be fatal because blood flow problems restrict the amount of oxygen going to the lungs," says Dr. Nathan, former physician-in-chief at Boston Children's Hospital and one of the world's top sickle-cell experts. "We are hopeful that the drug will reduce pulmonary injury and extend the lives of sickle cell patients."

In sickle cell anemia, the oxygen-carrying red blood cells become stiff, sticky and misshapen or "sickled" in appearance. This leads to poor blood flow that deprives tissues of oxygen and causes symptoms such as severe pain, difficulty breathing and damage to multiple organs. An inherited disorder, sickle cell anemia occurs in individuals who inherit two copies of the sickle cell geneone from each parent. It is most common in persons of African descent, but is also found in Hispanics of Caribbean ancestry, and people of Middle Eastern, Asian, Indian, Latin American, Native American, and Mediterranean heritage.

"I treat patients with sickle cell anemia in my clinic every day," says Dr. Field. "It is a devastating disease and therapies for the two most common complications, pain and acute chest syndrome, are very limited. Lexiscan has the potential to help people with sickle cell anemia by decreasing the severity of these life-th
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Contact: Bonnie Ward
contact@liai.org
619-303-3160
La Jolla Institute for Allergy and Immunology
Source:Eurekalert

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