Sickle Cell Disease

Sickle cell anemia is a disease passed
down through families in which red
blood cells form an abnormal crescent
shape. (Red blood cells are normally
shaped like a disc.)



L-GLUTAMINE TESTED IN SICKLE
CELL TRIAL
Researchers throughout the U.S. have
begun administering the sickle cell
treatment developed by investigators
led by Yutaka Niihara, MD,  at LA
BioMed and licensed to Emmaus
Medical, Inc. The patented drug
treatment involves the oral
administration of L-glutamine, which is
the most common amino acid in the
body. This is one of a very few
experimental treatments for sickle cell
disease to reach the Phase 3 clinical
trial stage.

Sickle cell disease is an inherited
disorder that causes red blood cells to
become oxidized, sticky and sickle
shaped instead of smooth, pliable and
round. Sickle cell disease leads to
anemia, organ damage, chronic and
acute pain and a host of other
problems.

In the Phase 3 clinical trial,
researchers at 20 to 25 sites around
the country will be seeking up to 225
research volunteers, age 5 years and
older, with a diagnosis of sickle cell
anemia or sickle beta O-thalassemia
who have a history of at least two
episodes of painful crisis during the
past 12 months. The trial is a 53-week
study requiring monthly visits to the
research facility. It is funded by
Emmaus Medical.



BONE MARROW TRANSPLANTS FOR
ADULTS TESTED IN SICKLE CELL
TRIAL
Bone Marrow Transplants May Cure
Sickle Cell in Adults not only in children.
John Tisdale of the US National
Institute of Diabetes and Digestive and
Kidney Diseases in Bethesda,
Maryland, and colleagues had done a
small study. A previously incurable
blood disorder – sickle-cell disease –
has been successfully treated in 9 of
10 adults who received stem cells
transplanted from tissue-matched
siblings.