Note from Door: I know it's not the same as thal, but I remember me a lady who had thal and sc. (it could be the wrong forum though, but it might be interested for us too)
Sickle Cell Treatment Developed At LA BioMed Approved For Phase 3 Clinical Trial
24 Apr 2009
The Food and Drug Administration (FDA) gave its approval this week for Emmaus Medical, Inc. to begin a Phase 3 clinical trial of an experimental treatment for sickle cell disease. The patented experimental treatment was originally developed by investigators led by Yutaka Niihara, MD, MPH, at the Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed) and is licensed to Emmaus Medical.
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.
"News of this clinical trial should be of great interest to the millions of people around the world who suffer from painful crises caused by sickle cell disease," said Dr. Niihara. "Phase 3 clinical trials are large, randomized studies conducted at multiple sites to determine the safety and efficacy of a potential treatment. Phase 3 clinical trials are usually the last clinical trials conducted before FDA approval is obtained and the treatment can be made widely available to the patient population."
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 our Phase 2 clinical trial, we observed an excellent safety profile and positive trends in reducing the number of crises and hospitalizations in sickle cell disease patients," said Dr. Niihara. "We look forward to initiating the Phase 3 clinical trial and remain committed to developing a treatment for sickle cell disease."
Emmaus Medical, Inc. was founded in Torrance, CA in 2000 as a private company to develop new treatments for sickle cell disease and other rare diseases. Emmaus Medical works closely with LA BioMed in developing these treatments. Emmaus' first product, NutreStore™ [L-glutamine powder for oral solution], together with Zorbtive® [somatropin (rDNA origin) for injection] and a specialized oral diet, is the only FDA-approved therapy for short bowel syndrome. For more information, please visit
http://www.emmausmedical.com or call 310-214-0065.
About LA BioMed
LA BioMed at Harbor-UCLA Medical Center is one of the country's largest nonprofit independent biomedical research institutes. It conducts biomedical research, trains young scientists and provides community services, including childhood immunization and nutrition assistance. The institute's researchers conduct studies in such areas as cardiovascular disease, emerging infections, cancer, diabetes, kidney disease, dermatology, reproductive health, vaccine development, respiratory disorders, inherited illnesses and neonatology. LA BioMed is academically affiliated with the David Geffen School of Medicine at UCLA. For more information, please visit our website at
http://www.LABioMed.orgSource
LA BioMed
--------------------------------------------------------------------------------
Article URL:
http://www.medicalnewstoday.com/articles/147414.php