Sickle Cell Treatment Developed At LA BioMed Approved For Phase 3 Clinical Trial
Main Category: Blood / HematologyAlso Included In: Regulatory Affairs / Drug Approvals; Clinical Trials / Drug Trials
Article Date: 24 Apr 2009 - 5:00 PDT
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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.org
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LA BioMed
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Sickle Cell Anemia
posted by Dan on 24 Apr 2009 at 5:41 amSickle Cell Anemia (SCA) has been called an autosomal recessive genetic disorder, which means that one is born missing a chromosome to share DNA with necessary for proper function.
SCA causes chronic hemolytic anemia, which unlike anemia itself, actually destroys the red blood cells within a SCA patient, and not simply is depleted of such cells as is the case with anemia in general.
SCA affects almost entirely the black population for reasons unclear as far as the race goes, but the disease is in fact due to a mutated gene assigned to regulate hemoglobin within red blood cells.
The red blood cells (RBCs) take on a rigid and sickle shape in the bloodstream of those affected. SCA was first discovered around 1950, and researched as well afterwards.
SCA typically presents itself in one’s childhood, as this anemia is rather ruthless in the damage it cause various areas of the human body.
The average life expectancy of A SCA patient is in the mid 40s presently.
The low life expectancy is due to the multiple clinical consequences that occur due to SCA. The hemolysis that occurs causes excessive destruction of one’s red blood cells.
As a result the spleen of the SCA patient becomes impaired due to the overload of red blood cells in now has to remove from the blood stream of the SCA victim.
And the SCA victim has to rely on his own bone marrow to reproduce additional red blood cells destroyed because of this sequence of events.
Approximately 70 thousand people are with SCA in the United States presently, and all experience this self destruction within them due to SCA.
Symptoms that happen due to SCA include what is called a pain crisis, anemia, infections, spleen inflammation and damage, and infections due to decreased blood flow to various parts of the body.
With SCA children, they are particularly at a high risk for both a stroke and lung disease as a result of this disorder.
Treatment options for SCA include hydroxyurea, which is an anti-metabolite that has the ability to reduce the number of sickle cells present in a SCA patient as well as increases their hemoglobin levels- both of which are needed with the SCA patient.
SCA patients report a deceased number of pain attacks on hdroxyurea as well. Blood transfusions occur at times with SCA patients- perhaps every few weeks. Pain management is needed for the SCA patient often at times.
There is ongoing research regarding sickle cell anemia, and there is hope that better treatment options, if not a cure, will be created sooner rather than later. Because in this certain disease, anemia does not only mean one who is without blood.
This anemia means the SCA victim is also without comfort, and often, without peace.
Dan Abshear
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