Originally published February 2 2006
New therapy gives hope to those suffering from sickle cell disease
by Mike Adams, the Health Ranger, NaturalNews Editor
At the Memorial Sloan-Kettering Cancer Center, researcher Selda Samakoglu and colleagues have pioneered a combination of gene therapy and RNA interference that purportedly cures sickle cell disease in lab conditions.
Lead researcher Selda Samakoglu and colleagues at the Memorial Sloan-Kettering Cancer Center noted the promising results after conducting an experiment using blood-producing stem cells from the bone marrow of patients with sickle cell anemia, then culturing the cells in the laboratory.
'I am quite hopeful that we will be able to start human trials in a little over a year,' Samokoglu told United Press International.
Sickle cell disease -- which affects about 80,000 people in the United States -- is a genetic condition that makes red blood cells produce abnormal hemoglobin and turn from their normal round disk shape to narrow sickle forms.
The only current treatment for the disease --which is mainly found in people of African, Mediterranean, Indian or Middle Eastern descent -- is a stem cell transplant from an outside donor.
After culturing the bone marrow-derived stem cells in the lab, the Sloan-Kettering researchers then put a small portion of RNA that interferes with the production of beta S-globin -- the part of hemoglobin that has been changed in people with sickle cell anemia -- and inserted it in a gene that promotes the production of healthy hemoglobin.
The finished product, called a transgene, was placed in a virus and introduced into the cell cultures, where the virus carried it into the stem cells of the sickle cell patients.
Samokoglu noted that her colleague Michel Satelain would start human trials on a similar procedure to treat thalassemia, another inherited form of anemia, in six months, and thought that performing those trials would help the team prepare for human studies of their sickle cell therapy.
'This sounds like a very innovative way to approach the problem of sickle cell disease, Minniti told UPI.
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