SICKLE CELL BREAKTHROUGH
Researchers at Duke University have made a potentially dramatic advance in the study of sickle cell disease.
The scientists now say they are beginning to understand what happens, on the cellular level, to patients with this often debilitating inherited disease.
For Gwendolyn Pittman and more than 70000 other Americans, living with sickle cell disease is living with pain that never quite goes away.
Gwendolyn Pittman, a sickle cell patient, says, “There is a constant pain in my chest, in my legs. I practically expect to see distortions in my body because the pain is so unbearable.”
The pain comes from small blood vessels clogged with sickle cells, or red blood cells unable to pass through because of their unusual crescent shape.
Now researchers at Duke University have discovered another key flaw of the sickle cell that could lead to a dramatic treatment.
Dr. Jonathan Stamler, lead researcher at Duke, says, “The latest study shows that a red blood cell from a sickle cell patient is unable to dilate a blood vessel and the reason for that is that it is unable to release nitric oxide.”
So the vessel stays constricted – the sickle cells clog it and the surrounding tissues are deprived of blood and oxygen.
It’s a catch-22 situation. The blood cells won’t sickle if the blood can flow and the oxygen can get through. At the same time the blood can’t flow unless the blood vessels remain open, and they can’t remain open because the sickle cells don’t produce the nitric oxide that keeps them open.
The promise in this discovery may lie in the fact that nitric oxide is a common gas found throughout the body.
“We think we may really have an avenue for the first time to make a contribution to the treatment of these patients,” says Stamler.
Dr. Jonathan Stamler and his colleagues believe that by simply adding nitric oxide to the patient’s blood, the clogging of the vessels might be avoided. Although it won’t change the shape of the sickle cells, there’s evidence it will help keep the vessels wide open, and wider vessels mean better blood flow, less pain and fewer complications.
Dr. Marilyn Telen, a sickle cell expert at Duke University Medical Center says, “We’re going to be seeing clinical trials of methods of affecting those pathways within the next few years. Realistically it may be some more time before we get these things FDA approved.”
The researchers say there are drugs right now that are on the market, used for other purposes, which are believed to be able to provide the nitric oxide to the blood cells. However, before they are used, research needs to be done on how to best utilize their power. Specifically, the drugs that could be used can become useless, because the patients get tolerant to them. This is something that the researchers are working on right now.
Regardless of the time frame, any relief from the pain would be welcome news to Gwendolyn Pittman and Andrew Powell.
“I never thought that something would come along that would make it go away,” says Gwendolyn.
“I sort of look at sickle cell as a challenge,” states Andrew.
Sickle cell disease certainly is a challenge- a life-long challenge that could just be getting a bit easier.
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