Drug could slash cost of treating sickle cell anemia

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Sickle cell anemia is primarily seen in people of African and Mediterranean descent. It can also be seen in folks from South and Central America, the Caribbean and the Middle East.

It is an inherited trait and blood tests can be taken to determine if a person carries that trait.

In the event that two people were contemplating having children and they found they both had the trait they would want to know each of their children would have a 25 percent chance of having sickle cell disease.

The normal red blood cell is round, but in sickle cell anemia it can be shaped like a crescent or sickle and is much more fragile than a normal red blood cell. The manifestations of the disease can be quite variable. It can include what has been called a crisis that may be accompanied with severe pain.

The only known cure at this time is a bone-marrow transplant however that in itself can pose serious problems so it is not often done. In the U.S. the cost for treating the disease can exceed $20,000 per year.

Although there is no known cure there is strong hope that a simple remedy may be able to cut the costs in half and reduce the pain the people experience.

A 1986 graduate of Loma Linda University School of Medicine, Dr. Yutaka Niihara is the lead investigator for a drug called L-glutamine. He grew up in Japan and was initially not serious about school. He stated, “I just wanted to fish all day long.” However upon hearing about the great medical missionary Albert Schweitzer he told his mother he wanted to study medicine.

She arranged for him to come to the U.S. and he ended up at Loma Linda University. After completing medical school he specialized in hematology and oncology — fields focusing on blood and cancer. During his training he saw many patients with sickle cell disease who reported episodes of severe pain and requested heavy narcotics.

At first he thought they were just drug seekers. But as time passed he began to understand how real their pain was.

The project to try L-glutamine begin 1992. At this point it should be noted that this substance is a simple amino acid that normally occurs in small amounts in a normal diet.

But it has been used for other things such as building muscle and enhancing sports performances. Some folks have wondered if such a simple drug would really relieve such intense pain. So far the answer seems to be yes.

The 1992 beginning for the use of L-glutamine for sickle cell anemia was phase I. Phase III appears to have been completed with good results. The Food and Drug Administration scheduled a meeting for Nov. 5 to discuss the results of Phase III and to consider what the next steps should be. I have not been able to determine what decisions have been made.

Dr. Niihara believes this drug will provide a way to alleviate the pain effectively and inexpensively, thus cutting treatment costs by half.

The information for this column was gleaned from the October-December 2012 issue of the Alumni Journal for the Loma Linda School of Medicine.

Dr. Don Casebolt of College Place is a retired physician who is passionate about preventive medicine. He spent four years as a medical officer in the U.S. Navy, the last 2 1/2 years as a flight surgeon. He also worked on the Navajo Reservation for 22 years.

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