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Sickle Cell Center is studying new drug

Rainier Ehrhardt/Staff
Ayukebangha Agbobock, of Elberton, Ga., suffers from sickle cell disease and has seen benefits from taking part in a clinical trial at the Medical College of Georgia Sickle Cell Center.
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Ayukebangha Agbobock of Elberton, Ga., has spent equal time in the United States and Africa and is grateful he is being treated here for his sickle cell disease.

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Trial study patient Ayuk Agbobock (right) sits in an examining room with nurse Latanya Bowman at the Sickle Cell Center at Georgia Health Sciences University.  Rainier Ehrhardt/Staff
Rainier Ehrhardt/Staff
Trial study patient Ayuk Agbobock (right) sits in an examining room with nurse Latanya Bowman at the Sickle Cell Center at Georgia Health Sciences University.

"Next week I will be 48 years old," said Agbobock, who spent 24 years in Cameroon before coming to the U.S. "That's a real blessing, because I saw a lot of my counterparts with sickle cell pass away" from complications.

He is trying to do his part by participating in a clinical study at the Medical College of Georgia Sickle Cell Center on a potential new drug to treat the disease.

Sickle cell disease affects an estimated 100,000 people in the U.S. and is most common in those of African, Central or South American, Caribbean or Mediterranean descent.

In those suffering from the disease, the red blood cells -- or hemoglobin cells -- are abnormally shaped (sickled) and sticky and can clog small blood vessels, leading to pain crises and organ damage, according to the National Heart Lung and Blood Institute.

Despite the relatively small number of patients, hospitalizations for sickle cell disease cost nearly $500 million in 2004, according to the U.S. Department of Health and Human Services' Agency for Healthcare Research and Quality.

"In terms of the medical economic impact, it's huge and definitely an important issue," said Dr. Abdullah Kutlar, a hematologist and the director of the sickle cell center.

The last major breakthrough drug to treat it was hydroxyurea, approved in 1997, and it is not without drawbacks. It is designed to fight cancerous tumors and inflammation and suppresses bone marrow production, Kutlar said. That can lead to different problems for patients -- Agbobock, for instance, gets ulcers on his left leg, some of which needed skin grafts to close.

Hydroxyurea is "a very effective drug," Kutlar said. "One thing it doesn't do is expedite the healing of these ulcers. In fact, some people think it might even cause some of the ulcers."

What it does do is help increase the amount of a certain kind of hemoglobin in the blood, called fetal hemoglobin or hemoglobin F, which is found in great quantities in newborns before production is switched to a different kind of hemoglobin after a few months.

In people who have a certain mutation that allows them to have higher levels of fetal hemoglobin, even if they have sickle cell disease, they don't get sick from it, said Dr. Steffen E. Meiler, the vice chairman for research in the Department of Anesthesiology and Perioperative Medicine.

"We have definitely clinical data from hydroxyurea that increasing hemoglobin F works," he said. "Because of all of that, there is a very ambitious search for novel agents to induce fetal hemoglobin."

That led researchers to pomalidomide, a derivative of thalidomide. Working in mice bred to have sickle cell disease, Meiler found that pomalidomide induced a higher level of fetal hemoglobin similar to hydroxyurea but without the suppression of other bone-marrow derived cells. That study was published recently in the journal Blood .

The sickle cell center is now conducting a Phase I clinical trial on pomalidomide, which Agbobock took part in, to find a safe and effective dose.

Whether the drug will work in humans, "the jury is out on that," Meiler said. But being able to connect with researchers such as Meiler is an important part of the center's work, Kutlar said.

"We can first try something in the mouse model, which this (Blood ) paper very nicely illustrates, and as a proof of principle show a response and then take it to the clinical study level," he said.

There might already be signs it is working in some patients, including Agbobock, whose leg ulcers healed while he was on the study drug, Kutlar said.

"We've had two or three other patients like that, whose ulcers healed. That's something rather new and interesting," he said.

Whether the new drug works out or not, Agbobock feels he is much better off since he started being treated by the center, even if it means a 100-mile trip to get there. He knows he has come much further than that.

"When I look at other sickle cell patients, I know that God has blessed me to be healthy, even though I live with sickle cell, and I continue to pray he gives me many more days until they come up with a cure or something that could really (help) a sickle cell patient," Agbobock said.


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