Opening doors to sickle cell cure

April 16, 2009 3:18:55 PM PDT
Sickle cell anemia is one of the most common genetic diseases in the United States, affecting 70,000 Americans. The only cure for the disease is a stem cell transplant, but the perfect donor is hard to find. A new type of transplant is opening to door to the thousands of children who don't have a donor in their family. For 14-year-old Albert Pinckney, the best experiences in life are on wheels.

"I like going fast," Pinckney said.

But having sickle cell anemia forced him to grow up too fast. Pinckney missed all of the first grade because of complications with his disease.

"My spleen, gall bladder, tonsils, adenoids -- I've had all those removed," Pinckney said.

Unfortunately, Pinckney's story isn't unusual among kids with severe sickle cell. The disease causes abnormally shaped blood cells that have trouble passing through vessels. Patients can get blood transfusions to replace the abnormal cells, but the fix is only temporary.

"We know that we have helped them for a short while, and then they are going to be back again," said Dr. Shalini Shenoy, Director of the Bone Marrow Transplant Unit at St. Louis Children's Hospital in St. Louis.

The only cure is a marrow or blood stem cell transplant, but until now, this has only taken place between family members. A new study is allowing patients to receive transplants from unrelated donors.

"The idea is to try to make transplants available to different groups of patients," Dr. Shenoy said.

The study is also trying to ease the long-term effects of the chemotherapy needed before the procedure. Lower doses could prevent organ damage, ovarian failure and sterility.

Although it's been a struggle, Pinckney's disease has made him who he is.

"It's really affected me and helped me to be a more mentally strong kid than most," Pinckney said.

A strong kid whose cure may be around the corner.

The stem cells used in the study come from the National Marrow Donor Program and cord blood registries across the country. Another possible cure for sickle cell disease is gene therapy, which has only been tested in mice.


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