The odds were against little Keone Penn from the start. Born with the most severe form of sickle-cell anemia, a hereditary blood disorder that afflicts more than 70,000 Americans, most of them of African descent, he experienced repeated episodes of racking pain and high fever as brittle, sickle-shaped red blood cells clogged his vessels. At age 5, he was temporarily paralyzed by a stroke. Since then he has bravely endured blood transfusions as often as every two weeks via a catheter attached to his chest. Still the threat of devastating pain and life-threatening infections continued to shadow him. Anything like a normal life was a distant dream.
Now Keone's dream seems about to come true, thanks to a pioneering medical treatment. Exactly a year ago last week, Keone, now 13, became the first sickle-cell patient to receive a transplant of blood cells from the umbilical cord of a newborn infant. In effect, he got a new bloodmaking system. Other young sickle-cell patients have undergone transplants, but these involved bone-marrow cells and had to be matched precisely with the recipients' own blood. In Keone's case, though, his half-sister could not offer matching marrow. So his doctors decided to turn to more easily available cord blood. Consisting largely of immature stem cells, it does not require precise matches between recipient and donor.
Cord-cell transplants have been performed for other blood diseases, such as leukemia, but they remain experimental and highly risky. Dr. Andrew Yeager, a transplant physician at Emory University medical school in Atlanta, warned the Penns that not only might Keone die, but there was not even more than a 50% chance the procedure would do any good. After seven years of blood transfusions that were becoming more and more painful and increasingly ineffective, Keone decided he had no other choice. "Mama, I might die anyway," he told his mother Leslie, a medical technician, who left the decision entirely up to him.
To prepare for the transplant, Keone had to undergo nine days of chemotherapy. The object was to kill his bone marrow, the source of his sickled blood cells, as well as to neutralize his immune system so it would accept the new cells. These came from an anonymous donor at the New York Blood Center and were fed intravenously into Keone on Dec. 11 last year by Yeager and his colleagues at the AFLAC Cancer Center of Children's Healthcare of Atlanta (formerly Egleston Children's Hospital).
The new cells seemed to take hold almost immediately, but for Keone the aftermath of the expensive ($200,000) treatment was like a death-defying roller-coaster ride. Again and again, he was readmitted to the hospital with fevers, diarrhea and loss of appetite, once for a six-week stay. Nine months after the transplant, his new immune system began attacking his own cells, inflaming his liver and intestines. Strong immunosuppressive drugs brought that emergency under control before any permanent damage occurred. Still, no one was breathing easy, least of all the physicians.