Umbilical stem cells
credited with cure

December 1, 2001

Stem cells from stored umbilical cord blood have been credited with curing a case of sickle cell anemia in a 15-year-old boy who had suffered from the disease since age 6 months.

The case, reported Wednesday by CBS News’ “60 Minutes II” program, involved Keone Penn of Atlanta, who – because of his ailment – even suffered a stroke at the age of 5.

“All I remember is I woke up and my mama was beside me and there was a basket beside me and a teddy bear,” he told “60 Minutes II.” “It was very scary, I mean, whew.”

Sickle cell anemia is a painful genetic blood disorder. According to medical information website WebMD, “The sickle cell disease process is triggered when red blood cells become deprived of oxygen. … The damage and durability of sickle cell disease occurs because the log jam that sickle cells cause in the capillaries slows the flow of blood and reduces the supply of oxygen to various tissues,” causing pain and “life-threatening conditions.”

In 1960, said WebMD, “most people with sickle cell disease were not expected to survive childhood.” But a major 1994 study found that “85 percent of children with full-blown sickle cell disease and 95 percent of those with a less serious variation were living into adulthood,” though still “only about half of all sickle cell patients live beyond 50 years.”

Keone had one of the most serious forms of the disease. In fact, “60 Minutes II” reported, for most of his life, he and his mother, Leslie Penn, were routinely in and out of the hospital to get blood transfusions they hoped would prevent another potentially deadly stroke.

“The pain is usually so intense that even morphine, Demerol, those heavy-duty medicines don’t really touch it,” Leslie Penn told the program. “All you can really do is pray that he’ll just go to sleep.”

The toll the disease took on young Keone could also be measured in his appearance; at 15 years old, he was far less developed physically than his peers. He looks much younger than his years and is only 4 feet, 9 inches in height. Plus, his activity is restricted.

“I was impaired from doing a lot of things that normal kids do,” he said, like “sports or anything or run.”

Then Dr. Andrew Yeager of the University of Pittsburgh, who had heard of the new treatment procedure involving cord stem cells, made contact with the Penns with the idea that they should give the procedure a try.

“He just basically said, ‘This is just a 50-50 chance, and it’s up to you all if you want to do it. I can’t offer you any guarantees,'” Leslie Penn recalled.

Said Yeager, “The goal here is that these stem cells, which are in a relatively high proportion in cord blood – higher than they would be in our own bone marrow and definitely higher than in our own circulating blood – could then be injected and would take hold and … make more of themselves, and make a whole new blood factory.”

Ordinarily, Keone would have to have had a bone marrow transplant for his advanced case of sickle cell. That was because until recently, scientists, researchers and doctors believed bone marrow was the only source for adult stem cells – which are far less controversial socially and politically than are stem cells derived from embryos.

Also, “60 Minutes II” reported, “bone marrow transplants can be tricky because there must be a precise match between the person donating the bone marrow and the patient receiving it.” In Keone’s case, no match could be found.

Yeager pointed out that stem cells from umbilical cord blood don’t require exact matches.

So, over the Christmas holiday in 1998 – and after extensive chemotherapy to cleanse Keone’s blood of toxicity due to the sickle cell – he was injected with the stem cells.

The results were miraculous.

Within two weeks, Keone’s blood type changed from type “O” to type “B,” the blood type of the umbilical cord stem cells. Within a year, doctors declared that the sickle cells in Keone’s body were all gone. And today, he is considered completely cured.

Such results are so dramatic, the program reported, that more and more new parents are asking doctors to retrieve the cord blood of their newborns – which amounts to, typically, about 4-6 ounces – and save it for future uses.

Keone’s cord blood came from the New York Public Blood Bank, which, since 1992, has steadily added donations of umbilical cord blood to its inventory. The blood is stored in a cryogenic tank at about 400 degrees below zero.

One of the country’s largest privately run cord blood banks is in Tucson, Ariz. – the Cord Blood Registry. So far, the firm has collected about 30,000 samples.

For a flat fee of $1,300 plus a $95-per-year fee, the firm will analyze and privately store cord blood for families.

“This is really where, I think, so much of biomedicine is going to be going in the 21st century,” Yeager told “60 Minutes II.”

To listen to him, he’s convinced that stem cells can create “blood factories” in just about anyone, replacing diseased blood with healthy blood and repairing damaged body tissue and muscle.

“It’s not just pie-in-the-sky speculation,” he said. “There are studies that would suggest that other organ dysfunction – nerve damage, heart damage, brain-cell damage – might actually be fixed.”

As for Keone, he is now doing things – like playing basketball – that he could never do before. But he’s not completely problem-free. Like most stem cell recipients, he has lingering medical problems.

He must take steroids and other medications because of the stem cell transplant, and that is causing some arthritis. He walks with a limp, the program said, and will eventually need to have his hip and knee joints replaced.

But the sickle cell disease that was killing him is gone.

“I love stem cells,” Keone told CBS. “I mean, they saved my life. If it weren’t for them, I wouldn’t … you never know. I probably wouldn’t be here today.”