Sickle Cell Cure

Adult Stem Cells Completely Cure Sickle Cell Patient

Pittsburgh, PA -- Stem cells are thought of as the Holy Grail of medicine. One young boy agrees with that. He made medical history because he's been cured of his life-threatening disease. The key to his cure did not come from a human embryo, where all the controversy is, but from something that is routinely toss in the garbage - an umbilical cord. Umbilical cords were always considered medical waste.

Not anymore.

That's why new parents like Pam Dorne and Stephen Ayers of suburban Chicago have decided to save their children's umbilical cord blood. Dorne gave birth last spring to a baby boy, Kyle.

After a baby is born, there is just a 15-minute window to retrieve the four to six ounces of blood in the umbilical cord. And in that blood are potentially lifesaving stem cells that can be saved for future use.

"This is really where, I think, so much of biomedicine is going to be going in the 21st century," says Dr. Andrew Yeager of the University of Pittsburgh.

For instance, when stem cells from umbilical cord blood are injected into a person's vein, they migrate to the bone marrow and can create what Dr. Yeager calls a blood factory, replacing diseased blood with healthy blood. According to the National Institutes of Health, stem cells may one day be able repair the body's tissue and muscle and cure everything from spinal cord injuries to Alzheimer's.

"It's not just pie-in-the-sky speculation," says Yeager. "There are studies that would suggest that other organ dysfunction - nerve damage, heart damage, brain-cell damage - might actually be fixed."

It has the potential to make paralyzed patients walk and make Alzheimer's sufferers remember.

That potential is what Dr. Yeager was counting on to cure a young patient named Keone Penn.

Keone suffers from a case of sickle cell, a painful genetic blood disease. He was diagnosed when he was 6 months old. He was 5 when his sickle cell caused a stroke.

"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 says. "It was very scary, I mean, whew."

For six years, Keone and his mother, Leslie Penn, were constantly in and out of an Atlanta hospital to receive transfusions to stave off another potentially deadly stroke. By the time Keone was 11, the transfusions were becoming less effective and he had excruciating pain in his joints and lower back.

"The pain is usually so intense that even morphine, Demerol, those heavy-duty medicines don't really touch it," Leslie Penn says. "All you can really do is pray that he'll just go to sleep."

Keone says he's tough, but at 15, he looks much younger. Sickle cell stunted his growth - he's just 4 feet, 9 inches, tall - and restricted what he could do.

"I was impaired from doing a lot of things that normal kids do, like sports or anything or run," he says. "Couldn't play basketball. Because, you know, some people like roughhousing when they play basketball and they can knock you over and push you and that could really hurt me."

The odds were that Keone had, at best, only five years to live. So Yeager decided to take a chance on a new procedure. Never before had stem cells from umbilical cord blood been used to treat sickle cell.

"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 again, make more of themselves. And make a whole new blood factory."

Yeager told the family he wasn't sure the procedure would work.

"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.'" recalls Leslie Penn.

Keone Penn remembers how his mother told him: "She came in the room looking very depressed. Pulled the chair up sat beside me in the bed and told me everything. And I almost started to cry. But she was very calm about it. She told me everything, said, 'You got-you may - have five years to live,' you know."

Ordinarily, patients with a severe case of sickle cell, like Keone's, would have had a bone-marrow transplant.That's because until recently bone marrow was the only source for stem cells.

But 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.

Stem cells from umbilical cord blood don't need an exact match.

Dr. Yeager and his team found a match that was close enough in a cryogenic tank at the New York Public Blood Bank, which since 1992 has slowly been collecting donations of umbilical cord blood.

Over Christmas vacation of 1998, after intensive chemotherapy to destroy Keone's bad blood, he was injected with the stem cells.

After a few weeks, something extraordinary happened - the stem cells changed his entire blood system from type O to type B.

"That concept there is the one that really blows my mind," says Leslie Penn. "The thought that your whole blood type is changed. The umbilical cord cell's donor, he took on their blood type.

A year later, doctors declared that the sickle cells in Keone's body had disappeared. Today, he is considered cured.

It was umbilical cord stem cells that cured Keone, not stem cells from human embryos. While the use of embryonic stem cells has generated fierce controversy, umbilical cord stem cells have attracted little attention and no political debate. And now it seems, more and more new parents have decided to bank their hopes on the stem cells in their newborn's cord blood.

Moments after Pam Dorne gave birth to a baby Kyle, his cord blood was sealed, packed in dry ice, and given to a courier. Within hours, the package was on a plane bound for Tucson, Arizona, where the largest privately run cord blood bank in the country is located.

There, a child's umbilical-cord blood is stored in a cryogenic tank at a temperature of minus 400 degrees Fahrenheit.

Dr. David Harris, laboratory director of the Cord Blood Registry, says it takes only a small vial of cord blood to change a person's entire system.

So far, Cord Blood Registry has collected about 30,000 samples from families willing to pay a $1,300 flat fee and $95 a year to analyze and privately store their baby's cord blood. The company has taken in over $40 million so far, selling a kind of biological insurance.

"Part of the issue when people bank," says Harris, "it is because they have a family history or they work or live in a place where there is a potential for cancer. But part of it is for peace of mind."

According to the American Academy of Pediatrics, that peace of mind isn't worth the money. The academy says the chances a family will ever need to use its frozen cord blood are very small. What they say makes more sense is to donate cord blood to a public bank, the kind where Keone Penn got his stem cells.

That is something Pam Dorne, an obstetrician, says she understands in her professional life. But her own personal choice was a private bank, she says, for one reason.

"If the American Academy of Pediatrics could tell me that none of my children would ever have a problem," she says. "Or that if they had a problem, I would be guaranteed that there would be enough donors and somebody would match them, that would be perfectly reasonable. But I don't think anybody has that crystal ball."

What saved Keone Penn's life, Dr. Yeager says, is a public blood bank and the umbilical cord blood from an anonymous donor.

"If they wish to pay, that's absolutely fine." He says of patients. "But to look at a larger, greatest good for greatest number, I would contend that a volunteer donation to a public blood bank would make the most sense."

Meanwhile, Keone, a pioneer, is doing things he's never done before.

"I discovered the other day that I like playing basketball, " Keone says. "I never played basketball, 'cause I've always been disabled to play it and to have fun."

Keone, who one day hopes to become a chef, still has some major health problems as a result of infections that occur in most stem-cell transplants. Because of steroids and other medication, he has arthritis, walks with a limp and will need joint replacement in his hips and knee. But the good news is the sickle cell that was killing him is gone.

"I love stem cells," he says. "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."

Keone doesn't know where the cord blood came from or who is the owner. He says he would like to know, just so he could say, "Thank You."