Umbilical cord transplant could save children

Kim and David Gosey: ,Daughters saved by transplant

Children suffering from potentially fatal disorders have been given hope of survival by the development of umbilical cord blood transplants.

US scientists have successfully used the technique to treat children suffering from genetic disorders.

Until now the only hope for these children was to give them a bone marrow transplant to stimulate their body to produce its own marrow.

But if a bone marrow transplant is to succeed the recipient and donor's blood type must be a perfect match or the transplanted cells will attack the host's body, causing potentially fatal disease.

Researchers from Duke University Medical Center in North Carolina have developed an alternative approach, using umbilical cord blood to stimulate bone marrow production.

Using this technique they have found that the recipient and donor do not have to have matching blood, although blood from an unrelated donor must be at least a two-thirds match.

If umbilical cord blood is taken from a brother or sister, then only half the blood proteins need match.

The Duke team treated 50 children suffering from genetic disorders with umbilical cord blood transplant from unrelated donors.

The disorders, all fatal without replacement of the children's bone marrow, included immune deficiencies, marrow failures and metabolic disorders.

Of the 50 children, 45 grew new marrow and 34 of the patients are still alive.

Sibling transplant

In a related study, four children with genetic disorders were given umbilical cord blood from a brother or sister.

Despite the fact that the recipient and donor's blood was only a half match, three out of four of the children survived.

Umbilical cord blood can substitute for marrow in a transplant because the blood is rich in immature cells, called stem cells, that generate developing and mature blood cells.

The immaturity of cells in the cord blood means that they are less likely to be rejected by the recipient.

Dr Rick Howrey, of the Duke team, said: "A lot of parents of children with these genetic disroders have been told by their doctors, 'There is nothing we can do,' which in the past has been true.

"But with this new form of treatment, there is now hope for children with many genetic diseases that in the past had been uniformly fatal."

Kim and David Gosey's daughter Hannah received an unrelated cord blood transplant in February 1997 to treat Hurler's Syndrome, a genetic disease that affects the metabolism.

Without a transplant, doctors said Hannah would have lived only seven to 10 years, and spent much of the time suffering.

The Gosey's second child Cassidy was born with the same syndrome. She received a transplant when she was three months old. Now two months past transplant, she is out of hospital and being treated on an outpatient basis.

Kim Gosey said: "Transplant has meant we have our family. With Hannah, we were first told to take her home and make her comfortable, that nothing that could be done.

"Now when we get up in the morning, we know that when we go to bed at night we will have our daughters."