LIFE BLOOD : 4-Year-Old Boy Fighting Leukemia Is First in Region to Get Transfusion From Umbilical Cord of Unrelated Infant

A 4-year-old leukemia patient Monday became the first person in Southern California to be infused with blood from an unrelated infant’s umbilical cord in what doctors consider a promising alternative to bone-marrow transplants.

In a room at Children’s Hospital of Orange County, as a pack of friends and family anxiously looked on, John Mash Jr. of Corona groggily received a two-hour “cord blood transplant” through an intravenous tube in his chest that doctors hope will rebuild his immune system and save his life.

“I think this provides some new options for us,” said Dr. Joel Weinthal, John’s primary physician. “We think . . . it’s going to be a good source and it will hopefully result in less complications” than marrow transplants.

John, a playful child whom his father calls “the Masher” because of his resilience, is a pioneer in researchers’ efforts to expand the treatment options for young patients with immune system and blood diseases whose lives are in danger because standard therapy has failed.

The boy, whose acute lymphoblastic leukemia was diagnosed more than two years ago, was being successfully treated with chemotherapy before he suffered a serious relapse last November.

For patients like him, the best choice usually is to undergo intensive chemotherapy and radiation that destroys the cancerous blood cells, then to undergo a bone-marrow transplant that allows generation of healthy cells.

But for John, finding a donor was not easy. Although he has plenty of willing relatives, no one in his family, including a 14-month old brother, was a close match.

He had other options: looking for an unrelated donor through a national registry or being infused with his own stored marrow after it had been cleansed of malignant cells. However, his parents and doctors at CHOC decided that the new therapy offered the greatest hope because it seemed to pose less risk of rejection or relapse.

In the past six years worldwide, the experimental technique has been used on about 50 patients who were related to the donor-infants, but only 21 who were unrelated. It involves infusing the patient with so-called “stem cells” from blood that has been extracted from the umbilical cord and placenta of a newborn, then frozen. The cells are the precursors to blood cells, and essentially give the recipient the machinery to replace diseased cells with healthy ones.

If the majority of recipients survive in the long term, the procedure will have proved itself an important option for young patients with cancer and other diseases, researchers said. It is both more efficient and less costly than marrow transplantation, said Dr. Mitchell Cairo, director of CHOC’s Blood and Bone-Marrow Transplant Program.

Arranging for a donation through the National Marrow Donor Program can take months, said Cairo, who performed Monday’s procedure. The donor must be contacted, the procedure arranged and the typing rechecked.

Cord blood, on the other hand, is already in storage and available in a matter of weeks, he said. And, because some of the immune cells from the cord blood are less developed than those that come from adult bone marrow, there is a lower risk of rejection.

The new procedure could save $30,000 in the search for a matching donor alone, Cairo said--more if the procedure results, as expected, in fewer complications.

The five ounces infused into John came from the New York Blood Center which, though it has only 3,700 units of frozen cord blood, has the largest such bank in the world. The donations come from Mount Sinai Medical Center, where new mothers routinely are asked if they would consent to donating their infants’ cord blood, said Dr. Pablo Rubinstein, head of immunogenetics at the New York Blood Center.

The procedure is considered less promising for adults, who in general are less resilient transplant patients, said Rubinstein, who flew out from New York to attended Monday’s infusion at CHOC. Also, children, because of their size, are less of a drain on still-limited supplies of cord blood, he said.

Cairo said John, who will turn 5 in two weeks, appeared to tolerate the infusion well, with no immediate adverse reactions. The critical test, he said, will be over the next 60 days, during which he will be closely observed at the hospital for any signs of rejection.

The donor pairing in John’s case was “almost perfect,” Cairo said; the two matched on all six major criteria. And the prognosis is much improved. The boy faced a 10% to 20% chance of survival without the procedure, and now the odds have been boosted to between 40% and 60%.

John Jr.’s parents, John and Chris Mash, repeatedly referred to the procedure as “a miracle.” While they acknowledged their son’s future was not assured, they said they were confident the child would fight for recovery with characteristic vigor. Only a day before the infusion, for example, he was jumping on his bed.

“He keeps us going,” Chris Mash said.

The Mash family’s church has set up a trust fund to cover John Jr.’s medical expenses. Donations may be sent to the Messiah Lutheran Church, 4861 Liverpool St., Yorba Linda, CA 92686.