Cord Blood Stem Cells Used to Help Boy with Fanconi Disorder

By Rick Allen, Ocala Star-Banner, April 16, 2007

Unlike the story of his brother Zac, four-year-old Logan’s story may not end as tragically.

Because of a transfusion of cord blood stem cells administered at the Shands Hospital - University of Florida on Thursday, doctors believe that Logan’s life will be saved.

Logan says the "oil change,'' as he calls the process, "is gonna make me big and strong.''

The entire family hopes that he is correct.

A disease that gradually erodes bone marrow, Fanconi anemia killed Logan’s brother Zac five years ago. If the rare genetic disorder is left untreated, eventually Logan’s body will no longer be able to create blood. He will suffer the same fate as his brother.

Via a transfusion port that was implanted in his abdomen last week, Logan had a small pouch of the maroon-colored cells attached to his body. This procedure was performed on Thursday in the Bone Marrow Transplant wing at Shands.

The process of rebuilding bone marrow and consequently a new immune system began as the fluid flooded into Logan’s 30-pound body. The entire process took the same amount of time as changing the oil in a car.

“The cells are smart and they know exactly where to go,” said Dr. Susan Staba, a pediatric oncologist caring for Logan.

Before white blood cell levels are considered to be in the “safe range”, the cells need about three weeks to build up in the blood and bone marrow.

The family tried to secure genetically matched blood for Logan two years ago. They made the news and Logan loved the attention. He was very talkative and at the time, he showed no outward signs of the deadly condition he had inherited.

Then his white blood cell count began to level off and then drop about a year ago. The Fanconi anemia had begun to show itself Staba said.

Swiss pediatrician Guido Fanconi first diagnosed the condition in 1927. By the time a child reaches the late teen or early adult years, the condition is considered 100 percent fatal.

According to the Fanconi Anemia Research Fund in Eugene, Oregon, the condition affects girls and boys of all races equally. There are about 3,000 cases of the condition worldwide; of those cases 300 to 500 are in the United States.

Giving hope to those who are afflicted with the condition are cord blood and bone marrow stem cells along with other recent advances. Before, the standard treatment lost effectiveness over time and consisted of whole blood and platelet transfusions.

When the 8-year-old Zach died of Fanconi complications in July of 2002, the transfusion was the only available treatment. Logan was born just a few weeks earlier.

"For Logan, this is a lifesaving option, because without this he'll die,'' Staba said.

A boy born only a few weeks ago provided the stem cells needed to regenerate Logan’s system said Logan’s grandmother Fran. The donor was anonymous and the family may never learn anything more about Logan’s genetic “twin”.

The donated blood is an exact 6-to-6 genetic match to Logan’s blood. That is the only absolute known about the cord blood.

"They look at six different HLA (human leukocyte) antigens,'' Staba said. "Less than 10 percent of cord bloods are a perfect match.'' Some cord blood transfusions can be done with a 4-to-6 match, but this 6-to-6 match offers extra optimism.

"If I didn't think it was going to work, I wouldn't have started with the chemotherapy,'' said Al, Logan's father, who hasn't left his son's side the past week. "We're up on the higher end of the scale and that makes us feel good.''

Added Staba: "It's a great cell dose, a perfect match, same blood type even - everything we could ask for.''

An 80 to 85 percent chance of success is usually the case wit ha 6-to-6 match.

To give the transplanted stem cells the best chance to establish themselves and thrive, pre-transplant preparations needed to be made. So, the marrow in Logan’s bones along with his existing immune system was destroyed over the last week using chemotherapy.

"Basically, he's empty right now, aplastic,'' Staba said Wednesday.

"We were told his hair would fall out,'' said Logan’s father. "This makes it a little less apparent,” he said, referring to the buzz cuts both he and Logan got prior to the procedure.

While changing the oil in his car recently, Logan’s father got the idea to call his sons treatment the same.

"It's a way Logan can understand what's happening,'' he said. And it helps that one of Logan's favorite movies of late is the animated "Cars.''

In order to give Logan the best chance, the medical team moved swiftly once the cord blood match was discovered.

"We've almost gotten through most of the viral season,'' Staba said. "We've got 'til next November until we hit the bad viral season again, and by then he'll be fine."

Blood from an umbilical cord and placenta traditionally is thrown away after birth. However, Staba says, it's rich in stem cells "just floating around in their blood because babies don't have a lot of room in their bones to store stem cells. There's enough blood left over to actually remake someone's whole immune system.''

According to the AABB (formerly the American Association of Blood Banks), it wasn’t until the 1990’s when cord blood was first used in a nonrelated transfusion, even though the practice of transfusions extends back to the early 1600’s.

“Public banking of cord blood is saving lives,” Staba said.

Logan will not be able to leave the safety of the transplant unit at Shands for several weeks because his immune system will be compromised while it is being rebuilt said Staba.

"He can't go to Wal-Mart or Chuck E. Cheese; the boy must avoid crowds,'' Staba added.

But Logan is looking forward to having fun with his little sister. After being tested three times, Tatiana was found to be 100% free of Fanconi anemia.

Tatiana and her mother Michelle are staying at a near by Ronald McDonald House, while Al is at the hospital with his son. The children’s grandmother May alternates time between both locations.

"We're all thinking he's going to come out of here with us hand-in-hand,'' May said. "Really, he's having a fantastic time. It's like camping and he's getting a lot of dad time."