When 9-year-old Barrett Ross was born, his parents chose to save the blood from his umbilical cord – ignoring the genetic counselor who told them there was no need for the expense. There was nothing in the family’s background to indicate potential disease for the newborn, the counselor told the new parents.
But Christine Ross, the boy’s mother, was thinking ahead. She thought by 2038, when her baby was 40, medical science surely would have use for the precious blood and its valuable stem cells if he were to develop cancer or another disease.
“I never expected he’d use it as a child,” she said from Barrett’s fourth-floor room at Shands at UF in the Clinical Research Center. Barrett was being prepped for an infusion of stem cells taken from his umbilical cord blood to see if the stem cells can slow – or even stop – the progress of his diabetes.
Barrett is one of 12 diabetic children who are taking part in a national pilot study at the University of Florida in which physicians are looking to see if a patient’s umbilical cord blood provides a potential source of cells that can help treat diabetes. Funded by the Juvenile Diabetes Research Foundation and National Institutes of Health, UF doctors hope the stem cells can neutralize the autoimmune process and help regenerate some pancreatic islet cells.
Dr. Michael Haller, a 2000 graduate from the UF College of Medicine, is a pediatric endocrinologist at UF who began the study approximately two years ago. He said participants include children recently diagnosed with type 1 diabetes whose families banked their umbilical cord blood at birth. Most are still producing a small amount of insulin.
“This is the first attempt at using cord blood as a potential therapy for type 1 diabetes,” Haller said. “We hope these cells can either lessen the immune system’s attack on the pancreas or possibly introduce stem cells that can differentiate into insulin-producing cells.”
The patients are evaluated for two years to measure how much insulin they make on their own and to assess blood sugar levels and the function of key immune system cells. According to preliminary results, the young patients have required significantly less insulin and maintained better control of blood sugar levels than children of comparable age with type 1 diabetes.
The idea for the cord blood study first came from the father of a patient, Haller explained. The parent read that diabetes had been reversed in a mouse after bone marrow from an identical sibling was infused into the diabetic animal.
“The father asked if there could be a positive effect from giving his daughter her own cord blood since the family had stored it at the time of her birth,” Haller said. “I thought it was a good question and not a bad idea to see how to apply this idea to humans.”
Haller stressed that the study is in its preliminary phase and could show that the valuable cord cells provide a supportive role in healing rather than actually repair damaged areas. He said researchers next will look at combining the cord blood with other agents to create more of an impact.
“Right now we are not manipulating the cells. We are simply infusing the cord blood,” he said. “In addition to adding other drugs, we may need to see if we can take the key cells from cord blood and safely manipulate them to improve on our findings.
“We know that no one thing is going to reverse diabetes,” Haller said. “It will be like AIDS or cancer; patients did not start getting better until a combination of drugs were administered. The same will most likely be true with diabetes. We think it will require a combination of treatments, and maybe one day it will have to do with these regulatory cells.”
Barrett and his family hope so. The boy from Lithia, a small town near Tampa, was diagnosed with diabetes right before Christmas, and in March he was the ninth child to receive an infusion as part of the study. According to Haller, Barrett is an ideal candidate for the experimental treatment because he’s a little older than most newly diagnosed kids, and his body still produces some insulin.
After the infusion, which was a less-than-dramatic event taking only a few minutes in the bone marrow transplant ward at Shands, Haller will keep tabs on Barrett’s production of insulin and his blood sugar levels for the next 24 months. The doctor can say for sure the experimental treatment won’t make Barrett’s diabetes worse.
“Since we happened to store his cord blood and we lived in Florida, it seems like destiny that we were to be a part of this study,” said Barrett’s mother, Christine. “If we can lessen the severity of his diabetes or extend the time that he’s doing well, why wouldn’t we do it?”