To find a superior treatment for juvenile diabetes, scientists have turned to a young boy.
Seven years ago, when Liam was born, his parents Steven and Beth opted to store his umbilical cord blood as an insurance to treat potential future illness. Now that Liam has diabetes, doctors have been injecting stem cells taken from the umbilical cord blood to try and slow, or possibly even stop, the progress of his diabetes.
For the national pilot study at the University of Florida, there are seven other children undergoing similar transplants using stem cells from their own umbilical cords.
Patients with juvenile diabetes, if left untreated, can fall into a coma or suffer kidney failure. Insulin-producing cells in the pancreas are destroyed by this autoimmune disease. More than 1.5 million Americans, including 125,000 children are afflicted with juvenile diabetes. Also known as type 1 diabetes, there is no cure for this disease.
Cancer and dozens of other blood disorders are regularly treated using stem cells taken from cord blood. Easily stored in blood banks, the stem cell rich cord blood can be extracted from the umbilical cord minutes after a baby’s birth.
The lead doctor in the study spends much of his time of the phone cautioning parents about keeping their expectations realistic due to the anticipation the stem cells can be successful in fighting juvenile diabetes.
“It’s important not to destroy their hope,” said Dr. Michael, a pediatric endocrinologist. “Everybody wants to cure diabetes. But this is a pilot study. It’s unlikely to be the Holy Grail.”
But it has helped Liam. His body is now fighting off the progression of the disease. His blood glucose levels decreased after the infusion. Prior to being injected, doctors were saying he would need regular injections of insulin because his blood glucose levels were rising.
A urine test detected excessive glucose in his system back in December, soon after the test, he was diagnosed with diabetes. Insulin, a hormone that regulates the body’s metabolism of carbohydrates, including glucose, was still being produced by his body in small quantities. This early diagnosis and circumstance is often called the “honeymoon” phase of the disease.
“There’s no magic pill to stop the process,” said Liam’s father, Steven. “As parents, you feel you’re helpless. You see what’s happening to your child.”
Surfing the web late on a December night, Liam’s mother discovered the Florida stem cell study while researching juvenile diabetes. She thought back to when Liam was born and remembered the cord blood bank that she paid to store his umbilical cord blood.
She enrolled her son in the four-year clinical trial.
“We feel God was with us,” she said.
Before taking Liam for his transplant at the university in Gainesville, the family visited the Kennedy Space Center and Sea World as a treat.
“You’ve got to have some sugar with your medicine,” Steven said.
Liam received the stem cells in an intravenous infusion after having some blood drawn. The procedure took only half an hour to complete.
To avoid rejection, Liam’s own cord blood needed to be used. There is a chance of rejection even with stem cells from a parent or sibling.
The clinical trial intends to enroll a total of 10 or more children. Currently there are five girls and three boys, all between the ages of 3 and 7.
The study requires using all of a child’s cord blood. This fact may deter some families because if the child gets another disease there won’t be any cord blood left to use. This will most likely change however, as storage facilities improve freezing and thawing processes that tend to destroy cells, and also begin keeping cord blood in more than one vial. Other methods of producing and preserving cells may also emerge.
At a cost of roughly $1,500, taking blood from a cut umbilical cord takes only a few minutes. A blood storage fee of $100 a year generally applies as well. Storage has become increasingly popular since blood banks began storing cord blood in the past decade. Most decide on the option in case a child or relative becomes ill.
Opposed to cells extracted from bone marrow, cord blood stem cells have more potential to develop into specialized cells needed to help a body fight disease. They are also easier to obtain than bone marrow cells and less controversial compared to those cells taken from human embryos.
The study at the university seeks to stop the autoimmune process but, “to cure diabetes, cord blood stem cells may be combined in the future with other medications, such as immune suppressant drugs, in a mix similar to “cocktails” given to cancer and HIV patients,” Dr. Michael stated.
Meanwhile, Liam’s parents are thankful to be able to hold off on having to give their son’s insulin shots.
“Liam should be getting worse, not better,” Steven said.
“You’re happy about that, right?” said Liam.
“We’re absolutely happy,” said his father, a medical equipment sales and service engineer.
Causes of the disease include genetic predisposition and other factors such as viruses or being overweight. Of the three siblings, two have diabetes, but their father does not.
“We’re not saying this is the end-all,” Beth said. “But it gives people hope. That’s why stem cell research is so important.”