SIOUX FALLS, S.D.- Children with the worst kind of diabetes never get a day off.
Dr. Kurt Griffin and the rest of his Sanford Research Center team are trying to change that and find a better way to treat Type I diabetes that affects about 2 in every 100,000 children in the U.S.
"The children and family really pay," Griffin, who works out of a small office in the Sioux Falls center, said.
Not only has the cost of a vial of insulin gone from about $50 only about 10 years ago to $400 now, Griffin said the shots themselves take a toll.
"It's too many too count," he said about the children who usually take a shot with every meal or snack and then before bed. It gets to be about four to eight shots a day.
Insulin pumps have helped some children and adults, but that's always attached to the body and then there's the blood pricks to the skin to check insulin levels.
"You can't ever take a break," Griffin said.
In the long term, the toll can be even heavier as diabetes is the No. 1 cause of blindness and also can cause amputations and kidney failures, Griffin said.
A new clinical study is well underway with 52 children from age 8 to 18 from Utah to Ohio, but most are from North Dakota and South Dakota as they are close to the sites in Sioux Falls and Fargo.
There are other sites, too, across the U.S. working with Griffin and his team to evaluate the children.
More children are needed for the study, however, as only those who are within the first 100 days of being diagnosed can participate. The goal is 111 participants.
"We're still recruiting," Griffin said.
What's done in the "T-Rex Study" as it's called, is that children with Type 1 diabetes have a loss of insulin-producing beta cells and researchers are trying to change that.
When a participant joins the study, a unit of blood is taken from the child and sent to a laboratory in California where in an "ultra pure' setting the number of regulatory T cells or "Tregs" are expanded in a child's blood from millions to billions.
In the lab, cells are sorted one by one in a process that takes about two week.
Those additional Treg cells, if the study goes as planned, will help prevent the immune system from mistakenly destroying those insulin-producing beta cells.
The blood is then put back into the child and hopefully the expanded number of Tregs and improved function will stop the attack on the beta cells that make insulin.
With the diabetes the "immune system is out of balance and we're trying to put it back in balance," Griffin said.
It would mean no more shots or insulin pumps. However that depends on another question researchers want to explore is whether the Tregs will be short-lived or last long enough in the body to keep enough insulin production over the long term.
Will it work?
That's the purpose of this second phase of the study. In the first phase, 18 adults underwent the blood work at the University of California-San Francisco mostly to check on the safety of the study.
It was then approved by the Federal Drug Administration to move onto this next phase.
Although the study is moving along with some testing already being done on the children, Griffin said he doesn't know if it's working.
As far as safety, there seems to have been no problems so far.
However, as is the norm in clinical trials, one-third of participants are getting a placebo, one-third a low dose of the expanded cells and one-third a high dose.
So Griffin and his Fargo counterpart, Dr. Luis Casas, don't know yet on if the process will work.
The best answers are likely to come in the spring of 2019, although some early indications could be found out as soon as early next year.
The therapy has received fast-track designation from the FDA, a first for any Type 1 diabetes intervention project. The status allows more frequent communication with the FDA, faster feedback about the therapy during the approval process and allows researchers to submit data and reports on a rolling basis.
Griffin said seeking a cure for Type 1 diabetes has been a top goal for Sanford since philanthropist Denny Sanford gave a gift of $400 million about 10 years ago. Thus, he feels some pressure to have something that can work.
Although he explains that he doesn't know if the new cell therapy is going to work, he does knows that any project takes time-in years.
If it does work, though, it may be a key to easing other autoimmune diseases such as arthritis, lupus and multiple sclerosis.