On June 29, he got an infusion of cells, grown from stem cells but just like the insulin-producing pancreas cells his body lacked.Now his body automatically controls its insulin and blood sugar levels.
Shelton, now 64, may be the first person cured of the disease with a new treatment that has experts daring to hope that help may be coming for many of the 1.5 million Americans suffering from Type 1 diabetes.
The study is continuing and will take five years, involving 17 people with severe cases of Type 1 diabetes.
It is not intended as a treatment for the more common Type 2 diabetes.
Irl Hirsch, a diabetes expert at the University of Washington who was not involved in the research.
Peter Butler, a diabetes expert at U.C.L.A.
Sam’s urine was brimming with sugar — a sign of diabetes.The disease, which occurs when the body’s immune system destroys the insulin-secreting islet cells of the pancreas, often starts around age 13 or 14.
Unlike the more common and milder Type 2 diabetes, Type 1 is quickly lethal unless patients get injections of insulin.
People with Type 1 diabetes are at risk of having their legs amputated and of death in the night because their blood sugar plummets during sleep.The only cure that has ever worked is a pancreas transplant or a transplant of the insulin-producing cell clusters of the pancreas, known as islet cells, from an organ donor’s pancreas.
O’Keefe had to prick Sam’s fingers and feet to check his blood sugar four times a day.
He turned to embryonic stem cells, which have the potential to become any cell in the body.
His goal was to turn them into islet cells to treat patients.Over the 20 years it took the lab of 15 or so people to successfully convert stem cells into islet cells, Dr.The challenge was figure out what sequence of chemical messages would turn stem cells into insulin-secreting islet cells.
The work involved unraveling normal pancreatic development, figuring out how islets are made in the pancreas and conducting endless experiments to steer embryonic stem cells to becoming islets.
They had put a dye into the liquid where the stem cells were growing.
For the first time, they had made functioning pancreatic islet cells from embryonic stem cells.
Then they had bright blue wool caps made for themselves with five circles colored red, yellow, green, blue and purple to represent the stages the stem cells had to pass through to become functioning islet cells.One challenge was to figure out how to grow islet cells in large quantities with a method others could repeat.The company, led by Bastiano Sanna, a cell and gene therapy expert, tested its cells in mice and rats, showing they functioned well and cured diabetes in rodents.“These are islet cells that we made at Semma,” he told Dr.
Employees working under scrupulously sterile conditions monitored vessels of solutions containing nutrients and biochemical signals where stem cells were turning into islet cells.
He says they cause him no side effects, and he finds them far less onerous or risky than constantly monitoring his blood sugar and taking insulin.
John Buse, a diabetes expert at the University of North Carolina who has no connection to Vertex, said the immunosuppression gives him pause.
“We need to carefully evaluate the trade-off between the burdens of diabetes and the potential complications from immunosuppressive medications.”.
He measured his blood sugar
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