Early this year, his ex-wife, Cindy Shelton, spotted a call for people with Type 1 diabetes to participate in a trial by Vertex Pharmaceuticals. The company was testing a treatment developed over decades by a scientist who vowed to find a cure after his kids got the disease. Shelton was the first patient. 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. “It’s a whole new life,” Shelton said.
Diabetes experts were astonished but urged caution. The study is continuing and will take five years, involving 17 people with Type 1 diabetes. It is not intended as a treatment for the more common Type 2 diabetes. “We’ve been looking for something like this to happen for decades,” said Dr Irl Hirsch, a diabetes expert at the University of Washington who was not involved in the study. He wants to see the result, not yet published in a peer-reviewed journal, replicated in more people. He also wants to know if there’ll be adverse effects and if the cells will last for a lifetime or if the treatment would have to be repeated. But, he said, “bottom line, it’s an amazing result”.
It all started with the quest of a Harvard University biologist, Doug Melton. He had never thought about diabetes until 1991 when his 6-month-old son, Sam, got sick and was diagnosed with Type 1 diabetes. . Type 1 is lethal unless patients get injections of insulin.
Patients are at risk of going blind. 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. In time, his daughter Emma, four years older than Sam, also developed the disease, when she was 14.
The only cure 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. But a shortage of organs makes this approach an impossibility. Melton started studying diabetes, determined to find a cure. 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.
The challenge was to figure out what sequence of chemical messages would turn stem cells into insulin-secreting islet cells. The work involved figuring out how islets are made in the pancreas and conducting experiments to steer embryonic stem cells to becoming islets. After years when nothing worked, a small team of researchers achieved a breakthrough in 2014. They put a dye into the liquid where the stem cells were growing. The liquid would turn blue if the cells made insulin. After hours of waiting, one researcher saw a faint blue tinge that got darker and darker. The team was ecstatic. For the first time, they had made functioning pancreatic islet cells from embryonic stem cells.
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November 28, 2021 at 07:01AM
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A cure for Type 1 diabetes? For one man, it seems to have worked - Times of India
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