When his two children were stricken with type 1 diabetes, Harvard stem cell scientist Douglas Melton says, he did what any father would want to do: He set out to cure the disease.
After 15 years of effort, including some false starts and regulatory hurdles, Melton has taken a major step toward that goal.
In a paper published in the journal Cell on Thursday, he reported a step-by-step procedure that starts with stem cells and results in hundreds of millions of the precious pancreatic cells that secrete the hormone insulin, keeping blood sugar levels in balance. It is the lack of insulin produced by those cells, called beta cells, that lies at the root of type 1 diabetes.
Ultimately, the hope is those cells could be transplanted into diabetes patients and allow them to create insulin naturally, creating a paradigm shift in treating a disease currently kept in check by insulin injections.
Melton cautions that the work is still years from being tested in patients and many challenges, scientific and practical, remain. But he is gratified to have reached this point and even more motivated to continue, so as not to disappoint the millions of people who suffer from type 1 diabetes, which is usually diagnosed in children and young adults.
“We’re tired of curing mice,” Melton said in an interview. “Most patients are sick of hearing that something’s just around the corner; I’m sick of thinking things are just around the corner. But I do believe in the big picture.”
Melton hopes the cells could be ready to be tested in people in a few years. Already, cells are being transplanted into primates through a collaboration with a researcher in Chicago.
Melton’s work is expected to energize the diabetes research community.
Dieter Egli, assistant professor in the pediatrics department at Columbia University Medical Center, said his laboratory will try to repeat Melton’s experiment immediately.
“It’s a wonderful result, something we’ve been waiting for quite awhile,” Egli said.
Earlier this year, Egli’s laboratory was able to create embryonic stem cells from a person with type 1 diabetes, through a process called somatic cell nuclear transfer. He now plans to use Melton’s procedure to create the beta cells that are affected by the disease.
Egli said that while he hopes the work will allow him to transplant cells into patients one day, the ability to generate large numbers of beta cells in the laboratory will also aid in the near-term search for diabetes drugs that work on beta cells in diseased patients.
Melton is also collaborating with a researcher at the Massachusetts Institute of Technology to surmount the other major challenge in treating type 1 diabetes: stopping the immune system from attacking and killing the beta cells within the pancreas that secrete insulin. Together with MIT bioengineer Daniel Anderson, he hopes to create an encapsulation technology that could protect the cells from the immune system.
Other attempts to use stem cells for diabetes therapy have been making steady progress. A clinical trial was announced this summer in which researchers will transplant into patients immature cells capable of developing into beta cells.
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SOURCE: Boston Globe – Carolyn Y. Johnson