Since the discovery of insulin, we have seen only incremental progress in the management of Type 1 Diabetes, but now, a breakthrough has now been made


B was a strapping young man, tall, handsome, rippling biceps, and an evangelist. No, not the religious type; he wouldn’t dare. This was UCSF in the 60’s, and the student body was radically areligious. His cause was Type 1 Diabetes, aka juvenile diabetes. He had it since infancy, and he was telling us, as part of a nutrition course, how he managed his blood sugar without adjusting his insulin while running marathon races! A personal feat to be sure, but his regimen of carbo loading became a household term among runners. Since then, in my professional life, I have come across many Type 1 Diabetics, from patients to activists, from scientists to bank presidents and company CEOs. One thing always stands out: they were all zealous in their determination to live a normal life, and contribute to the fight against this debilitating disease.

The mission statement of the Juvenile Diabetes Research Fund (JDRF) reflects this community’s dogged determination:

“JDRF is the leading diabetes foundation funding T1D research. JDRF’s goal is to progressively remove the impact of T1D from people’s lives until we achieve a world without T1D.”

 

What is Type 1 Diabetes?

Type 1 Diabetes, once known as juvenile diabetes or insulin-dependent diabetes, is a chronic condition in which the insulin-producing beta cells of the pancreas are destroyed. Insulin is needed to allow glucose to enter cells to produce energy. Far more common, Type 2 Diabetes occurs when the body becomes resistant to insulin or doesn’t make enough insulin to keep up with the body’s needs.

Before the discovery of insulin, young patients simply withered away and died. In 1923, Banting and Macleod received the Nobel Prize in Medicine for the discovery of insulin, saving countless children’s lives.

[Gossip break: Banting was only 32 when he got the prize, for work he and Charles Best, his collaborator, did in isolating the hormone. Macleod was the chairman of the department, was passionately hated by Banting, and didn’t contribute to the discovery one iota except putting his name on the papers and claiming the credit. Banting was outraged by the exclusion of Charles Best, and being Canadian and a gentleman, shared the prize money with him.]

Since that breakthrough, science advanced by small increments. Isolation of pig insulin was an improvement over cow insulin (less immunological reaction), formulations of insulin to better control its release rate, sequencing human insulin, synthesis of insulin using classical protein chemistry methods, generating human insulin by fermentation using bioengineered bacteria. But the basic fact remains: diabetics still have to closely monitor their blood sugar and inject themselves with doses of insulin several times a day.That does not provide the kind of exquisite fine tuning necessary to properly control metabolism, and that lack of control leads to devastating complications ranging from blindness to kidney failure to loss of limbs. and more.

 

A Breakthrough: Insulin-producing Cells from Stem Cells

Harvard stem cell researchers announced on October 9, 2014, that they have made a giant leap forward in the quest to find a truly effective treatment for Type 1 Diabetes, a condition that affects an estimated 3 million Americans at a cost of about $15 billion annually. Their seminal work was published in Cell. With human embryonic stem cells as a starting point, the scientists are for the first time able to produce, in the kind of massive quantities needed for cell transplantation and pharmaceutical purposes, human insulin-producing beta cells equivalent in most every way to normally functioning beta cells.

Here is a touching human aspect of the story: Doug Melton who led the work and who 23 years ago, when his then infant son Sam was diagnosed with Type 1 Diabetes, dedicated his career to finding a cure for the disease, said he hopes to have human transplantation trials using the cells to be underway within a few years. The stem cell-derived beta cells are presently undergoing trials in animal models, including non-human primates.

 

Why not use it now?

Type 1 Diabetes is a result of autoimmune attack on the insulin-producing beta cells. If the newly generated stem cells were to be injected they would be subjected to the same autoimmune attack and would die in short order. So a method has to be developed to administer the 150 million cells in a way that would protect them from immune attack.

Melton is collaborating on the development of an implantation device to protect the cells with Daniel G. Anderson, Professor of Applied Biology, Associate Professor in the Department of Chemical Engineering, the Institute of Medical Engineering and Science, and the Koch Institute at MIT. The device has been implanted in several animal species, including monkeys, and the beta cells have so far continued to secrete insulin.

Once animal results meet FDA requirements, clinical trials will begin. And those will take several years, many patients and lots of money. Despite this long process, we can’t but be amazed at this breakthrough. It has been equated to the invention of antibiotics. I totally agree.


This post is dedicated to Karl Piez, mentor, colleague and friend, and a brilliant scientist who struggled with Type 1 Diabetes to his last day.

Dov Michaeli, MD, PhD
Dov Michaeli, MD, PhD loves to write about the brain and human behavior as well as translate complicated basic science concepts into entertainment for the rest of us. He was a professor at the University of California San Francisco before leaving to enter the world of biotech. He served as the Chief Medical Officer of biotech companies, including Aphton Corporation. He also founded and served as the CEO of Madah Medica, an early stage biotech company developing products to improve post-surgical pain control. He is now retired and enjoys working out, following the stock market, travelling the world, and, of course, writing for TDWI.

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