Who Said Bigger is Better?

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By Nora Saul, M.S., R.D., C.D.E., Manager of Nutritional Services at Joslin

It is not just insulin pumps that keep getting smaller and more intelligent.  Researchers have started to experiment with nanotherapeutics as a way to prevent and treat type 1 diabetes. Clinical trials for this technology may be years away, but small and targeted might be the way to go on the road to better clinical care.

Researchers at Wyss Institute for Biologically Inspired Engineering at Harvard University and Children’s Hospital in Boston have developed “smart” injectable nanoparticles that can be programmed to selectively deliver drugs or stem cells to the pancreas.  Only between 1 and 100 nanometers (nm) in size (a pin head is 1,500,000 nm), these cells are highly specific cargo carriers with sophisticated homing devices on board.  They deliver drugs directly to the disease-affected areas.

Nanotherapeutics has been used successfully to treat solid cell cancers such as lung cancer, but this technique is new to the diabetes world. In cancer therapy the nano messengers focus on the presence of leaky blood vessels to identify their target. But developing a similar device for diabetes is more difficult because it isn’t entirely clear how to select the tissues of interest.

The Wyss Institute and Children’s Hospital research team addressed this problem by using a homing peptide molecule to create “smart” nanoparticles.  These particles are capable of binding to the capillary blood vessels in the pancreas that feed those insulin-producing cells most at risk during disease onset.

In research experiments, drugs carried by the nanoparticles to tissues increased the efficacy of the delivered drugs by 200-fold in vitro (cell studies).  They did this by protecting the drug from degradation and concentrating it at the pancreatic beta cells (the insulin producing cells in the pancreas.)

Sending drugs precisely to the areas they are needed reduces the amount of the drug required to produce the same effects, creating the possibility of significantly lower toxic side effects, as well as lower treatment cost.

This treatment could one day be applied to people with diabetes, but also to people who are at risk. Since the drugs would be delivered directly to the pancreas, they could have the potential to jump-start ailing insulin production and prevent, or at least delay, full-blown type 1.

Type I diabetes strikes both children and adults—approximately 3 million people in the United States have it, and about 80 people per day are diagnosed with this incurable disease.

Risk factors for type 1 include family history, having a parent or sibling with the disease, certain genetic markers and geography. Living further away from the equator increases your risk of developing the disease (for example, people in Finland have 400 times the risk of developing type 1 than people in Venezuela), although the causation behind this still isn’t clear.

Knowing you may be at risk for type 1 doesn’t provide any therapeutic advantage presently, and the treatments discussed here are years away from human trials. But with a bit of luck, these new therapies could provide hope for future generations.

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