Targeted nanoparticles deliver diabetes drugs directly to pancreas
6 February 2012
Lab studies have shown that nanoparticles containing protein
molecules that bind to pancreas cells can effectively deliver diabetes
drugs directly to the pancreas, the organ that produces insulin and is
at most risk during onset of diabetes.
The research was a collaboration between the Wyss Institute for
Biologically Inspired Engineering at Harvard University and
Children's Hospital Boston, in the US.
Although this nanotechnology will need significant additional
testing and development before being ready for clinical use, it
could potentially improve treatment for Type I diabetes by
increasing therapeutic efficacy and reducing side effects.
The approach was found to increase drug efficacy by 200-fold in
in vitro studies based on the ability of these nanomaterials to both
protect the drug from degradation and concentrate it at key target
sites, such as regions of the pancreas that contain the
insulin-producing cells. The dramatic increase in efficacy also
means that much smaller amounts of drugs would be needed for
treatment, opening the possibility of significantly reduced toxic
side effects, as well as lower treatment costs.
Type I diabetes, which often strikes children and young adults,
is a debilitating disease in which the body's immune system
progressively destroys the cells in the pancreas that produce
insulin. According to the Juvenile Diabetes Research Foundation, as
many as 3 million Americans have the disease and some 30,000 new
cases are diagnosed every year. The risk of developing Type I
diabetes, which can lead to serious health complications such as
kidney failure and blindness, can be predicted with 90% accuracy.
Therapeutic intervention for people identified as high risk has
been limited because many systemic treatments are barred from
clinical use due to the severe side effects they produce when used
at the high doses required to achieve a therapeutic response.
"The consequences of Type I diabetes are felt in both the people
who live with the disease and in the terrible strain that treatment
costs put on the economy," said Ingber. "In keeping with our vision
at the Wyss Institute, we hope that the programmable nanotherapy we
have developed here will have a major positive impact on people's
lives in the future."
Using nanoparticles that can be programmed to deliver drug or
stem cell therapies to specific disease sites is an excellent
alternative to systemic treatments because improved responses can be
obtained with significantly lower therapeutic doses and hence, fewer
side effects. To date, such nanotherapeutics have been developed
primarily to treat cancer, since they can home in on the tumor via
its leaky blood vessels. The challenge has been to develop ways to
selectively deliver drugs to treat other diseases in which the
tissues of interest are not as easily targeted.
The research team addressed this problem by using a unique homing
peptide molecule to create "smart" nanoparticles that can seek out
and bind to the capillary blood vessels in the islets of the
pancreas that feed the insulin-producing cells most at risk during
The research was supported by the Wyss Institute and a SysCODE
(Systems-Based Consortium for Organ Design and Engineering) grant
from the National Institutes of Health that supports a group of
seven clinical and academic institutions working to develop new ways
to induce regeneration of organs, including the pancreas.