0:00
Welcome to the seminar on "Nanotechnology for CNS
Delivery of Biological
Therapeutics."
My name is Mansoor Amiji.
I'm a Distinguished Professor
and Chairman of the Department
of Pharmaceutical Sciences
in the School of Pharmacy
at Northeastern University
in Boston, Massachusetts.
I'm also the director of the
laboratory of Biomaterials
and Advanced Nano-Delivery Systems
here at Northeastern University.
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The diseases of the central
nervous system, or CNS,
currently represent 11% of
the global disease burden.
This number will increase
to 14% in the year 2020,
largely due to the
aging demographics,
both here in the United
States and around the world.
CNS diseases are
especially devastating,
as they are predominant in older
adults with other morbidities.
In neurodegenerative
disease, for example,
such as Alzheimer's and
Parkinson's disease,
lack of early diagnosis
and current treatment
strategies that are
purely symptomatic
are also additional challenges.
A piece published
in 2010 in Science,
showed that CNS drug development
takes a long time for approval,
has poor success rate, and costs
more than any other diseases.
As such, currently
there are very few
major pharmaceutical and
biotechnology companies having
a program in CNS drug development.
Biological therapies based on
peptides, proteins, nucleic acid,
and even cells have
shown tremendous benefit
in the pre-clinical evaluation for a
variety of CNS diseases, especially
neurodegenerative diseases.
Many of these agents have disease
modifying effects, rather than just
providing symptomatic treatment.
However, delivery of therapeutic
agents, especially biologicals that
are highly hydrophilic, large
molecular weight, sometimes
charged, and labile
molecules to the brain,
especially upon administration
through the bloodstream,
is impossible.
