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Team Develops Targeted Therapies for Pain

and Inflammation
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The two most common ways to administer drugs are oral ingestion or intravenous
injection. These methods disperse medication systemically, and only a small portion
of the dosage actually reaches the part of the body that is in need of therapy.

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Targeted drug delivery aims to get therapeutic medication directly to the site in the body that
needs it, without exposure to healthy tissues and the resulting side effects.
UC San Francisco researcher Aditi Bhargava, an associate
professor in the Department of Surgery who has expertise
in molecular biology and neuroendocrinology, is working on
the development of targeted therapeutics in areas of bowel
disease and pain. She has teamed up with others from
UCSF, including collaborators Peter Ohara, PhD, a
neuroanatomist, and Luc Jasmin, MD, PhD, a
neurosurgeon, to develop a method for delivering smallmolecules to a specific target group of cells for treatment of
pain.
We came up with a technique that could be used like a
Trojan horse, encapsulating and delivering RNA-based
therapies or small drug-based therapies to specific
neurons, says Bhargava of the initial project collaboration
involving targeted pain management.
Bhargava has received support for her research from the
Catalyst Awards, a program managed by UCSFs Clinical
and Translational Science Institute (CTSI) that aims at
accelerate the translation of scientific advances to
improvements in healthcare.

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She shares her experiences in navigating translational

science in a new series of educational resources featured
on LaunchPad, a new online resource for investigators also
managed by CTSI. She describes how Catalyst Awards advisors helped her better understand
how to take her work through the next steps of the process, such as teaming up with strategic
partners, and worked with her to expand her goals and create a broader delivery platform that
also includes non-pain models.

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also includes non-pain models.

Making a Strong Development Plan

It is helpful for investigators to gain exposure not only to the process of moving their work into
the marketplace, but also to the perspective of those who have come from that world, said
Ruben Rathnasingham, PhD, associate director of the Early Translational Research program
at CTSI, which manages LaunchPad and the Catalyst Awards. Oftentimes during a Catalyst
Awards consultation, investigators change course after becoming aware of the challenges and
limitations of their current line of inquiry.
A consultation provided by Catalyst Awards advisor Alex Bajamonde, PhD, led Bhargava to
also pursue a targeted therapeutic approach for inflammatory bowel disease (IBD), which she
is actively researching. Pain is an integral component associated with severe inflammation and
people with IBD suffer significant abdominal pain when areas of the colon or intestine become
inflamed due to attacks by the bodys immune system. As an immune disorder, IBD is
commonly treated with drugs aimed at suppressing the immune system. However, because
the bodys immune system is critical for overall health, systemic delivery of these drugs can
lead to a variety of unwanted side effects such as infections and lymphoma. In addition, as
IBD can affect different regions of the gut, the ability to locally target drug delivery specifically
to affected regions would be extremely beneficial, says Bhargava.
My hope is that this treatment would allow people with IBD to be treated as soon as the
disease is identified and we can intervene early enough and get them to a stage where they
would be disease-free, explains Bhargava. She also anticipates that her targeted delivery
platform could be applied to other healthcare domains.
Bhargava is currently in talks with strategic industry partners who are interested in using her
technology to improve the delivery of their drugs aimed at treating IBD. This partnership would
present Bhargava and her colleagues with a clear path towards providing an alternative
treatment to patients who urgently need it.
UCSF's CTSI is a member of the Clinical and Translational Science Awards network funded
through the National Center for Advancing Translational Sciences (grant Number UL1
TR000004) at the NIH Health. Under the banner of "Accelerating Research to Improve Health,"
CTSI provides a wide range of resources and services for researchers, and promotes online
collaboration and networking tools such as UCSF Profiles.
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