Team Develops Targeted Therapies for Pain and Inflammation

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.

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.

LaunchPad, a project of UCSF's Clinical and Translational Science Institute, is designed to highlight the experiences and accomplishments of UCSF’s translational researchers, and to support them in their efforts to develop beneficial medical products.

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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 small-molecules 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 UCSF’s Clinical and Translational Science Institute (CTSI) that aims at accelerate the translation of scientific advances to improvements in healthcare.

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.

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 body’s immune system. As an immune disorder, IBD is commonly treated with drugs aimed at suppressing the immune system. However, because the body’s 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.