Duke CTSA Regulatory Resources Assist Researchers Throughout Projects

March 3, 2020

Jim McNamara & Bruce Burnett
Jim McNamara, MD, and Bruce Burnett, PhD

Jim McNamara, MD, was stuck. In his laboratory within the Department of Neurobiology, McNamara and his team have spent decades working to elucidate the
cellular and molecular mechanisms underlying epileptogenesis, the process by which a normal brain becomes epileptic.  The epilepsies constitute a group of common, serious neurological disorders, among which temporal lobe epilepsy (TLE) is the most prevalent and devastating. As part of this work, they are developing peptide and small molecule inhibitors of the BDNF receptor tyrosine kinase (TrkB) signaling in an effort to prevent or cure temporal lobe epilepsy.

McNamara, a Duke School of Medicine Professor in Neuroscience and a co-principal investigator of Duke’s Clinical and Translational Science Award (CTSA), thought
a molecule he was studying in the lab was ready to study via a phase 1 trial.  And this is where he got stuck.

“Quite frankly, I had no earthly idea where to start in an effort to advance this peptide to the clinic.”

McNamara needed to know the steps to advancing his molecule (his idea) from the lab to filing an Investigational New Drug (IND) application for FDA authorization to getting approval to administer an investigational drug to humans (e.g., to conduct a phase 1, first-in-human, study). 

Fortunately, Duke’s CTSA is home to nationally recognized experts in regulatory guidance for everything from preclinical requirements to first-in-human studies and beyond. The Office of Regulatory Affairs and Quality serves as a resource facilitating regulatory strategy development, IND/IDE preparation and maintenance, GCP/GLP/GMP requirements, and interfacing with regulatory authorities, such as the FDA. 

In his quest for answers, McNamara contacted Bruce Burnett, PhD, the director of Regulatory Affairs at Duke and the Regulatory Core senior leader within Duke’s Clinical and Translational Science Institute.

“In one brief conversation with Bruce, I learned an immense amount.  He told me what needed to be done and how to go about it,” said McNamara. “Bruce provided the blueprint for what I needed to do and how I needed to do it.  After that conversation, all the pieces fell into place!”

Burnett has been with Duke since 2008, but has also been on loan to the NIH for the past few years, developing and building regulatory knowledge and resources at the national level. With his long-distance oversight and guidance, Duke, via the CTSI, continued to strengthen the Office of Regulatory Affairs and Quality to provide resources and guidance to Duke scientists and researchers in all disciplines across campus. While ORAQ is now housed within the School of Medicine, the staff continue to provide core support on behalf of the CTSI.

The senior directors and regulatory staff work tirelessly to support the work required to move a molecule or biologic into a translational drug development phase to safely test in humans and eventually translate into medical practice and ultimately healthcare improvements.

Burnett and the ORAQ leadership team, Amanda Parrish, PhD, and Erika Segear, PhD, love what they do. They are incredibly well-connected within the research community at Duke, and work hard to provide just-in-time support as well as longer term training and education.

“We love the enthusiasm of the Duke investigators and enjoy the collaboration,” said Burnett. “Our goal is to be there for the long haul to see each project through to success.”

There are very real barriers associated with the translation of an idea from the lab bench through the appropriate regulatory processes and into human studies. Most often, the lack of familiarity with the process or the knowledge to know which questions to ask or the path to take can present obstacles that discourage researchers from advancing their idea beyond the lab.

“What’s exciting is that we have the knowledge and expertise here at Duke to transform an idea to IND in a day!” says Burnett. “We run our office just as is done in an industry setting. We have vast coverage across disciplines, and we’ve supported hundreds of molecules through the process, licensed products with the FDA, and everything in between.  Our office is unlike any other ‘regulatory’ office in an academic setting.”

In many cases, scientists aren’t even aware how close they might be from taking their “basic” concept to a translational clinical discovery. 

“That’s where we can provide the essential knowledge and expertise.  Our team can stay with the project, at the pace of the research, providing the critical and just-in-time quality assurance function at every milestone along the translational journey.”

In McNamara’s case, due to the “hugely useful input” from Burnett and some hard work on Jim’s part, his team submitted their application to a funding agency seeking support for additional preclinical IND enabling studies. Success with this application will jumpstart future NIH funding for the remainder of the project.