Robert Nussbaum, MDBy Kate Rauch
With the goal of slowing or stopping the spread of Parkinson’s disease, UCSF geneticist and neuroscientist Robert Nussbaum, MD, and a team of researchers have developed genetically-engineered mice to test drugs and medical therapies. In particular, he has his eye on Posiphen, a drug already under study with Alzheimer’s patients that blocks the production of alpha-synuclein, a central player in the disease.
The mice Nussbaum is testing express a rare form of the human alpha-synuclein gene associated with Parkinson’s disease. The mice are already showing early signs of the disease, as well as the tell-tale presence of excess alpha-synuclein, a protein that collects in the nerve cells of people with Parkinson’s.
However, Nussbaum says that laboring in his lab and navigating the world of pharmaceutical product development are as different as two languages. He admits that the world of drug development is an area he is still awkwardly getting to know.
With this in mind, Nussbaum applied for the T1 Translational Catalyst Award, offered by UCSF’s Clinical and Translational Science Institute (CTSI). The award is designed to help drive promising research through the complexities of early stage development by connecting UCSF scientists to customized expertise and capabilities outside of UCSF and the traditional academic realm. The phased award offers up to $100,000 of funding but more importantly, customized consultation and support for medical and health product development including legal, financial, and regulatory issues.
After Nussbaum’s proposal review that included a round-table project discussion by a panel of experts from UCSF and industry, he was offered consultation with industry and business community experts who provided guidance and helped develop a future strategy. During this phase, his consultants made what Nussbaum calls a “wise” suggestion. The result is the formation of a new, national collaboration to study Posiphen and Parkinson’s.
“Steve completely turned the process on its head,” Nussbaum said, of his primary T1 consultant, Stephen Freedman, PhD, an industry expert in neurodegenerative diseases with extensive experience in licensing programs and therapeutics from academia and brokering industry-academia partnerships. “He’s a very straightforward guy. In a forthright way he helped me swim through the complexities, and made it very clear.”
Posiphen is owned by a small biotech company, QR Pharma, which holds intellectual property rights. Nussbaum’s original proposal was to use small amounts of the drug for animal testing with permission from the company, an interested partner.
Citing the time-consuming challenges of doing research with drugs owned by a different entity, Freedman outlined a partnership between Nussbaum, QR Pharma, Harvard University, and the National Institutes of Health (NIH), with the biotech company retaining Posiphen rights. Nussbaum would act as a research consultant to the company. Freedman also helped broker the deal as a facilitator. “Steve asked all the right questions,” Nussbaum said.
The T1 Catalyst Award provided additional, extended consultation with Freedman and a grant writer, Terry Reisine, PhD, to guide the new team on applications for significant funding from the Michael J. Fox Foundation and the NIH. “Terry helped us organize things, he kept us on time tables, and made sure we met our targets,” Nussbaum said. “Until now, I’ve been completely ignorant about drug development. Now I know a little more, and it’s been interesting.”
CTSI at UCSF is a member of the national, NIH-funded CTSA network focusing on accelerating research to improve health. The T1 Translational Catalyst Award is among a wide range of CTSI resources and services that support research at every stage.
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