In 2011, Steve Finkbeiner, of the University of California, San Francisco Gladstone Institutes and Taube-Koret Center, participated in the Bay Area SciCafé following publication of his paper describing small molecules that stimulate autophagy as possible treatments for neurodegenerative disease (Nat. Med. 16, 1227, 2010). Key to this discovery was the invention of a patented high-throughput single-cell imaging platform that makes it possible to track the development of brain cells from patient-derived induced pluripotent stem cells.

Nature Biotechnology : How have you built on the work described in the Nature Medicine paper?

Steve Finkbeiner: Initially, our efforts were directed at developing leads from our internal academic programs far enough that they warranted industry partnerships, using financial support from philanthropists or other non-dilutive funding sources. The goal was to catalyze the discovery of therapeutics by carrying out the early-stage discovery and development work necessary to de-risk the leads. However, as we developed innovative tools and deep biology expertise to do this work, industry sought access to our platform to advance their own programs.

NBT: What types of challenges does commercialization of neuroscience research pose?

SF: Early-stage central nervous system drug discovery is viewed as risky, so the extent to which discoveries must be de-risked is especially high. Collaboration and open innovation are ways to manage risk because it reduces the investment necessary to have an effective development infrastructure. Philanthropy is absolutely critical as well. It makes it possible to carry out the development of promising leads without adding encumbrances that would ultimately make those leads difficult to partner out. Industry partnerships are essential because they are uniquely resourced to afford and execute clinical trials. My impression is that philanthropy in this area is growing, and I hope that the message that philanthropists have the opportunity to make a major difference and can see the impact of their efforts entices even greater investment.

NBT: What led you to pursue translational applications as well as fundamental research?

SF: Part of my work as an academic scientist led naturally to a focus on mechanisms of disease, which in turn led to the discovery of potential therapeutic targets. A few years ago, I was fortunate to be approached by philanthropists interested in one of the diseases we study, and with their help, created an infrastructure for developing discoveries with therapeutic potential from the academic research program. We raise about $5 from other sources for every $1 we receive in philanthropy. For example, the invention of a first-generation high-throughput stem cell platform was made possible with philanthropy. Our early successes using it attracted the resources to develop the technology further and attract pharma partnerships and sponsored research agreements.