Updated 6.15.13: A Stanford Medicine article further describes the vismodegib research.
When I first interviewed Brian Kobilka, MD, winner of the 2012 Nobel Prize for Chemistry in October, I was struck by an off-hand comment about his motivation for his near-obsessive two-decades long research quest to uncover the workings of GPCRs, or G-protein-coupled receptors, which serve as one of the main methods of molecular communication within the body.
The research, which it’s believed will lead to the creation of new drugs for clinical care, was not originally done for this purpose. It was motivated by simple scientific curiosity - the kind that often leads to amazing discoveries that help cure suffering or save lives. Initially, Kobilka just really wanted to know how it worked.
In a story published in today’s Inside Stanford Medicine, I describe the success of research based on scientific curiosity in leading to clinical care breakthroughs. The story describes the 30-year history of scientific breakthroughs that led to the approval of a new drug called vismodegib that is used to treat inoperable basal cell carcinomas, and how the drug helped save the eyesight of 101-year-old Winnie Bazurto of San Mateo, Calif. It’s a story that begins with a similar motivation - basic scientific interest - and ends with discoveries that help patients in a very practical way. As Jean Tang, MD, PhD, Bazurto's dermatologist and vismodegib researcher, says in the article:
If a patient only knew the whole story — how the happenstance of science led to their treatment... If they could go back to when this molecular pathway was first discovered in fruit flies, they'd be amazed. It's not until the dots are connected 30 years later that it begins to make sense.
Stanford's Matthew Scott, PhD, one of the key players in this basic-science success story, commented to me in an e-mail just how essential it is for future clinical discoveries that basic science continues to be funded. He expressed concern about a current trend toward conservatism in funding that requires much quicker results that lead to treatment options for patients saying, "Current conservatism in funding asks for translational work that gives cures in a few years (which never happens). Far-sighted funding of basic science ... pays off big time.”
The vismodegib story illustrates just how essential basic science is to the future of clinical discoveries:
For many of the basic scientists involved in this research, the clinical use of hedgehog-inhibiting drugs to treat patients like Bazurto — while not the original goal of their research — is the ultimate success.
Previously: Why basic research is the venture capital of the biomedical world, Future of medical research is at risk, says Stanford medical school dean and The economic benefits of publicly funded medical research
Photo of Winnie Bazurto by Leslie Williamson