Learning to Appreciate the Process
Anne Fung, Medical Director, Genentech Ophthalmology
My father is a retina specialist. In high school, he planted the first kernels of curiosity about medicine. He spoke with wide eyes about ‘recombinant DNA.’ And later about a substance I later learned to understand as ‘VEGF.’
While the dinner table talk may have been difficult for an outsider to follow, it taught me that while healing through medicine can make people’s lives better it sometimes comes up short – Mrs. Shimada’s joy as she recovered vision after retinal detachment surgery… but Mr. Brown’s frustration with vision loss from age-related macular degeneration (AMD).
Eventually, I would learn that my father had been audacious enough in the 1980s to try a novel medical therapy, interferon alpha, for neovascular macular degeneration when blinding laser treatment was the standard of care. And while further research did not support its use, the experience put AMD and the possibility of a medical therapy on my radar.
Fast-forward two decades… Dad and I had been practicing retina together for 10 years when I asked him what he thought about my taking a position with Genentech to help interpret and communicate clinical data and foster my colleagues’ work through the supported research program. His eyes brightened again – decades of awe and wonder bubbling to the surface.
Inspired, I happily joined Genentech in March 2014.
Researching AMD has been a passion of mine since the early 2000s. I always thrived on running our clinical research program when I was in private practice. When I wasn’t participating in a study, I kept up with the latest published papers and loved learning from my colleagues about the newest devices and medicines that could help our patients’ vision.
During my fellowship, I was a sub-investigator on one of Genentech’s first AMD studies and learned first-hand about the rigorous standards applied in clinical trials. I also participated in three investigator-sponsored studies on the nuances of AMD. (Investigator-sponsored means that the idea for the study is developed by the physician and the pharmaceutical company helps by providing drug and/or financial support.)
When there was an opportunity to become a lead investigator for another one of Genentech’s AMD studies, I was excited to participate. However, despite those experiences, I often wondered why biotech and pharmaceutical companies couldn’t make a drug better overnight, or last longer, or get us other medicines that our patients needed faster. As a physician who wanted to provide the best care, I found this frustrating.
It wasn't until I started my new role that I realized a new appreciation for what it takes to bring a medicine from the lab to the patient. What I discovered was that each step is more complex than expected - involving everything from asking the right question to figuring out how to answer it and then understanding what we found. But each arduous step has purpose, and our patients are at the centre of each decision.
There is an art, as well as a science, to deciding which lines of research to pursue for treating a disease. That art is what has given Genentech a reputation for making good decisions about what to study, when to pause, and how to proceed.
What most people may not know is that there are several steps — several is probably an understatement here — before we can even begin research on a potential compound.
In ophthalmology, a team first needs to discover what has gone awry in the normal workings of the eye that led to disease, and then design a drug that is effective enough to stop it - yet safe for everything else. Next, a small study is conducted to test the drug's safety. If it passes, then a larger study is conducted to find the appropriate dose and get an early sense of its effectiveness. And if that works, then we embark on large Phase 3 trials comparing the new drug to see if it is better than the current standard of care.
Another team organises and coordinates clinical trials for upwards of 100 research sites – ensuring each one is ready with certified cameras, staff and physicians – and all in compliance with federal regulations that govern human studies. Then we need to manage the FDA’s requirements with our expectations of what we hope to accomplish in the study (for instance…is the compound safe to use? Does it improve someone’s disease? Does it improve someone’s symptoms? Are there particular patients who respond better to the therapy than others? Is it better than anything else on the market to date?)
As a physician investigator, I respected Genentech for its scientific standards, and some of the cleanest protocols in the industry. Now that I’m part of this team, I get to see the rigor first-hand. For instance, we have an entire team and building completely dedicated to making the liquid in which our drug-producing cells grow. Without the dedication and expertise of this group, cells would not grow at the correct rate, or they wouldn’t get the right nutrients at the right time.
Our patients rely on us to deliver novel therapeutics and if we can’t precisely and safely manufacture those therapeutics and replicate that process in other facilities, in other countries, then medicines aren’t getting to the people that need them. And that is unacceptable. This sometime forgotten but vital step is just one of the many important parts of the process of developing medicine that I have come to appreciate.
Our approach to drug development likely makes us more cautious than other companies. But it is this deliberate care in how we make medicine that reflects our commitment and a long-term vision for making people’s lives better through healing.
Adapted from an article on www.gene.com which first appeared in print on 8 Aug 2014. https://www.gene.com/stories/learning-to-appreciate-the-process?topic=ophthalmology