Growing up on the East Coast, I was always near the water. The ocean and the natural world were really important to me, and I used to go out on whale watching boats pretty often when I lived in New England. The marine biologists on those boats seemed like they had the coolest job—they explained to everybody what the creatures were and why they’re so important in the ecosystem, how each of them worked together. I grew up thinking I was going to be a marine biologist.
I was in high school when my family moved to Arizona—we were landlocked, and marine biology wasn't a course of study at my state school. In my first year, I took the freshman general biology intro course. It was a giant class, so big, in fact, that we had to take our exams in the basketball stadium. The curriculum was divided into three parts—ecology, evolutionary biology, and molecular biology.
That first year of college, at seventeen years old, was my first real exposure to molecular biology. I loved it. I was fascinated with how these teeny, tiny molecules in every single cell could have so much impact. That fascination set me on the path to studying molecular biology.
In graduate school, I worked on the basic research for a newly discovered human enzyme, running on the premise that if we could understand this enzyme better, we could harness its activity in improving outcomes and quality for patients receiving a certain class of chemotherapy drug. Gene therapy was in the news, and we were just getting our arms around other things like RNA interference. It seemed like all of these advanced therapeutics were going to cure everything before I could even get into the field. Obviously, we’re still not quite there! The world is still refining those tools, but with some of them now approved for human use, we're on the right track.
After my postdoc training, I then spent some time in drug discovery. In a sense, that was the boat that led me through bench research to clinical research. Being on teams who moved compounds into clinical trials was exciting, and opened my eyes to that whole realm of where the molecules go once we discovered them. It got me thinking about doing the science versus treating the people. I spent several more years on the discovery side, deep-diving into all kinds of areas - from infectious disease to cancer to neurology.
During this time my mom became unwell. I took a year off from my career track to spend her last year with her. Once it was time to return to a career, I focused on going into the clinic with other people's molecules, transitioning to drug development.
My mom’s condition was advanced enough that she didn’t qualify for some of the more probable treatment candidates in later phases, but she wanted to contribute to science by participating in clinical trials. Every Monday there was a 12 to 14-hour day we would spend together in the clinic doing pharmacokinetic sampling. To live that trial with my family forever changes the way I think about involving patients, and the way it's all going to seem through their eyes.
Like those marine biologists I admired as a kid, our team is thinking about patient communities in their natural habitat, not just in the research clinic. That's what we need to be doing more of in the clinical development space – bringing the trials to the actual lives the patients are currently living, making that research a care option they can access without disrupting everything else they have going on.
I think that everybody's path involves a certain amount of luck, but between college and graduate school I was very, very fortunate. I spent a couple of years in pediatric oncology, working with clinical scholars. They cared deeply about their patients, and these doctors strived for improved clinical care to give these kids. They were a part of a healthy ecosystem of patients, their care, and their treatments.
They had a research program separate from their clinical work, but we researchers were often in the clinic to discuss our results with them. The whole point of the research program was to develop technologies that could be moved to companies for clinical development, hopefully bringing new treatments to these kids. It was a transformative experience for me to spend a certain portion of the day and week on the pediatrics patient floor. You become engaged with these kids and are part of the social community that they created. It really brought to life why we did what we did in the lab. It showed the importance of everyone’s role in the system.
Over 20 years ago, researchers like me were working on things that we thought could help stimulate the immune system to help them fight cancer. I wish I'd had the foresight to keep that rejection letter from this high-profile journal, telling us “there is no such thing as cancer immunotherapy.”
Now, cancer immunotherapy has changed the world, improved countless lives, given so many families more time together. It was exciting to be part of the teams who got technology and compounds into trials. It’s still kind of like being on a boat, we are constantly seeing landscapes change and shift. I like to think of my work now in a similar way—we change the landscape of treatments.
That’s what I want to do for the rest of my life—the research that changes human health.
RARE DISEASES NEWSLETTER Volume 6, May 2017
This quarterly publication will keep you up to date on PRA’s Rare Disease Team focus, achievements, and new initiatives.
Rare Diseases and the Promise of Gene Therapy: What You Need to Know
Cross-Border Enrollment of Rare Disease Patients
Clinical trials in rare diseases present unique challenges unseen in trials for more common conditions. Cross-border enrollment can be key to…