By: Chris Lowe
Friday, January 15, 2016 was landmark day for a group of 35 young scientists at Rutgers University. While the typical pathway to FDA review of a new drug, including discovery, development, preclinical studies, and clinical trials, requires more than a decade of work and hundreds of dedicated chemists, scientists, and clinicians, the students of the 3rd Annual Interdisciplinary Boot Camp in Quantitative Biology were able to take a drug through this process in just two weeks.
The Center for Integrative Proteomic Research hosts their annual boot camp as an intensive, interdisciplinary workshop, providing participants the tools to integrate concepts of quantitative biology to further advance their disciplines and better prepare them to pioneer solutions to the world’s most challenging problems. The topic for this year’s boot camp was Drug Discovery and Development, and throughout the two-week course, experts long entrenched in the field narrated detailed perspectives on their life’s work. The course was powerfully communicated through a comprehensive case study of the development of Erlotinib (trade name Tarceva), a small molecule oncology drug indicated for non-small cell lung cancer (NSCLC). Boot camp participants were asked to turn back the clock, assimilating and assembling all the data necessary for a simulated presentation to the FDA’s Oncological Drug Advisory Committee (ODAC) panel as if they were seeking approval for Erlotinib as a first line therapy for NSCLC. Participants were broken up into 9 teams that spanned the entire drug development process, from target biology of the epidermal growth factor receptor’s kinase domain, all the way through phase III clinical trials. Together these 9 teams comprised a mock small biotechnology firm, Organic Dynamics, and worked throughout the two weeks to prepare for the 90-minute presentation during the simulated ODAC meeting.
"The small molecule drug candidate is not discovered, because it does not already exist...Drugs are invented by the work and creativity of scientists." - Dr. David Kimball, Rutgers ORED
The technical topics covered are far too numerous to describe, but included lectures and case studies regarding all of the activities critical to a drug’s evolution from initial lead to postmarketing surveillance, as well as workshops offering specific training and tools to effectively guide development through those steps. The breadth and depth of topics covered was simply astounding, and the lineup of speakers who are at the forefront of this field on a daily basis was incredible, featuring speakers from Bristol-Myers Squibb, Merck, Novartis, Colgate-Palmolive, the Cancer Institute of New Jersey, the Office of Research and Economic Development at Rutgers, Protein Data Bank and the Cambridge Crystallographic Data Center, among others.
Fortunate enough to be a full participant in this boot camp, I consistently found two themes clearly impressed upon me as a participant. Dr. David Kimball from the Rutgers Office of Research and Economic Development (ORED), armed with 30 years’ experience in the pharmaceutical industries, spoke on the first day of the program and mentioned his dislike for the term “drug discovery“. He impressed that drugs are no longer truly discovered, but instead invented by the hard work and creativity of chemists. This sentiment was echoed by many other experts who took part in the boot camp. While high throughput screening of massive libraries of chemical compounds can yield a preliminary hit, that is just the first building block for the rigorous, iterative science that follows as chemists build out from that initial molecule to achieve more and more effective variations to pass forward. The process requires as much science and technical expertise as it does creativity and innovation by clever scientists.
Even seemingly marginal improvements over the current standard of care can have substantial and lasting benefits for patients and their families.
A second critical takeaway instilled was the ethical responsibility we have to patients as emerging scientists with an eye towards medicine and the pharmaceutical industry. It was made abundantly clear that the enormous amount of time and cost that is requisite of this process is insignificant in comparison to the impact that this work has on the lives of patients affected by disease and those closest to them. As the focus of the boot camp was an oncological drug, the devastating effects of cancer were never far from mind. It was surprising at first to delve into efficacy studies for some of the drugs discussed, as the improvement in survival for new drugs was perhaps only a few months over existing recommended therapies. It may not seem like much benefit, but especially in a disease as life altering as cancer, the added time to spend with loved ones and the potential to witness family milestones can be considered completely invaluable from the patient perspective. Further, even slight improvements in quality of life or alleviating symptoms can contribute overwhelmingly to patient morale and well-being. Even seemingly marginal improvements over the current standard of care can have substantial and lasting benefits for patients and their families. Altogether it’s a severe, but critical, perspective to have gained moving forward into our careers.
The 2016 Interdisciplinary Boot Camp in Quantitative Biology was a rousing success for all lucky enough to take part. The challenging, intensive course was transformative for its 35 participants and its rewards are sure to be reaped over the course of many, successful careers where this diverse group puts what they’ve learned into practice. An enormous thanks to all who donated their time and energy to ensure the success of the boot camp, but in particular to both Dr. Gail Ferstandig Arnold and Dr. Steven Burley from the Institute of Quantitative Biomedicine at Rutgers whose passion for the subject matter, commitment to teaching, and dedication to hosting such a comprehensive experience was evident each and every day of the program. If you missed out on this year’s program, mark your calendars for next January!
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The young team of scientists of Organic Dynamics before their simulated ODAC meeting Friday, January 15, 2016[/caption]