The long slog of medical R&D and finding inspiration

Author: Elizabeth Csaszar, 03/27/17

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Research and development (R&D) of a medical therapeutic is a long slog. This isn’t news to anyone working in the field. The average time to bring a new drug product to market is over a decade. Moreover, this is the timeline when everything progresses well – funding and business decisions align, manufacturing processes come together, and regulatory submissions don’t face any substantial setbacks. For less established cell and gene therapies where the clinical translation road is less clearly paved, the path can look daunting.

I’ve often thought of this process in contrast to medical breakthroughs of the past. We hear brief sound bites of discoveries of cures and new treatments that seem so sudden and dramatic: Alexander Fleming’s discovery of penicillin; Frederick Banting‘s isolation of insulin; Jonas Salk’s development of the polio vaccine. Often these accounts are of the lone scientist or clinician having an eureka moment and, mere months later, lives being saved. It’s easy to romanticize these stories and the way at which they seem to have led to improved health outcomes so rapidly and effortlessly.

I recently read two non-fiction books back-to-back that provided some perspective. The first book was The Philadelphia Chromosome: A Genetic Mystery, a Lethal Cancer, and the Improbable Invention of a Lifesaving Treatment by Jessica Wapner. This is a detailed account of the discovery and development of the blockbuster drug Gleevec (Imatinib), a tyrosine kinase inhibitor primarily used to treat Chronic Myeloid Leukemia (CML), which was approved by the FDA in 2001.

The story is complex and winding, as one might expect with modern drug development. It chronicles the many stakeholders, tricky academic-industry relationships, tough market decisions, passionate but frustrated champions, bruised egos, and seemingly endless setbacks. It is clear that the individuals involved often felt that they were caught in the long slog. And yet, Gleevec was indeed brought to market, and many thousands of patient lives have been dramatically improved and extended as a result. The book left me feeling inspired, but also a bit overwhelmed by the drug development process.

Then I read Breakthrough: Banting, Best, and the Race to Save Millions of Diabetics, by Thea Cooper and Arthur Ainsburg. This book recounts the famous discovery of Insulin in the early 1920s, and its subsequent productization by Eli Lilly. As a Canadian scientist (who until very recently went to work everyday in The Banting Institute in Toronto), I am certainly familiar with Banting’s work, but had never read deeply about it.

Fascinatingly, I learned that the discovery and development of Insulin was also a path full of many stakeholders, tricky academic-industry relationships, tough market decisions, passionate but frustrated champions, bruised egos, and seemingly endless setbacks. Although occurring 80 years before the development of Gleevec, the parallels were striking. Banting may have had his eureka moment that led to the purification of Insulin from the pancreases of dogs, but he and all involved certainly felt the frustration of the long slog.

I find it refreshing to realize that the development of Insulin wasn’t as quick and easy as I had imagined, and that it had a great deal in common with the Gleevec story. Despite the challenges surrounding both discoveries, they also had much in common that was positive. For example, there was the buzz in the conference halls when early findings were presented and scientists and clinicians in the audience realized the potential clinical impact. There was excitement when legal agreements were signed and stakeholders confirmed that their partners were ready to take a leap. There were follow-up appointments with early patients, who spoke of returning to their regular life as they continued to set new records for the length of time their disease had been managed by the new drug. These are the moments that keep scientists and others engaged and motivated.

As I read these two very different yet similar accounts, I wondered what future stories will be written of the cell and gene therapy field. As we see new clinical successes emerging and excitement growing in our field, it’s inspiring to think that we are the protagonists of these stories to come.

 

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Elizabeth Csaszar

Elizabeth Csaszar

Elizabeth (Liz) Csaszar is a development manager for the BridGE team at CCRM. Her areas of interest are cell manufacturing, clinical translation of cell based therapies, and blood cell technologies. Liz obtained her PhD at the University of Toronto, focusing on the strategies to expand and self-renew hematopoietic stem cells in vitro. Liz is interested in STEM outreach and education. Follow her on twitter at @lizcsaszar
Elizabeth Csaszar

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