Shreya Shukla is a Development Scientist at CCRM. Her scientific interests lie at the intersection of T cell immunotherapy, gene editing and stem cell bioengineering. Shreya obtained her BSc in Biomedical Engineering from Georgia Institute of Technology and PhD from Dr. Peter Zandstra’s laboratory at University of Toronto (UofT) focusing on the scalable generation of progenitor T cells from pluripotent stem cells and hematopoietic stem cells. Her interests in science communication and education inspired her to co-found the UofT chapter of Engineering World Health (@EWHUofT) that aims to mobilize the UofT student community to innovate novel solutions to tackle global health challenges in low- and middle-income countries. You can follow Shreya on Twitter @shukshre.
Imagine you are a scientist who has worked most weekends in the lab for the past several years performing meticulous experiments to test a brand-new approach for treating a disease. Imagine that after all this time, and after building upon the years of work of other scientists before you, the proof-of-concept experiment finally works. Congrats! You have finished a long marathon and deserve a short respite. But don’t rest too long. If you are truly motivated to bring this novel therapy to patients who really need it, you are signing yourself up for another challenging marathon that will require not just you, but an entire army of experts working hard along with you.
The Bench to Bedside for Biotherapeutics (B3) workshop was held on October 5th in Toronto as an introductory overview for scientists interested in undertaking the daunting task of translating their fundamental science discoveries to the clinic. Held for the very first time, the B3 workshop was made possible by the collaborative efforts of seven Canadian organizations comprising the Clinical Translation Education Group: BioCanRx, CCRM, CellCAN, Foundation Fighting Blindness, OICR, OIRM and Stem Cell Network. The objective of the workshop was to expose scientists to the complexities of designing pre-clinical studies and putting together a clinical trial application (CTA), as well as providing networking opportunities with others dedicated to the same task.
One of the main lessons from the day-long workshop was the importance of teamwork in taking therapies from bench to bedside. This concept was highlighted by Dr. Lauralyn McIntyre, Ottawa Hospital Research Institute (OHRI), and Josée Champagne, OHRI, who worked together on a Phase 1 clinical trial translating the use of MSCs for the treatment of septic shock (also known as the Cellular Immunotherapy for Septic Shock (CISS) trial). The first and most important step in initiating a Phase 1 trial, according to Dr. McIntyre, is to build a multi-disciplinary team of scientists, clinicians, clinical trial managers, etc., dedicated to patient recruitment, follow-up and overseeing daily operations to ensure that the trial is conducted in accordance with Health Canada regulations. Josée further underscored the point by describing the extensive volume of data that are incorporated from multiple team members into a CTA to Health Canada and defining the acronym TEAM as “Together Everyone Achieves More.”
Dr. David Kaplan, SickKids Research Institute, described how in his clinical translation experience, he had worked on a multi-centre $8 million team grant with collaborators scattered all across Canada working together to discover novel drugs for the treatment of glioblastoma. Teamwork and collaboration are indispensable for getting novel therapies to pass all the necessary clinical trial checkpoints and be approved for clinical use.
Another main take-away from the workshop was the massive quantities of pre-clinical data and documentation that need to be collected to formulate a CTA and comply with Good Manufacturing Practices (GMP) standards. Gayle Piat, Alberta Cell Therapy Manufacturing, jokingly defined GMP as “Great Mounds of Paper.”
The bar for clinical translation, according to Dr. Kaplan, is if your therapeutic has demonstrated efficacy on cultured primary cells, efficacy in a pre-clinical animal model for the disease, has preliminary toxicity data (which may need to be done in a different animal model than the one used to demonstrate efficacy) and if you understand the mechanism of action on the disease target. He described how for “repurposed” drugs that have a known safety profile in humans, the pathway to the clinic is short; however, new drugs can take years to get approval due to the amount of time necessary to collect the required pre-clinical data.
Friederike Pfau, a Quality Assurance Manager for Laboratory of Experimental Organogenesis, stressed that, for auditors, if one step of the production process has not been documented, it didn’t happen. Friederike described how a quality management system (QMS) is necessary to keep track of documentation in exquisite detail for all reagents and test records used in a production batch, down to certificates ensuring GMP quality of carbon dioxide that is fed to cells growing inside incubators! Every measure is taken to meet the highest quality standards for patient safety.
The day ended with a great discussion between Drs. Mary Sunderland, Foundation Fighting Blindness, and Dawn Richards, Clinical Trials Ontario, on the importance of patient engagement. Together, they urged scientists to engage with patients early when drafting clinical trials to understand their unmet needs and increase public awareness about the lengthy clinical trial process. Dawn stressed how science is cool and gives people living with illnesses hope and inspiration in their daily lives. It is the responsibility of every scientist to convey to the very taxpayers funding their research how novel therapies are steadily and excitingly moving towards the clinic.
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