Catalyzing the translation of stem cell therapies to the clinic

The People Driving Innovation in Canada’s Regenerative Medicine and Cell Therapy Sector

TRADITIONALLY POWERED BY STEM CELLS, REGENERATIVE MEDICINE (RM) focuses on replacing, repairing or regenerating human cells, tissues and organs. In the coming decades, regenerative medicine therapies and technologies will revolutionize patient care, generating economic and health benefits for all.

Beginning with the discovery of stem cells by Drs. James Till and Ernest McCulloch in the early 1960s, Canada has played a leading role in moving cell and gene therapies and technologies to the bedside. The creation of the Stem Cell Network (SCN) in 2001 bolstered our nations strengths across the stem cell and RM field, establishing an international reputation for impactful research and high-quality talent. Today, through its suite of research funding programs, SCN is powering stem cell and RM research in more than 26 disease areas, including heart disease, diabetes, cancer and neurological diseases, and fueling the talent pipeline by training the nextgeneration to work in life sciences and biomanufacturing. Recently, SCN had the opportunity to speak with a few key Canadian innovators working to bring novel therapies to patients.

ACCELERATING THE DEVELOPMENT OF ‘MADE-IN-CANADA’ CELL AND GENE THERAPIES

In the summer of 2000, University of Alberta scientists captured the world’s attention by achieving what until then had seemed impossible –developing a successful islet cell transplant protocol to treat type I diabetes.

Dr. Greg Korbut was one of those scientists, and building on his early achievements, applied for and received $26.2 million in funding* in 2009 to build a state-of-the-art Good Manufacturing Practices (GMP) cleanroom facility. The Alberta Cell Therapy Manufacturing (ACTM) is one of the first of its kind and offers a range of services from technology transfer, process and assay development through to GMP manufacturing for clinical trials.

“Our goal is to help researchers and industry worldwide bring more innovative cell and gene therapies to the clinic,” said Dr. Korbutt. “We are driven to help clients navigate and optimize the bench-to-bedside process, ultimately bringing safe, timely, and high-quality therapies to patients in need.”

In 2011, Gayle Piat joined the University of Alberta to oversee the construction and equipment procurement for ACTM. Following its completion, Gayle moved into a management role, currently serving as facility Director. One of her earliest challenges was finding qualified and experienced personnel. While this remains a global challenge in the GMP and cell culture space, training programs like SCN’s are addressing this talent gap to build capacity and support the growth of the sector.

“It has been said that it takes a village to raise a child, and, in the case of cell and gene therapies, it takes a community to raise a new product,” said Ms. Piat. “Community-based networks like the Stem Cell Network have been essential to this ecosystem. They facilitate interactions across the diverse stakeholders in the sector, take strategic risks to move research forward, and, importantly, recognize that a well-trained workforce is a requisite for accelerating translational research.”

ACTM recently celebrated a major milestone: manufacturing their first clinical product, a CAR-T cell cancer therapy, infused into a patient in 2021. They remain committed to bringing innovative therapies to patients for years to come.

Accelerating Drug Discovery By Harnessing The Potential Of Muscle Stem Cells

Professor Penney Gilbert grew up in a small town in New England and became the first in her family to attend university. After earning a Bachelor of Science degree, Penney worked as a research technician at the University of Pennsylvania under the guidance of Dr. Chris Burd.

“It was in this lab that I discovered my passion for research and desire to pursue a career in academia,” said Dr. Gilbert. “While I was always curious about how the natural world worked, my path to becoming a professor and leading a team of researchers was the result of having supportive parents and mentors at every stage of my career.”

Dr. Gilbert attended graduate school and completed her postdoctoral training before opening the Gilbert Lab at the University of Toronto in 2012. Her research team focuses on keeping muscle healthy and strong throughout life by finding ways to stimulate muscle stem cells and harness their potential. Her lab is renowned for engineering “muscle tissue in a dish”, used to predict how patients may respond to therapies. This invention is accelerating the translation of novel therapies from the bench to the bedside.

Penney accredits the Stem Cell Network as being integral to her success.

“The mechanisms embedded within the Stem Cell Network introduced and integrated me into the community of stem cell researchers across the country,” said Dr. Gilbert. “They selflessly supported me throughout my career.

For others following in her footsteps, Dr. Gilbert shared these inspiring words.

“Surround yourself with creative and energetic people who have a passion for science. Identify individuals who enjoy demystifying academia and take advantage of opportunities to get outside your comfort zone. Most importantly, be kind to yourself.”