Harnessing the potential of customizable stem cells

- Ottawa, Ontario

New international collaboration aims to accelerate therapies for cancer and other diseases using precisely engineered cells from the patient's own body.

A reprogrammed human induced pluripotent stem (iPSC) cell colony expressing a key stem cell marker.

Imagine treating blindness, diabetes and Parkinson's disease without drugs, or replacing diseased heart and kidney tissues without waiting for compatible donors. It's not just a utopian dream.

Scientists in Canada and Japan are collaborating to design stem cells for regenerative medicine and cancer treatments. These cells have the potential to serve as "universal donors" for repairing or replacing damaged cells and tissues without the usual risk of rejection.

In Japan, in the early 2000s, Dr. Shinya Yamanaka made a major breakthrough in this field. He found the key to generating patient-derived stem cells that can be used to better understand and develop treatments for a number of diseases. By adding just 4 genes to adult skin cells, they became like embryonic stem cells, which can produce any type of cell in the body. He called them induced pluripotent stem (iPS) cells and won the Nobel Prize in Medicine in 2012 for the discovery.

Looking to capitalize on the possibilities, the National Research Council of Canada (NRC), Concordia University and Kyoto University's Center for iPS Cell Research and Application (CiRA) in Japan launched a powerful international alliance in January 2022 to accelerate research toward the use of iPS cells in therapeutic applications.

"This collaboration is bridging the Pacific Ocean to bring together experts in stem cells, genome engineering tools and technologies, and robotics," says Dr. Knut Woltjen, Principal Investigator at CiRA. "We share the dream of developing powerful, robust, accessible and safe iPS cell-derived therapies."

NRC researchers see infinite potential in iPS cells, which can be customized to develop treatments for many hard-to-treat conditions and disorders, such as neurodegenerative diseases and cancer. And, because cells are living entities, they can replace lost function and be armed to fight cancer.

Uniting complementary strengths

Each partner agency brings a slightly different expertise to the table, making for an ideal collaboration. CiRA researchers will develop novel genome editing tools with improved editing precision and efficiency. Concordia University's Centre for Applied Synthetic Biology (CASB), Canada's genome foundry, will automate the platform using its pioneering robotics technology. The NRC will prepare and provide supporting technologies such as novel iPS cell lines and CRISPR/Cas9-based genome engineering tools.

"This 3-way partnership is of tremendous value to Canadians and our life sciences ecosystem," says Dr. Vincent Martin, professor and CASB director. "It enables us to leverage different expertise, tools and investments in synthetic biology to help mobilize solutions to rapidly advance cell and gene therapy development."

Bridging the translational gap

Cell-based therapies offer vast therapeutic potential for treating diseases that don't respond well to conventional therapies. But, as these are still relatively new medical treatments, a significant amount of time and effort is still needed to move a scientific breakthrough into something practical that can be used with actual patients. The industry term for that time and effort is "translational gap" and it is particularly challenging for cell and gene therapies.

"Driven by the NRC's Cell and Gene Therapy Challenge program and brokered by our International Innovation Office, this collaboration with CiRA and Concordia is a crucial step in minimizing the large translational gap for new engineered cell and gene therapies," says Dr. Kelley Parato, the NRC's Cell and Gene Therapy Challenge program director. "It will help make these new treatment opportunities available faster to both Canadian and international patients."

Bridging that gap starts with the Cell and Gene Therapy Challenge program's Precision Engineering master project, led by NRC researchers Dr. Simon Drouin and Dr. Anna Jezierski. They have already begun developing the tools and iPS cells required to bring stem-cell therapy into the mainstream.

Although this is a complex and challenging endeavour, Dr. Drouin says the NRC's expertise in genomics and cell engineering is critical to supporting the collaboration. "In addition, our experience developing and scaling-up the processes for creating customized engineered cell solutions will enable us to make a significant contribution to advancing iPS cell-based therapies in Canada and around the world."

Dr. Jezierski adds that by developing its own novel iPS cell lines and optimizing the production of the most useful cell types for these therapies, the NRC is making real progress toward validating and translating the research tools into tangibles outcomes.

Strengthening international collaborations

With satellite offices in Tokyo and Munich, this new alliance is another step in the NRC's path to increasing international research collaborations for finding creative, relevant and sustainable solutions to current and future economic, social and environmental challenges.

"This partnership is the cornerstone of an important and strategic Canada-Japan collaboration," says Renato Caldart, Senior Advisor of International Relations responsible for the NRC's Japan engagement at the time. "It will serve as a foundation for future international efforts in this space and will be an important milestone in the development of clinically relevant iPS cell-derived engineered cell-based therapeutic approaches."

Ultimately, this type of collaboration will develop the foundation upon which safer, more affordable and accessible cell therapy products will be developed. While this project is still in its early stages, the collaborators anticipate it will lead to the development of accurate, robust and safe genome engineering tools and platforms to produce powerful cell and gene therapies.

For thousands of Canadian patients, and thousands more around the world who live with difficult-to-treat conditions like diabetes, Parkinson's disease or cancer, these new therapies may be exactly what they've been waiting for.

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