Harnessing the potential of customizable stem cells

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

A colony of reprogrammed human induced pluripotent stem cells (iPSCs) expressing a key stem cell marker.

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

Scientists from 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” to repair or replace 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 area. He has 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 have become like embryonic stem cells, which can produce any type of cell in the body. He called them induced pluripotent stem cells (iPS) and won the Nobel Prize in Medicine in 2012 for this discovery.

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

“This collaboration bridges the Pacific Ocean to bring together experts in stem cells, genome engineering tools and technologies, and robotics,” says Dr. Knut Woltjen, senior researcher at CiRA. “We share the dream of developing potent, robust, accessible and safe iPS cell-derived therapies.”

NRC researchers see endless 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 functions and be armed to fight cancer.

Join complementary forces

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

“This three-way partnership is invaluable to Canadians and our life sciences ecosystem,” said Dr Vincent Martin, professor and director of the CASB. “This allows us to leverage different expertise, tools and investments in synthetic biology to help mobilize solutions to rapidly advance the development of cell and gene therapies.”

Bridging the translational gap

Cell therapies offer vast therapeutic potential for treating diseases that do not respond well to conventional therapies. But, since these are still relatively new medical treatments, it still takes a lot of time and effort to turn a scientific breakthrough into something practical that can be used with real patients. The industry term for this time and effort is “translation gap” and it is particularly difficult for cell and gene therapies.

“Led by the NRC’s Cell and Gene Therapies Challenge program and brokered by our International Office of Innovation, this collaboration with CiRA and Concordia is a critical step in closing the large translational gap for new engineered cell and gene therapies. “, said Dr. Kelley Parato, director of the NRC’s Cell and Gene Therapy Challenge program. “This will help make these new treatment options more quickly available to Canadian and international patients.”

Bridging that gap begins with the Cell and Gene Therapy Challenge program’s master’s project in precision engineering, led by NRC researchers Dr. Simon Drouin and Dr. Anna Jezierski. They have already started developing the tools and iPS cells needed to make stem cell therapy mainstream.

Although it is a complex and demanding undertaking, Dr. Drouin says NRC’s expertise in genomics and cell engineering is essential to support the collaboration. “Furthermore, our experience in developing and scaling processes to create custom engineered solutions will allow us to make a significant contribution to the advancement of iPS cell therapies in Canada and around the world.”

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

Strengthening of international collaborations

With satellite offices in Tokyo and Munich, this new alliance is another step in NRC’s journey to increase international research collaborations to find creative, relevant and sustainable solutions to current and future economic, social and environmental challenges.

“This partnership is the cornerstone of an important and strategic collaboration between Canada and Japan,” said Rene Caldart, Senior International Relations Advisor responsible for NRC’s engagement in Japan at the time. “It will serve as a foundation for future international efforts in this area and will be an important step in the development of engineered cell therapeutic approaches derived from clinically relevant iPS cells.

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

For thousands of Canadian patients and thousands more around the world living with hard-to-treat diseases like diabetes, Parkinson’s or cancer, these new treatments could be just what they’ve been waiting for.

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