Success Story

Game-Changing Research Finds New Class of Stem Cells, Could Help Treat Diseases from Cancer to Diabetes

A global team led by Mount Sinai’s Dr. Andras Nagy has discovered a new class of stem cells that could help to treat a myriad of degenerative diseases. This four-year project will profoundly affect future research, world-wide, in stem cells, cancer, fetal development and regenerative medicine.

It doesn’t get much bigger than this. Research spanning four continents and spearheaded by Dr. Andras Nagy at Mount Sinai Hospital’s Lunenfeld-Tanenbaum Research Institute has culminated in a detailed molecular map of major stages in the transformation of specialized cells, such as skin cells, into stem cells.

This research also led to a game-changing discovery: a new stem cell type, previously dismissed as “junk,” that’s easier to grow than others. It could help to treat and cure currently incurable and frequently devastating diseases such as blindness, Parkinson’s, Alzheimer’s, spinal cord injury, stroke, diabetes, blood and kidney diseases and many others that are associated with tissue damage and cell loss.

This massive, four-year project, nicknamed “Project Grandiose,” involved close to 50 researchers in Canada, The Netherlands, South Korea and Australia. Its ramifications extend into many different areas of research, including stem cells, cancer, fetal development and regenerative medicine.

Mount Sinai
Team at Mount Sinai Hospital’s Lunenfeld-Tanenbaum Research Institute (left-to-right): Drs. Peter Tonge, Ian Rogers, Samer Hussein, Andras Nagy and Mira Puri. Photograph courtesy of Mount Sinai.

Even the way in which this news burst on to the global research scene was dramatic and unprecedented: No less than five articles were concurrently published on December 11, 2014 in the highly respected journals Nature and Nature Communications.

Dr Andras Nagy
Dr. Andras Nagy, Mount Sinai Hospital’s Lunenfeld-Tanenbaum Research Institute. Photograph courtesy of Mount Sinai.

“We are incredibly proud of Dr. Nagy’s leadership of this extraordinary team. [This work] reinforces how strategic investment in the best scientific research can truly transform our understanding and lead to new therapeutic possibilities,” says Dr. Jim Woodgett, Director, Lunenfeld-Tanenbaum Research Institute.

The Honourable Dr. Eric Hoskins, Ontario’s Minister of Health and Long-Term Care, present at the announcement on December 10, 2014, concurred: “This is a fine example of the groundbreaking research that takes place here in Ontario. Stem cell research was pioneered here in the province, and I am proud that we continue to make world-class breakthroughs in this life-saving area of research.”

The Honourable Reza Moridi, Ontario’s Minister of Research and Innovation, underscored the implications of this work: “This discovery has the potential to lead to improved disease treatments not only for millions of Ontarians, but people worldwide, and demonstrates Ontario’s ongoing leadership in the field of stem cell research.”

A Vast and Comprehensive Biological Map Now Possible

From the moment stems cells were discovered half a century ago, they have held great promise for possibly repairing damaged or diseased tissue, due to their ability to create all of the cells in our body. The best-known example of a stem cell-based therapy is bone marrow transplantation, which has been performed for more than 40 years.

In this context, Nagy’s team has taken a huge step forward in solving the mystery of how a cell, taken from a person’s skin, can be reprogrammed into an embryonic-like stem cell, from which any other cell type in the body can be generated.

The genesis of his study was global. Through provincial and federal funding, Nagy, whose career spans 25 years at Mount Sinai, was able to assemble a global team of experts that could go farther than his team alone.

“It was an enormously enlightening feeling that a single scientific question was able to transcend geographical distances, time zones, international borders and cultural differences,” he says. “Each of the scientists with unique expertise contributed to a unified product which none of us as individuals could even get close to,” he adds.

With this collective knowledge, Nagy’s team was able to completely explain the process involved in the formation of stem cells from specialized cells, such as skin cells: The team produced a detailed account of how 17,000 genes change during reprogramming to stem cells.

The end product of this research is the first dataset that functions as a vast and comprehensive biological map that catalogues the major biological stages of the reprogramming process.

New Class of Stem Cells Discovered

In this process, Nagy’s team discovered a new type of stem cell called ‘F-class,’ that have a fuzzy appearance compared to other embryonic-like stem cell types, and are also easier, less expensive and faster to grow. Because of these properties, the new F-class stem cells can be produced more economically in very large quantities, which will speed up drug screening efforts, disease modeling and eventually the development of treatments for different illnesses.

This discovery could lead to new avenues for generating useful “designer” cells, which might not necessarily exist in the body or during development, but could be safer and more efficient when used for therapy. Therefore this research is a critical step forward in the road to generating safe and highly efficient sources for therapeutic cell production.

The knowledge gained through this research will be used across the globe for countless generations of future researchers.

Nagy initiated the project in 2010 with a $4.5-million grant from the Ontario Ministry of Research and Innovation’s Global Leadership Round 2 in Genomics & Life Sciences Competition Program for enhancing genome and stem cell research in Ontario. Additional funding came from the Canadian Institutes for Health Research, the McEwen Centre postdoctoral fellowship, and Pfizer.

To read more about this research, go here: http://bit.ly/1GdGFQb. To read more about Dr. Nagy’s work, go here: http://bit.ly/1ChzUxm.