Game-Changing Discovery will “Rapidly Accelerate” the Development of Cell-Based Therapies

November 14th, 2018 by FFB Canada

Today, we are excited to announce a truly transformative discovery that will “rapidly accelerate” the development of cell-based therapies—not just for blinding eye diseases, but for all kinds of regenerative medicine!

One of the main hurdles to developing stem cell therapies is ensuring that they are safe. Stem cells hold incredible therapeutic potential because they can divide and create additional stem cells, and they can form all the different types of cells that are present in the human body.

Unfortunately, the characteristics that impart stem cells’ incredible therapeutic potential are also shared by a dangerous cell type: cancer cells. Like stem cells, cancer cells can divide (self-replicate) and create different cell types. These shared characteristics have caused many experts in the stem cell field to proceed with caution toward the clinic, where experimental treatments are tested on people.

“The first principle in medicine is to not cause harm to the patient,” says FFB-funded researcher, Dr. Andras Nagy, who is a Senior Scientist at Sinai Health System’s Lunenfeld-Tanenbaum Research Institute. Dr. Nagy has dedicated his life’s work to creating useful cells for therapy and holds a Canada Research Chair in Stem Cells and Regeneration. “As cell therapy moves into clinical practice, we must avoid causing harm by this new medicine.”

This mantra has motivated Dr. Nagy to develop a research agenda that looks for discoveries that can truly accelerate the development of cell-based therapies into the clinic.

At the FFB, we are proud to be funding Dr. Nagy’s research that aims to develop a combined stem cell and gene therapy to treat age-related macular degeneration, the leading cause of blindness in people over the age of 55. Indeed, we recently announced Dr. Nagy as the inaugural recipient of the Cedric Ritchie Fund to Cure Blindness—Dr. Nagy is a worthy recipient because of his clear commitment to translate his discoveries from the laboratory into the clinic where they can impact patients.

Dr. Nagy’s recent discovery, published today in the prestigious scientific journal, Nature, describes the new “Safe-Cell System” that was developed in his laboratory. The Safe-Cell System genetically engineers stem cells to have a remotely controlled killer switch, which enables dangerous cells, such as cancer cells, to be eliminated. Moreover, his team developed a unique approach to accurately assess when things might go wrong with a new cell therapy. Quantifying risk in this tangible way will accelerate the development of new treatments that depend on a clear understanding of potential hazards.

We are thrilled that this high-impact paper featured very compelling data from experiments that were done in the eye that demonstrate the effectiveness of the Safe-Cell System.

Fueling the development of safe and effective stem-cell therapies to restore sight is a top priority at the FFB and we are so thankful that Dr. Nagy shares this goal!

Read More:

Liang et al. 2018. Linking a cell division and a suicide gene to define and improve cell therapy safety. Nature.

New Discovery: A built-in killer switch eliminates dividing cells from lab-grown transplant tissue to improve patient safety