New stem cell technology gives new hope for high-risk leukemia patients

Few people get a second chance at life. And fewer still are forced to take it before their 25th birthday. Tyler Rabey is one of those people. He was diagnosed with an aggressive form of leukemia at the prime of his life; a cancer that defied all standard treatments. Forced to put his plans for a Master’s education on hold, Rabey slipped into cancer’s spiral. Within a year, this outdoor enthusiast was confined to a hospital bed, facing a terminal prognosis.

But Rabey was fortunate to be in Montreal, and to have a doctor on his medical team that wouldn’t give up on him. This doctor found a drug that paused the cancer’s rapid advance long enough for Rabey to qualify for an innovative stem cell clinical trial.

The results of this clinical trial, supported by the Stem Cell Network, are now published in the medical journal, The Lancet Hematology. A total of 27 patients were recruited to the trial and were treated under the supervision of the paper’s lead author, Dr. Sandra Cohen, a hematologist at the Maisonneuve-Rosemont Hospital/University of Montreal. Patients received a transplant using stem cells that had been expanded from umbilical cord blood using a new therapeutic technology, UM171. The findings from this early phase clinical trial demonstrated that this technology is feasible and appears to decrease the incidence of severe post-transplant complications, thus contributing to better outcomes for the patients after an average of two years of follow-up.

“Under current standard of care, especially in the context of high risk blood cancers, about half of patients who receive a blood stem cell transplant either suffer a relapse or succumb to life-threatening post-transplant complications,” said Dr. Cohen. “Remarkably, amongst the 22 patients who received a single UM171-modified cord blood transplant, the rates of graft rejection (0%), severe acute graft versus host disease (9%), severe chronic graft versus host disease (0%) and mortality linked to transplant complications (5%) were lower than expected. However, these promising results remain preliminary and need to be confirmed in additional studies with more patients and longer follow-up.”

Since 2003, the Stem Cell Network has provided over $9 million for research related to the discovery and development of a blood stem cell expansion program, which has principally been led in more recent years by Dr. Guy Sauvageau at the University of Montreal. This support includes $1.5 million for two clinical trials.

We are pleased to be supporting clinical trials like this one, which is making tangible improvements in the lives of people like Tyler, and we congratulate the research team for this landmark achievement.

Dr. Michael Rudnicki, CEO and Scientific Director of the Stem Cell Network

“SCN’s investments in programs such as this will make a difference in how disease and illness are treated in this country,” Rudnicki continued. “Dr. Cohen’s work typifies Canadian research excellence and demonstrates the potential of what stem cell research has to offer. We can look forward to more advances like this across different disease areas in the years ahead.”

The technology used in this clinical trial is being advanced by Montreal-based biotechnology company ExCellThera Inc, using the small molecule, UM171, discovered at the University of Montreal. The technology is capable of expanding stem and immune cells in cord blood exponentially, making this life-saving therapy available to a greater number of patients. It has the potential to improve how health care is delivered to those fighting blood diseases such as leukemias and lymphomas, and other severe disorders.

For Rabey, who is now able to walk in the outdoors again and has returned to school after a year-long recovery, the opportunity to be one of the patients receiving the enhanced transplant was nothing short of life-altering.

“I’m certain this technology and the opportunity I had to be part of a clinical trial saved my life. I will forever be grateful to all those who made it possible,” said Rabey. “I hope that someday soon this treatment will be made available to anybody who could benefit from it.”

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