A molecule that saves lives – Innovita Research

Cancer survivors Monique Russier and Tyler Rabey owe their lives to a molecule called UM-171.

In 2014, on a hiking trip in the mountains of Savoie, in France, Russier came down with a high fever and started coughing. A blood test and X-ray of her lungs later revealed that the 66-year-old had double leukamia, a cancer that is particularly difficult to treat.

“It was very brutal,” she recalled.

After receiving a transplant of umbilical-cord blood, Russier returned to Quebec and was followed by UdeM assistant medical professor Sandra Cohen, a clinical researcher in hematology and medical oncology at Maisonneuve-Rosemont Hospital.

A few months later, she relapsed. “It was really the sky falling on my head,” she said.

But then Dr. Cohen put Russier in touch with Dr. Guy Sauvageau, principal investigator in the Molecular Genetics of Stem Cells research unit at UdeM's Institute for Research in Immunology and Cancer (IRIC). Thanks to the UM-171 molecule developed in his laboratory, Sauvageau and his team have found a way to multiply the stem cells in umbilical cord blood and make transplants like Russier's more effective.

“There were side effects that could be fatal, but I thought if I had a chance to be saved, I would take it,” Russier recalled. “And if it didn't work for me, at least it would contribute to the advance of science.”

She got her UM-171 graft in September 2016. “I felt the energy coming back much faster than during my first transplant,” she said. After the operation, encouraged by her husband and two daughters, she tried very hard to get her strength back, and it worked. After four or five months, she had recovered, feeling as energetic as she did before her cancer diagnosis two years before.

“I am very honoured to have been able to take part in this research, conducted by an extraordinary team,” she now says. “I wouldn't have had a chance to live without it.”

Palliative care at 24

Rabey is another UM-171 survivor. After being diagnosed with lymphoblastic lymphoma, a rare and aggressive type of leukemia, the 24-year-old did not respond to initial treatment and was told to consider palliative care to keep him alive a few more months.

“I refused that,” he recalled. “But I agreed to one more chemotherapy session as a last resort. The chance that it would work was only 10 to 15 per cent, and if it did, they said I would then need a stem-cell transplant. In the end, I did respond to treatment. That's how I wound up participating in the UM-171 study.”

Nerve damage from his last chemotherapy put the young man in a wheelchair for four months and took more than a year to heal, but he recovered. “I couldn't have done it without the progress of medicine,” he said.

That progress is due in large part to the collaborative work between Sauvageau, his team and other collaborators. Their goal was to find a molecule that would multiply stem cells in cord blood, and to reach it Sauvageau sought the help of UdeM professor Anne Marinier, principal investigator and director of medicinal chemistry at IRIC.

“My team looked at our collection of molecules synthesized by UdeM researchers and we identified one that we optimized,” said Marinier, who worked in the pharmaceutical industry for 15 years before joining IRIC.

And so UM-171, named after UdeM, was born.

Exciting for researchers

The particularly promising results of the team's clinical studies so far are exciting for researchers.

“Of 22 patients treated, the mortality rate has been less than 5 per cent,” said Sauvageau. “Even though some patients were in palliative care at the start of their treatment, there was very little recurrence of the cancer afterwards.”

It's every doctor's ambition to put research to good use, and that's true for the team at IRIC.

“When you choose to go into drug research, you want to make a difference and have an impact on people – just thinking you can cure a patient is huge,” said Marinier. Added Sauvageau:  “Since I was very young, I had a dream of having an impact on cancer.”

The research could soon have other benefits, too.

“We know the UM-171 molecule can increase stem cells, but we also know that it does other things,” said Sauvageau. “And from there, we understand that it can help us to cure other kinds of diseases. What we just experienced is only the first chapter.”

Source: University of Montreal