Exploring stem cells and gene therapy

In 1999, Science magazine declared the newly emerging therapeutic promise of embryonic stem cells - the cells that give rise to all the organs in the developing fetus - to be its breakthrough of the year.

And, in so doing, quite unintentionally unleashed a research tidal wave in Ottawa.

That is because Ron Worton, then recently appointed head of research at what is now the Ottawa Health Research Institute, had been considering how to reconfigure his institution's efforts. What he was looking for was a major theme to focus on, something that would galvanize interest on the inside and draw attention and funding from the outside.

He had been considering stem cells, which he had studied in the 1960s in the lab of their Toronto-based discoverers, Ernest McCulloch and James Till.

But even though it was known for decades that stem cells act as a repair system for the mature body, the persistent difficulties in obtaining viable cells led to a basic problem. Stem cells had slid to the bottom of almost everyone's research agenda.

But when Science magazine anointed the cells' promise, Worton had what he calls his "bingo moment." "I said, 'Okay, that sort of confirms all of the things that I've been thinking about.' I walked over to my colleagues on the floor and I said, 'Okay, that should be our next big wave of activity. We can build something around this,'" he recalls.

What he wanted was not just research, but therapeutic applications. And what he got? Consider the following.

On the research front, Worton was the initial scientific director of the Stem Cell Network - a network that linked 80 researchers across the country. They had been looking at potential therapies for the gamut of diseases where cells fail or malfunction; for example, cancer, type 1 diabetes, heart and lung disease, stroke, spinal cord injury and more.

In 2005, this mantle was passed on to Michael Rudnicki, who had been recruited from McMaster University to head up stem cell research at the hospital. On Worton's behalf, Rudnicki and his colleagues began to take a long, hard look at the fundamental mechanisms that trigger stem cells and allow them to complete their repair efforts. One quite promising result has been the research that has grown out of a transgenic mouse that was engineered to have an exceptionally thin-walled and poorly functioning heart.

Reasoning that this is exactly the kind of defect that stem cells were designed to correct, Rudnicki's laboratory moved on to discover stem cells in adult muscle and were able to define the genes that are important for muscle regeneration.

However, by far the most spectacular results made have been the work of Harry Atkins with multiple sclerosis, a disease in which the body's immune system attacks and destroys the brain and spinal cord.

What Atkins did was purify and freeze patients' bone marrow stem cells and then destroy their old immune system with chemotherapy. The stem cells were then re-implanted with the hope they would eventually form a new immune system.

In almost all cases, stem cells have slowed or halted the progression of the disease, and in one woman apparently reversed it entirely.

And there also has been a very different kind of success. The almost daily discussions in the media of the possibilities of stem cell therapy combined with what is the Ottawa Health Research Institute's skill in expanding these possibilities have inspired a flood of private donations from people wanting to associate themselves with stem cell therapies.

The most notable is a $7 million donation to support operating costs from Eric and Vizma Sprott, after whom the OHRI stem cell research centre is now named.

But they are not the only ones lending support.

"People understand that the kind of research going on here is going to be part of their future," says Duncan Stewart, who in 2007 took over from Worton.

"And if they have the means, they want to make that future happen faster."

Worton nods and adds a mantra that was true when he moved his institution into stem cell research and is even truer today: "Success creates excitement."