The Thrill of Discovery-The Satisfaction of Success

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Most days Michael Rudnicki, PhD can be found in the bowels of his laboratory at the Ottawa Health Research Institute (OHRI) (Ottawa, ON), guiding the research that has helped put Canada at the forefront of molecular medicine and on the verge of harnessing the capacity of stem cells for treating disease.

"What motivates me as a basic scientist is primarily the thrill of trying to figure out how things work," he says in a leisurely way that gives no inkling of the busy life he leads.

"But there are potential health benefits that also come from this work," he continues. "A lot of satisfaction comes from contributing to the larger good."
That contribution is likely to be enormous, given Rudnicki is at the helm of an impressive list of initiatives in stem cell research and collaboration.

But alongside a research career in full throttle is a small biotechnology company, StemPath Inc., which Rucknicki co-founded in 2002 as an OHRI spinoff with his research colleague Lynn Megeney, PhD to apply their bench research to a range of diseases and conditions.

In a nutshell, their research revolves around the molecular mechanism involved in regulating proliferation and differentiation of stem cells during embryonic development and tissue regeneration. "Adult" stem cells appear to be the basis of the regenerative capacity that exists within most tissues throughout the human body.

Rudnicki and his team hope they can exploit the body's ability to heal itself by developing drugs that can modulate these adult stem cells endogenously, rather than using the invasive exogenous stem cell approach championed by many groups. (www.stempath.com)

This ability to encourage the body's own regenerative capacity could revolutionize the treatment of a wide range of conditions such as muscle wasting in cancer patients, scarring and fibrosis of heart muscle cause by a heart attack, muscular dystrophy, complications associated with AIDS and cirrhosis of the liver.

Making the Transition
Trained at the University of Ottawa (Ottawa, ON) and the Massachusetts Institute of Technology (Cambridge, MA), Rudnicki began his career at McMaster University (Hamilton, ON). In 2000, he returned to head the molecular medicine program at the University of Ottawa and now holds the Canada Research Chair in Molecular Genetics.

"About four or five years ago, the research lab decided to take a more strategic approach and embark on a patenting strategy," he explains. "Rather than having 105 scientists looking at things one at a time, we decided to focus as a group on several areas — stem cells was one area, cancer was another . . .we built up a portfolio in the stem cell area of four patent families."

Rudnicki says that, fortunately for the StemPath team, some OHRI staff had the necessary business skills and patent experience to walk them through the conception stage. "We had regular staff meetings, had audits, did Gantt charts — all the business things we had to do." But at some point a company needs expertise and capital that can’t be found in a lab.

StemPath's research attracted the attention of Genesys Capital Partners Inc. (Toronto, ON), a major investor in biotechnology and health-care technology.
Genesys has been StemPath's sole partner in what Rudnicki calls "quite an exciting ride so far," and recently announced $1 million in funding for the next stage of the company’s development. StemPath expects to use these funds to bring forward its first drug candidates - aimed at treating acute myocardial infarction and skeletal muscle myopathies - into the preclinical testing phase which, according to Rudnicki, could begin next year.

"The heart (research) is quite well-advanced and just going to pigs now . . . this set of experiments should provide us with the data to move forward with Series A financing," he says with a sense of anticipation.

A Cultural Adjustment
Genesys has invested over $2 million in StemPath thus far. According to Rudnicki, good investor relationships and understanding the commercial viewpoint are keys to success.

For the post-doctoral fellows who joined the company this has meant undergoing "a cultural adjustment," Rudnicki says.
"It's not about understanding how something works, it's about understanding whether you can make it work . . . we need a 'go or no-go decision' on what direction to take . . . it doesn't matter what you learn, and how many science-based patents you have. You have to have something that you can put into the clinic that you can sell.

"It's a different way of approaching one's work," Rudnicki admits. "Rather than simply doing science and finding out how things work, one can actually make a difference . . . see your findings move from the bench to the bedside and actually be applied. That is satisfying and rewarding too."

What has he learned from the adventure? "Keep the investors happy," he quips without hesitation. "Communication is key and certainly the glass is always 'half full' in one's communications."

Though StemPath has had some delays and still operates "on a shoestring" compared to research in the U.S., things are moving "very quickly in spite of this," Rudnicki says. "What works well is having it all in one lab."

Rudnicki also thinks that having one primary investor relationship is the best strategy at the early stage. "(Genesys) has a certain degree of ownership (in the project), an investment in moving forward. If we had multiple investors at this stage I think managing those interactions would take up a lot of time. By keeping it simple at this early stage, it works well for investors and it works well for us.
"At the next stage of Series A financing we’ll be building, growing, moving out of the lab. There will be several investors involved. So the relationship for me will change from being more 'hands on' to more of a consultant. That's normal and healthy, just like a teenage heading off to school," he says. "You give birth to it, but it's important not to feel that this is your company and try to micromanage all aspects of it - you really have to take a team approach and the company will evolve. If it's successful, it will evolve up and out."

Juggling all these balls seems to come naturally to Rudnicki. He is looking forward to moving into OHRI's new Sprott Centre for Stem Cell Research (set to open in November), and directing the largest centre in Canada devoted specifically to stem cell research. He also continues as scientific director of the Stem Cell Network, one of Canada's Networks of Centres of Excellence.

Despite this busy workload, Rucknicki seems to keep the challenge of balancing it all in perspective. "There are lots of other people who are busy out there too," he says modestly. "I don't find it a challenge . . . it is a continuum of activities.

One piece of advice Rudnicki has for others stepping into a similar role: "One has to be patient," he says.
"Certainly there are a lot of stakeholders in the process. Understanding the investor's point of view, having patience with that is worth the trouble. It is, after all, their money - they have to have absolute confidence in what you're doing. Communication and building those personal relationship is an important aspect to building investor confidence . . . and keeping it."