Of the $3.6 million in research funding awarded in May 2017 by the Ontario Institute of Regenerative Medicine (OIRM) and supported through funding from the Ontario Ministry of Research, Innovation, and Science, $3 million went to five large OIRM Disease Team grants. These projects focus on delivering new curative therapies for conditions that drive large costs to the healthcare system, as well as forging global opportunities for economic development based on stem cell based therapies.
“Currently in Ontario, there are at least 13 clinical trials underway or about to begin that are assessing stem-cell based treatments for debilitating illnesses and injuries such as heart disease, septic shock, knee osteoarthritis, malignant brain tumours and more,” says Dr. Duncan Stewart, OIRM president, and scientific director. “Twelve of these trials are led by OIRM member researchers, which is a testament to the depth and breadth of expertise we have in this province, and the level of support that exists for regenerative medicine research here.”
Three years ago, there were only five trials in the province – an increase of more than 150 per cent.
Launched in 2014, OIRM is dedicated to the translation of stem cell research into curative therapies for major degenerative diseases. More than 210 research programs at universities and institutions across the province are involved with OIRM, with additional contributions from key clinical and health charity partners and from OIRM’s commercialization partner, CCRM.
“The potential to address some of these very complex diseases, some of which are common and some rare, is enormous. However, we also make these treatments as cost-effective as possible, and therefore widely accessible through Ontario’s health system,” Dr. Stewart explains. “Therefore, we need to emphasize high-quality translational research so we can identify which therapies work in which patients, and how the healthcare system can fully adopt their use.”
At The Ottawa Hospital, a research team led by neonatologist Dr. Bernard Thébaud is working on a stem cell therapy that may mitigate the damage caused by bronchopulmonary dysplasia (BPD), a chronic lung disease that often develops following ventilator and oxygen treatment in extremely premature babies. Dr. Thébaud was awarded an OIRM Disease Team grant in May to conduct preclinical work, that, if successful, will lead to a human clinical trial as soon as 2018.
The appropriately named “INCuBATOR” project uses mesenchymal stem cells (MSCs) from discarded umbilical cord tissue – a relatively untapped source of stem cells – to stimulate growth and repair in the lungs. Dr. Thébaud’s team has worked extensively with these cells, having both proven their effectiveness in rats while developing a system to produce large quantities of the cells that would meet the stringent requirements of human treatment.
Before a single stem cell is given as a therapy, Dr. Thébaud says it is essential to answer as many questions as possible. Poor design or a lack of comprehensive answers is a common problem that can halt trials in their tracks. But for developing infants in the first months of life, whose vision, cognitive and motor development, and digestive systems are all dependent on the ability to get enough oxygen, time is of the essence.
“The idea is to accelerate the translation from the bench to the bedside, in an evidence-based fashion. We want to show proof-of-concept that a cell product may work for babies with BPD, then streamline everything so that we can move quickly from the bench into patients.”
Fragile lives require a good deal of care, which is why the project includes both the necessary pre-clinical work (in an animal model that very closely mimics the human condition of BPD) as well as a survey of the infants’ parents and health care professionals to assess the level of risk they are willing to accept.
“The goal is to find obstacles or facilitators for clinical trials. In this way, we’ll be able to design a trial that makes sense, but that will also have buy-in from the parents and clinicians,” Dr. Thébaud explains.
For Dr. Stewart, this work is representative of the burgeoning stem cell research and commercialization community in Ontario, led by OIRM and its partners.
“Ontario has a deep history of excellence in regenerative medicine – stem cells were first identified at Princess Margaret Hospital in Toronto, and today we are ranked third in the world in terms of the impact factor of discoveries made here,” he says.
“I think we are building toward a critical mass, where we’ll see frequent, significant outcomes that improve human health. But, to get there we need to ensure we have the resources to conduct trials in a meaningful way.”