NeuroDevNet investigators' review article addresses knowledge gap surrounding CP & stem cells

May 27, 2013

Lack of knowledge and misconception about stem cells and the state of the science among both clinicians and families of people affected by neurological injury has prompted NeuroDevNet investigators to publish a comprehensive review article on the subject.

An early view version of "The potential for stem cell therapies to have an impact on cerebral palsy: opportunities and limitations" appeared on-line May 16, 2013 in Developmental Medicine and Child Neurology, co-authored by NeuroDevNet post-doctoral fellows Dr. Crystal Ruff and Dr. Stuart Faulkner, along with Dr. Michael Fehlings of our CP research group.

'We undertook this review because of the urgency behind the quest for meaningful treatment," says Dr. Ruff.  "Neurological injury is often so personally and financially devastating, and at the same time, cells have been unscrupulously touted as a 'cure all' that in turn has driven a significant stakeholder push for 'stem cell therapies.' "

"This review provides a crucial bridge which describes clearly for everyone what stem cells are, what types exist, what they can and cannot do, what trials are currently available, and where the state of the science is heading," adds Dr. Fehlings. "It is important to emphasize that stem cells and in particular neural stem cells hold significant promise as a therapeutically relevant treatment strategy for cerebral palsy, and that this work could be translated in the next 5-10 years."

Early phase safety trials with neural stem cells have been launched in a number of neurodevelopmental disorders, including Batten's disease and Pelizaeus Merzbacher disease (PMD). "It is important to drive forward clinical trials," continues Dr. Fehlings, "while at the same time having a robust pre-clinical research strategy which is examining ways to perfect the neural stem cell technology and to optimize the application in disease-relevant animal models."

"On the one hand," says Dr. Falkner, "there is the necessity to understand scientifically what the stem cells are, how they work and what they are capable of. This way, we will be able to optimize treatment in the future and combine treatment strategies to obtain the bests outcomes." Scientists and physicians have to be certain that the therapies will be effective - or at least, not cause damage each time, Faulkner stresses, "because once stem cells are put in, they cannot be removed.

Given the tremendous challenges of daily living with neurological injury, the trio feel a moral and ethical obligation to translate any therapy that is effective and safe as soon as possible.

"That said, there is no stem cell therapy that has always and consistently worked in preclinical models," says Dr. Ruff. MSC therapies, although safest in humans, are inconsistent in terms of efficacy, and we are working on determining what properties of these cells are different between the cells in studies that improve function, and the cells in those that do not.

"NPCs are perhaps the most promising cell type for use in neurological injury, and over the years," Dr. Ruff continues. "Scientists have been able to purify and refine these cells to the point where they are being used in the first human populations. However, we do not know yet if these cells are safe."

The most frequent questions regenerative neuroscientists hear are, "how close are we," or "when?" 

"Unfortunately, says Dr. Fehlings, "nobody can predict this. A successful stem cell treatment strategy is not likely to arrive overnight - it will be the result of incremental research. As a cooperative group, we are progressing, step by step, toward our goals, and Canada is establishing itself as one of the world's leaders in stem cell science.

"The formation of large collaborative networks like NeuroDevNet has increased the pace of our discovery," adds Dr. Fehlings, "by linking our nation's leaders in regenerative neuroscience. Through this linkage, we have multiplied our individual productivities exponentially in efforts to distribute the most effective stem cell replacement strategies to those who need them."

Click here to read an abstract of Ruff, C. A., Faulkner, S. D. and Fehlings, M. G. (2013), The potential for stem cell therapies to have an impact on cerebral palsy: opportunities and limitations. Developmental Medicine & Child Neurology. doi: 10.1111/dmcn.12166