A unique pilot study has established a safe pathway for using bone-marrow stem cells to slow down and potentially treat Amyotrophic Lateral Sclerosis (ALS), a fatal neurodegenerative disease without cure.
The study, published in the journal, Muscle & Nerve and led by Dr. Neil Cashman, professor of neurology at The University of British Columbia and director of the ALS program at Vancouver Coastal Health and VCH Research Institute, tested the use of a growth factor stimulant in ALS patients and found that bone-marrow stem cells became activated with no adverse effects to patients.
“Our idea was to use a growth factor stimulant to increase the number of circulating stem cells from within the body’s bone marrow where they would have the potential to travel to the site of injury and begin repair, slowing down the progression of ALS,” says Cashman, who also holds the Canada Research Chair in Neurodegeneration and Protein Misfolding Diseases at UBC and is a member of the Brain Research Centre at UBC Hospital.
“This pathway, if one day successful, may provide a new therapy that will avoid the ethical debate surrounding embryonic stem cells,” says Cashman.
Growth factors are proteins that can stimulate cell division. They occur naturally in the human body and can also be developed in a laboratory. Stem cells serve as a “repair system” in the human body and have the potential to develop and divide into many different cell types.
“The project was complex because growth factors have the potential to activate the wrong cells in the brain and spinal cord, which could be harmful to ALS patients” says Cashman.
The researchers identified Granulocyte Colony Stimulating Factor (G-CSF) as the safest possible growth factor to use. They then conducted the pilot trial to establish safety and measure stem cell mobilization.
“We were able to measure a prominent effect on stem cell mobilization and found no adverse effects in the patients,” said Cashman. “There have been many misgivings in using stem cell stimulators in ALS patients but now we know we can safely do this. This is an important first step in providing a new treatment for ALS.”
The research team is now developing a larger scale multicentre trial to look at therapeutic effect. This trial is at least one year away from beginning.
ALS is a progressive and ultimately fatal neurodegenerative disease that produces weakness, atrophy – partial or complete wasting away of a part of the body, and spasticity – continuous contracting of certain muscles. It results from progressive degeneration of motor neurons in the brain, brainstem, and spinal cord. There is no cure for ALS and to date the only registered pharmacological treatment is riluzole, which slows the progression of the disease on average by 10-15 per cent. New effective therapies are greatly needed to slow or halt this disease.
The Webster Foundation in Montreal through the VGH & UBC Hospital Foundation in Vancouver, as well as the Temerty Family Foundation in Toronto provided funding for this study. The co-authors include Dr. Andy Eisen (senior author), professor Emeritus, Neurology, University of British Columbia and former director Vancouver Coastal Health ALS program; and Dr. Charles Krieger, associate professor of kinesiology, Simon Fraser University, professor, neurology, clinical associate professor, Neurology, University of British Columbia, and clinician researcher VCH ALS program.
VCHRI is the research body of Vancouver Coastal Health Authority. In academic partnership with UBC, the institute advances health research and innovation across B.C., Canada, and beyond. www.vchri.ca
The Faculty of Medicine at UBC provides innovative programs in the health and life sciences, teaching students at the undergraduate, graduate and postgraduate levels, and generates more than $200 million in research funding each year.
The Brain Research Centre at UBC Hospital is a multidisciplinary centre dedicated to improving understanding and finding new treatments for brain diseases. The centre is a partnership of the University of British Columbia and Vancouver Coastal Health Research Institute.
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