Dr. Vic Rafuse pursues stem cell treatments for Lou Gehrig’s Disease (ALS)
Dr. Vic Rafuse and his team are on the trail of a promising way to restore motor neurons destroyed by Lou Gehrig’s disease (ALS). The cell bodies of these specialized neurons reside in the spinal cord and send long tendril-like axons to connect to every muscle in the body. In this way, motor neurons connect our brains to our bodies and enable us to move and breathe.
“In ALS, the motor neurons start dying in one part of the spinal cord, but the cell death spreads from there,” Dr. Rafuse says. “Eventually, motor neurons in the brain start to die, which affects lung function. Many people die within a few years of diagnosis, most often from respiratory failure.”
Dr. Rafuse has already proven that embryonic stem cells can be engineered into functional motor neurons. He and his team have also found a way to make these engineered motor neurons send their axons to the appropriate muscle fibres, taking them ever closer to a potential cell transplant therapy for ALS, as well as for spinal cord and peripheral nerve injuries.
Now, Dr. Rafuse and his team have shown that a new source of stem cells—called iPS cells, or ‘induced pluripotent stem cells’—can also be turned into motor neurons. Motor neurons derived from iPS cells offer several advantages.
“We can harvest iPS cells from any part of a person’s body,” Dr. Rafuse explains. “We’re making our motor neurons from iPS cells taken from the skin. This provides us with a non-invasive source of cells that will not provoke an immune rejection response, because they come from the patient’s own body. This approach also solves ethical problems with using embryonic stem cells.”
Dr. Rafuse and Dr. Rob Brownstone have initiated a project aimed to restore hand movement to people with peripheral nerve injuries. Their ultimate goal is to transplant engineered motor neurons into the damaged area and then implant an electrical stimulator that the person can activate by clicking their teeth to open and close their hand.