Dr. Victor Rafuse
Practical approaches to ALS:
Dr. Victor Rafuse is exploring innovative approaches to diagnosing and treating ALS (amyotrophic lateral sclerosis), a neurodegenerative disease that tends to progress rapidly, paralyzing muscles throughout the body and often ending in respiratory failure.
“We’re learning what happens in ALS, as motor neurons and the synapses that connect them to the muscles die,” says Dr. Rafuse, a Professor in Dalhousie’s Department of Medical Neuroscience and Director of the Brain Repair Centre. “We must understand the disease to fight it.”
Dr. Rafuse and his team turn stem cells from people’s skin into working motor neurons that make appropriate synaptic connections to muscles - creating a “test tube” model of ALS that allows them to study the disease in a Petri dish. He and his colleague, Dr Ying Zhang, have translated this into a technology for rapidly screening compounds that could save both synapses and motor neurons to slow the progress of ALS. They’re also working with a Canadian-led team that’s using this new technology to test potential treatments.
At the same time, Dr. Rafuse and his team are working towards a simple test for diagnosing ALS in its early stages, when treatments have the best chance of altering the course of the disease. “ALS is difficult to diagnose,” he notes. “It can take a year or more to get a definitive diagnosis, and by this time many motor neurons and their synapses have died. Early diagnosis is essential to stopping this disease.”
Dr. Rafuse is also working on a gene therapy to help people with ALS breathe more easily, and launching new projects to explore the relationship between metabolism and ALS. “It’s an exciting time for ALS research Dalhousie and the Brain Repair Centre,” says Dr. Rafuse. “New researchers are coming to Halifax to join our growing ALS research group, so we’re able to study the disease from many new angles.”
The planned expansion of the Maritime Brain Tissue Bank - to be funded through the proceeds of the 2014 Molly Appeal - presents another opportunity. “Greater access to human tissues opens a new window into the mechanisms of disease that will help us in our search for diagnostics and cures,” Dr. Rafuse says. An expanded brain bank will be a great asset to our neuroscience community as a whole.”