There is no cure for amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease, but there is some good news: in a few years’ time, it may be possible to stop its progression. The team led by Jean-Pierre Julien, researcher in the Faculty of Medicine at Université Laval and CERVO Brain Research Centre, has found a way to improve the cognitive and motor function of mice with the neuromuscular disease, which leads to paralysis and death less than five years after diagnosis.
Jean-Pierre Julien hopes to stop the progress of the disease when a patient is diagnosed.
It was found that 98% of ALS patients have aggregated TDP-43 protein in their cytoplasm—the substance that surrounds the nuclei of their nerve cells. These aggregates are responsible for the sufferer’s loss of function over time. The researchers relied on a virus to carry tiny synthesized antibodies into the cells to erode the protein. As a result, the cognitive and motor function of the mice improved.
This innovative approach opens the door to the development of a therapy without adverse effects. However, the challenge is to adapt it to the human body. Reluctant to use a virus to transport the synthesized antibodies to a human nervous system, the experts tested a miniature pump and catheter system instead. Their experiments in mice proved conclusive. The next step is now to humanize the antibodies to correspond to human genetic sequences to avoid rejection.
In two to three years, Jean-Pierre Julien believes he and his colleagues will be able to begin clinical trials in patients. At best, he hopes to stop the progress of the disease when a patient is diagnosed. The treatment could also be used to slow certain types of dementia that involve the TDP-43 protein in seniors.