5th Symposium of the Fondation André-Delambre on Amyotrophic Lateral Sclerosis

Photo: Pascal Huot

Left to right:
Dr. Guy A.Rouleau, MD, PhD, FRCP(C), OQ
Director of the Molecular Neurogenetics Laboratory at McGill University and professor in the Department of Medicine at Université de Montréal. He is also Director of the CHU Sainte-Justine Research Centre and the Centre for Excellence in Neuromics at Université de Montréal
Dr. Jean-Pierre Julien, PhD
Professor in the Department of Anatomy and Physiology in the Faculty of Medicine at Université Laval and researcher in the CHU universitaire de Québec (CRCHUQ) Research Centre.
Dr. Angela Genge, MD, FRCP(C)
Director of the ALS Clinic at the Montreal Neurological Institute (MNI)
Dr. Nicolas Dupré, MD, neurologist at Hôpital de l’Enfant-Jésus in Québec City and professor at Université Laval
Researchers looking for a cure for amyotrophic lateral sclerosis (ALS) are faced with an even greater challenge: finding the causes of this disease that attacks the cells of the nervous system and paralyzes the most vital muscles of the body one by one. This year the 5th Symposium of the Fondation André-Delambre for ALS brought together the top names in ALS research and students to report on scientific and therapeutic developments.

5th Symposium of the Fondation André-Delambre on Amyotrophic Lateral Sclerosis

Research outlook

By Monica Lalancette

“Researchers need to meet each year to advance the cause of research into ALS.” This was the message of Monica Lalancette, neurologist and researcher at CHU Université Laval, and of Jean-Pierre Julien, a professor at Université Laval. The gathering of experts from around the world became a reality at the 5th Symposium on Amyotrophic Lateral Sclerosis. Some 100 invited guests attended a series of talks on the clinical promise of stem cells, organogenesis and genetics. “The symposium also included students,” Nicolas Dupré, neurologist at Hôpital de l’Enfant-Jésus and associate professor at Université Laval, points out. “It’s a way of encouraging them to consider a career in ALS research, rather than in another field.”

Amyotrophic lateral sclerosis can be explained by defining the three words. “Sclerosis” refers to the hardening of the regions affected by the disease, in this case the muscles. “Lateral” refers to the affected region, that is, the spinal cord that carries the nerve impulses. Finally, “amyotrophic” means “starved muscle,” which refers to the atrophy of the muscles due to degeneration of the motor neurons responsible for the nerve impulses. “Amyotrophic lateral sclerosis shocks the imagination of people who are closely affected by the disease, especially when someone they know with ALS dies in just a few years,” says Dupré. “The disease spread quickly by attacking the most vital muscles, such as the lungs and heart, and leads to death after making patients prisoners of their own body.” François Berthod, an expert in organogenesis at Université Laval, adds: “In most cases, patients are lucid from the beginning to the end and witness the progressive deterioration of their body.”
Little is known today about this disease, but two factors have become evident to researchers: the disease rarely appears before the age of 35 and it requires other cells, in addition to motor neurons, to develop. “Transgenic mouse models whose motor neurons are affected by amyotrophic lateral sclerosis do not develop the disease,” Julien says. “The entire nervous system, therefore, needs to be toxic.” Research is currently moving toward the artificial production of ALS using stem cells. The hope is that this technique will enable researchers to study the process of degeneration that the cells undergo.

Research outlook

Organogenesis and the manipulation of stem cells and induced cells were on the list of topics discussed at the symposium. A promising development for research is organogenesis that, among other things, can transform skin cells into stem cells to eventually recreate the environment of the disease or reproduce new motor neurons. At the clinical level, researchers are developing strategies to slow the progression of the disease and to uncover all the sporadic causes possible. “The eventual goal will be to compare healthy cells with degenerated cells after transformation,” Berthod explains. At the symposium, Julien announced that an organogenesis centre will soon be established in Quebec.
While it is possible to imagine the grafting of neurons one day, Dupré believes that it is less risky to first attempt to restore the environment of the nervous system. Julien asks, “If the whole environment of the nervous system is toxic, as we now believe it is, then what good will it be to graft neurons that will only end up becoming diseased?” Another avenue of research is the manipulation of infused stem cells, harvested from any cell body. By adding a specific gene to them, researchers can transform them into embryonic cells. “We’re making the cell do a virtual U-turn from the adult stage to the embryonic stage,” Berthod explains. The success of this method will apparently improve research into the causes of ALS because all the cells will have the potential to become stem cells, making them easier to access. “There’s still a lot we don’t know, and nothing is being done in this area in Quebec yet,” the neurologist adds.

For the moment, research is also literally at the embryonic stage. “Amyotrophic lateral sclerosis is caused by a multitude of small effects that we still need to uncover,” Dupré acknowledges. For the neurologist, the guiding principle over the next few years will be to put “order” into basic research and, above all, “to concentrate on uncovering the causes of the disease through the use of stem cells.”