Drug Development

Drug discoveries start with fundamental (basic) research in the lab, studying animal and cellular models. Fundamental research aims to better understand the biology of ALS and discover new ways to treat it. ALS researchers will study cells from people living with ALS and replicate the disease in animal models, to better understand the biology of ALS in a living system, with the hope that it will represent (model) what happens in people.

Promising therapeutic compounds identified in these models need to then be tested in humans, as the presentation of ALS in people is significantly more complex than in animals and cells in the lab.

New potential compounds will undergo rigorous testing with multiple clinical trial phases that are necessary to assess if the drug is safe, tolerable, and effective in humans.

ALS drug development has come a long way in the past few decades, with a great number of clinical trials globally currently testing potential therapies for the treatment and management of ALS. Despite these numbers, many therapies that show promise in animal models often fail to replicate those results in clinical trials, which is a major challenge in developing treatments for ALS. However, even unsuccessful clinical trials provide key insights into pathways and targets for new drugs, and the ongoing research around the world offers hope for advancements in ALS treatment and management.

As the only national dedicated source of funding for ALS research across Canada, the ALS Canada Research Program aims to accelerate drug development by providing funding for the most promising ALS projects focused on translating scientific discoveries into treatments for ALS.