ALS is a rare, fatal neurodegenerative disease characterized by motor neuron loss in the brain and spinal cord. At present, about 10 percent of ALS cases are caused by a known genetic mutation that can be passed from one generation to another, while a staggering 90 percent have no known cause. Nevertheless, scientists believe it is likely that additional genetic mutations have yet to be identified – underscoring the importance of Biogen’s continued work in understanding ALS genetics.
For nearly a decade, Biogen has remained unwavering in its commitment to furthering ALS research in an effort to bring therapies to people living with this rapidly progressing neurological condition. Starting in 2012, the company began a discovery and development program, carrying out a Phase 3 clinical trial called EMPOWER for broad ALS. Unfortunately, the trial failed to meet its primary endpoint and no efficacy was seen in individual components of function or survival – though much valuable information on the disease course in individual patients was collected.
It was this data, and the insights they provided on the individual level, that shifted Biogen’s focus to genetic ALS, with the goal of bringing a potential therapy to patients in need.
“Our understanding of ALS has really exploded over the last 10 years,” explains Chris Henderson, senior vice president and head of research at Biogen. “We now understand the genetics of many human cases, which has provided us with the ability to directly target the causal genes in those patients. These genetics have allowed scientists to create animal models in the laboratory that quite faithfully represent several of the degenerative processes going on in ALS patients. By studying these models, as well as human tissue samples, researchers around the world have brought to light new biologies that we now believe to be involved in all forms of the disease. And each new biology is a potential target for a therapeutic, even in patients that have ALS with no known genetic cause.”
Watch Jeremy’s story here.