Many ALS clinical trials, including those testing promising drugs, have fallen short of expectations—often because the extent of the disease can vary, and patients don’t respond the same way to medications.
But a new study led by scientists at Case Western Reserve University used stem cells created from ALS patients to target a specific gene as a kind of shut-off valve for what stresses nerve cells—and it worked.
Although the research involved a very rare type of ALS, the research team was optimistic the positive results could provide clues for potentially treating the devastating disorder more broadly.