Scientists Extend Lifespan in ALS Mice by 30%

Scientists at Northwestern Medicine just made a discovery that could change how we treat ALS and other devastating brain diseases.

The research team, led by Dr. Robert Kalb at the Les Turner ALS Center, found that a protein called RAD23A might be doing more harm than good in neurodegenerative diseases. Instead of helping cells clean up dangerous protein clumps, it was actually protecting them.

Every neurodegenerative disease shares one deadly feature: misfolded proteins that pile up in brain cells like toxic waste. These twisted proteins damage cells and eventually kill them, causing diseases like ALS, Parkinson's, Alzheimer's, and Huntington's.

Dr. Kalb's team started by studying tiny worms with ALS-like symptoms. They discovered that RAD23 proteins play a confusing double role: they help break down some proteins while keeping others stable. When the scientists removed RAD23 from cells, harmful proteins broke down faster.

The real breakthrough came when they tested this idea in mice with ALS. Using special gene-silencing tools called antisense oligonucleotides, or ASOs, they reduced levels of RAD23A in sick mice. The results were remarkable.

The treated mice lived significantly longer than expected. They kept their muscle strength while untreated mice grew weaker. Most importantly, the toxic protein clumps in their cells shrank dramatically.

"We found this really robust effect on survival, behavior and rescued cell death," Dr. Kalb explained. The therapy worked by removing a roadblock that was preventing cells from disposing of dangerous proteins properly.

Think of RAD23A as an overprotective security guard that won't let the cleanup crew do their job. By reducing this protein, cells could finally clear out the toxic buildup that was killing them.

The Bright Side

This discovery matters beyond ALS. The toxic protein TDP43, which RAD23A affects, shows up in almost every neurodegenerative disease. That means this approach could help millions of people facing different diagnoses.

The ASO technology used in this study already exists and is being refined for other conditions. This gives researchers a clear path from lab bench to patient bedside, unlike discoveries that require building entirely new treatment methods.

Dr. Kalb and his team aren't stopping here. They're continuing to study how protein clumps form and how cutting-edge tools can stop them. "We have to be willing to serve up a lot of ideas and investigate every option," he said.

The research, published in Nature Communications, represents years of work looking at brain disease from every possible angle. For families touched by ALS and similar conditions, it offers something precious: scientific hope backed by real results in living animals.