Vantage Point

SOD1 mice. SOD1 rats. Anyone on top of ALS research has heard about these animal models. Both, of course, have rodent genes. But they also carry human genes that somehow trigger ALS because they’re flawed. The animals, then, are marked by the same gene errors as people with a rare, inherited form of ALS.

Yet it’s that very rareness that puzzles those who know how heavily research leans on these models. Why would you study a form of the disease that the vast majority of ALS sufferers (those with sporadic ALS) don’t have?

The answer is simple: You use what’s available as a starting place, as long as it makes sense. And as ALS Alert shows, what SOD1 animals are telling us does make sense for laying out the basics of ALS. More important, those insights push us closer to therapy.

Take the cover story, which tells how an agent called IGF-1 dramatically extends the life of motor neurons in model mice under attack. We believe IGF-1’s good effects come about because it blocks a toxic path activated in any motor neuron, whether it’s in an SOD1 mouse or someone’s mother with sporadic ALS.

Read about our mitochondria studies done largely with SOD1 mice. The way cells decline begins in animals may not be exactly the same in people with sporadic ALS, but in both cases, our Center scientists tell us, mitochondria are likely the first cell structure that’s injured. Since damaged mitochondria can switch on the process of cell death, studying them in SOD1 animals makes sense.

Sure, we use other models when they’re useful. Some of the potential drug therapies we’re starting to test in patients (see A Tale of Three Drugs) came from work with cell or organ cultures, as well as with SOD1 animals. But we know we’ve no time to waste. We use every tool we can—mice and screening cultures—to build an answer and a therapy.

Jeffrey D. Rothstein, M.D., Ph.D.
Director, The Robert Packard Center for ALS Research

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