ALS Alert - Robert Packard Center for ALS Research

Vantage Point

Jeffrey D. Rothstein, M.D., Ph.D.

One of the most satisfying things about directing the Center has been watching the shift in our thinking about the biology of ALS. The change has been gradual but sure—with much of it coming from work from the Center’s member laboratories worldwide. Because the shift opens new targets for therapy, that’s all the more exciting. We describe several of the studies in this first edition of ALS Alert.

Traditional ideas on ALS suggest problems lie exclusively in motor neurons.

But a couple of things have made us waver, things that called our attention to the cells— astrocytes—that surround motor neurons. Several years ago, for example, our Hopkins labs showed that ALS patients’ astrocytes don’t clear the neurotransmitter glutamate efficiently from nerve tissues.

Now we’ve found that astrocytes apparently become abnormal before motor neurons do. The clincher is Margaret Sutherland’s work that shows “fixing” flaws in astrocytes brings a huge increase in mouse survival.

Do we know what causes ALS? No. But we now realize that mere “bystander” cells are important. Astrocytes aren’t as downstream as we thought. So we’re shifting our therapeutic approach to include tactics that boost astrocyte function, including drugs like Celebrex and stem cells.

Over the next few months, I will tell you about projects under way at the Center, including new stem cell work, studies on novel proteins that might be responsible for motor neuron death, and new ideas about how a mutant version of the enzyme SOD may slow the disease.

All of this edges toward our goal—finding new therapies to stop or reverse ALS.

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

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