Vantage Point

For two perfect spring days last month, Center scientists were inside a windowless conference room and, from what I could tell, they didn’t mind. Year-round, we report our research in bits and pieces. But there’s nothing for perspective like hearing all the results at once. That’s why our annual symposium works so well. Plus, it’s a check on our progress which, this year, has been considerable.

On the therapy front, we’re close to human trials with several drugs from last year’s large-scale screening (see Report Cards that Shine). The drugs appear to compensate for certain ALS cell errors we identified a decade ago. And minocycline, an anti-inflammatory drug, is being shepherded by Center scientist Jean-Pierre Julien, who’s avidly exploring the role inflammation plays in ALS (Report Cards). Then, late this summer, we’ll learn how well Celebrex slows ALS. Earlier work Dan Drachman and I did laid the groundwork for the two-and-a-half-year nationwide trial.

But more than this, we’ve strengthened our understanding of neuromuscular disease. Work from Jon Glass’s lab, for example, suggests that death of motor neurons begins in axons, rather than in the cell bodies. He has two mouse studies to show that blocking axon death can keep cell bodies alive. And Kurt Fischbeck—one of our scientific advisors—has found that warping certain proteins key to a neuron’s internal transport system brings motor neuron death.

We’ve gained insight on stem cells and on how axons grow—studies we’ll feature in coming issues. And finally, we’re exploring new mouse models of ALS, based on newly found genes for inherited disease. It’s all part of our looking for what’s common to motor neuron disease. We pounce on these new genes. They hold the fine print that explains what’s wrong. This year, our vision for it has gotten sharper.

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

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