Vantage Point

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

With approaches to ALS therapy, progress seems to move one step forward and a half step back. That’s sure to be the case with the encouraging results of a three-drug trial in ALS mice by Center researcher Jean-Pierre Julien. And it’s true for the 12 potential drugs we’re waiting to test—products of a huge screening the FDA sponsored last year.

These results are great. But there’s a quiet dilemma: The more drugs that reputable science offers us to try, the more we’re aware that available ways to test them early on aren’t as conclusive as we’d like.

Take creatine, for example. A few years back, when the over-the-counter supplement was fed to mouse models of ALS, the animals resisted becoming ill and lived 18 percent longer than those without creatine-laced chow. “Go for it!” said the research community. And because creatine is pretty benign stuff, it wasn’t long before human trials began.

But this winter, a well-done Dutch study showed oral creatine holds no benefits for ALS patients. And while results of two U.S. studies with a lower dosage are still to come, we wonder how something with a fair amount of mouse-power can be so disappointing in humans. It could be the dose in patients wasn’t equivalent. Perhaps differences in mouse metabolism are to blame. Maybe the point where creatine works in the mouse model is tied to the inherited ALS that the mouse mimics and not to the more common sporadic type addressed in clinical trials.

But such results don’t necessarily waste time. Other people’s no-shows tell you not to go down blind alleys. And because the Center is aggressive, a new team is bent on rapidly finding sure, early signposts that a drug will work. That’s as important to therapy as finding the drugs themselves.

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

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