Vantage Point

If you’ve read this column before, you’re no stranger to my feelings that even the most fundamental research we do—studies even more basic than how ALS begins—should have some clear connection to therapy. At the Center, we've always put our basic studies and therapy searches on equal pedestals because it can't be any other way: Short-change basic research and ideas for therapy dry up; put all your eggs in the fundamentals basket and you may delay potentially valuable treatments.

So this issue’s two scientific articles reflect our need to be in both places. One shows how Don Cleveland and his colleagues took basic information they’d gathered on the mutant SOD1 gene and its behavior—plus even more basic insights on how genes get expressed—and turned that into an intriguing possible therapy for familial ALS patients. We haven’t waited until we understood everything about familial or sporadic ALS. We’ve gone just as far as we needed with the basics and stopped to let the therapy studies catch up.

Jean-Pierre Julien’s discovery that cells selectively boot out flawed SOD1 protein, putting it adjacent to motor neurons where it can make trouble, builds on basic facts of cell secretion. No treatment has yet surfaced, but the fact that a mutant molecule waits outside motor neurons like a sitting duck makes it a tempting drug target.

A last comment. Recently, I’ve watched the two pedestals move closer together. It may be because of huge scientific technology advances. Or, within the Center, more likely, it’s a closer sharing of information. But whatever we use to shed light on basic science is also pulling treatments closer. The potential therapy from Cleveland’s team, for example, works by turning off a “bad” gene. But that technique originally had more basic purposes, to see what particular genes do.

So we encourage such work, then step out of the way. And expect the best.

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

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