The Robert Packard Center for ALS Research at Johns Hopkins

Our 5K run photo may be misleading. Yes, we’re in a race against time to stop ALS and heal its damage, but it’s not a single race. More than a dozen of them are taking place in our laboratories, and, as science works, they’re on track. Actually, because of our efforts to streamline research support and collaboration, they’re well ahead of where they’d be on the usual scientific path.

When the Center came together seven years ago, we had only two true standout clues to ALS:

We knew patients’ motor neurons were more vulnerable to death by overstimulation, called excitotoxicity.
And we knew that in people with a rare familial version of the disease, a mutated gene (mSOD1) was the cause.

So we took that and began running. Early Packard scientists were quick to develop animal models based on mSOD1, for example. They went on to describe resulting molecular, cell and whole-animal changes, always with an eye on therapy. Along the way, clues arose beyond SOD1 and excitotoxicity. And, guided by our science advisors, we funded investigations into those with promise.

It’s good that we did.

Even though Riluzole, the only drug approved for ALS, tested well on the SOD1 mice, that model hasn’t predicted other therapies. We know that. So, using other genes and characteristics of ALS, we’ve shifted to more varied models, aiming for one that reflects all types of the disease (see page 9). What’s quickened the models’ appearance and let us integrate them into the therapy search? Research diversity. Progress in our “races,” include finding that both sporadic and familial ALS warp a motor neuron’s internal transport system, that damping inflammation slows ALS damage, that stem cells can lead to healing in animal models and that we can turn up tissues’ built-in protective systems.

This year was one of advancement in the astrocyte race—showing that those central nervous cells are the main source of neurodegeneration in both types of ALS, even if they don’t necessarily trigger the disease. And our newest studies—uncovering very early flaws in nerve-muscle junctions and in muscles themselves—are focusing our efforts. We’re heightening our sensitivity for the one or two findings that will snap things into place. Then all of our races will converge into a clear route for a cure.

Meanwhile, we’re an accomplished group (page 2). Packard scientists have contributed more than 75 percent of recent ALS scholarship, publishing in journals such as Nature, Science and The Proceedings of the National Academy of Sciences. So far, we’ve awarded $11,888,528 in grants, often with as little as six weeks between applying and having the check in hand. We’re strong. We’re on track.

There’s no stopping us now.