From the Director

In that time, the Packard Center has gone from little more than a wish list from a handful of ALS researchers—and the hopes of patients’ friends and families—to a world-class, self-supporting body that’s advanced knowledge of ALS while moving potential treatment forward as no other.

We’ve become known in the ALS community for high-quality fundamental research that leads quickly to new drugs and therapeutic approaches—four have made it to clinical trials and others are in the pipeline. We, ourselves, believe that borders on amazing.

What else have we accomplished? As you’ll read, we’ve been strong in creating animal models of ALS. Models clarify cell pathology; they’re also crucial to testing therapies. And since no model is perfect, we’ve devised four new ones. The chimeric mouse, for example (see In Five Short Years), let us make the major discovery that ALS probably doesn’t begin in motor neurons.

Our scientists helped find a new familial gene for ALS—ALS4. And one Packard researcher has set up a nationwide screening for genes tied to the common sporadic disease.

Many of us work with patients, and we’re well aware that they need help now. While we never stop seeking a cure, we see value in making ALS a chronic, manageable disease. So we’ve advanced studies to protect both nerves and muscles after ALS has started. Research continues to shed light on how nerve cells regrow, how synapses might be protected, how to keep muscles from deteriorating (see Jeff Johnson and Kathryn Wagner in Our Researchers). As for stem cells? We’re proud that we’re the world’s premier center for stem cell research for ALS.

Our partnerships with the organizations ALSA, MDA and Project ALS and, this year, our joining The Brain Trust Collaborative, a new union of four brain research philanthropies, all extend our reach. So do our NIH collaborations. And tapping into the clinical trials expertise of the Northeast ALS Consortium has sped well-designed testing of possible therapies.

The Center’s approach is working. Scientists’ own measuring sticks, NIH grants and journal publications, tell us that. We focus solely on ALS and unite handpicked scientists from around the world, giving them quick funding for targeted studies. We ask that they share pre-publication findings as well as their expertise. These first five years have seen our 30-or-so Packard researchers publish more than 297 ALS-related papers in leading journals. And Packard financial help or scientific expertise has supported the initial research they needed to justify at least 34 hard-to-come-by NIH RO1 grants. The latter add up to millions of dollars’ worth of additional research “muscle,” the sort that hastens a cure.

This year, our donor base has swelled to 1,344 good people who’ve helped us raise $2.8 million. Since we’ve opened, we’ve offered scientists more than $9 million in support.

At five years, our infrastructure is strong, our scientists skilled and dedicated, our program focused. And our steps to the cure are sure.

Jeffrey Rothstein