Vantage Point

ALS scientists haven’t spent much time at the middle ground. By that, I mean there’s not been much medical research into ways to make things better for those who have the disease, to use biology to enhance quality of life, even though we can’t make ALS go away. The low ground concerns itself with “outside” strategies to help—special forks, better speech devices. The high ground aims for a cure; it’s what laps up most of our time and energy here at the Packard Center.

Of course, nobody’s intentionally neglected “palliative care,” as it’s called, but until recently, not enough was known about ALS’s biology, or even about the give-and-take between healthy nerve and muscle for us to find anything helpful. So could ALS become a chronic disease? Sure. With the right drugs you could slow it substantially and ease patients’ symptoms.

The approach isn’t so far from what Parkinson’s disease patients experience: PD still progresses, but we can manage it significantly using drugs or surgery to stop the tremor and slowness of movement. Then, good quality of life extends longer than ever before.

So the Center starts with a specific question: Can drugs that enhance a muscle’s function (see Of Mighty Mice and Men) fill in the gaps? A new study hopes—ultimately—to make the most of patients’ muscles.

Far from diverting our search for the cure, exploring middle ground may deliver unexpected benefits. What if increasing muscle size in patients early on actually helps their motor neurons resist the disease? We’d be exploring what’s going on there in a hurry. You never know where the break that will deliver the cure comes from. For that reason alone, the middle ground bears investigating.

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

Next > On Center
Charity Begins on the Course