ALS Alert mastheadALS Alert mastheadFall 2002 - Science. Scope. Speed

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In This Issue:

New Drug Screen: Uncle Sam’s Grand Present
Late this summer, 12 potential new ALS drugs—a jaw-dropping number—made their way into the Center’s first stages of animal testing thanks to an unusual project linking Center scientists and researchers across the country.

Center Scientist Eyes Key Step in Cells’ ‘Death March’
A current hot spot in als research centers on something long ignored as a source of trouble in the disease: the mitochondria. The plentiful, often jellybean-shaped cell bodies are dubbed the cell’s powerhouses because they generate most of its energy.

Accentuate the Positive
Some Center scientists, eying a cure, seek the cause of ALS. Others study how it damages cells. But a third group’s work may lessen immediate misery: They’re learning the basics of damage control and repair.

The Aggregate Dilemma: Too Obvious to Ignore
For years, scientists have noted obvious clumps of protein in motor neurons of patients with both sporadic and inherited forms of ALS—those who have a mutated gene for the SOD1 enzyme.

A Wedding to Remember
“I’ve come to realize what good friends are. People you knew but didn’t know have become friends. There’s a depth to it I hadn’t experienced before.”

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About ALS Alert

Vantage Point

photo - Jeffrey D. Rothstein, M.D., Ph.D.  
Jeffrey D. Rothstein, M.D., Ph.D.
   

What, exactly, does ALS do to motor neuron cells? In this issue, several of our articles feature the Center’s efforts to answer that crucial question. Most of our studies use animal models of the disease—they’re close mimics. And using them to find how ALS damages cells will help us understand how the disease occurs in the first place.

Read why Center investigator Valina Dawson, for example, asks: Are harmful molecules leaking out of the cell’s mitochondria? And follow Dave Borchelt’s steps to answer: Are damaging proteins accumulating in motor neurons?

A mounting body of evidence says “yes” to each question. That’s why these two areas—how mitochondria play a part in cell death and how the build-up of certain proteins is linked to motor neurons’ decline—are hot in ALS research.

Benefits of these basic studies also come in identifying specific targets we need for ALS therapy. And because our research continues under one roof, so to speak, we increase the likelihood of combining helpful approaches—the sort of tactic that’s made such a difference to AIDS patients—while we seek the cure.

So the Center does basic research. But, as our drug-screening story on this page shows, we also push investigations that could translate relatively quickly into therapy. We feel we must follow a variety of pathways because time isn’t on our side. Is Dr. Borchelt right? Dr. Dawson? Each basic science lab works in parallel as we also test possible therapies as aggressively as possible. We’re not going to wait to finish one study before we start the next; that time would be a luxury we can’t afford.

In other words, we cast our nets wide, but we make sure we’ve solid reasons for where we’re casting.

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

Next > On Center: New Name and ‘Wings’ Give Center Zing
Put 1,700 Wall Street investment bankers, ALS patients and supporters in one of the New York Marriott Marquis’ grand ballrooms, add hors d’oeuvres and some Hollywood “biggies” and you have this year’s Wings Over Wall Street.

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Special Features:

Vantage point
What, exactly, does ALS do to motor neuron cells? In this issue, several of our articles feature the Center’s efforts to answer that crucial question.

On Center: New Name and ‘Wings’ Give Center Zing
Put 1,700 Wall Street investment bankers, ALS patients and supporters in one of the New York Marriott Marquis’ grand ballrooms, add hors d’oeuvres and some Hollywood “biggies” and you have this year’s Wings Over Wall Street.

Insider’s View
Nicholas Maragakis, M.D., is a Hopkins neurologist/researcher who specializes in neuromuscular diseases. In this column he answers questions.

From the Clinic
Lora Clawson, M.S.N., C.R.N.P., manages Johns Hopkins’ ALS clinic. She also oversees its clinical trials. In this column she answers typical patients’ questions.

A Friend Indeed
With ALS Support, It’s Never ‘Too Many Cooks’

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