This month, The ALS Association (ALSA), a major sponsor of ALS research
worldwide, has announced joint support of two projects with the Robert
Packard Center for ALS Research at Johns Hopkins. The sponsorship marks
the first the two institutions have shared. "This will certainly help
extend our reach to understand the disease, something we expect to move
therapy closer," says Jeffrey Rothstein, the Center's director.

As of February 1, ALSA will help fund the work of three Center
scientists, Philip Wong, Ph.D., Huaibin Cai, Ph.D., and David Borschelt,
Ph.D. All are basic scientists whose work focuses on understanding
precisely how ALS harms motor neuron cells.

Wong and Cai are focusing on the role of the abnormally short protein
alsin, which is the product of a mutant gene associated with a rare form
of juvenile ALS. Because it's abnormal, the alsin protein apparently
cannot function in cells.

By knocking out the alsin gene altogether, the researchers are
developing an animal model that mimics the human condition. They'll
follow the animals for signs of disease, specifically observing changes
in motor neurons. Additionally, the team is creating ways to track
what's going on molecularly in the animal model cells. "For a variety of
reasons, it's challenging work," Wong says.

While the function of alsin is a mystery, its structure has given
scientists clues about how it brings about disease. "Figuring out how
alsin leads to the death of cells should be a major step in
understanding all types of ALS, not just this very rare juvenile form,"
says Lucie Bruijn, Ph.D., the science director for The ALS Association.
"As new genes linked to the disease are discovered," she adds,
"scientists may find that common disease pathways exist. Such paths
would make natural targets for therapy."

* * * *

In the second jointly-funded project, David Borschelt will work with
the more traditional ALS model, that of SOD1 mice ---- animals that
carry a mutant human gene linked with a slightly more common inherited
form of ALS. Borchelt will systematically remove parts of the abnormal
SOD1 protein, trying to find which part it is responsible for the
protein's known ability to form large clumps in motor neuron cells. The
work could shore up earlier studies that suggest the clumping or
aggregation is tied to cell disease.

The role of aggregation in disease has gained increasing attention
during the past few years. Such clumping may be a part of the most
common sporadic form of ALS as well as the more unusual familial forms.

Once Borschelt or others pinpoint SOD1's "hot spot" for aggregation,
the scientists believe, designing a drug or other means to put it out of
commission would be the next step of choice.

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The Center for ALS Research at Johns Hopkins is a collaborative effort
by some of the best ALS and non-ALS scientists to aggressively and
rapidly develop new treatments and find a cure for ALS, also known as
Lou Gehrig's disease. It's the only institution of its kind dedicated
solely to the disease. Research conducted by the Center is meant to
translate from bench to bedside in an expedited time frame. Center
scientists from institutions around the world have made some of the most
important discoveries in ALS, leading to advances in understanding and
treatment of the disease.

The nature of ALS shapes the Center's aggressive, results-oriented
scientific approach. ALS is a devastating, progressive neuromuscular
disease that causes complete paralysis and loss of function -- including
the ability to eat, speak and breathe -- and eventually, death. ALS
progresses quickly and is not curable. Most patients die within five
years of diagnosis.

To learn more about The Center for ALS Research at Johns Hopkins,
including the latest information on ALS research and treatment, log on
to www.alscenter.org.