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December 10, 2002
SECOND ANNUAL RESEARCH SYMPOSIUM:
PROGRESS IS SURE
One advantage of requiring 24 ALS researchers to present their scientific
advances once a year is that you quickly see what's hot in the field and
how fast things are moving. With their second annual symposium just finished
in Baltimore this fall, scientists with the Robert Packard Center for
ALS Research have realized genuine progress in their path to a cure.
Their broad advances lie in understanding how ALS upsets normal cell biology,
unleashing damage on motor neurons - the disease's main casualty. The
researchers have offered credible ideas on how that damage could begin.
And some have shed light on ways the body normally repairs a damaged nervous
system, or could, given stem cells or exposure to certain natural cell
substances.
But it's the specifics that are most interesting. Many of the Center's
studies focus on the animal models of ALS, the rodents that get a version
of ALS when they carry the same mutant human genes as people with the
less-common inherited form of the disease. More than a half-dozen researchers
are trying to determine just why those animals sicken and die.
In his symposium talk, researcher Larry Goldstein from the University
of California San Diego, discussed strong evidence that ALS doesn't necessarily
begin in the motor neurons ---- the cells whose death is a hallmark of
the disease. He described new evidence that glial cells, which surround
motor neurons, bring about the healthy cells' death. Such news could provide
new approaches to therapy by increasing the ratio of healthy to sick cells
via stem cells, for example, or by switching on helpful genes or using
drugs that mimic their effects.
David Borchelt at Johns Hopkins is closer to explaining an odd clumping
of protein that appears in motor neuron cells of some people with an inherited
form of ALS. Meanwhile Jeffrey Elliott at the University of Texas is homing
in on the specific mutations that trigger the clumping. He's also designing
studies to show if protein clumps are toxic.
From work at McGill University, Jean-Pierre Julien reported signs that
a three-drug "cocktail" may be more therapeutic in animal models than
the sole FDA-approved agent for ALS. He also reported signs that inflammation
pays a clear role in the disease-one that could, in theory, be lessened
with drugs.
And, from Hopkins, Vassilis Koliatsos showed his evidence that certain
human stem cells, when introduced into damaged rodent nervous systems,
appear capable of differentiating into spinal cord cells. Like true nerve
cells, these have connections with other nerve cells and they express
nerve transmitters. Koliatsos' studies help answer questions on whether
immature (stem) cells might help repair an established, adult nervous
system.
Work by Hopkins scientist Philip Wong and UCSD researcher Don Cleveland
is well under way to create a new animal model of ALS with the second,
recently-discovered ALS gene, the one for a juvenile form of the disease.
And David Irani, also of Johns Hopkins, reported on an easy-to-create
animal model, using Sindbis virus that mimics several key features of
common forms of ALS.
Most exciting, director Jeffrey Rothstein announced that a large, national
screening of existing drugs for a variety of disorders has resulted in
a half-dozen new possibilities for ALS. This year, those potential drugs
will be put on a fast-track for animal testing as a collaboration between
the Packard Center, Project ALS, the ALS Association and the NIH. Should
they warrant it, the drugs will then move into clinical trials.
more Recent News
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| Recent news from the Robert Packard Center
for ALS Research: |
| Packard Center Welcomes Its First Dedicated Science Director - July 30, 3008 |
| In ALS, It’s Not the Number of Ailing Astrocytes That Counts - June 12, 2008 |
| Leaky Blood Vessels Add To ALS Damage, Could Offer New Repair Site - June 10, 2008 |
| William H. Adams Foundation Pumps New Energy, Funds into Search for ALS Cure - May 6, 2008 |
| Tell-Tale Protein Clumping in ALS is Less Complex Than Expected - April 10, 2008 |
ALS Mouse Study Highlights Astrocytes' Strong Potential as Therapy Target - February 7, 2008 |
| Exciting New Human ALS Trial: Lithium and Riluzole - February 7, 2008 |
| ALS Treatment: A Matter of Cleaning House? - December 19, 2007 |
New Study Brings What Goes Wrong in Inherited ALS into Focus - September 18, 2007 |
| New ALS Protein Could Be a Keystone - August 9, 2007 |
| Muscles More Than Passive Victims in ALS, Study Suggests - June 29, 2007 |
| Saer and O’Neill Named Packard Center Board Co-Chairs - June 28, 2007 |
Self-Attack? Self-Repair? First Real Look at Gene Activity in ALS Models Sparks Thirst for Answers - May 3, 2007 |
| Model of Accelerated Familial ALS Sheds Light on Disease Process - April 6, 2007 |
| Early News From First Large Search for Sporadic ALS Genes - February 20, 2007 |
| Human Stem Cell Transplants Mature Into Neurons and Make Contacts in Rat Spinal Cord - February 14, 2007 |
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